Evidence map›Paper›PMID 29987133›Full record

ArticleNeurology2018

Clinical trial design for cutaneous neurofibromas.

Ashley Cannon, Kurt Jarnagin, Bruce Korf, Brigitte C Widemann, Denise Casey, Hon-Sum Ko, Jaishri O Blakeley, Sharad K Verma, Dominique C Pichard

Open access · hybridAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Neurology, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
1.0field-weighted citation impact, top 24% of its field
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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

3 citing papers in PubMed, 15 citations in OpenAlex.

  1. Article
  2. Article
  3. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

9 authors at 3 institutions in 1 country.

Ashley CannonFrom the Department of Genetics (A.C., B.K.), University of Alabama at Birmingham; BioPharm Tech (K.J.), San Mateo, CA; Pediatric Oncology Branch (B.C.W.) and Dermatology Branch, Center for Cancer Research (D.C.P.), National Cancer Institute, NIH, Bethesda; Division of Oncology Products (D.C.) and Division of Dermatology and Dental Products (H.-S.K.), Food and Drug Administration, Silver Spring; and Department of Neurology (J.O.B., S.K.V.), The Neurofibromatosis Therapeutic Acceleration Program, The Johns Hopkins University School of Medicine, Baltimore, MD.
Kurt JarnaginFrom the Department of Genetics (A.C., B.K.), University of Alabama at Birmingham; BioPharm Tech (K.J.), San Mateo, CA; Pediatric Oncology Branch (B.C.W.) and Dermatology Branch, Center for Cancer Research (D.C.P.), National Cancer Institute, NIH, Bethesda; Division of Oncology Products (D.C.) and Division of Dermatology and Dental Products (H.-S.K.), Food and Drug Administration, Silver Spring; and Department of Neurology (J.O.B., S.K.V.), The Neurofibromatosis Therapeutic Acceleration Program, The Johns Hopkins University School of Medicine, Baltimore, MD.
Bruce KorfFrom the Department of Genetics (A.C., B.K.), University of Alabama at Birmingham; BioPharm Tech (K.J.), San Mateo, CA; Pediatric Oncology Branch (B.C.W.) and Dermatology Branch, Center for Cancer Research (D.C.P.), National Cancer Institute, NIH, Bethesda; Division of Oncology Products (D.C.) and Division of Dermatology and Dental Products (H.-S.K.), Food and Drug Administration, Silver Spring; and Department of Neurology (J.O.B., S.K.V.), The Neurofibromatosis Therapeutic Acceleration Program, The Johns Hopkins University School of Medicine, Baltimore, MD.
Brigitte C WidemannFrom the Department of Genetics (A.C., B.K.), University of Alabama at Birmingham; BioPharm Tech (K.J.), San Mateo, CA; Pediatric Oncology Branch (B.C.W.) and Dermatology Branch, Center for Cancer Research (D.C.P.), National Cancer Institute, NIH, Bethesda; Division of Oncology Products (D.C.) and Division of Dermatology and Dental Products (H.-S.K.), Food and Drug Administration, Silver Spring; and Department of Neurology (J.O.B., S.K.V.), The Neurofibromatosis Therapeutic Acceleration Program, The Johns Hopkins University School of Medicine, Baltimore, MD.
Denise CaseyFrom the Department of Genetics (A.C., B.K.), University of Alabama at Birmingham; BioPharm Tech (K.J.), San Mateo, CA; Pediatric Oncology Branch (B.C.W.) and Dermatology Branch, Center for Cancer Research (D.C.P.), National Cancer Institute, NIH, Bethesda; Division of Oncology Products (D.C.) and Division of Dermatology and Dental Products (H.-S.K.), Food and Drug Administration, Silver Spring; and Department of Neurology (J.O.B., S.K.V.), The Neurofibromatosis Therapeutic Acceleration Program, The Johns Hopkins University School of Medicine, Baltimore, MD.
Hon-Sum KoFrom the Department of Genetics (A.C., B.K.), University of Alabama at Birmingham; BioPharm Tech (K.J.), San Mateo, CA; Pediatric Oncology Branch (B.C.W.) and Dermatology Branch, Center for Cancer Research (D.C.P.), National Cancer Institute, NIH, Bethesda; Division of Oncology Products (D.C.) and Division of Dermatology and Dental Products (H.-S.K.), Food and Drug Administration, Silver Spring; and Department of Neurology (J.O.B., S.K.V.), The Neurofibromatosis Therapeutic Acceleration Program, The Johns Hopkins University School of Medicine, Baltimore, MD.
Jaishri O BlakeleyFrom the Department of Genetics (A.C., B.K.), University of Alabama at Birmingham; BioPharm Tech (K.J.), San Mateo, CA; Pediatric Oncology Branch (B.C.W.) and Dermatology Branch, Center for Cancer Research (D.C.P.), National Cancer Institute, NIH, Bethesda; Division of Oncology Products (D.C.) and Division of Dermatology and Dental Products (H.-S.K.), Food and Drug Administration, Silver Spring; and Department of Neurology (J.O.B., S.K.V.), The Neurofibromatosis Therapeutic Acceleration Program, The Johns Hopkins University School of Medicine, Baltimore, MD.
Sharad K VermaFrom the Department of Genetics (A.C., B.K.), University of Alabama at Birmingham; BioPharm Tech (K.J.), San Mateo, CA; Pediatric Oncology Branch (B.C.W.) and Dermatology Branch, Center for Cancer Research (D.C.P.), National Cancer Institute, NIH, Bethesda; Division of Oncology Products (D.C.) and Division of Dermatology and Dental Products (H.-S.K.), Food and Drug Administration, Silver Spring; and Department of Neurology (J.O.B., S.K.V.), The Neurofibromatosis Therapeutic Acceleration Program, The Johns Hopkins University School of Medicine, Baltimore, MD.
Dominique C PichardFrom the Department of Genetics (A.C., B.K.), University of Alabama at Birmingham; BioPharm Tech (K.J.), San Mateo, CA; Pediatric Oncology Branch (B.C.W.) and Dermatology Branch, Center for Cancer Research (D.C.P.), National Cancer Institute, NIH, Bethesda; Division of Oncology Products (D.C.) and Division of Dermatology and Dental Products (H.-S.K.), Food and Drug Administration, Silver Spring; and Department of Neurology (J.O.B., S.K.V.), The Neurofibromatosis Therapeutic Acceleration Program, The Johns Hopkins University School of Medicine, Baltimore, MD. picharddc@nih.gov.
Johns Hopkins University · USUnited States Food and Drug Administration · USUniversity of Alabama at Birmingham · US

Funding

Therapy for NF1-Related Tumors and other Genetic Tumor Predisposition SyndromesZIABC010801 · NCI · DIVISION OF BASIC SCIENCES - NCI · PI WIDEMANN, BRIGITTE · 2009 to 2020
$13.8M
6 · The paper itself

Abstract

objectiveSeveral clinical trials targeting cutaneous neurofibromas (cNF) have been conducted; however, none has resulted in meaningful changes to care. The Clinical Trial Design and Development subgroup's goals were to (1) define key considerations in the design of clinical trials for cNF, (2) summarize existing data in relation to these considerations, and (3) provide consensus recommendations about key elements of trial design to accelerate the clinical development of therapies for cNF.

methodsThe subgroup, with experts from genetics, dermatology, neurology, oncology, and basic science, spanning academia, government research, and regulatory programs, and industry, reviewed published and unpublished data on clinical trials for cNF and other diseases in the skin. Discussions of these data resulted in formulation of a list of priority issues to address in order to develop efficient and effective clinical trials for cNF.

resultsThe subgroup identified 2 natural history studies of cNF, 4 priority outcome measures, and 6 patient-reported outcome tools for potential use in efficacy trials of cNF. Time to initiate intervention, patient eligibility, mechanism of action, route of administration, safety monitoring, and regulatory agency interactions were identified as key factors to consider when designing clinical trials for cNF.

conclusionsAlignment on endpoints and methods for the measurement and quantification of cNF represent a priority for therapeutic development for cNF. Advances in technological methods and outcome tools utilized in other skin diseases may be applicable to cNF studies. Patient age is an important factor guiding trial design and clinical development path.

Indexed as

Research DesignClinical Trials as TopicHumansNeurofibromaOutcome Assessment, Health CarePatient Reported Outcome MeasuresSkin Neoplasms

Identifiers

PMID29987133
PMCPMC9703339
OpenAlexW2865847389

What Socratic holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

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.