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

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:  Part A (Multiple Ascending Dose (MAD)) which will be conducted to evaluate the safety and tolerability of SRP-5051 at multiple ascending dose levels to determine the maximum tolerated dose (MTD); Part B (Dose Expansion) will be conducted to evaluate SRP-5051 administered at the MTD in patients who have completed Part A.

Interventions: Drug: SRP-5051
NCT Number: NCT04004065
  • Canada
  • United States
Age Limits:
7-21
years
Overall Recruitment Status: Recruiting

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

Official Title:

A 48-Week, Randomized, Double-Blind, Placebo-Controlled, Systemic, Gene-Delivery Clinical Trial for Duchenne Muscular Dystrophy Using SRP-9001 With a 96-Week Extension 

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 two parts:  a 48-week randomized, double-blinded, placebo-controlled period (Part 1), and a 96-week, double-blinded extension period (Part 2).

Interventions: Drug: SRP-9001; Drug: Placebo
NCT Number: NCT03769116
  • United States
Age Limits:
4-7
years
Overall Recruitment Status: Recruiting

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

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 will be conducted to evaluate the safety and tolerability of two doses (high dose level 1 and high dose level 2) of eteplirsen in approximately 8 patients; Part 2 will be conducted for the selection of a high dose (high dose level 1 vs high dose level 2) (dose finding phase), and its comparison with the 30 mg/kg dose of eteplirsen (dose comparison phase), in approximately 144 DMD patients with genetically confirmed deletion mutations amenable to treatment by skipping exon 51. 

Interventions: Drug: Eteplirsen
NCT Number: NCT03992430
  • Not Provided
Age Limits:
7-13
years
Overall Recruitment Status: Not Yet Recruiting

A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Patients With Duchenne Muscular Dystrophy

Official Title:

An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients With Duchenne Muscular Dystrophy Who Have Completed Study 4658-102

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

The purpose of this extension study is to evaluate the ongoing safety and tolerability of additional treatment with eteplirsen administered once weekly by intravenous (IV) infusion in male DMD patients who have successfully completed the 96-week eteplirsen study:  Study 4658-102 (NCT03218995)

Interventions: Drug: Eteplirsen
NCT Number: NCT03985878
  • Belgium
  • France
  • Italy
  • United Kingdom
Age Limits:
2-5
years
Overall Recruitment Status: Enrolling by Invitation

A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy (DMD)

Official Title:

A Phase 1 Trial to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Treatment 

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

The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of 5 escalating doses of SRP-5051 administered as a single dose to patients with DMD amenable to exon 51 skipping treatment.

Interventions: Drug: SRP-5051
NCT Number: NCT03375255
  • Canada
  • United States
Age Limits:
12+
years
Overall Recruitment Status: Completed

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
  • Italy
  • Spain
  • United Kingdom
  • United States
Age Limits:
7-23
years
Overall Recruitment Status: Enrolling by Invitation

An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051

Official Title:

An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051

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

The purpose of this extension study is to evaluate the safety, tolerability, and pharmacokinetics of repeat administrations of SRP-5051 in patients with Duchenne muscular dystrophy (DMD) who participated in studies of SRP-5051.

Interventions: Drug: SRP-5051
NCT Number: NCT03675126
  • Canada
  • United States
Age Limits:
7+
years
Overall Recruitment Status: Recruiting

Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients

Official Title:

Clinical Study to Assess the Safety of AVI-4658 in Subjects With Duchenne Muscular Dystrophy Due to a Frame-shift Mutation Amenable to Correction by Skipping Exon 51. 

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

The specific aim of this Phase I/II study is to assess the safety of intravenous administered Morpholino oligomer directed against exon 51 (AVI-4658 PMO). 

Interventions: Drug: AVI-4658 for Injection
NCT Number: NCT00844597
  • United Kingdom
Age Limits:
5-15
years
Overall Recruitment Status: Completed

Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients

Official Title:

A Randomized, Double-Blind, Placebo-Controlled, Dose-Titration, Safety, Tolerability, and Pharmacokinetics Study Followed by an Open-Label Safety and Efficacy Evaluation of SRP-4045 in Advanced-Stage Patients With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping

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

This is a first-in-human dose-titration and open-label extension study to assess safety, tolerability, and pharmacokinetics of SRP-4045 in advanced-stage Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon 45 skipping.

Interventions: Drug: SRP-4045; Drug: Placebo
NCT Number: NCT02530905
  • United States
Age Limits:
7-21
years
Overall Recruitment Status: Completed

Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients

Official Title:

A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Efficacy, Safety, Tolerability and Pharmacokinetics Study of AVI-4658 (Eteplirsen), in the Treatment of Ambulant Subjects With Duchenne Muscular Dystrophy

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

This study is designed to assess the efficacy, safety, tolerability, and pharmacokinetics (PK) of AVI-4658 (eteplirsen) in both 50.0 mg/kg and 30.0 mg/kg doses administered over 24 weeks in subjects diagnosed with Duchenne muscular dystrophy (DMD). 

Interventions: AVI-4658 (Eteplirsen); Other: Placebo
NCT Number: NCT01396239
  • United States
Age Limits:
7-13
years
Overall Recruitment Status: Completed

Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy

Official Title:

Open-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy Who Participated in Study 4658-US-201

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

The primary objective of this study is to assess the ongoing efficacy, safety, and tolerability of an additional 212 weeks of treatment with eteplirsen injection in Duchenne muscular dystrophy (DMD) subjects who have successfully completed the 28 week eteplirsen study:  Study 4658-us-201. This study will also evaluate the correlation between biomarkers for DMD and the clinical status of participating DMD subjects.

Interventions: Drug: AVI-4658 (Eteplirsen)
NCT Number: NCT01540409
  • United States
Age Limits:
7-13
years
Overall Recruitment Status: Completed

Gene Delivery Clinical Trial of SRP-9003 for Patients With 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 LGMD2E patients. Six total subjects will be enrolled. 

Interventions: Drug: SRP-9003
NCT Number: NCT03652259
  • United States
Age Limits:
4-15
years
Overall Recruitment Status: Recruiting

Phase I/II Study of SRP-4053 in DMD Patients

Official Title:

A 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose-Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping 

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

This is a first-in-human, multiple-dose 2-part study to assess the safety, tolerability, efficacy, and pharmacokinetics of SRP-4053 in Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon 53 skipping. 

Interventions: Drug: Placebo; Drug: SRP-4053
NCT Number: NCT02310906
  • France
  • Italy
  • United Kingdom
  • United States
Age Limits:
6-15
years
Overall Recruitment Status: Completed

Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy

Official Title:

An Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients With Advanced Stage Duchenne Muscular Dystrophy 

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

The primary objective of this study is to explore safety and tolerability of eteplirsen in participants with advanced stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. 

Interventions: Drug: Eteplirsen
NCT Number: NCT02286947
  • United States
Age Limits:
7-21
years
Overall Recruitment Status: Completed

Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy

Official Title:

An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early Stage Duchenne Muscular Dystrophy 

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

This is an open-label study to assess the safety, tolerability, efficacy and pharmacokinetics of eteplirsen in patients with early stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. 

Interventions: Drug: eteplirsen
NCT Number: NCT02420379
  • United States
Age Limits:
4-6
years
Overall Recruitment Status: Completed

Study of Eteplirsen in DMD Patients

Official Title:

An Open-Label, Multi-Center, Study With a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy 

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

The main objective of this study is to provide evidence of efficacy of eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) patients that are amenable to skipping exon 51. Additional objectives include evaluation of safety, biomarkers and the long-term effects of eteplirsen up to 96 weeks, followed by a safety extension (not to exceed 48 weeks). 

Interventions: Drug: eteplirsen
NCT Number: NCT02255552
  • United States
Age Limits:
7-16
years
Overall Recruitment Status: Completed

Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 Skipping

Official Title:

An Open-label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping 

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

This is a multicenter, open-label, dose-escalation study to evaluate the safety, tolerability, PK, and efficacy of once-weekly IV infusions of eteplirsen in approximately 12 male patients, ages 6 months to 48 months (inclusive), who have genotypically confirmed DMD with a deletion mutation amenable to exon 51 skipping.

Interventions: Drug: Eteplirsen
NCT Number: NCT03218995
  • Belgium
  • France
  • Italy
  • United Kingdom
Age Limits:
6-48
months
Overall Recruitment Status: Recruiting

Study of SRP-4045 and SRP-4053 in DMD Patients

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 and SRP-4053 compared to placebo in Duchenne muscular dystrophy (DMD) patients 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
  • Australia
  • Belgium
  • Bulgaria
  • Canada
  • Czechia
  • France
  • Germany
  • Hungary
  • Israel
  • Italy
  • Poland
  • Spain
  • Sweden
  • United Kingdom
  • United States
Age Limits:
7-13
years
Overall Recruitment Status: Recruiting

Systemic Gene Delivery Clinical Trial for Duchenne Muscular Dystrophy

Official Title:

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

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

The proposed clinical trial is a single-dose controlled trial using rAAVrh74.MHCK7.micro-dystrophin for DMD subjects. Cohort A will include six subjects ages 3 months to 3 years, and Cohort B will include six subjects ages 4 to 7 years old. All subjects will receive intravenous micro-dystrophin vector (2X10e14 vg/kg in 10mL/kg) 

Interventions: Drug: rAAVrh74.MHCK7.micro-dystrophin
NCT Number: NCT03375164
  • United States
Age Limits:
3-7
months
Overall Recruitment Status: Active, Not Recruiting