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A Phase 1/2a, Multi-center, Open-label Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of PBGENE-DMD in Participants With Duchenne Muscular Dystrophy (FUNCTION-DMD)
The purpose of this Phase 1/2a trial is to evaluate the safety, tolerability, and preliminary efficacy of PBGENE-DMD in patients with DMD harboring mutations a…
Clinical Trial Readiness for the Dystroglycanopathies
The purpose of the study is to describe the early signs and symptoms of the dystroglycanopathies, and to gather information that will be required for future cl…
Power Forward Study: A Cross-Sector, Multisite Clinical Trial of a Powered Knee-Ankle-Foot Orthosis
The goal of this clinical trial is to evaluate the impact of using the Nomad powered KAFO in people who have had a musculoskeletal or neurological injury that …
Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds)
The primary goal of this study is to validate motor and functional outcomes and refine clinical trial strategies for pediatric-onset FSHD
CureDuchenne Link®: A Resource to Support Research Studies in Duchenne and Becker Muscular Dystrophy (DMD/BMD)
CureDuchenne link is a data hub comprised of integrated biospecimens, clinical data, and self- and/or caregiver-reported information from participants. Anyone …
Congenital Muscle Disease Patient and Proxy Reported Outcome Study
The Congenital Muscle Disease Patient and Proxy Reported Outcome Study (CMDPROS) is a longitudinal 10 year study to identify and trend care parameters, adverse…
Clenbuterol to Target DUX4 in FSHD (Target FSHD): Open Label Safety and Tolerability Study of 3 Doses of Clenbuterol
The purpose of this study is to determine if Clenbuterol is a therapeutic option for FSHD by determining the safety and tolerability of the medication at three…
A Phase 1, Double-blinded, Randomized, Dose-repeating, Placebo-controlled, Cross-over Study to Assess the Safety and Preliminary Efficacy of Allogeneic ULSC on Disease Severity in Facioscapulohumeral Muscular Dystrophy (FSHD)
The goal of this clinical trial is to learn about how an umbilical cord lining-derived stem cell product (ULSC) performs when treating Facioscapulohumeral Musc…
An Open-label Phase 1b/2 Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy
This is a Phase 1b/2 open-label study to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and clinical effects of intravenous (IV…
A Two-part Multicenter Study: a Randomized, Double-blind, Placebo-controlled Dose-escalation Safety Phase (Part 1) Followed by Double-blind, Placebo-controlled, Adaptive Phase (Part 2) Study to Evaluate the Safety and Efficacy of AB-1003 in Adult Subjects With LGMD2I/R9 Mutations in the Gene Encoding Fukutin Related Protein (FKRP)
The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular …
Anti-AAV8 Antibody Assessment Study of Males With Duchenne Muscular Dystrophy Aged 0 to <25 Years
This is an observational screening study to evaluate the prevalence of anti-adeno-associated serotype 8 (AAV8) antibodies in participants with Duchenne muscula…
Extracellular RNA Biomarkers of Duchenne Muscular Dystrophy
Current methods of measuring the response to new treatments for muscular dystrophies involve the examination of small pieces of muscle tissue called biopsies. …