Evidence map›Paper›PMID 38828955›Full record

Trial reportThe New England journal of medicine2024

Phase 3 Trial of Crinecerfont in Adult Congenital Adrenal Hyperplasia.

Richard J Auchus, Oksana Hamidi, Rosario Pivonello, Irina Bancos, Gianni Russo, Selma F Witchel, Andrea M Isidori, Patrice Rodien, Umasuthan Srirangalingam, Florian W Kiefer and 11 more

2 registry-linked trialsAbstract readRandomized Controlled TrialClinical Trial, Phase IIIMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Trial report in The New England journal of medicine, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 32 papers.

0numbers the graph read from it
0cells of the map it votes in
32citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT04490915 phase3active not recruitingnot on this map

A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Adult Subjects With Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment

TypeinterventionalSponsorNeurocrine BiosciencesRan2020 to 2027Enrolled182ConditionsCongenital Adrenal HyperplasiaArmsCrinecerfont, Placebo
NCT07099456 recruitingnot on this map

Channelling Fertility And Sexual Function In Congenital Adrenal Hyperplasia. CALLIOPE: An Observational, Longitudinal Pilot Study

TypeobservationalSponsorUniversity of Roma La SapienzaRan2024 to 2030Enrolled50ConditionsCongenital Adrenal Hyperplasia (CAH)
3 · Its place in the literature

Who cites it

32 citing papers in PubMed.

  1. Trial
  2. Review
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  4. Article
  5. Integrated mapping resolves pathogenicity of 11Journal of the Endocrine Society · 2026
    Article
  6. Article
  7. [Congenital adrenal hyperplasia].Problemy endokrinologii · 2026
    Review
  8. Review
  9. Article
  10. Article
  11. Review
  12. Article
  13. Review
  14. Article
  15. Review
  16. Article
  17. Article
  18. Review
  19. [Transition in cases of complex endocrine and diabetic diseases].Innere Medizin (Heidelberg, Germany) · 2025
    Review
  20. Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

21 authors.

