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A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study (EXPEDITION)

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ClinicalTrials.gov Identifier: NCT05967351
Recruitment Status : Enrolling by invitation
First Posted : August 1, 2023
Last Update Posted : April 23, 2024
Sponsor:
Collaborator:
Hoffmann-La Roche
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Study Description
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Brief Summary:
The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants who received delandistrogene moxeparvovec in a previous clinical study. No study drug will be administered as part of this study. Pre-infusion baseline will be defined as the timepoint just prior to infusion of delandistrogene moxeparvovec from a previous clinical study. Each participant will be followed for a minimum of 5 years post-infusion of delandistrogene moxeparvovec from a previous clinical study. The duration of participation in this study is dependent on the length of follow-up the participant completed post-infusion of delandistrogene moxeparvovec from a previous clinical study.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Genetic: delandistrogene moxeparvovec Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 400 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Other
Official Title: A Phase 3, Multinational, Long-term Follow-up Study to Evaluate Safety and Efficacy in Subjects Who Have Previously Received SRP-9001 in a Clinical Study
Actual Study Start Date : September 27, 2023
Estimated Primary Completion Date : November 30, 2030
Estimated Study Completion Date : November 30, 2030

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Arms and Interventions
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Arm Intervention/treatment
Experimental: Delandistrogene Moxeparvovec
Participant received delandistrogene moxeparvovec in a previous clinical study.
 Genetic: delandistrogene moxeparvovec
No study drug will be administered as part of this study. Eligible participants who received treatment with delandistrogene moxeparvovec during a previous clinical study will be included.
Other Names:
  • SRP-9001
  • delandistrogene moxeparvovec-rokl
  • ELEVIDYS


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI) [ Time Frame: Up to 5 years after dosing ]

Secondary Outcome Measures :
  1. Change in the North Star Ambulatory Assessment (NSAA) Total Score From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec [ Time Frame: Baseline, 5 years ]
  2. Change in Time to Rise From Floor From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec [ Time Frame: Baseline, 5 years ]
  3. Change in the Time of 10-meter Walk/Run (10MWR) From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec [ Time Frame: Baseline, 5 years ]
  4. Change in Performance of Upper Limb (PUL) (Version 2.0) Total Scores From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec [ Time Frame: Baseline, 5 years ]
  5. Change in PUL (Version 2.0) Domain Specific Scores From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec [ Time Frame: Baseline, 5 years ]
  6. Change in Forced Vital Capacity Percent (FVC%) Predicted From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec [ Time Frame: Baseline, 5 years ]
  7. Change in Peak Expiratory Flow Percent (PEF%) Predicted From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec [ Time Frame: Baseline, 5 years ]
  8. Change in Cardiac Magnetic Resonance Imaging (MRI) Findings From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec [ Time Frame: Baseline, 5 years ]
  9. Change in Musculoskeletal MRI Findings From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec [ Time Frame: Baseline, 5 years ]

Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Received delandistrogene moxeparvovec for Duchenne muscular dystrophy in a previous clinical study.
  • Has (a) parent(s) or legal caregiver(s) or is ≥18 years of age and able to understand and comply with the study visit schedule and all other protocol requirements.

Exclusion Criteria:

  • Participant or family does not want to disclose participation with general practitioner/primary care physician and other medical providers.

Other inclusion/exclusion criteria may apply.

Contacts and Locations
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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): NCT05967351


Locations
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United States, California
UCLA Medical Center
Los Angeles, California, United States, 90095
Lucile Packard Children's Hospital Stanford (LPCH)
Palo Alto, California, United States, 94304
United States, Missouri
Washington University School of Medicine
Saint Louis, Missouri, United States, 63110
United States, New York
University of Rochester Medical Center
Rochester, New York, United States, 14642
United States, Ohio
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
United States, Virginia
Children's Hospital of The King's Daughters
Norfolk, Virginia, United States, 23510
United States, Wisconsin
Children's Hospital Wisconsin
Milwaukee, Wisconsin, United States, 53226
Sponsors and Collaborators
Sarepta Therapeutics, Inc.
Hoffmann-La Roche
More Information
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Responsible Party: Sarepta Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT05967351    
Other Study ID Numbers: SRP-9001-305
2023-505043-39-00 ( Other Identifier: Clinical Trials Information System (CTIS) )
First Posted: August 1, 2023    Key Record Dates
Last Update Posted: April 23, 2024
Last Verified: April 2024

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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
Pediatric
Long-term
 Follow-up
Safety
Efficacy
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