Study for Dose Determination of SRP-5051, Then Dose Expansion in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (MOMENTUM)
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ClinicalTrials.gov Identifier: NCT04004065 |
Recruitment Status :
Recruiting
First Posted : July 1, 2019
Last Update Posted : January 7, 2021
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Condition or disease | Intervention/treatment | Phase |
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Duchenne Muscular Dystrophy | Drug: SRP-5051 | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 70 participants |
Allocation: | Non-Randomized |
Intervention Model: | Sequential Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official 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 |
Actual Study Start Date : | June 26, 2019 |
Estimated Primary Completion Date : | May 31, 2022 |
Estimated Study Completion Date : | May 31, 2022 |

Arm | Intervention/treatment |
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Experimental: Part A: SRP-5051
Patients will be sequentially assigned to receive 1 of the 5 escalating dose levels of SRP-5051, monthly, via intravenous (IV) infusion for at least 12 weeks during Part A. Once the maximum tolerated dose (MTD) has been determined in Part A, all patients who have completed Part A will transition to Part B.
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Drug: SRP-5051
SRP-5051 injection, for IV use |
Experimental: Part B: SRP-5051
Patients will receive SRP-5051 at the MTD determined in Part A, or if applicable, at a second dose introduced in Part B, monthly, via IV infusion, for 24 weeks. This includes the patients who roll over from Part A, as well as expansion cohort patients who will enroll in the study at the beginning of Part B.
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Drug: SRP-5051
SRP-5051 injection, for IV use |
- Part A: Incidence of Adverse Events (AEs) [ Time Frame: Part A: approximately 68 weeks ]Incidence of adverse events includes clinically significant laboratory abnormalities.
- Part B: Change From Baseline in Dystrophin Protein Level [ Time Frame: Baseline, Week 12 or Week 24 ]
- Part A: Pharmacokinetics (PK): Plasma Concentration of SRP-5051 [ Time Frame: Pre-dose and at multiple time points (up to 32 hours) after end of infusion ]
- Part A: PK: Urine Concentration of SRP-5051 [ Time Frame: Pre-dose and at multiple time periods (up to 48 hours) after end of infusion ]
- Part B: Change From Baseline in Exon-Skipping Levels [ Time Frame: Baseline, Week 12 or Week 24 ]
- Part B: Incidence of Adverse Events (AEs) [ Time Frame: Part B: approximately 44 weeks ]Incidence of adverse events includes clinically significant laboratory abnormalities.
- Part B: Change From Baseline in Forced Vital Capacity (FVC) (percent predicted) [ Time Frame: Baseline, Week 12 or Week 24 ]
- Part B: Change From Baseline in the North Star Ambulatory Assessment (NSAA) [ Time Frame: Baseline, Week 12 or Week 24 ]
- Part B: Change From Baseline in the Performance of Upper Limb (PUL) Scores [ Time Frame: Baseline, Week 12 or Week 24 ]
- Part B: Change From Baseline in the Brooke Upper Extremity Scale score (Brooke score) [ Time Frame: Baseline, Week 12 or Week 24 ]
- Part B: PK: Plasma Concentration of SRP-5051 [ Time Frame: Part B predose and at multiple timepoints (up to 24 hours) after end of infusion ]

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Ages Eligible for Study: | 4 Years to 21 Years (Child, Adult) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04004065
Contact: Medical Information | +1 800-690-2003 | clinicaltrials@sarepta.com |
United States, Florida | |
Northwest Florida Clinical Research Group, LLC | Recruiting |
Gulf Breeze, Florida, United States, 32561 | |
Contact: Genei Bougher, APRN 850-934-1299 info@nwflcrg.com | |
Principal Investigator: Weldon Mauney, MD | |
United States, Georgia | |
Rare Disease Research, LLC | Recruiting |
Atlanta, Georgia, United States, 30318 | |
Contact 678-883-6897 Info@rarediseaseresearch.com | |
Principal Investigator: Han Phan, MD | |
United States, Iowa | |
University of Iowa Hospitals and Clinics | Recruiting |
Iowa City, Iowa, United States, 52242 | |
Contact: Bryn Miller 319-335-5836 bryn-miller@uiowa.edu | |
Principal Investigator: Katherine Mathews, MD | |
United States, Pennsylvania | |
Children's Hospital of Pittsburgh of UPMC | Recruiting |
Pittsburgh, Pennsylvania, United States, 15224 | |
Contact: Jennifer Monahan 412-692-5176 jennifer.monahan@chp.edu | |
Principal Investigator: Hoda Abdel-Hamid, MD | |
United States, Texas | |
Austin Neuromuscular Center | Active, not recruiting |
Austin, Texas, United States, 78756 | |
Belgium | |
Universitair Ziekenhuis Gent | Recruiting |
Gent, Belgium, 9000 | |
Contact: Elke De Vos elke.devos@uzgent.be | |
Principal Investigator: Nicolas Deconinck, MD | |
Canada, Ontario | |
Children's Hospital - London Health Sciences Centre (LHSC) | Recruiting |
London, Ontario, Canada, N6A 5W9 | |
Contact: Chelsea Huisman Chelsea.Huisman@lhsc.on.ca | |
Principal Investigator: Craig Campbell, MD | |
Spain | |
Hospital Sant Joan de Déu. U.B. | Recruiting |
Barcelona, Spain, 08950 | |
Contact: Andres Nascimento Osorio anascimento@hsjdbcn.org | |
Principal Investigator: Andres Nascimento Osorio, MD | |
Hospital Universitari I Politecnic La Fe de Valencia | Recruiting |
Valencia, Spain, 46026 | |
Contact: Juan Jesús Vilchez Padilla vilchez_jje@gva.es | |
Principal Investigator: Juan Jesús Vilchez Padilla, MD | |
United Kingdom | |
Alder Hey Children's NHS Foundation Trust | Recruiting |
Liverpool, United Kingdom, L12 2AP | |
Contact: Clinical Research Facility +44 151-252-5115 neuromuscularresearch@alderhey.nhs.uk | |
Principal Investigator: Stephan Spinty, MD |
Study Director: | Medical Director | Sarepta Therapeutics, Inc. |
Responsible Party: | Sarepta Therapeutics, Inc. |
ClinicalTrials.gov Identifier: | NCT04004065 |
Other Study ID Numbers: |
5051-201 2019-000601-77 ( EudraCT Number ) |
First Posted: | July 1, 2019 Key Record Dates |
Last Update Posted: | January 7, 2021 |
Last Verified: | January 2021 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
DMD Duchenne Dystrophy Dystrophin Exon Skipping Ambulatory |
Duchenne Muscular Dystrophy Exon 51 Nonambulatory Pediatric Peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) |
Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases |
Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked |