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A Phase 2 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 : July 1, 2019
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Brief Summary:
This study will be comprised of 2 parts: Part A (Multiple Ascending Dose (MAD)) which will be conducted to evaluate the safety and tolerability of SRP-5051 at multiple ascending dose levels to determine the maximum tolerated dose (MTD); Part B (Dose Expansion) will be conducted to evaluate SRP-5051 administered at the MTD in patients who have completed Part A.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: SRP-5051 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 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 : March 2021
Estimated Study Completion Date : March 2021


Arm Intervention/treatment
Experimental: Part A: SRP-5051
Patients will be sequentially assigned to receive 1 of the 4 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.
Drug: SRP-5051
SRP-5051 IV infusion

Experimental: Part B: SRP-5051
Patients who will complete Part A will receive SRP-5051 at the MTD determined in Part A, monthly, via IV infusion, for 24 weeks.
Drug: SRP-5051
SRP-5051 IV infusion




Primary Outcome Measures :
  1. Part A: Incidence of Adverse Events (AEs) [ Time Frame: From signing of informed consent until 28 days after last dose of study drug administered (up to 60 weeks) ]
    Incidence of adverse events includes clinically significant laboratory abnormalities.

  2. Part B: Change From Baseline Biopsy in Exon-Skipping Levels [ Time Frame: Baseline, Part B Week 24 ]
  3. Part B: Change From Baseline Biopsy in Dystrophin Protein Production Levels [ Time Frame: Baseline, Part B Week 24 ]

Secondary Outcome Measures :
  1. Part A: Pharmacokinetic (PK) Plasma Concentration of SRP-5051 [ Time Frame: Predose and at multiple timepoints (up to 24 hours) after end of infusion ]
  2. Part B: Incidence of Adverse Events (AEs) [ Time Frame: Up to 28 weeks in Part B ]
    Incidence of adverse events includes clinically significant laboratory abnormalities.

  3. Part B: Change From Baseline in Forced Vital Capacity (FVC) (percent predicted) [ Time Frame: Baseline, Part B Week 24 ]
  4. Part B: Change From Baseline in the North Star Ambulatory Assessment (NSAA) [ Time Frame: Baseline, Part B Week 24 ]
  5. Part B: Change From Baseline in the Performance of Upper Limb (PUL) Scores [ Time Frame: Baseline, Part B Week 24 ]
  6. Part B: Change From Baseline in the Brooke Upper Extremity Scale score (Brooke score) [ Time Frame: Baseline, Part B Week 24 ]
  7. Part B: PK Plasma Concentration of SRP-5051 [ Time Frame: Part B predose and at multiple timepoints (up to 12 hours) after end of infusion ]


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Ages Eligible for Study:   7 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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.

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) 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 24 weeks prior to Screening, or with any experimental gene therapy for the treatment of DMD at any time.

Other inclusion/exclusion criteria apply.


Information from the National Library of Medicine

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


Contacts
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Contact: Medical Information +1 888-727-3782 clinicaltrials@sarepta.com

Locations
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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         
Sponsors and Collaborators
Sarepta Therapeutics, Inc.
Investigators
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Study Director: Medical Director Sarepta Therapeutics, Inc.

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Responsible Party: Sarepta Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT04004065     History of Changes
Other Study ID Numbers: 5051-201
2019-000601-77 ( EudraCT Number )
First Posted: July 1, 2019    Key Record Dates
Last Update Posted: July 1, 2019
Last Verified: June 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Sarepta Therapeutics, Inc.:
Duchenne Muscular Dystrophy
DMD
Duchenne
Dystrophy
Dystrophin
Exon Skipping
Ambulatory
Exon 51
Nonambulatory
Pediatric
Peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO)
Momentum
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked