Trial reportMuscle & nerve2013
The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study.
Trial report in Muscle & nerve, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT07502989 (Convenient Quantification of Myopathic Change in Muscle Via Electrical Impedance Myography), which is not on this map. Cited by 175 papers, 9 of them syntheses that pooled it.
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.
Convenient Quantification of Myopathic Change in Muscle Via Electrical Impedance Myography
Who cites it
175 citing papers in PubMed, 9 syntheses or guidelines pooled it, 349 citations in OpenAlex.
- Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A Systematic Review.Paediatric drugs · 2024Pooled it
- Antioxidants to prevent respiratory decline in people with Duchenne muscular dystrophy and progressive respiratory decline.The Cochrane database of systematic reviews · 2021Pooled it
- Antioxidants to prevent respiratory decline in people with Duchenne muscular dystrophy and progressive respiratory decline.The Cochrane database of systematic reviews · 2021Pooled it
- Measuring quality of life in Duchenne muscular dystrophy: a systematic review of the content and structural validity of commonly used instruments.Health and quality of life outcomes · 2020Pooled it
- Characterizing health state utilities associated with Duchenne muscular dystrophy: a systematic review.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2020Pooled it
- Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta-analysis of disease progression rates in recent multicenter clinical trials.Muscle & nerve · 2020Pooled it
- Exon skipping for Duchenne muscular dystrophy: a systematic review and meta-analysis.Orphanet journal of rare diseases · 2018Pooled it
- Minimal clinically important difference for the 6-min walk test: literature review and application to Morquio A syndrome.Orphanet journal of rare diseases · 2017Pooled it
- A systematic review to investigate the measurement properties of goal attainment scaling, towards use in drug trials.BMC medical research methodology · 2016Pooled it
- Vamorolone for Duchenne Muscular Dystrophy: A Cross-Trial Efficacy Comparison With Classic Corticosteroids From the FOR-DMD Trial.Neurology · 2026Trial
- Long-Term Evaluation of Givinostat in Duchenne Muscular Dystrophy, and Natural History Comparisons.Annals of clinical and translational neurology · 2025Trial
- Confirmatory long-term efficacy and safety results of ataluren in patients with nmDMD from Study 041, an international, randomized, double-blind, placebo-controlled, Phase III trial.Journal of comparative effectiveness research · 2025Trial
- Effect of creatine monohydrate on motor function in children with facioscapulohumeral muscular dystrophy: A multicenter, randomized, double-blind placebo-controlled crossover trial.Pharmacotherapy · 2025Trial
- Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status.Journal of comparative effectiveness research · 2023Trial
- A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment.Journal of neuromuscular diseases · 2022Trial
- Trial
- Mexiletine in Myotonic Dystrophy Type 1: A Randomized, Double-Blind, Placebo-Controlled Trial.Neurology · 2021Trial
- Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial.JAMA neurology · 2020Trial
- Serum biomarkers associated with baseline clinical severity in young steroid-naïve Duchenne muscular dystrophy boys.Human molecular genetics · 2020Trial
- Early phase 2 trial of TAS-205 in patients with Duchenne muscular dystrophy.Annals of clinical and translational neurology · 2020Trial
115 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
13 authors at 6 institutions in 2 countries.
Funding
Abstract
introductionAn international clinical trial enrolled 174 ambulatory males ≥5 years old with nonsense mutation Duchenne muscular dystrophy (nmDMD). Pretreatment data provide insight into reliability, concurrent validity, and minimal clinically important differences (MCIDs) of the 6-minute walk test (6MWT) and other endpoints.
methodsScreening and baseline evaluations included the 6-minute walk distance (6MWD), timed function tests (TFTs), quantitative strength by myometry, the PedsQL, heart rate-determined energy expenditure index, and other exploratory endpoints.
resultsThe 6MWT proved feasible and reliable in a multicenter context. Concurrent validity with other endpoints was excellent. The MCID for 6MWD was 28.5 and 31.7 meters based on 2 statistical distribution methods.
conclusionsThe ratio of MCID to baseline mean is lower for 6MWD than for other endpoints. The 6MWD is an optimal primary endpoint for Duchenne muscular dystrophy (DMD) clinical trials that are focused therapeutically on preservation of ambulation and slowing of disease progression.
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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.