Richard J AuchusFrom the Departments of Pharmacology and Internal Medicine, Division of Metabolism, Endocrinology, and Diabetes, University of Michigan Medical School, and the Endocrinology and Metabolism Section, Medicine Service, Lieutenant Colonel Charles S. Kettles Veterans Affairs Medical Center - both in Ann Arbor (R.J.A.); the Division of Endocrinology and Metabolism, University of Texas Southwestern Medical Center, Dallas (O.H.); Dipartimento di Medicina Clinica e Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologia e Nutrizione, Università Federico II di Napoli, Naples (R.P.), the Department of Pediatrics, Endocrine Unit, IRCCS San Raffaele Scientific Institute, Endo-ERN Center for Rare Endocrine Conditions, Milan (G.R.), and the Department of Experimental Medicine, Sapienza University of Rome, Rome (A.M.I.) - all in Italy; the Division of Endocrinology and Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester (I.B.), and the Departments of Pediatrics and Experimental and Clinical Pharmacology, Divisions of Endocrinology and Genetics and Metabolism, University of Minnesota Medical School, Minneapolis (K.S.) - both in Minnesota; the Division of Pediatric Endocrinology, Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine, Pittsburgh (S.F.W.); the Department of Endocrinology, Diabetology, and Nutrition, Endo-ERN Center for Rare Endocrine Conditions, Centre Hospitalier Universitaire d'Angers and Laboratoire Physiopathologie Cardiovasculaire et Mitochondriale, Université d'Angers, Angers, France (P.R.); the Departments of Endocrinology and Diabetes, University College London Hospital, London (U.S.); the Division of Endocrinology and Metabolism, Department of Medicine III, Medical University of Vienna, Vienna (F.W.K.); the Department of Molecular Medicine and Surgery, Karolinska Institutet, and the Department of Endocrinology, Karolinska University Hospital, Stockholm (H.F.); the National Institutes of Health Clinical Center and Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD (D.P.M.); the Department of Endocrinology, Internal Medicine IV, Ludwig Maximilians Universität München, Munich, Germany (N.R.); Gordon Cutler Consultancy, Deltaville, VA (G.B.C.); and Neurocrine Biosciences, San Diego, CA (J.S., E.R., V.H.L., J.L.C., R.H.F.).
Oksana HamidiFrom the Departments of Pharmacology and Internal Medicine, Division of Metabolism, Endocrinology, and Diabetes, University of Michigan Medical School, and the Endocrinology and Metabolism Section, Medicine Service, Lieutenant Colonel Charles S. Kettles Veterans Affairs Medical Center - both in Ann Arbor (R.J.A.); the Division of Endocrinology and Metabolism, University of Texas Southwestern Medical Center, Dallas (O.H.); Dipartimento di Medicina Clinica e Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologia e Nutrizione, Università Federico II di Napoli, Naples (R.P.), the Department of Pediatrics, Endocrine Unit, IRCCS San Raffaele Scientific Institute, Endo-ERN Center for Rare Endocrine Conditions, Milan (G.R.), and the Department of Experimental Medicine, Sapienza University of Rome, Rome (A.M.I.) - all in Italy; the Division of Endocrinology and Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester (I.B.), and the Departments of Pediatrics and Experimental and Clinical Pharmacology, Divisions of Endocrinology and Genetics and Metabolism, University of Minnesota Medical School, Minneapolis (K.S.) - both in Minnesota; the Division of Pediatric Endocrinology, Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine, Pittsburgh (S.F.W.); the Department of Endocrinology, Diabetology, and Nutrition, Endo-ERN Center for Rare Endocrine Conditions, Centre Hospitalier Universitaire d'Angers and Laboratoire Physiopathologie Cardiovasculaire et Mitochondriale, Université d'Angers, Angers, France (P.R.); the Departments of Endocrinology and Diabetes, University College London Hospital, London (U.S.); the Division of Endocrinology and Metabolism, Department of Medicine III, Medical University of Vienna, Vienna (F.W.K.); the Department of Molecular Medicine and Surgery, Karolinska Institutet, and the Department of Endocrinology, Karolinska University Hospital, Stockholm (H.F.); the National Institutes of Health Clinical Center and Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD (D.P.M.); the Department of Endocrinology, Internal Medicine IV, Ludwig Maximilians Universität München, Munich, Germany (N.R.); Gordon Cutler Consultancy, Deltaville, VA (G.B.C.); and Neurocrine Biosciences, San Diego, CA (J.S., E.R., V.H.L., J.L.C., R.H.F.).
Rosario PivonelloFrom the Departments of Pharmacology and Internal Medicine, Division of Metabolism, Endocrinology, and Diabetes, University of Michigan Medical School, and the Endocrinology and Metabolism Section, Medicine Service, Lieutenant Colonel Charles S. Kettles Veterans Affairs Medical Center - both in Ann Arbor (R.J.A.); the Division of Endocrinology and Metabolism, University of Texas Southwestern Medical Center, Dallas (O.H.); Dipartimento di Medicina Clinica e Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologia e Nutrizione, Università Federico II di Napoli, Naples (R.P.), the Department of Pediatrics, Endocrine Unit, IRCCS San Raffaele Scientific Institute, Endo-ERN Center for Rare Endocrine Conditions, Milan (G.R.), and the Department of Experimental Medicine, Sapienza University of Rome, Rome (A.M.I.) - all in Italy; the Division of Endocrinology and Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester (I.B.), and the Departments of Pediatrics and Experimental and Clinical Pharmacology, Divisions of Endocrinology and Genetics and Metabolism, University of Minnesota Medical School, Minneapolis (K.S.) - both in Minnesota; the Division of Pediatric Endocrinology, Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine, Pittsburgh (S.F.W.); the Department of Endocrinology, Diabetology, and Nutrition, Endo-ERN Center for Rare Endocrine Conditions, Centre Hospitalier Universitaire d'Angers and Laboratoire Physiopathologie Cardiovasculaire et Mitochondriale, Université d'Angers, Angers, France (P.R.); the Departments of Endocrinology and Diabetes, University College London Hospital, London (U.S.); the Division of Endocrinology and Metabolism, Department of Medicine III, Medical University of Vienna, Vienna (F.W.K.); the Department of Molecular Medicine and Surgery, Karolinska Institutet, and the Department of Endocrinology, Karolinska University Hospital, Stockholm (H.F.); the National Institutes of Health Clinical Center and Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD (D.P.M.); the Department of Endocrinology, Internal Medicine IV, Ludwig Maximilians Universität München, Munich, Germany (N.R.); Gordon Cutler Consultancy, Deltaville, VA (G.B.C.); and Neurocrine Biosciences, San Diego, CA (J.S., E.R., V.H.L., J.L.C., R.H.F.).
