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Trial record 3 of 3 for:    SRP-5051

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

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ClinicalTrials.gov Identifier: NCT04004065
Recruitment Status : Active, not recruiting
First Posted : July 1, 2019
Last Update Posted : November 24, 2021
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Brief 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 MAD levels to determine doses to be administered in Part B, and 2) Part B will be conducted to further evaluate the SRP-5051 doses selected in Part A. Participants enrolling in Part B will be those who completed Part A or Study 5051-102 and meet applicable eligibility criteria for Part B, as well as additional participants who meet applicable eligibility criteria for enrollment at the beginning of Part B.

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 : 60 participants
Allocation: 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 : September 30, 2023
Estimated Study Completion Date : August 31, 2024


Arm Intervention/treatment
Experimental: Part A: 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.
Drug: SRP-5051
SRP-5051 injection, for IV use

Experimental: Part B: SRP-5051
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.
Drug: SRP-5051
SRP-5051 injection, for IV use




Primary Outcome Measures :
  1. Part A: Number of Adverse Events (AEs) [ Time Frame: Part A: Baseline up to 75 weeks ]
    Number of adverse events includes clinically significant laboratory abnormalities.

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

Secondary Outcome Measures :
  1. Part A: Pharmacokinetics (PK): Plasma Concentration of SRP-5051 and Metabolite (SRP-5051A) [ Time Frame: Pre-dose and at multiple time points (up to 32 hours) after end of infusion ]
  2. 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 ]
  3. Part B: Change From Baseline in Exon-Skipping Levels [ Time Frame: Part B: Baseline, Week 28 ]
  4. Part B: Number of Adverse Events (AEs) [ Time Frame: Part B: Baseline up to Week 104 ]
    Number of adverse events includes clinically significant laboratory abnormalities.

  5. Part B: PK: Plasma Concentration of SRP-5051 and Metabolite (SRP-5051A) [ Time Frame: Part B predose and at multiple timepoints (up to 48 hours) after end of infusion ]
  6. Part B: PK: Urine Concentration of SRP-5051 [ Time Frame: Part B predose and at multiple timepoints (up to 48 hours) after end of infusion ]
  7. Part B: Change from Baseline in Percent Dystrophin-Positive Fibers (PDPF) and Mean Intensity, as Measured by Immunofluorescence Assay [ Time Frame: Part B: Baseline, Week 28 ]
    Change from baseline in PDPF and mean intensity as measured by immunofluorescence assay will be reported.



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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 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).

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 12 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


Locations
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United States, Florida
Northwest Florida Clinical Research Group, LLC
Gulf Breeze, Florida, United States, 32561
United States, Georgia
Rare Disease Research, LLC
Atlanta, Georgia, United States, 30318
United States, Iowa
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States, 52242
United States, Pennsylvania
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15224
United States, Texas
Austin Neuromuscular Center
Austin, Texas, United States, 78756
Belgium
Universitair Ziekenhuis Gent
Gent, Belgium, 9000
Canada, Ontario
Children's Hospital - London Health Sciences Centre (LHSC)
London, Ontario, Canada, N6A 5W9
Spain
Hospital Sant Joan de Déu. U.B.
Barcelona, Spain, 08950
Hospital Universitari I Politecnic La Fe de Valencia
Valencia, Spain, 46026
United Kingdom
Alder Hey Children's NHS Foundation Trust
Liverpool, United Kingdom, L12 2AP
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    
Other Study ID Numbers: 5051-201
2019-000601-77 ( EudraCT Number )
First Posted: July 1, 2019    Key Record Dates
Last Update Posted: November 24, 2021
Last Verified: November 2021

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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.:
DMD
Duchenne
Dystrophy
Dystrophin
Exon Skipping
Ambulatory
Duchenne Muscular Dystrophy
Exon 51
Nonambulatory
Pediatric
Peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO)
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