Clinical Trials List
Protocol Number4658-402
2019-08-31 - 2024-06-03
Phase III
Recruiting2
ICD-10G71.0
Muscular dystrophy
ICD-9359.1
Hereditary progressive muscular dystrophy
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
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Trial Applicant
PPD DEVELOPMENT (HK) LIMITED
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Sponsor
Sarepta Therapeutics, Inc.
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Trial scale
Multi-Regional Multi-Center
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Update
2025/08/20
Investigators and Locations
The Actual Total Number of Participants Enrolled
0 Recruiting
Co-Principal Investigator
- WEN-CHIN WENG Division of Pediatrics
The Actual Total Number of Participants Enrolled
0 Recruiting
Condition/Disease
Duchenne Muscular Dystrophy
Objectives
Primary Outcome Measures :
Part 1 and Part 2 (Dose Finding): Incidences of Adverse Events (AEs)
Part 2 (Dose Finding): Dystrophin Expression in Biopsied Muscle Tissue
Part 2 (Dose Finding): Pharmacokinetic (PK) Plasma Concentration of Eteplirsen
Part 2 (Dose Finding): Tissue Concentration of Eteplirsen From Biopsied Muscle Tissue
Part 2 (Dose Comparison): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score
Secondary Outcome Measures :
Part 2 (Dose Comparison): Change From Baseline in the Total Distance Walked During 6-Minute Walk Test (6MWT)
Part 2 (Dose Comparison): Change From Baseline in Time to Complete Walk/run, Stairs and Time to Rise
Part 2 (Dose Comparison): Annual Rate in Decline of Forced Vital Capacity Percent Predicted (FVC%p)
Part 2 (Dose Comparison): Time to Loss of Ambulation (LOA)
Part 2 (Dose Comparison): Change From Baseline in Skeletal Muscle Dystrophin Expression
Part 2 (Dose Comparison): Incidence of Adverse Events (AEs)
Test Drug
Eteplirsen
Active Ingredient
Eteplirsen
Dosage Form
concentrate for solution for infusion
Dosage
100mg/2mL; 500mg/10mL
Endpoints
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.
Inclution Criteria
Inclusion Criteria:
Be a male with an established clinical diagnosis of DMD and an out-of-frame deletion mutation of the DMD gene amenable to exon 51 skipping.
Have achieved a mean 6-minute walk test (6MWT) distance of greater than equal to (>=) 300 and less than equal to (<=) 450 meters.
Have intact right and left biceps muscles or an alternative upper arm muscle group.
Have been on a stable dose or dose equivalent of oral corticosteroids for at least 12 weeks prior to randomization.
Have stable pulmonary function (forced vital capacity >= 50 percent (%) of predicted and no requirement for nocturnal ventilation).
Be a male with an established clinical diagnosis of DMD and an out-of-frame deletion mutation of the DMD gene amenable to exon 51 skipping.
Have achieved a mean 6-minute walk test (6MWT) distance of greater than equal to (>=) 300 and less than equal to (<=) 450 meters.
Have intact right and left biceps muscles or an alternative upper arm muscle group.
Have been on a stable dose or dose equivalent of oral corticosteroids for at least 12 weeks prior to randomization.
Have stable pulmonary function (forced vital capacity >= 50 percent (%) of predicted and no requirement for nocturnal ventilation).
Exclusion Criteria
Exclusion Criteria:
Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks prior to randomization.
Current or previous treatment with gene therapy or any other experimental pharmacologic treatment for DMD; some exceptions apply.
Previous treatment with Ezutromid in the last 1 week prior to first dose or Drisapersen in the last 36 weeks prior to first dose.
Major surgery within 3 months prior to randomization.
Presence of any other significant neuromuscular or genetic disease other than DMD.
Presence of any known impairment of renal function and/or other clinically significant illness.
Has evidence of cardiomyopathy, as defined by left ventricular ejection fraction less than (<) 50% on the screening Echocardiogram or QTcF >= 450 millisecond based on the screening ECGs.
Other inclusion/exclusion criteria apply.
Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks prior to randomization.
Current or previous treatment with gene therapy or any other experimental pharmacologic treatment for DMD; some exceptions apply.
Previous treatment with Ezutromid in the last 1 week prior to first dose or Drisapersen in the last 36 weeks prior to first dose.
Major surgery within 3 months prior to randomization.
Presence of any other significant neuromuscular or genetic disease other than DMD.
Presence of any known impairment of renal function and/or other clinically significant illness.
Has evidence of cardiomyopathy, as defined by left ventricular ejection fraction less than (<) 50% on the screening Echocardiogram or QTcF >= 450 millisecond based on the screening ECGs.
Other inclusion/exclusion criteria apply.
The Estimated Number of Participants
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Taiwan
8 participants
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Global
154 participants
