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Found 96 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
eteplirsen
forced vital capacity
corticosteroids
deletion mutation
  • 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
MABs Therapy m.3243A>G Mutation Carriers

Rationale: Mitochondrial disorders are progressive, often fatal multisystem disorders, in 20-25% of the cases caused by heteroplasmic mutations in the mitochondrial DNA (mtDNA). At this moment, there is no effective treatment known to influence the disease process or manifestation. Myogenic stem cell-based therapies complementing defective muscle cells and fibres, are …

  • 0 views
  • 07 Oct, 2021
  • 1 location
Variability of the Unique Fiber Potentials of a Spastic Muscle Treated With Botulinum Toxin (JITTOX)

The main objective is to study the variability and evolution of single-fiber jitter and fiber density (FD) electrophysiological parameters in a spastic muscle during botulinum toxin type A (BTA) treatment in hemiplegic patients after stroke, according to primary or multi-injected status. The secondary objectives of this study are: To establish …

Accepts healthy volunteers
  • 15 views
  • 26 Mar, 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.

forced vital capacity
corticosteroids
duchenne muscular dystrophy
deletion mutation
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
duchenne muscular dystrophy
corticosteroids
prednisolone
calcium
  • 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 …

duchenne muscular dystrophy
corticosteroids
drug test
deletion mutation
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 …

muscle biopsy
muscular dystrophy
corticosteroids
  • 85 views
  • 05 Feb, 2021
  • 2 locations