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Active clinical trials for "Muscular Dystrophies"

Results 11-20 of 545

Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)

Becker Muscular Dystrophy

A study of EDG-5506 in Becker muscular dystrophy (known as CANYON) and pivotal cohort (known as GRAND CANYON). The EDG-5506-201 CANYON study was expanded to include an additional 120 adult participants in a cohort called GRAND CANYON, that is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of EDG-5506 in adults with Becker. CANYON is fully enrolled; GRAND CANYON is currently enrolling.

Recruiting12 enrollment criteria

A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular...

Duchenne Muscular Dystrophy

This Phase II study is an open-label, multiple dose study to evaluate the safety, tolerability, PK, PD, clinical efficacy, behavior and neuropsychology, and physical functioning vamorolone over a treatment period of 12 weeks in steroid-naïve boys ages 2 to <4 years, and glucocorticoid-treated and currently untreated boys ages 7 to <18 years with DMD.

Recruiting28 enrollment criteria

Phase 1/2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD)

FSHDFSHD133 more

A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Adult Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

Recruiting15 enrollment criteria

A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)

Duchenne Muscular Dystrophy

The LYNX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics and biomarkers in children with Duchenne muscular dystrophy including a randomized, double-blind, placebo-controlled part A, followed by an open-label part B.

Recruiting16 enrollment criteria

Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients

Duchenne Muscular Dystrophy

This Phase I/II of the clinical trial is to investigate whether the transplantation of normal myoblasts throughout one muscle (the extensor carpi radialis) of the patients is safe and will improve the strength of that muscle. During this Phase I/II, the patients will be transplanted with myoblasts grown from the muscle biopsy of a donor and kept frozen in liquid nitrogen. Thirty million myoblasts will be injected per cm cube in a progressively higher surface of the radialis (i.e., 3, 6 and 9 cm2). The contralateral muscle will be injected with saline to serve as a control. The strength of both muscles will be measured at 3 months post transplantation to verify whether the myoblast transplantation improved the strength of the muscle. If there is no significant strength improvement, the protocol will be terminated immediately for that patient. If there is a significant strength improvement, the patient will be maintained under immunosuppression until 6 months post transplant and his strength will be re-evaluated.

Recruiting31 enrollment criteria

Feasibility of Virtual Reality in Children With Neuromuscular Disease, Effectiveness of Virtual...

Neuromuscular DiseaseDuchenne Muscular Dystrophy3 more

Our study is a randomized controlled study and the subjects included in the study will be divided into three groups as virtual reality training, biofeedback training, and conventional rehabilitation.

Recruiting9 enrollment criteria

A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

Muscular DystrophiesMuscular Dystrophy10 more

HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy (DMD) and impaired skeletal muscle function. Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses during the first 12-months of the study. All participants will be eligible to receive 4 doses of CAP-1002 for an additional 12 months as part of an open-label extended assessment period.

Recruiting33 enrollment criteria

A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy(REACH-DMD)

Duchenne Muscular Dystrophy

The purpose of this study is to evaluate the efficacy and safety of TAS-205 in patients with Duchenne muscular dystrophy

Recruiting38 enrollment criteria

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec...

Duchenne Muscular Dystrophy

The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2.

Recruiting11 enrollment criteria

Sleep Intervention in Young Boys With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

This project will systematically plan and evaluate the implementation of the Transdiagnostic Sleep and Circadian Intervention for youth (TranS-CY). As an early stage study, investigators will focus on recruitment strategies to reach the target population and collection of preliminary data on primary and secondary effects of the TranS-CY. Weekly remote (video web conferencing) parent training sessions will allow investigators to explore adoption through parent adherence and examine whether the essential elements of the TranS-CY intervention (e.g., motivational interviewing, goal setting, problem solving, sleep routine scheduling, monitoring) can be consistently taught by clinicians and implemented by parents into the home setting.

Recruiting6 enrollment criteria
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