Irina BancosFrom the Departments of Pharmacology and Internal Medicine, Division of Metabolism, Endocrinology, and Diabetes, University of Michigan Medical School, and the Endocrinology and Metabolism Section, Medicine Service, Lieutenant Colonel Charles S. Kettles Veterans Affairs Medical Center - both in Ann Arbor (R.J.A.); the Division of Endocrinology and Metabolism, University of Texas Southwestern Medical Center, Dallas (O.H.); Dipartimento di Medicina Clinica e Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologia e Nutrizione, Università Federico II di Napoli, Naples (R.P.), the Department of Pediatrics, Endocrine Unit, IRCCS San Raffaele Scientific Institute, Endo-ERN Center for Rare Endocrine Conditions, Milan (G.R.), and the Department of Experimental Medicine, Sapienza University of Rome, Rome (A.M.I.) - all in Italy; the Division of Endocrinology and Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester (I.B.), and the Departments of Pediatrics and Experimental and Clinical Pharmacology, Divisions of Endocrinology and Genetics and Metabolism, University of Minnesota Medical School, Minneapolis (K.S.) - both in Minnesota; the Division of Pediatric Endocrinology, Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine, Pittsburgh (S.F.W.); the Department of Endocrinology, Diabetology, and Nutrition, Endo-ERN Center for Rare Endocrine Conditions, Centre Hospitalier Universitaire d'Angers and Laboratoire Physiopathologie Cardiovasculaire et Mitochondriale, Université d'Angers, Angers, France (P.R.); the Departments of Endocrinology and Diabetes, University College London Hospital, London (U.S.); the Division of Endocrinology and Metabolism, Department of Medicine III, Medical University of Vienna, Vienna (F.W.K.); the Department of Molecular Medicine and Surgery, Karolinska Institutet, and the Department of Endocrinology, Karolinska University Hospital, Stockholm (H.F.); the National Institutes of Health Clinical Center and Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD (D.P.M.); the Department of Endocrinology, Internal Medicine IV, Ludwig Maximilians Universität München, Munich, Germany (N.R.); Gordon Cutler Consultancy, Deltaville, VA (G.B.C.); and Neurocrine Biosciences, San Diego, CA (J.S., E.R., V.H.L., J.L.C., R.H.F.).
Gianni RussoFrom the Departments of Pharmacology and Internal Medicine, Division of Metabolism, Endocrinology, and Diabetes, University of Michigan Medical School, and the Endocrinology and Metabolism Section, Medicine Service, Lieutenant Colonel Charles S. Kettles Veterans Affairs Medical Center - both in Ann Arbor (R.J.A.); the Division of Endocrinology and Metabolism, University of Texas Southwestern Medical Center, Dallas (O.H.); Dipartimento di Medicina Clinica e Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologia e Nutrizione, Università Federico II di Napoli, Naples (R.P.), the Department of Pediatrics, Endocrine Unit, IRCCS San Raffaele Scientific Institute, Endo-ERN Center for Rare Endocrine Conditions, Milan (G.R.), and the Department of Experimental Medicine, Sapienza University of Rome, Rome (A.M.I.) - all in Italy; the Division of Endocrinology and Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester (I.B.), and the Departments of Pediatrics and Experimental and Clinical Pharmacology, Divisions of Endocrinology and Genetics and Metabolism, University of Minnesota Medical School, Minneapolis (K.S.) - both in Minnesota; the Division of Pediatric Endocrinology, Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine, Pittsburgh (S.F.W.); the Department of Endocrinology, Diabetology, and Nutrition, Endo-ERN Center for Rare Endocrine Conditions, Centre Hospitalier Universitaire d'Angers and Laboratoire Physiopathologie Cardiovasculaire et Mitochondriale, Université d'Angers, Angers, France (P.R.); the Departments of Endocrinology and Diabetes, University College London Hospital, London (U.S.); the Division of Endocrinology and Metabolism, Department of Medicine III, Medical University of Vienna, Vienna (F.W.K.); the Department of Molecular Medicine and Surgery, Karolinska Institutet, and the Department of Endocrinology, Karolinska University Hospital, Stockholm (H.F.); the National Institutes of Health Clinical Center and Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD (D.P.M.); the Department of Endocrinology, Internal Medicine IV, Ludwig Maximilians Universität München, Munich, Germany (N.R.); Gordon Cutler Consultancy, Deltaville, VA (G.B.C.); and Neurocrine Biosciences, San Diego, CA (J.S., E.R., V.H.L., J.L.C., R.H.F.).
Selma F WitchelFrom the Departments of Pharmacology and Internal Medicine, Division of Metabolism, Endocrinology, and Diabetes, University of Michigan Medical School, and the Endocrinology and Metabolism Section, Medicine Service, Lieutenant Colonel Charles S. Kettles Veterans Affairs Medical Center - both in Ann Arbor (R.J.A.); the Division of Endocrinology and Metabolism, University of Texas Southwestern Medical Center, Dallas (O.H.); Dipartimento di Medicina Clinica e Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologia e Nutrizione, Università Federico II di Napoli, Naples (R.P.), the Department of Pediatrics, Endocrine Unit, IRCCS San Raffaele Scientific Institute, Endo-ERN Center for Rare Endocrine Conditions, Milan (G.R.), and the Department of Experimental Medicine, Sapienza University of Rome, Rome (A.M.I.) - all in Italy; the Division of Endocrinology and Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester (I.B.), and the Departments of Pediatrics and Experimental and Clinical Pharmacology, Divisions of Endocrinology and Genetics and Metabolism, University of Minnesota Medical School, Minneapolis (K.S.) - both in Minnesota; the Division of Pediatric Endocrinology, Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine, Pittsburgh (S.F.W.); the Department of Endocrinology, Diabetology, and Nutrition, Endo-ERN Center for Rare Endocrine Conditions, Centre Hospitalier Universitaire d'Angers and Laboratoire Physiopathologie Cardiovasculaire et Mitochondriale, Université d'Angers, Angers, France (P.R.); the Departments of Endocrinology and Diabetes, University College London Hospital, London (U.S.); the Division of Endocrinology and Metabolism, Department of Medicine III, Medical University of Vienna, Vienna (F.W.K.); the Department of Molecular Medicine and Surgery, Karolinska Institutet, and the Department of Endocrinology, Karolinska University Hospital, Stockholm (H.F.); the National Institutes of Health Clinical Center and Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD (D.P.M.); the Department of Endocrinology, Internal Medicine IV, Ludwig Maximilians Universität München, Munich, Germany (N.R.); Gordon Cutler Consultancy, Deltaville, VA (G.B.C.); and Neurocrine Biosciences, San Diego, CA (J.S., E.R., V.H.L., J.L.C., R.H.F.).
Andrea M IsidoriFrom the Departments of Pharmacology and Internal Medicine, Division of Metabolism, Endocrinology, and Diabetes, University of Michigan Medical School, and the Endocrinology and Metabolism Section, Medicine Service, Lieutenant Colonel Charles S. Kettles Veterans Affairs Medical Center - both in Ann Arbor (R.J.A.); the Division of Endocrinology and Metabolism, University of Texas Southwestern Medical Center, Dallas (O.H.); Dipartimento di Medicina Clinica e Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologia e Nutrizione, Università Federico II di Napoli, Naples (R.P.), the Department of Pediatrics, Endocrine Unit, IRCCS San Raffaele Scientific Institute, Endo-ERN Center for Rare Endocrine Conditions, Milan (G.R.), and the Department of Experimental Medicine, Sapienza University of Rome, Rome (A.M.I.) - all in Italy; the Division of Endocrinology and Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester (I.B.), and the Departments of Pediatrics and Experimental and Clinical Pharmacology, Divisions of Endocrinology and Genetics and Metabolism, University of Minnesota Medical School, Minneapolis (K.S.) - both in Minnesota; the Division of Pediatric Endocrinology, Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine, Pittsburgh (S.F.W.); the Department of Endocrinology, Diabetology, and Nutrition, Endo-ERN Center for Rare Endocrine Conditions, Centre Hospitalier Universitaire d'Angers and Laboratoire Physiopathologie Cardiovasculaire et Mitochondriale, Université d'Angers, Angers, France (P.R.); the Departments of Endocrinology and Diabetes, University College London Hospital, London (U.S.); the Division of Endocrinology and Metabolism, Department of Medicine III, Medical University of Vienna, Vienna (F.W.K.); the Department of Molecular Medicine and Surgery, Karolinska Institutet, and the Department of Endocrinology, Karolinska University Hospital, Stockholm (H.F.); the National Institutes of Health Clinical Center and Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD (D.P.M.); the Department of Endocrinology, Internal Medicine IV, Ludwig Maximilians Universität München, Munich, Germany (N.R.); Gordon Cutler Consultancy, Deltaville, VA (G.B.C.); and Neurocrine Biosciences, San Diego, CA (J.S., E.R., V.H.L., J.L.C., R.H.F.).
