ArticleAnnals of clinical and translational neurology2020
Refining clinical trial inclusion criteria to optimize the standardized response mean of the CMTPedS.
Article in Annals of clinical and translational neurology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06630650 (A Single Center Prospective Natural History and Outcome Measure Validation Study of Congenital Myasthenic Syndromes), which is not on this map. Cited by 21 papers.
What it found
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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.
A Single Center Prospective Natural History and Outcome Measure Validation Study of Congenital Myasthenic Syndromes
Who cites it
21 citing papers in PubMed.
- Article
- Clinical Development of Therapies for Charcot-Marie-Tooth Disease: Recommendations for Trial Design, Endpoints, and Regulatory Pathways.Journal of the peripheral nervous system : JPNS · 2026Review
- Daily-Life, Sensor-Derived Tremor Measures Are Sensitive to Progression in Early Parkinson's Disease.Annals of neurology · 2026Article
- Axonal-demyelinating differences in functional change in pediatric Charcot-Marie-Tooth and related neuropathies.BMC neurology · 2026Article
- Impact of inactivated influenza vaccination on health-related quality of life for young children using Japanese experimental EuroQol toddler and infant populations (EQ-TIPS) v2.0.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2026Article
- Charcot-Marie-Tooth disease type 1E: clinical natural history and molecular impact of PMP22 variants.Brain : a journal of neurology · 2026Article
- Article
- Effects of Levodopa on Gait in People With Parkinson's Disease With and Without a History of Falls.Movement disorders clinical practice · 2025Article
- Measurement properties of the Comprehensive Quality-of-Life Measure for Acne (CompAQ): a cohort study at three international sites.The British journal of dermatology · 2025Article
- iHOT-12 item analysis: correlations between individual items and overall score within and across time points.Journal of hip preservation surgery · 2025Article
- Charcot-Marie-Tooth disease type 1E: Clinical Natural History and Molecular Impact ofmedRxiv : the preprint server for health sciences · 2025Article
- Review
- Nonsurgical Knee Osteoarthritis Treatments for Reducing Inflammation as Measured on MRI Scans: A Systematic Review of Randomized Controlled Trials.Orthopaedic journal of sports medicine · 2024Review
- Article
- Observational
- Clinical Characteristics of Charcot-Marie-Tooth Disease Type 4J.Neurology · 2024Article
- Comparison of the Measurement Properties of EQ-5D-5L and SF-6Dv2 in COVID-19 Patients in China.Applied health economics and health policy · 2024Article
- Cross-Cultural Adaptation and Measurement of Psychometric Properties of the Lithuanian Version of the Western Ontario Shoulder Instability Index (WOSI).Medicina (Kaunas, Lithuania) · 2024Article
- Trials for Slowly Progressive Neurogenetic Diseases Need Surrogate Endpoints.Annals of neurology · 2023Review
- Clinically Relevant Changes for Cognitive Outcomes in Preclinical and Prodromal Cognitive Stages: Implications for Clinical Alzheimer Trials.Neurology · 2022Article
Corrections and comments
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Authors and funding
19 authors.
Funding
Abstract
The CMT Pediatric Scale (CMTPedS) is a reliable, valid, and responsive clinical outcome measure of disability in children with CMT. The aim of this study was to identify the most responsive patient subset(s), based on the standardized response mean (SRM), to optimize the CMTPedS as a primary outcome measure for upcoming clinical trials. Analysis was based on a 2-year natural history data from 187 children aged 3-20 years with a range of CMT genetic subtypes. Subsets based on age (3-8 years), disability level (CMTPedS score 0-14), and CMT type (CMT1A) increased the SRM of the CMTPedS considerably. Refining the inclusion criteria in clinical trials to younger, mildly affected cases of CMT1A optimizes the responsiveness of the CMTPedS.
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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 OpenQuestion graph.