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

• Status: Active, not recruiting
• Trial end date: 2022-05-31
• Target enrollment: 0
• Participant gender: Male

Summary

This study will be comprised of 2 parts: 1) Part A (Multiple Ascending Dose [MAD]) will be conducted to evaluate the safety and tolerability of SRP-5051 at multiple ascending dose levels to determine the maximum tolerated dose (MTD), and 2) Part B (Dose Expansion) will be conducted to evaluate SRP-5051 administered at the MTD determined in Part A or, if applicable, at a second dose introduced in Part B. Patients enrolling in Part B will be those who completed Part A as well as additional expansion cohort patients who will enroll at the beginning of Part B.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Sarepta Therapeutics; Sarepta Therapeutics, Inc.

Criteria

Inclusion Criteria:

• Has a genetic diagnosis of DMD and an out-of-frame deletion mutation of the DMD gene amenable to exon 51-skipping treatment.

• Has been on a stable dose of oral corticosteroids for at least 12 weeks prior to study drug administration, or has not received corticosteroids for at least 12 weeks prior to study drug administration.

• Part A age group (ages 7 to 21)

• Part B age group (ages 4 to 21)

Exclusion Criteria:

• Has a left ventricular ejection fraction (LVEF) less than (<) 40.0 percent (%) based on an echocardiogram (ECHO) performed within 12 weeks prior to Screening or at the Screening visit.

• Has a FVC < 40.0% of predicted value within 12 weeks prior to Screening or at Screening.

• Initiation or change of dosing (except for modifications to accommodate changes in weight or changes in standard of care) within 12 weeks prior to Screening for any of the following: angiotensin-converting enzyme inhibitors, angiotensin receptor-blocking agents, β-blockers, or potassium.

• Initiation or change of dosing within 12 weeks prior to Screening for over-the-counter preparations, such as herbal/nonherbal supplements, vitamins, minerals, and homeopathic preparations.

• Treatment with any exon 51-skipping therapy within 12 weeks prior to Screening, or with any experimental gene therapy for the treatment of DMD at any time.

Other inclusion/exclusion criteria apply