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Found 42 clinical trials
A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)

This study will be comprised of 2 parts: Part 1 (dose escalation) will be conducted to evaluate the safety and tolerability of 2 doses (high dose level 1 and high dose level 2) of eteplirsen in approximately 10 participants with DMD; Part 2 (dose finding and dose comparison) will be …

walk test
muscular dystrophy
deletion mutation
forced vital capacity
corticosteroids
  • 11 views
  • 19 Apr, 2022
  • 6 locations
A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

This study is designed to evaluate safety, tolerability, and pharmacokinetics (PK) in male children with nmDMD aged ≥6 months to <2 years treated daily for 24 weeks with orally administered ataluren 10, 10, and 20 milligrams/kilogram (mg/kg) (morning, mid-day, and evening dose, respectively).

  • 2 views
  • 13 May, 2022
  • 1 location
Study of SRP-4045 and SRP-4053 in Participants With Duchenne Muscular Dystrophy (DMD) (ESSENCE)

The main objective of this study is to evaluate the efficacy of SRP-4045 and SRP-4053 compared to placebo in participants with DMD with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53, respectively.

muscular dystrophy
deletion mutation
forced vital capacity
corticosteroids
duchenne muscular dystrophy
  • 131 views
  • 25 Jan, 2022
  • 45 locations
Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53)

This is a phase II, open-label study where weekly doses of 80 mg/kg viltolarsen is administered intravenously over a 48-week treatment period to ambulant and non-ambulant DMD patients over the age of 8 years.

  • 0 views
  • 19 Apr, 2022
  • 6 locations
Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)

The main objective of this study is to evaluate the efficacy of Viltolarsen compared to placebo in Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping.

muscular dystrophy
duchenne muscular dystrophy
  • 315 views
  • 21 Apr, 2022
  • 32 locations
A Phase 3 Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids, in Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD)

To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids administered every two weeks in ambulatory subjects with Duchenne muscular

serum electrolyte
corticosteroids
duchenne muscular dystrophy
calcium
prednisolone
  • 2 views
  • 29 Apr, 2022
  • 45 locations
Two-Part Study for Dose Determination of SRP-5051 (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (MOMENTUM)

This study will be comprised of 2 parts: 1) Part A (Multiple Ascending Dose [MAD]) will be conducted to evaluate the safety and tolerability of SRP-5051 at MAD levels to determine doses to be administered in Part B, and 2) Part B will be conducted to further evaluate the SRP-5051 …

muscular dystrophy
deletion mutation
corticosteroids
drug test
duchenne muscular dystrophy
  • 236 views
  • 15 May, 2022
  • 9 locations
Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients

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 …

muscular dystrophy
muscle biopsy
corticosteroids
  • 85 views
  • 05 Feb, 2021
  • 2 locations
Sleep Intervention in Young Boys With Duchenne Muscular Dystrophy (DMD)

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 …

muscular dystrophy
duchenne muscular dystrophy
  • 0 views
  • 25 Mar, 2022
  • 1 location
Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy (DMD)

Duchenne muscular dystrophy (DMD) is a devastating X-linked disease which leads to loss of ambulation between ages 7 and 13, respiratory failure and cardiomyopathy (CM) at any age, and

ejection fraction
angiotensin converting enzyme
beta blockers
angiotensin
aldosterone antagonists
  • 7 views
  • 26 Apr, 2022
  • 10 locations