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A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Duchenne Muscular Dystrophy (DMD) Patients
study id #: NCT03992430
condition: Muscular Dystrophy, Duchenne
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.
mechanism of action: Exon-skipping to promote dystrophin production
last updated: August 26, 2020
start date: July 13, 2020
estimated completion: October 2024
phase of development: Phase 3
size / enrollment: 152
- Part 1 and Part 2 (Dose Finding): Incidences of Adverse Events (AEs) [ Time Frame: Baseline up to Week 148 ]
- Part 2 (Dose Finding): Dystrophin Expression in Biopsied Muscle Tissue [ Time Frame: Up to Week 48 ]
- Part 2 (Dose Finding): Pharmacokinetic (PK) Plasma Concentration of Eteplirsen [ Time Frame: Multiple timepoints up to Week 48 ]
- Part 2 (Dose Finding): Tissue Concentration of Eteplirsen From Biopsied Muscle Tissue [ Time Frame: Up to Week 48 ]
- Part 2 (Dose Comparison): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score [ Time Frame: Baseline, Week 144 ]
- Part 2 (Dose Comparison): Change From Baseline in the Total Distance Walked During 6-Minute Walk Test (6MWT) [ Time Frame: Baseline up to Week 144 ]
- Part 2 (Dose Comparison): Change From Baseline in Time to Complete Walk/run, Stairs and Time to Rise [ Time Frame: Baseline up to Week 144 ]
- Part 2 (Dose Comparison): Annual Rate in Decline of Forced Vital Capacity Percent Predicted (FVC%p) [ Time Frame: Up to Week 144 ]
- Part 2 (Dose Comparison): Time to Loss of Ambulation (LOA) [ Time Frame: Baseline up to Week 144 ]
- Part 2 (Dose Comparison): Change From Baseline in Skeletal Muscle Dystrophin Expression [ Time Frame: Baseline and Week 48 ]
- Part 2 (Dose Comparison): Incidence of Adverse Events (AEs) [ Time Frame: Baseline up to Week 144 ]
• Eligible Sexes: male
• 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 24 weeks prior to randomization.
• Have stable pulmonary function (forced vital capacity >= 50 percent (%) of predicted and no requirement for nocturnal ventilation).
• 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 drisapersen, ezutromid, or domagrozumab in the last 24 weeks prior to study enrollment.
• Major surgery within 3 months prior to randomization.
• Presence of any other significant neuromuscular or genetic disease other than DMD.
• Presence of 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.
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