Evidence map›Paper›PMID 39579284›Full record

Trial reportJournal of autism and developmental disorders2026

Parent-Reported Outcome Measures for Individuals with Fragile X Syndrome: Clinically Meaningful Change Thresholds.

Meredith A Nelson, Lauren M Schmitt, Paul S Horn, Elizabeth Berry-Kravis, David Hessl, Rebecca C Shaffer, Randall Carpenter, Dejan B Budimirovic, Paul Wang, Debra L Reisinger and 2 more

Registry-linked trialAbstract readClinical Trial, Phase III
PubMed Publisher
In one paragraph

Trial report in Journal of autism and developmental disorders, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT07654114 (Evaluation of the Safety, Tolerability, and Effectiveness of CTH120 in Adult Males With Fragile X Syndrome), which is not on this map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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.

NCT07654114 phase2recruitingnot on this map

Evaluation of the Safety, Tolerability, and Effectiveness of CTH120 in Adult Males With Fragile X Syndrome

TypeinterventionalSponsorConnecta Therapeutics, S.L.Ran2026 to 2027Enrolled30ConditionsFragile X Syndrome (FXS), Fragile X SyndromeArmsCTH120, CTH120 placebo hard capsules
3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

12 authors.

Meredith A NelsonCincinnati Children's Hospital Medical Center, Cincinnati, USA. Meredith.Nelson@cchmc.org.ORCID http://orcid.org/0000-0002-6887-9790
Lauren M SchmittCincinnati Children's Hospital Medical Center, Cincinnati, USA.
Paul S HornCincinnati Children's Hospital Medical Center, Cincinnati, USA.
Elizabeth Berry-KravisDepartment of Pediatrics, Rush University Medical Center, Chicago, USA.
David HesslMIND Institute, University of California Davis, Davis, USA.
Rebecca C ShafferCincinnati Children's Hospital Medical Center, Cincinnati, USA.
Randall CarpenterAllos Pharma, Cambridge, USA.
Dejan B BudimirovicDepartment of Psychiatry, Fragile X Clinic, Kennedy Krieger Institute, Baltimore, USA.
Paul WangClinical Research Associates, LLC, New York, USA.
Debra L ReisingerCincinnati Children's Hospital Medical Center, Cincinnati, USA.
Karen Walton-BowenClinical Research Associates, LLC, New York, USA.
Craig A EricksonCincinnati Children's Hospital Medical Center, Cincinnati, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Estimating meaningful change thresholds (MCT) on clinical outcome assessments is an important consideration when evaluating treatments. In fragile X syndrome (FXS) research, there has been no consensus on how to define MCT's on several commonly used outcome measures. The purpose of the current study was to determine clinically relevant MCT's of caregiver-rated assessments using data from a phase 3 clinical trials of arbaclofen (Berry-Kravis et al., 2017). Data were collected as a part of previous phase 3, double-blind, placebo-controlled studies of arbaclofen in individuals with FXS (Berry-Kravis et al., 2017). The two studies enrolled age groups of 5-11-years (n = 159) and 12-50-years (n = 119). The current study examines meaningful within-patient change thresholds from baseline to treatment week 8 across several measures: ABC-C

Indexed as

BaclofenFragile X SyndromeGABA-B Receptor AgonistsOutcome Assessment, Health CareParentsAdolescentAdultChildChild, PreschoolDouble-Blind MethodFemaleHumansMaleMiddle AgedPatient Reported Outcome MeasuresTreatment OutcomeBaclofenGABA-B Receptor AgonistsFragile X syndromeMeaningful change thresholdsOutcome assessments

Identifiers

PMID39579284

What Socratic holds

Textmetadata
Read underepoch 390

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.