ArticleJournal of developmental and behavioral pediatrics : JDBP2013
Outcome measures for clinical trials in fragile X syndrome.
Article in Journal of developmental and behavioral pediatrics : JDBP, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03196323 (Development of a Behavioral Outcome Measure for Rett Syndrome), which is not on this map. Cited by 90 papers, 3 of them syntheses that pooled 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.
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.
Development of a Behavioral Outcome Measure for Rett Syndrome (RettBe)
Who cites it
90 citing papers in PubMed, 3 syntheses or guidelines pooled it, 159 citations in OpenAlex.
- Is trofinetide a future treatment for Rett syndrome? A comprehensive systematic review and meta-analysis of randomized controlled trials.BMC medicine · 2024Pooled it
- Safety and efficacy of trofinetide in Rett syndrome: a systematic review and meta-analysis of randomized controlled trials.BMC pediatrics · 2024Pooled it
- Placebo Responses in Genetically Determined Intellectual Disability: A Meta-Analysis.PloS one · 2015Pooled it
- Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study.Nature medicine · 2023Trial
- Effects of mavoglurant on visual attention and pupil reactivity while viewing photographs of faces in Fragile X Syndrome.PloS one · 2019Trial
- Effect of the mGluR5-NAM Basimglurant on Behavior in Adolescents and Adults with Fragile X Syndrome in a Randomized, Double-Blind, Placebo-Controlled Trial: FragXis Phase 2 Results.Neuropsychopharmacology : official publication of the American College of Neuropsychopharmacology · 2018Trial
- Distance delivery of a spoken language intervention for school-aged and adolescent boys with fragile X syndrome.Developmental neurorehabilitation · 2018Trial
- Clinical Trial Designs for Rare Disorders: A Scoping Review of the Effectiveness of Pharmacologic Interventions in Fragile X Syndrome.Neurology. Genetics · 2026Review
- From Discovery to Innovative Translational Approaches in 80 Years of Fragile X Syndrome Research.Biomedicines · 2025Review
- The Relationships, Employment, Autonomy, and Life Satisfaction (REALS) Measures for Autistic Adults and Adults With Other Intellectual and Developmental Disabilities: Psychometric Testing of the Self-Report and Proxy Versions.Autism research : official journal of the International Society for Autism Research · 2025Article
- Psychometric Assessment of the Rett Syndrome Caregiver Assessment of Symptom Severity (RCASS).Journal of autism and developmental disorders · 2025Article
- Developing a Measure of Key Adult Outcomes in Adults with Developmental Disabilities: Conceptual Model and Item Generation of the REALS (Relationships, Employment, Autonomy, and Life Satisfaction).Journal of autism and developmental disorders · 2025Article
- Assessing Communication Impairments in a Rare Neurodevelopmental Disorder: TheNeurology. Clinical practice · 2025Article
- Sensory Symptoms and Signs of Hyperarousal in Individuals with Fragile X Syndrome: Findings from the FORWARD Registry and Database Multisite Study.Journal of autism and developmental disorders · 2024Article
- Adult Inception of Ketogenic Diet Therapy Increases Sleep during the Dark Cycle in C57BL/6J Wild Type and Fragile X Mice.International journal of molecular sciences · 2024Article
- Article
- Ketogenic Diet Affects Sleep Architecture in C57BL/6J Wild Type and Fragile X Mice.International journal of molecular sciences · 2023Article
- Antisense oligonucleotide rescue of CGG expansion-dependentProceedings of the National Academy of Sciences of the United States of America · 2023Article
- Expressive language sampling and outcome measures for treatment trials in fragile X and down syndromes: composite scores and psychometric properties.Scientific reports · 2023Article
- Brief intensive social gaze training reorganizes functional brain connectivity in boys with fragile X syndrome.Cerebral cortex (New York, N.Y. : 1991) · 2023Article
30 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors at 4 institutions in 2 countries.
Funding
Abstract
objectiveProgress in basic neuroscience has led to identification of molecular targets for treatment in fragile X syndrome (FXS) and other neurodevelopmental disorders; however, there is a gap in translation to targeted therapies in humans. One major obstacle to the demonstration of efficacy in human trials has been the lack of generally accepted endpoints to assess improvement in function in individuals with FXS. To address this problem, the National Institutes of Health convened a meeting of leading scientists and clinicians with the goal of identifying and standardizing outcome measures for use as potential endpoints in clinical trials in FXS.
methodsParticipants in the meeting included FXS experts, experts in the design and implementation of clinical trials and measure development, and representatives from advocacy groups, industry, and federal agencies.
resultsThe group generated recommendations for optimal outcome measures in cognitive, behavioral, and biomarker/medical domains, including additional testing and validation of existing measures and development of new measures in areas of need. Although no one endpoint or set of endpoints could be identified that met all criteria as an optimal measure, recommendations are presented in this report.
conclusionThe report is expected to guide the selection of measures in clinical trials and lead to the use of a more consistent battery of measures across trials. Furthermore, this will help to direct research toward gaps in the development of validated FXS-specific outcome measures and to assist with interpretation of clinical trial data by creating templates for measurement of treatment efficacy.
Indexed as
Identifiers
What OpenQuestion holds
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 OpenQuestion graph.