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

NCT ID: NCT05967351

Last Updated: 2025-01-17

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ENROLLING_BY_INVITATION

Clinical Phase

PHASE3

Total Enrollment

400 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-09-27

Study Completion Date

2030-11-30

Brief Summary

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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

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Conditions

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Duchenne Muscular Dystrophy

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

OTHER

Blinding Strategy

NONE

Study Groups

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Delandistrogene Moxeparvovec

Participant received delandistrogene moxeparvovec in a previous clinical study.

Group Type EXPERIMENTAL

delandistrogene moxeparvovec

Intervention Type GENETIC

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.

Interventions

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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.

Intervention Type GENETIC

Other Intervention Names

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SRP-9001 delandistrogene moxeparvovec-rokl ELEVIDYS

Eligibility Criteria

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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.
Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Hoffmann-La Roche

INDUSTRY

Sponsor Role collaborator

Sarepta Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Arkansas Children's Hospital

Little Rock, Arkansas, United States

Site Status

UCLA Medical Center

Los Angeles, California, United States

Site Status

Lucile Packard Children's Hospital Stanford (LPCH)

Palo Alto, California, United States

Site Status

University of California, Davis

Sacramento, California, United States

Site Status

Rady Children's Hospital

San Diego, California, United States

Site Status

Children's Hospital of Colorado

Aurora, Colorado, United States

Site Status

University of Florida Clinical Research Center

Gainesville, Florida, United States

Site Status

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Site Status

University of Iowa ICTS Clinical Research Unit

Iowa City, Iowa, United States

Site Status

The Johns Hopkins Hospital, Rubenstein Child Health Bldg

Baltimore, Maryland, United States

Site Status

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

Washington University School of Medicine

St Louis, Missouri, United States

Site Status

Columbia University Pediatric Neuromuscular Center

New York, New York, United States

Site Status

University of Rochester Medical Center

Rochester, New York, United States

Site Status

Lenox Baker Children's Hospital (Duke University)

Durham, North Carolina, United States

Site Status

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

Oregon Health and Science University

Portland, Oregon, United States

Site Status

Chiildren's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Children's Health Specialty Center

Dallas, Texas, United States

Site Status

University of Utah Health

Salt Lake City, Utah, United States

Site Status

Children's Hospital of The King's Daughters

Norfolk, Virginia, United States

Site Status

Children's Hospital Wisconsin

Milwaukee, Wisconsin, United States

Site Status

Universitair Ziekenhuis Gent, Neuromuscular Reference Centre (NMRC)

Ghent, East Flanders, Belgium

Site Status

Universitätsklinikum Essen - Klinik für Kinderheilkunde I

Essen, North Rhine-Westphalia, Germany

Site Status

Hong Kong Children's Hospital

Hong Kong, , Hong Kong

Site Status

UOC Neurologia Pediatrica e Malattie Muscolari, Istituto G. Gaslini, Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico

Genova, , Italy

Site Status

UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, , Italy

Site Status

UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gemelli IRCCS, Universita Cattoclica del Sacro Cuore

Roma, , Italy

Site Status

Kobe University Hospital

Kobe, Hyōgo, Japan

Site Status

National Center of Neurology and Psychiatry

Kodaira, Tokyo, Japan

Site Status

Hospital Sant Joan de Deu

Esplugues de Llobregat, