Duchenne Muscular Dystrophy (DMD) Clinical Trial
Official title:
A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The study consists of 3 periods: a multiple-ascending dose (MAD) / placebo-controlled period (24 weeks), an open-label period (24 weeks) and a long-term extension period (96 weeks).
n/a
Status | Clinical Trial | Phase | |
---|---|---|---|
Terminated |
NCT02814019 -
A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids
|
Phase 3 | |
Completed |
NCT01478022 -
To Compare the Pharmacokinetics Profiles of ISO 20, IBU 200 and IBU Plus ISO Combinations 200 + 20
|
Phase 1 | |
Completed |
NCT00654784 -
Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy
|
Phase 2 | |
Recruiting |
NCT02614820 -
The Safety, Efficacy and Tolerability of Remote Ischemic Preconditioning as a Therapy to DMD
|
N/A | |
Withdrawn |
NCT02036463 -
A Trial of Chronotherapy of Corticosteroids in Duchenne Muscular Dystrophy
|
Phase 2 | |
Completed |
NCT02255552 -
Study of Eteplirsen in DMD Patients
|
Phase 3 | |
Completed |
NCT01982695 -
Cardiomyopathy in DMD: Lisinopril vs. Losartan
|
N/A | |
Recruiting |
NCT05516745 -
E-monitoring of PULMonary Function in Patients With Duchenne Muscular Dystrophy at Home"
|
N/A | |
Completed |
NCT02420379 -
Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
|
Phase 2 | |
Active, not recruiting |
NCT05135663 -
Extension Study of NS-089/NCNP-02 in DMD
|
Phase 2 | |
Completed |
NCT01380964 -
Research of Biomarkers in Duchenne Muscular Dystrophy Patients
|
N/A | |
Completed |
NCT05990608 -
Peabody Developmental Motor Scaling In Children With Period Duchenne Muscular Dystrophy
|
||
Recruiting |
NCT04322357 -
Twice Weekly Steroids and Exercise as Therapy for DMD
|
Phase 2 | |
Not yet recruiting |
NCT06392724 -
A Study to Evaluate the Safety and Tolerability of GEN6050X in Duchenne Muscular Dystrophy.
|
Early Phase 1 | |
Completed |
NCT01540409 -
Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy
|
Phase 2 | |
Terminated |
NCT04184882 -
A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD)
|
Phase 1 | |
Terminated |
NCT04254172 -
A Low Interventional Study to Monitor Activity Using Wearable Sensors in Duchenne Muscular Dystrophy
|
||
Completed |
NCT01761292 -
A Study to Assess Safety/Tolerability, pk, Effects on Histology, Clinical Parameters of Givinostat in Children With DMD
|
Phase 1/Phase 2 |