Patrice RodienFrom the Departments of Pharmacology and Internal Medicine, Division of Metabolism, Endocrinology, and Diabetes, University of Michigan Medical School, and the Endocrinology and Metabolism Section, Medicine Service, Lieutenant Colonel Charles S. Kettles Veterans Affairs Medical Center - both in Ann Arbor (R.J.A.); the Division of Endocrinology and Metabolism, University of Texas Southwestern Medical Center, Dallas (O.H.); Dipartimento di Medicina Clinica e Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologia e Nutrizione, Università Federico II di Napoli, Naples (R.P.), the Department of Pediatrics, Endocrine Unit, IRCCS San Raffaele Scientific Institute, Endo-ERN Center for Rare Endocrine Conditions, Milan (G.R.), and the Department of Experimental Medicine, Sapienza University of Rome, Rome (A.M.I.) - all in Italy; the Division of Endocrinology and Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester (I.B.), and the Departments of Pediatrics and Experimental and Clinical Pharmacology, Divisions of Endocrinology and Genetics and Metabolism, University of Minnesota Medical School, Minneapolis (K.S.) - both in Minnesota; the Division of Pediatric Endocrinology, Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine, Pittsburgh (S.F.W.); the Department of Endocrinology, Diabetology, and Nutrition, Endo-ERN Center for Rare Endocrine Conditions, Centre Hospitalier Universitaire d'Angers and Laboratoire Physiopathologie Cardiovasculaire et Mitochondriale, Université d'Angers, Angers, France (P.R.); the Departments of Endocrinology and Diabetes, University College London Hospital, London (U.S.); the Division of Endocrinology and Metabolism, Department of Medicine III, Medical University of Vienna, Vienna (F.W.K.); the Department of Molecular Medicine and Surgery, Karolinska Institutet, and the Department of Endocrinology, Karolinska University Hospital, Stockholm (H.F.); the National Institutes of Health Clinical Center and Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD (D.P.M.); the Department of Endocrinology, Internal Medicine IV, Ludwig Maximilians Universität München, Munich, Germany (N.R.); Gordon Cutler Consultancy, Deltaville, VA (G.B.C.); and Neurocrine Biosciences, San Diego, CA (J.S., E.R., V.H.L., J.L.C., R.H.F.).
Umasuthan SrirangalingamFrom the Departments of Pharmacology and Internal Medicine, Division of Metabolism, Endocrinology, and Diabetes, University of Michigan Medical School, and the Endocrinology and Metabolism Section, Medicine Service, Lieutenant Colonel Charles S. Kettles Veterans Affairs Medical Center - both in Ann Arbor (R.J.A.); the Division of Endocrinology and Metabolism, University of Texas Southwestern Medical Center, Dallas (O.H.); Dipartimento di Medicina Clinica e Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologia e Nutrizione, Università Federico II di Napoli, Naples (R.P.), the Department of Pediatrics, Endocrine Unit, IRCCS San Raffaele Scientific Institute, Endo-ERN Center for Rare Endocrine Conditions, Milan (G.R.), and the Department of Experimental Medicine, Sapienza University of Rome, Rome (A.M.I.) - all in Italy; the Division of Endocrinology and Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester (I.B.), and the Departments of Pediatrics and Experimental and Clinical Pharmacology, Divisions of Endocrinology and Genetics and Metabolism, University of Minnesota Medical School, Minneapolis (K.S.) - both in Minnesota; the Division of Pediatric Endocrinology, Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine, Pittsburgh (S.F.W.); the Department of Endocrinology, Diabetology, and Nutrition, Endo-ERN Center for Rare Endocrine Conditions, Centre Hospitalier Universitaire d'Angers and Laboratoire Physiopathologie Cardiovasculaire et Mitochondriale, Université d'Angers, Angers, France (P.R.); the Departments of Endocrinology and Diabetes, University College London Hospital, London (U.S.); the Division of Endocrinology and Metabolism, Department of Medicine III, Medical University of Vienna, Vienna (F.W.K.); the Department of Molecular Medicine and Surgery, Karolinska Institutet, and the Department of Endocrinology, Karolinska University Hospital, Stockholm (H.F.); the National Institutes of Health Clinical Center and Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD (D.P.M.); the Department of Endocrinology, Internal Medicine IV, Ludwig Maximilians Universität München, Munich, Germany (N.R.); Gordon Cutler Consultancy, Deltaville, VA (G.B.C.); and Neurocrine Biosciences, San Diego, CA (J.S., E.R., V.H.L., J.L.C., R.H.F.).
Florian W KieferFrom the Departments of Pharmacology and Internal Medicine, Division of Metabolism, Endocrinology, and Diabetes, University of Michigan Medical School, and the Endocrinology and Metabolism Section, Medicine Service, Lieutenant Colonel Charles S. Kettles Veterans Affairs Medical Center - both in Ann Arbor (R.J.A.); the Division of Endocrinology and Metabolism, University of Texas Southwestern Medical Center, Dallas (O.H.); Dipartimento di Medicina Clinica e Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologia e Nutrizione, Università Federico II di Napoli, Naples (R.P.), the Department of Pediatrics, Endocrine Unit, IRCCS San Raffaele Scientific Institute, Endo-ERN Center for Rare Endocrine Conditions, Milan (G.R.), and the Department of Experimental Medicine, Sapienza University of Rome, Rome (A.M.I.) - all in Italy; the Division of Endocrinology and Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester (I.B.), and the Departments of Pediatrics and Experimental and Clinical Pharmacology, Divisions of Endocrinology and Genetics and Metabolism, University of Minnesota Medical School, Minneapolis (K.S.) - both in Minnesota; the Division of Pediatric Endocrinology, Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine, Pittsburgh (S.F.W.); the Department of Endocrinology, Diabetology, and Nutrition, Endo-ERN Center for Rare Endocrine Conditions, Centre Hospitalier Universitaire d'Angers and Laboratoire Physiopathologie Cardiovasculaire et Mitochondriale, Université d'Angers, Angers, France (P.R.); the Departments of Endocrinology and Diabetes, University College London Hospital, London (U.S.); the Division of Endocrinology and Metabolism, Department of Medicine III, Medical University of Vienna, Vienna (F.W.K.); the Department of Molecular Medicine and Surgery, Karolinska Institutet, and the Department of Endocrinology, Karolinska University Hospital, Stockholm (H.F.); the National Institutes of Health Clinical Center and Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD (D.P.M.); the Department of Endocrinology, Internal Medicine IV, Ludwig Maximilians Universität München, Munich, Germany (N.R.); Gordon Cutler Consultancy, Deltaville, VA (G.B.C.); and Neurocrine Biosciences, San Diego, CA (J.S., E.R., V.H.L., J.L.C., R.H.F.).
Henrik FalhammarFrom the Departments of Pharmacology and Internal Medicine, Division of Metabolism, Endocrinology, and Diabetes, University of Michigan Medical School, and the Endocrinology and Metabolism Section, Medicine Service, Lieutenant Colonel Charles S. Kettles Veterans Affairs Medical Center - both in Ann Arbor (R.J.A.); the Division of Endocrinology and Metabolism, University of Texas Southwestern Medical Center, Dallas (O.H.); Dipartimento di Medicina Clinica e Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologia e Nutrizione, Università Federico II di Napoli, Naples (R.P.), the Department of Pediatrics, Endocrine Unit, IRCCS San Raffaele Scientific Institute, Endo-ERN Center for Rare Endocrine Conditions, Milan (G.R.), and the Department of Experimental Medicine, Sapienza University of Rome, Rome (A.M.I.) - all in Italy; the Division of Endocrinology and Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester (I.B.), and the Departments of Pediatrics and Experimental and Clinical Pharmacology, Divisions of Endocrinology and Genetics and Metabolism, University of Minnesota Medical School, Minneapolis (K.S.) - both in Minnesota; the Division of Pediatric Endocrinology, Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine, Pittsburgh (S.F.W.); the Department of Endocrinology, Diabetology, and Nutrition, Endo-ERN Center for Rare Endocrine Conditions, Centre Hospitalier Universitaire d'Angers and Laboratoire Physiopathologie Cardiovasculaire et Mitochondriale, Université d'Angers, Angers, France (P.R.); the Departments of Endocrinology and Diabetes, University College London Hospital, London (U.S.); the Division of Endocrinology and Metabolism, Department of Medicine III, Medical University of Vienna, Vienna (F.W.K.); the Department of Molecular Medicine and Surgery, Karolinska Institutet, and the Department of Endocrinology, Karolinska University Hospital, Stockholm (H.F.); the National Institutes of Health Clinical Center and Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD (D.P.M.); the Department of Endocrinology, Internal Medicine IV, Ludwig Maximilians Universität München, Munich, Germany (N.R.); Gordon Cutler Consultancy, Deltaville, VA (G.B.C.); and Neurocrine Biosciences, San Diego, CA (J.S., E.R., V.H.L., J.L.C., R.H.F.).ORCID 0000-0002-5622-6987
Deborah P MerkeFrom the Departments of Pharmacology and Internal Medicine, Division of Metabolism, Endocrinology, and Diabetes, University of Michigan Medical School, and the Endocrinology and Metabolism Section, Medicine Service, Lieutenant Colonel Charles S. Kettles Veterans Affairs Medical Center - both in Ann Arbor (R.J.A.); the Division of Endocrinology and Metabolism, University of Texas Southwestern Medical Center, Dallas (O.H.); Dipartimento di Medicina Clinica e Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologia e Nutrizione, Università Federico II di Napoli, Naples (R.P.), the Department of Pediatrics, Endocrine Unit, IRCCS San Raffaele Scientific Institute, Endo-ERN Center for Rare Endocrine Conditions, Milan (G.R.), and the Department of Experimental Medicine, Sapienza University of Rome, Rome (A.M.I.) - all in Italy; the Division of Endocrinology and Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester (I.B.), and the Departments of Pediatrics and Experimental and Clinical Pharmacology, Divisions of Endocrinology and Genetics and Metabolism, University of Minnesota Medical School, Minneapolis (K.S.) - both in Minnesota; the Division of Pediatric Endocrinology, Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine, Pittsburgh (S.F.W.); the Department of Endocrinology, Diabetology, and Nutrition, Endo-ERN Center for Rare Endocrine Conditions, Centre Hospitalier Universitaire d'Angers and Laboratoire Physiopathologie Cardiovasculaire et Mitochondriale, Université d'Angers, Angers, France (P.R.); the Departments of Endocrinology and Diabetes, University College London Hospital, London (U.S.); the Division of Endocrinology and Metabolism, Department of Medicine III, Medical University of Vienna, Vienna (F.W.K.); the Department of Molecular Medicine and Surgery, Karolinska Institutet, and the Department of Endocrinology, Karolinska University Hospital, Stockholm (H.F.); the National Institutes of Health Clinical Center and Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD (D.P.M.); the Department of Endocrinology, Internal Medicine IV, Ludwig Maximilians Universität München, Munich, Germany (N.R.); Gordon Cutler Consultancy, Deltaville, VA (G.B.C.); and Neurocrine Biosciences, San Diego, CA (J.S., E.R., V.H.L., J.L.C., R.H.F.).ORCID 0000-0002-3746-0460
Nicole ReischFrom the Departments of Pharmacology and Internal Medicine, Division of Metabolism, Endocrinology, and Diabetes, University of Michigan Medical School, and the Endocrinology and Metabolism Section, Medicine Service, Lieutenant Colonel Charles S. Kettles Veterans Affairs Medical Center - both in Ann Arbor (R.J.A.); the Division of Endocrinology and Metabolism, University of Texas Southwestern Medical Center, Dallas (O.H.); Dipartimento di Medicina Clinica e Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologia e Nutrizione, Università Federico II di Napoli, Naples (R.P.), the Department of Pediatrics, Endocrine Unit, IRCCS San Raffaele Scientific Institute, Endo-ERN Center for Rare Endocrine Conditions, Milan (G.R.), and the Department of Experimental Medicine, Sapienza University of Rome, Rome (A.M.I.) - all in Italy; the Division of Endocrinology and Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester (I.B.), and the Departments of Pediatrics and Experimental and Clinical Pharmacology, Divisions of Endocrinology and Genetics and Metabolism, University of Minnesota Medical School, Minneapolis (K.S.) - both in Minnesota; the Division of Pediatric Endocrinology, Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine, Pittsburgh (S.F.W.); the Department of Endocrinology, Diabetology, and Nutrition, Endo-ERN Center for Rare Endocrine Conditions, Centre Hospitalier Universitaire d'Angers and Laboratoire Physiopathologie Cardiovasculaire et Mitochondriale, Université d'Angers, Angers, France (P.R.); the Departments of Endocrinology and Diabetes, University College London Hospital, London (U.S.); the Division of Endocrinology and Metabolism, Department of Medicine III, Medical University of Vienna, Vienna (F.W.K.); the Department of Molecular Medicine and Surgery, Karolinska Institutet, and the Department of Endocrinology, Karolinska University Hospital, Stockholm (H.F.); the National Institutes of Health Clinical Center and Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD (D.P.M.); the Department of Endocrinology, Internal Medicine IV, Ludwig Maximilians Universität München, Munich, Germany (N.R.); Gordon Cutler Consultancy, Deltaville, VA (G.B.C.); and Neurocrine Biosciences, San Diego, CA (J.S., E.R., V.H.L., J.L.C., R.H.F.).
Kyriakie SarafoglouFrom the Departments of Pharmacology and Internal Medicine, Division of Metabolism, Endocrinology, and Diabetes, University of Michigan Medical School, and the Endocrinology and Metabolism Section, Medicine Service, Lieutenant Colonel Charles S. Kettles Veterans Affairs Medical Center - both in Ann Arbor (R.J.A.); the Division of Endocrinology and Metabolism, University of Texas Southwestern Medical Center, Dallas (O.H.); Dipartimento di Medicina Clinica e Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologia e Nutrizione, Università Federico II di Napoli, Naples (R.P.), the Department of Pediatrics, Endocrine Unit, IRCCS San Raffaele Scientific Institute, Endo-ERN Center for Rare Endocrine Conditions, Milan (G.R.), and the Department of Experimental Medicine, Sapienza University of Rome, Rome (A.M.I.) - all in Italy; the Division of Endocrinology and Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester (I.B.), and the Departments of Pediatrics and Experimental and Clinical Pharmacology, Divisions of Endocrinology and Genetics and Metabolism, University of Minnesota Medical School, Minneapolis (K.S.) - both in Minnesota; the Division of Pediatric Endocrinology, Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine, Pittsburgh (S.F.W.); the Department of Endocrinology, Diabetology, and Nutrition, Endo-ERN Center for Rare Endocrine Conditions, Centre Hospitalier Universitaire d'Angers and Laboratoire Physiopathologie Cardiovasculaire et Mitochondriale, Université d'Angers, Angers, France (P.R.); the Departments of Endocrinology and Diabetes, University College London Hospital, London (U.S.); the Division of Endocrinology and Metabolism, Department of Medicine III, Medical University of Vienna, Vienna (F.W.K.); the Department of Molecular Medicine and Surgery, Karolinska Institutet, and the Department of Endocrinology, Karolinska University Hospital, Stockholm (H.F.); the National Institutes of Health Clinical Center and Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD (D.P.M.); the Department of Endocrinology, Internal Medicine IV, Ludwig Maximilians Universität München, Munich, Germany (N.R.); Gordon Cutler Consultancy, Deltaville, VA (G.B.C.); and Neurocrine Biosciences, San Diego, CA (J.S., E.R., V.H.L., J.L.C., R.H.F.).
Gordon B CutlerFrom the Departments of Pharmacology and Internal Medicine, Division of Metabolism, Endocrinology, and Diabetes, University of Michigan Medical School, and the Endocrinology and Metabolism Section, Medicine Service, Lieutenant Colonel Charles S. Kettles Veterans Affairs Medical Center - both in Ann Arbor (R.J.A.); the Division of Endocrinology and Metabolism, University of Texas Southwestern Medical Center, Dallas (O.H.); Dipartimento di Medicina Clinica e Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologia e Nutrizione, Università Federico II di Napoli, Naples (R.P.), the Department of Pediatrics, Endocrine Unit, IRCCS San Raffaele Scientific Institute, Endo-ERN Center for Rare Endocrine Conditions, Milan (G.R.), and the Department of Experimental Medicine, Sapienza University of Rome, Rome (A.M.I.) - all in Italy; the Division of Endocrinology and Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester (I.B.), and the Departments of Pediatrics and Experimental and Clinical Pharmacology, Divisions of Endocrinology and Genetics and Metabolism, University of Minnesota Medical School, Minneapolis (K.S.) - both in Minnesota; the Division of Pediatric Endocrinology, Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine, Pittsburgh (S.F.W.); the Department of Endocrinology, Diabetology, and Nutrition, Endo-ERN Center for Rare Endocrine Conditions, Centre Hospitalier Universitaire d'Angers and Laboratoire Physiopathologie Cardiovasculaire et Mitochondriale, Université d'Angers, Angers, France (P.R.); the Departments of Endocrinology and Diabetes, University College London Hospital, London (U.S.); the Division of Endocrinology and Metabolism, Department of Medicine III, Medical University of Vienna, Vienna (F.W.K.); the Department of Molecular Medicine and Surgery, Karolinska Institutet, and the Department of Endocrinology, Karolinska University Hospital, Stockholm (H.F.); the National Institutes of Health Clinical Center and Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD (D.P.M.); the Department of Endocrinology, Internal Medicine IV, Ludwig Maximilians Universität München, Munich, Germany (N.R.); Gordon Cutler Consultancy, Deltaville, VA (G.B.C.); and Neurocrine Biosciences, San Diego, CA (J.S., E.R., V.H.L., J.L.C., R.H.F.).
Julia SturgeonFrom the Departments of Pharmacology and Internal Medicine, Division of Metabolism, Endocrinology, and Diabetes, University of Michigan Medical School, and the Endocrinology and Metabolism Section, Medicine Service, Lieutenant Colonel Charles S. Kettles Veterans Affairs Medical Center - both in Ann Arbor (R.J.A.); the Division of Endocrinology and Metabolism, University of Texas Southwestern Medical Center, Dallas (O.H.); Dipartimento di Medicina Clinica e Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologia e Nutrizione, Università Federico II di Napoli, Naples (R.P.), the Department of Pediatrics, Endocrine Unit, IRCCS San Raffaele Scientific Institute, Endo-ERN Center for Rare Endocrine Conditions, Milan (G.R.), and the Department of Experimental Medicine, Sapienza University of Rome, Rome (A.M.I.) - all in Italy; the Division of Endocrinology and Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester (I.B.), and the Departments of Pediatrics and Experimental and Clinical Pharmacology, Divisions of Endocrinology and Genetics and Metabolism, University of Minnesota Medical School, Minneapolis (K.S.) - both in Minnesota; the Division of Pediatric Endocrinology, Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine, Pittsburgh (S.F.W.); the Department of Endocrinology, Diabetology, and Nutrition, Endo-ERN Center for Rare Endocrine Conditions, Centre Hospitalier Universitaire d'Angers and Laboratoire Physiopathologie Cardiovasculaire et Mitochondriale, Université d'Angers, Angers, France (P.R.); the Departments of Endocrinology and Diabetes, University College London Hospital, London (U.S.); the Division of Endocrinology and Metabolism, Department of Medicine III, Medical University of Vienna, Vienna (F.W.K.); the Department of Molecular Medicine and Surgery, Karolinska Institutet, and the Department of Endocrinology, Karolinska University Hospital, Stockholm (H.F.); the National Institutes of Health Clinical Center and Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD (D.P.M.); the Department of Endocrinology, Internal Medicine IV, Ludwig Maximilians Universität München, Munich, Germany (N.R.); Gordon Cutler Consultancy, Deltaville, VA (G.B.C.); and Neurocrine Biosciences, San Diego, CA (J.S., E.R., V.H.L., J.L.C., R.H.F.).
Eiry RobertsFrom the Departments of Pharmacology and Internal Medicine, Division of Metabolism, Endocrinology, and Diabetes, University of Michigan Medical School, and the Endocrinology and Metabolism Section, Medicine Service, Lieutenant Colonel Charles S. Kettles Veterans Affairs Medical Center - both in Ann Arbor (R.J.A.); the Division of Endocrinology and Metabolism, University of Texas Southwestern Medical Center, Dallas (O.H.); Dipartimento di Medicina Clinica e Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologia e Nutrizione, Università Federico II di Napoli, Naples (R.P.), the Department of Pediatrics, Endocrine Unit, IRCCS San Raffaele Scientific Institute, Endo-ERN Center for Rare Endocrine Conditions, Milan (G.R.), and the Department of Experimental Medicine, Sapienza University of Rome, Rome (A.M.I.) - all in Italy; the Division of Endocrinology and Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester (I.B.), and the Departments of Pediatrics and Experimental and Clinical Pharmacology, Divisions of Endocrinology and Genetics and Metabolism, University of Minnesota Medical School, Minneapolis (K.S.) - both in Minnesota; the Division of Pediatric Endocrinology, Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine, Pittsburgh (S.F.W.); the Department of Endocrinology, Diabetology, and Nutrition, Endo-ERN Center for Rare Endocrine Conditions, Centre Hospitalier Universitaire d'Angers and Laboratoire Physiopathologie Cardiovasculaire et Mitochondriale, Université d'Angers, Angers, France (P.R.); the Departments of Endocrinology and Diabetes, University College London Hospital, London (U.S.); the Division of Endocrinology and Metabolism, Department of Medicine III, Medical University of Vienna, Vienna (F.W.K.); the Department of Molecular Medicine and Surgery, Karolinska Institutet, and the Department of Endocrinology, Karolinska University Hospital, Stockholm (H.F.); the National Institutes of Health Clinical Center and Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD (D.P.M.); the Department of Endocrinology, Internal Medicine IV, Ludwig Maximilians Universität München, Munich, Germany (N.R.); Gordon Cutler Consultancy, Deltaville, VA (G.B.C.); and Neurocrine Biosciences, San Diego, CA (J.S., E.R., V.H.L., J.L.C., R.H.F.).
