Trial reportContemporary clinical trials2017
Developing standardized corticosteroid treatment for Duchenne muscular dystrophy.
Trial report in Contemporary clinical trials, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 40 papers, 2 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
40 citing papers in PubMed, 2 syntheses or guidelines pooled it, 65 citations in OpenAlex.
- Efficacy and safety of different doses of vamorolone in boys with Duchenne muscular dystrophy: a systematic review and network meta-analysis.Frontiers in neurology · 2024Pooled it
- A Decade of Progress in Gene Targeted Therapeutic Strategies in Duchenne Muscular Dystrophy: A Systematic Review.Frontiers in bioengineering and biotechnology · 2022Pooled it
- Trial
- Relationships betweenNeurology · 2019Trial
- Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function.Neurology · 2019Trial
- Deflazacort versus prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post HOC analysis from the ACT DMD trial.Muscle & nerve · 2018Trial
- Development and Validation of a Novel, Disease-Specific, Caregiver-Reported Outcome Measure: The Duchenne Muscular Dystrophy Caregiver Reported-Health Index (DMDCR-HI).Muscle & nerve · 2026Article
- Current and Emerging Therapeutic Strategies for the Treatment of Duchenne Muscular Dystrophy.Genes · 2026Review
- Survey of Adrenal Insufficiency Management for Duchenne muscular dystrophy in Italy.Endocrine · 2026Article
- Preferences for gene therapy in Duchenne muscular dystrophy: insights from patient and caregiver interviews and attribute development.Frontiers in pharmacology · 2025Article
- Safety and Tolerability of Wharton's Jelly-Derived Mesenchymal Stem Cells for Patients With Duchenne Muscular Dystrophy: A Phase 1 Clinical Study.Journal of clinical neurology (Seoul, Korea) · 2025Article
- Use of the experience sampling method in adolescents with Duchenne muscular dystrophy: a feasibility study.European child & adolescent psychiatry · 2024Article
- Moving Beyond the 2018 Minimum International Care Considerations for Osteoporosis Management in Duchenne Muscular Dystrophy (DMD): Meeting Report from the 3rd International Muscle-Bone Interactions Meeting 7th and 14th November 2022.Journal of neuromuscular diseases · 2024Article
- Association between neurodevelopmental impairments and motor function in Duchenne muscular dystrophy.Annals of clinical and translational neurology · 2023Article
- Genetic Neuromuscular Disorders and Health Services Access, Utilization, and Needs in Zambia.Pediatric neurology · 2023Article
- Therapeutic opportunities and clinical outcome measures in Duchenne muscular dystrophy.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2022Review
- Cardiac care of children with dystrophinopathy and females carrying DMD-gene variations.Open heart · 2022Review
- Corticosteroid use and bone health management for Duchenne muscular dystrophy in South Korea.Scientific reports · 2022Article
- Clinical and Genetic Characteristics in Young, Glucocorticoid-Naive Boys With Duchenne Muscular Dystrophy.Neurology · 2022Article
- Comparing Deflazacort and Prednisone in Duchenne Muscular Dystrophy.Journal of neuromuscular diseases · 2022Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
47 authors at 20 institutions in 5 countries.
Funding
Abstract
Despite corticosteroids being the only treatment documented to improve strength and function in boys with Duchenne muscular dystrophy (DMD) corticosteroid prescription is inconsistent and in some countries, corticosteroids are not prescribed. We are conducting a clinical trial that (1) compares the 3 most frequently prescribed corticosteroid regimes; (2) standardizes treatment of DMD complications; and (3) standardizes prevention of corticosteroid side effects. Investigators at 38 sites in 5 countries plan to recruit 300 boys aged 4-7 who are randomly assigned to one of three regimens: daily prednisone; daily deflazacort; or intermittent prednisone (10days on/10days off). Boys are followed for a minimum of 3years to assess the relative effectiveness and adverse event profiles of the different regimens. The primary outcome is a 3-dimensional variable consisting of log-transformed time to rise from the floor, forced vital capacity, and subject/parent satisfaction with treatment, each averaged over all post-baseline visits. The study protocol includes evidence- and consensus-based treatment of DMD complications and of corticosteroid side effects. This study seeks to establish a standard corticosteroid regimen for DMD. Since all new interventions for DMD are being developed as add-on therapies to corticosteroids, defining the optimum regimen is of importance for all new treatments.
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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.