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

Phase 3

Recruiting

• Conditions: DMD; muscular dystrophy; 10028396

• Registration Number: NL-OMON52516
• Lead Sponsor: Sarepta Therapeutics, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 24 June 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: Not specified
• Target Recruitment: 1

Inclusion 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.
• Ambulatory participant, able to perform TTRISE in 10 seconds or less at the
time of screening visit.
• Able to walk independently without assistive devices.
• 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 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.
• 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:
• Use of any pharmacologic treatment (other than corticosteroids) within 12
weeks prior to randomization.
• 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).
• 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 Fridericia's
correction formula (QTcF) >=450 millisecond based on the screening
electrocardiograms (ECGs).
Other inclusion/exclusion criteria apply.

Study & Design

• Study Type: Interventional
• Study Design: Not specified