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

Phase 3

• Conditions: Health Condition 1: G710- Muscular dystrophy

• Registration Number: CTRI/2022/09/045928
• Lead Sponsor: Sarepta Therapeutics Inc

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Last Update Posted: 25 July 2023

Recruitment & Eligibility

• Status: Open to Recruitment
• Sex: Not specified
• Target Recruitment: 0

Inclusion Criteria

Please note that India will be participating in Part 2 (Double-Blind Dose Finding and Dose Comparison) of the study.

Inclusion Criteria for Part 2 of study:

1. 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.

2. Ambulatory participant, able to perform TTRISE in 10 seconds or less at the time of screening visit.

3. Able to walk independently without assistive devices.

4. Have intact right and left biceps muscles or an alternative upper arm muscle group.

5. Have been on a stable dose or dose equivalent of oral corticosteroids for at least 12 weeks prior to randomization and the dose is expected to remain constant (except for modifications to accommodate changes in weight and stress-related needs as per the recently published guidelines throughout the study.

6. For ages 7 years and older, has stable pulmonary function (forced vital capacity >=50 percent (%) of predicted and no requirement for nocturnal ventilation). For ages 4 to 6 years, does not require support from ventilator or non-invasive ventilation at time of screening

Exclusion Criteria

Exclusion Criteria for Part 2 of study:

1. Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks prior to randomization.

2. Current or previous treatment with any other experimental pharmacologic treatment for DMD or any prior exposure to antisense oligonucleotide, gene therapy or gene editing; except the following: Ezutromid in the last 12 weeks prior to first dose; Drisapersen in the last 36 weeks prior to first dose; Suvodirsen in the last 12 weeks prior to first dose; Vamorolone in the last 12 weeks prior to first dose; and Eteplirsen (previous or current use).

3. Major surgery within 3 months prior to randomization.

4. Presence of any other significant neuromuscular or genetic disease other than DMD.

5. Presence of any known impairment of renal function and/or other clinically significant illness.

6. Has evidence of cardiomyopathy, as defined by left ventricular ejection fraction less than 50% on the screening echocardiogram.

7.Other inclusion/exclusion criteria apply.

Study & Design

• Study Type: Interventional
• Study Design: Not specified