Two-Part Study for Dose Determination of SRP-5051 (Vesleteplirsen) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (MOMENTUM)
Duchenne Muscular Dystrophy
About this trial
This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring DMD, Duchenne, Dystrophy, Dystrophin, Exon Skipping, Ambulatory, Duchenne Muscular Dystrophy, Exon 51, Nonambulatory, Pediatric, Peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO)
Eligibility Criteria
Inclusion Criteria for participants previously treated with SRP-5051:
- Has received prior SRP-5051 treatment in Part A of this study or in Study 5051-102
Exclusion Criteria for participants previously treated with SRP-5051 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, 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.
- 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.
Sites / Locations
- Univertisty of California Davis Health
- Connecticut Children's
- Northwest Florida Clinical Research Group, LLC
- Rare Disease Research, LLC
- University of Iowa Hospitals and Clinics
- University of Kansas Medical Center Research Inst.
- University of Massachusetts
- UPMC Children's Hospital of Pittsburgh
- Austin Neuromuscular Center
- Children's Health Ambulatory Pavilion
- Seattle Children's
- Universitair Ziekenhuis Gent
- UZ Leuven
- Children's Hospital - London Health Sciences Centre (LHSC)
- University of Essen - Children's Hospital
- Klinikum der Universität München
- Fondazione Policlinico Universitario A Gemelli
- A.O.U. Citta della Salute e della Scienza di Torino - SS Malattie Neuromuscolari, Department of Neurosciences
- Leiden University Medical Center
- Hospital Sant Joan de Déu. U.B.
- Hospital Universitari I Politecnic La Fe de Valencia
- Royal Hospital for Children (Glasgow)
- Alder Hey Children's NHS Foundation Trust
- Great Ormond Street Hospital for Children NHS Foundation Trust
- Oxford University Hospial NHS Foundation Trust, John Radcliffe Hospital
Arms of the Study
Arm 1
Arm 2
Experimental
Experimental
Part A: SRP-5051
Part B: SRP-5051
Participants received escalating dose levels of SRP-5051, every 4 weeks, via intravenous (IV) infusion for up to 75 weeks during Part A. Once the doses have been selected for Part B, all participants who have completed Part A will transition to Part B.
Participants will receive SRP-5051 at the doses selected based on data from Part A every 4 weeks, via IV infusion, for up to 2 years. This includes the participants who rollover from Part A, as well as the additional participants who will be enrolled at the beginning of Part B.