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A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 (Delandistrogene Moxeparvovec) in Participants With Duchenne Muscular Dystrophy (DMD) (EMBARK)

Official Title:

A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Patients With Duchenne Muscular Dystrophy (EMBARK)

Condition/disease: Duchenne Muscular Dystrophy
Study Phase: Phase III
Brief Summary:

The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene transfer therapy at the beginning of the second year.

Interventions: Genetic: SRP-9001; Genetic: Placebo
NCT Number: NCT05096221
  • Belgium
  • Germany
  • Hong Kong
  • Italy
  • Japan
  • Spain
  • Taiwan
  • United Kingdom
  • United States
Age Limits:
4-7
years
Overall Recruitment Status: Active, Not Recruiting

A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9001 (Delandistrogene Moxeparvovec) in Participants With Duchenne Muscular Dystrophy (DMD)

Official Title:

Systemic Gene Delivery Phase I/IIa Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MHCK7.Micro-dystrophin (microDys-IV-001)

Condition/disease: Duchenne Muscular Dystrophy
Study Phase: Phase I/II
Brief Summary:

This is an open-label single-dose gene transfer therapy study evaluating the safety of SRP-9001 (delandistrogene moxeparvovec) intravenous (IV) administration in boys with DMD. This study will consist of 2 Cohorts. Cohort A will include participants ages 3 months to 3 years, and Cohort B will include participants ages 4 to 7 years old. All participants in the study will receive IV SRP-9001.

Interventions: Genetic: SRP-9001
NCT Number: NCT03375164
  • United States
Age Limits:
3-84
months
Overall Recruitment Status: Active, Not Recruiting

A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 (Delandistrogene Moxeparvovec) for Duchenne Muscular Dystrophy (DMD)

Official Title:

A Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial for Duchenne Muscular Dystrophy Using SRP-9001

Condition/disease: Duchenne Muscular Dystrophy
Study Phase: Phase II
Brief Summary:

The purpose of this study is to evaluate the safety and efficacy of exogenous gene transfer in DMD patients by measuring biological and clinical endpoints in three parts: two 48-week randomized, double-blinded, placebo-controlled periods (Part 1 and Part 2), and an open-label follow-up period (Part 3). Patients who are randomized to placebo in Part 1 will have the opportunity for treatment with SRP-9001 (delandistrogene moxeparvovec) in Part 2.

Interventions: Drug: SRP-9001; Drug: Placebo
NCT Number: NCT03769116
  • United States
Age Limits:
4-7
years
Overall Recruitment Status: Active, Not Recruiting

A Study of the Natural History of Patients With LGMD2E/R4, LGMD2D/R3, and LGMD2C/R5, ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice

Official Title:

Journey: A Global, Multicenter, Longitudinal Study of the Natural History of Subjects With Limb Girdle Muscular Dystrophy (LGMD) Type 2E (LGMD2E/R4), Type 2D (LGMD2D/R3), and Type 2C (LGMD2C/R5)

Condition/disease: Limb-girdle Muscular Dystrophy
Study Phase: N/A
Brief Summary:

This study will follow patients who are screened and confirmed with a genetic diagnosis of Limb-girdle muscular dystrophy type 2E (LGMD2E/R4), Limb-girdle muscular dystrophy type 2D (LGMD2D/R3), or Limb-girdle muscular dystrophy type 2C (LGMD2C/R5). These enrolled patients will be followed to evaluate mobility and pulmonary function for up to 3 years after enrollment. Additional patient data will be collected from the time the individual began experiencing LGMD symptoms to the present.

Interventions: Not Provided
NCT Number: NCT04475926
  • United States
Age Limits:
4+
years
Overall Recruitment Status: Recruiting

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

Official Title:

A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients With Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping

Condition/disease: Duchenne Muscular Dystrophy
Study Phase: Phase III
Brief Summary:

This study will be comprised of 2 parts: Part 1 (dose escalation) will be conducted to evaluate the safety and tolerability of 2 doses (100 milligrams/kilogram [mg/kg] and 200 mg/kg) of eteplirsen in approximately 10 participants with DMD; Part 2 (dose finding and dose comparison) will be conducted for the selection of a high dose (100 mg/kg versus 200 mg/kg) and its comparison with the 30 mg/kg dose of eteplirsen, in approximately 144 participants with genetically confirmed deletion mutations amenable to treatment by skipping exon 51.

Interventions: Drug: Eteplirsen
NCT Number: NCT03992430
  • Canada
  • Czechia
  • France
  • Greece
  • Ireland
  • Korea, Republic of,
  • New Zealand
  • Poland
  • Spain
  • Taiwan
  • Turkey
  • United Kingdom
  • United States
Age Limits:
4-13
years
Overall Recruitment Status: Recruiting

An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy

Official Title:

Long-term, Open-label Extension Study for Patients With Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen

Condition/disease: Duchenne Muscular Dystrophy
Study Phase: Phase III
Brief Summary:

The main objective of this study is to evaluate the safety and tolerability of long-term treatment with casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD).

Interventions: Drug: Casimersen; Drug: Golodirsen
NCT Number: NCT03532542
  • Belgium
  • Bulgaria
  • Canada
  • Czechia
  • France
  • Germany
  • Israel
  • Italy
  • Poland
  • Spain
  • Sweden
  • United Kingdom
  • United States
Age Limits:
7-23
years
Overall Recruitment Status: Enrolling by invitation

Gene Delivery Clinical Trial of SRP-9003 for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)

Official Title:

A Single-Center, Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9003 Administered by Systemic Infusion in Subjects With LGMD2E (β-Sarcoglycan Deficiency)

Condition/disease: Limb-Girdle Muscular Dystrophy, Type 2E
Study Phase: Phase I/II
Brief Summary:

The proposed clinical trial is the first-in-human, single-center, open-label, gene delivery study of SRP-9003 in participants with LGMD2E.

Interventions: Drug: SRP-9003
NCT Number: NCT03652259
  • United States
Age Limits:
4-15
years
Overall Recruitment Status: Active, Not Recruiting

Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD) (ESSENCE)

Official Title:

A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy

Condition/disease: Duchenne Muscular Dystrophy
Study Phase: Phase III
Brief Summary:

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

Interventions: Drug: SRP-4045; Drug: SRP-4053; Drug: Placebo
NCT Number: NCT02500381
  • Argentina
  • Australia
  • Belgium
  • Bulgaria
  • Canada
  • Czechia
  • Denmark
  • France
  • Germany
  • Greece
  • Hungary
  • Ireland
  • Israel
  • Italy
  • Mexico
  • Poland
  • Russian Federation
  • Serbia
  • Spain
  • Sweden
  • United Kingdom
  • United States
Age Limits:
6-13
years
Overall Recruitment Status: Recruiting

Two-Part Study for Dose Determination of SRP-5051 (Vesleteplirsen) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (MOMENTUM)

Official Title:

A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, Then Dose Expansion, in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

Condition/disease: Duchenne Muscular Dystrophy
Study Phase: Phase II
Brief Summary:

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 (vesleteplirsen) 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 doses selected in Part A. Participants enrolling in Part B will be those who completed Part A or Study 5051-102 and meet applicable eligibility criteria for Part B, as well as additional participants who meet applicable eligibility criteria for enrollment at the beginning of Part B.

Interventions: Drug: SRP-5051
NCT Number: NCT04004065
  • Belgium
  • Canada
  • Germany
  • Italy
  • Netherlands
  • Spain
  • United Kingdom
  • United States
Age Limits:
7-21
years
Overall Recruitment Status: Recruiting