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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05967351
Recruitment Status : Enrolling by invitation
First Posted : August 1, 2023
Last Update Posted : May 16, 2024
Sponsor:
Collaborator:
Hoffmann-La Roche
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Tracking Information
First Submitted Date  ICMJE July 21, 2023
First Posted Date  ICMJE August 1, 2023
Last Update Posted Date May 16, 2024
Actual Study Start Date  ICMJE September 27, 2023
Estimated Primary Completion Date November 30, 2030   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 21, 2023)		 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 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: July 21, 2023)
  • 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 ]
  • Change in Time to Rise From Floor From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec [ Time Frame: Baseline, 5 years ]
  • 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 ]
  • 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 ]
  • 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 ]
  • 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 ]
  • 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 ]
  • 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 ]
  • Change in Musculoskeletal MRI Findings From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec [ Time Frame: Baseline, 5 years ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
Official Title  ICMJE 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
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Other
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE 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
Study Arms  ICMJE Experimental: Delandistrogene Moxeparvovec
Participant received delandistrogene moxeparvovec in a previous clinical study.
Intervention: Genetic: delandistrogene moxeparvovec
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Enrolling by invitation
Estimated Enrollment  ICMJE
 (submitted: July 21, 2023)		 400
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE November 30, 2030
Estimated Primary Completion Date November 30, 2030   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT05967351
Other Study ID Numbers  ICMJE SRP-9001-305
2023-505043-39-00 ( Other Identifier: Clinical Trials Information System (CTIS) )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party Sarepta Therapeutics, Inc.
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Sarepta Therapeutics, Inc.
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Hoffmann-La Roche
Investigators  ICMJE Not Provided
PRS Account Sarepta Therapeutics, Inc.
Verification Date May 2024

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP