Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

• Register: ClinicalTrials.gov
• Last refreshed on: 11 December 2023
• Main ID: NCT04004065
• Date of registration: 27/06/2019
• Prospective Registration: No
• Primary sponsor: Sarepta Therapeutics, Inc.
• Public title: Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment MOMENTUM
• Scientific title: A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, Then Dose Expansion, in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
• Date of first enrolment: June 26, 2019
• Target sample size: 62
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT04004065
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Belgium, Canada, Germany, Italy, Netherlands, Spain, United Kingdom, United States

Contacts

• Name: Medical Director
• Affiliation: Sarepta Therapeutics, Inc.

Key inclusion & exclusion criteria

Inclusion Criteria for participants previously treated with Vesleteplirsen:

- Has received prior Vesleteplirsen treatment in Part A of this study or in Study 5051-102.

Exclusion Criteria for participants previously treated with Vesleteplirsen and new
participants enrolling into Part B:

- Presence of other clinically significant illness, including cardiac, pulmonary, hepatic,
renal, hematologic, immunologic, or behavioral disease, or infection or malignancy or any
other condition that, in the Investigator's opinion, could interfere with participation in
the trial.

Inclusion Criteria for treatment-naïve participants enrolling into Part B:

- Has a genetic diagnosis of Duchenne muscular dystrophy (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 and the dose is expected to remain constant throughout the study
(except for modifications to accommodate changes in weight), or has not received
corticosteroids for at least 12 weeks prior to study drug administration.

- Has stable pulmonary function (forced vital capacity [FVC] =40% of predicted and no
requirement for nocturnal ventilation).

Exclusion Criteria for treatment-naive participants enrolling into Part B:

- History of hypomagnesemia within 12 weeks prior to 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, ß-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.

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

- Treatment with any exon 51-skipping therapy within 4 weeks prior to Screening, or with
any experimental gene therapy for the treatment of DMD at any time.

Other inclusion/exclusion criteria apply.



Age minimum: 7 Years
Age maximum: 21 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Drug: Vesleteplirsen

Primary Outcome(s)

Part B: Change From Baseline in Dystrophin Protein Level at Week 28 [Time Frame: Part B: Baseline, Week 28]

Part A: Incidence of Adverse Events (AEs) [Time Frame: Part A: Baseline up to 75 weeks]

Secondary Outcome(s)

Part B: Change from Baseline in Percent Dystrophin-Positive Fibers (PDPF) and Mean Intensity, as Measured by Immunofluorescence Assay at Week 28 [Time Frame: Part B: Baseline, Week 28]

Part A: Pharmacokinetics (PK): Plasma Concentration of Vesleteplirsen [Time Frame: Pre-dose and at multiple time points (up to 32 hours) after end of infusion]

Part B: PK: Urine Concentration of Vesleteplirsen [Time Frame: Part B predose and at multiple timepoints (up to 48 hours) after end of infusion]

Part B: Change From Baseline in Exon-Skipping Levels at Week 28 [Time Frame: Part B: Baseline, Week 28]

Part B: PK: Plasma Concentration of Vesleteplirsen [Time Frame: Part B predose and at multiple timepoints (up to 48 hours) after end of infusion]

Part A: PK: Urine Concentration of Vesleteplirsen [Time Frame: Pre-dose and at multiple time periods (up to 48 hours) after end of infusion]

Part B: Incidence of Adverse Events (AEs) [Time Frame: Part B: Baseline up to Week 304]

Secondary ID(s)

5051-201

2019-000601-77

Source(s) of Monetary Support

Please refer to primary and secondary sponsors