Vivian H LinFrom the Departments of Pharmacology and Internal Medicine, Division of Metabolism, Endocrinology, and Diabetes, University of Michigan Medical School, and the Endocrinology and Metabolism Section, Medicine Service, Lieutenant Colonel Charles S. Kettles Veterans Affairs Medical Center - both in Ann Arbor (R.J.A.); the Division of Endocrinology and Metabolism, University of Texas Southwestern Medical Center, Dallas (O.H.); Dipartimento di Medicina Clinica e Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologia e Nutrizione, Università Federico II di Napoli, Naples (R.P.), the Department of Pediatrics, Endocrine Unit, IRCCS San Raffaele Scientific Institute, Endo-ERN Center for Rare Endocrine Conditions, Milan (G.R.), and the Department of Experimental Medicine, Sapienza University of Rome, Rome (A.M.I.) - all in Italy; the Division of Endocrinology and Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester (I.B.), and the Departments of Pediatrics and Experimental and Clinical Pharmacology, Divisions of Endocrinology and Genetics and Metabolism, University of Minnesota Medical School, Minneapolis (K.S.) - both in Minnesota; the Division of Pediatric Endocrinology, Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine, Pittsburgh (S.F.W.); the Department of Endocrinology, Diabetology, and Nutrition, Endo-ERN Center for Rare Endocrine Conditions, Centre Hospitalier Universitaire d'Angers and Laboratoire Physiopathologie Cardiovasculaire et Mitochondriale, Université d'Angers, Angers, France (P.R.); the Departments of Endocrinology and Diabetes, University College London Hospital, London (U.S.); the Division of Endocrinology and Metabolism, Department of Medicine III, Medical University of Vienna, Vienna (F.W.K.); the Department of Molecular Medicine and Surgery, Karolinska Institutet, and the Department of Endocrinology, Karolinska University Hospital, Stockholm (H.F.); the National Institutes of Health Clinical Center and Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD (D.P.M.); the Department of Endocrinology, Internal Medicine IV, Ludwig Maximilians Universität München, Munich, Germany (N.R.); Gordon Cutler Consultancy, Deltaville, VA (G.B.C.); and Neurocrine Biosciences, San Diego, CA (J.S., E.R., V.H.L., J.L.C., R.H.F.).
Jean L ChanFrom the Departments of Pharmacology and Internal Medicine, Division of Metabolism, Endocrinology, and Diabetes, University of Michigan Medical School, and the Endocrinology and Metabolism Section, Medicine Service, Lieutenant Colonel Charles S. Kettles Veterans Affairs Medical Center - both in Ann Arbor (R.J.A.); the Division of Endocrinology and Metabolism, University of Texas Southwestern Medical Center, Dallas (O.H.); Dipartimento di Medicina Clinica e Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologia e Nutrizione, Università Federico II di Napoli, Naples (R.P.), the Department of Pediatrics, Endocrine Unit, IRCCS San Raffaele Scientific Institute, Endo-ERN Center for Rare Endocrine Conditions, Milan (G.R.), and the Department of Experimental Medicine, Sapienza University of Rome, Rome (A.M.I.) - all in Italy; the Division of Endocrinology and Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester (I.B.), and the Departments of Pediatrics and Experimental and Clinical Pharmacology, Divisions of Endocrinology and Genetics and Metabolism, University of Minnesota Medical School, Minneapolis (K.S.) - both in Minnesota; the Division of Pediatric Endocrinology, Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine, Pittsburgh (S.F.W.); the Department of Endocrinology, Diabetology, and Nutrition, Endo-ERN Center for Rare Endocrine Conditions, Centre Hospitalier Universitaire d'Angers and Laboratoire Physiopathologie Cardiovasculaire et Mitochondriale, Université d'Angers, Angers, France (P.R.); the Departments of Endocrinology and Diabetes, University College London Hospital, London (U.S.); the Division of Endocrinology and Metabolism, Department of Medicine III, Medical University of Vienna, Vienna (F.W.K.); the Department of Molecular Medicine and Surgery, Karolinska Institutet, and the Department of Endocrinology, Karolinska University Hospital, Stockholm (H.F.); the National Institutes of Health Clinical Center and Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD (D.P.M.); the Department of Endocrinology, Internal Medicine IV, Ludwig Maximilians Universität München, Munich, Germany (N.R.); Gordon Cutler Consultancy, Deltaville, VA (G.B.C.); and Neurocrine Biosciences, San Diego, CA (J.S., E.R., V.H.L., J.L.C., R.H.F.).
Robert H FarberFrom the Departments of Pharmacology and Internal Medicine, Division of Metabolism, Endocrinology, and Diabetes, University of Michigan Medical School, and the Endocrinology and Metabolism Section, Medicine Service, Lieutenant Colonel Charles S. Kettles Veterans Affairs Medical Center - both in Ann Arbor (R.J.A.); the Division of Endocrinology and Metabolism, University of Texas Southwestern Medical Center, Dallas (O.H.); Dipartimento di Medicina Clinica e Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologia e Nutrizione, Università Federico II di Napoli, Naples (R.P.), the Department of Pediatrics, Endocrine Unit, IRCCS San Raffaele Scientific Institute, Endo-ERN Center for Rare Endocrine Conditions, Milan (G.R.), and the Department of Experimental Medicine, Sapienza University of Rome, Rome (A.M.I.) - all in Italy; the Division of Endocrinology and Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester (I.B.), and the Departments of Pediatrics and Experimental and Clinical Pharmacology, Divisions of Endocrinology and Genetics and Metabolism, University of Minnesota Medical School, Minneapolis (K.S.) - both in Minnesota; the Division of Pediatric Endocrinology, Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine, Pittsburgh (S.F.W.); the Department of Endocrinology, Diabetology, and Nutrition, Endo-ERN Center for Rare Endocrine Conditions, Centre Hospitalier Universitaire d'Angers and Laboratoire Physiopathologie Cardiovasculaire et Mitochondriale, Université d'Angers, Angers, France (P.R.); the Departments of Endocrinology and Diabetes, University College London Hospital, London (U.S.); the Division of Endocrinology and Metabolism, Department of Medicine III, Medical University of Vienna, Vienna (F.W.K.); the Department of Molecular Medicine and Surgery, Karolinska Institutet, and the Department of Endocrinology, Karolinska University Hospital, Stockholm (H.F.); the National Institutes of Health Clinical Center and Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD (D.P.M.); the Department of Endocrinology, Internal Medicine IV, Ludwig Maximilians Universität München, Munich, Germany (N.R.); Gordon Cutler Consultancy, Deltaville, VA (G.B.C.); and Neurocrine Biosciences, San Diego, CA (J.S., E.R., V.H.L., J.L.C., R.H.F.).
CAHtalyst Adult Trial Investigators

Funding

TRAINING PROGRAM IN ENDOCRINOLOGY AND METABOLISM.T32DK007245 · UNIVERSITY OF MICHIGAN AT ANN ARBOR · 1986 to 2005
$1.4M
Study of a corticotropin releasing factor-1 receptor antagonist for the treatment of congenital adrenal hyperplasiaZIACL090093 · CLINICAL CENTER · 2025 to 2025
$0k
NIDDK NIH HHS T32 DK007245
6 · The paper itself

Abstract

backgroundAdrenal insufficiency in patients with classic 21-hydroxylase deficiency congenital adrenal hyperplasia (CAH) is treated with glucocorticoid replacement therapy. Control of adrenal-derived androgen excess usually requires supraphysiologic glucocorticoid dosing, which predisposes patients to glucocorticoid-related complications. Crinecerfont, an oral corticotropin-releasing factor type 1 receptor antagonist, lowered androstenedione levels in phase 2 trials involving patients with CAH.

methodsIn this phase 3 trial, we randomly assigned adults with CAH in a 2:1 ratio to receive crinecerfont or placebo for 24 weeks. Glucocorticoid treatment was maintained at a stable level for 4 weeks to evaluate androstenedione values, followed by glucocorticoid dose reduction and optimization over 20 weeks to achieve the lowest glucocorticoid dose that maintained androstenedione control (≤120% of the baseline value or within the reference range). The primary efficacy end point was the percent change in the daily glucocorticoid dose from baseline to week 24 with maintenance of androstenedione control.

resultsAll 182 patients who underwent randomization (122 to the crinecerfont group and 60 to the placebo group) were included in the 24-week analysis, with imputation of missing values; 176 patients (97%) remained in the trial at week 24. The mean glucocorticoid dose at baseline was 17.6 mg per square meter of body-surface area per day of hydrocortisone equivalents; the mean androstenedione level was elevated at 620 ng per deciliter. At week 24, the change in the glucocorticoid dose (with androstenedione control) was -27.3% in the crinecerfont group and -10.3% in the placebo group (least-squares mean difference, -17.0 percentage points; P<0.001). A physiologic glucocorticoid dose (with androstenedione control) was reported in 63% of the patients in the crinecerfont group and in 18% in the placebo group (P<0.001). At week 4, androstenedione levels decreased with crinecerfont (-299 ng per deciliter) but increased with placebo (45.5 ng per deciliter) (least-squares mean difference, -345 ng per deciliter; P<0.001). Fatigue and headache were the most common adverse events in the two trial groups.

conclusionsAmong patients with CAH, the use of crinecerfont resulted in a greater decrease from baseline in the mean daily glucocorticoid dose, including a reduction to the physiologic range, than placebo following evaluation of adrenal androgen levels. (Funded by Neurocrine Biosciences; CAHtalyst ClinicalTrials.gov number, NCT04490915.).

Indexed as

Adrenal Hyperplasia, CongenitalAminesAndrostenedioneThiazolesAdultCRF Receptor, Type 1Dose-Response Relationship, DrugDouble-Blind MethodDrug Therapy, CombinationFatigueFemaleGlucocorticoidsHeadacheHumansHydrocortisoneMaleAminesAndrostenedioneCRF Receptor, Type 1crinecerfontGlucocorticoidsHydrocortisoneReceptors, Corticotropin-Releasing HormoneThiazoles

Identifiers

PMID38828955
PMCPMC11309900

What Socratic holds

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.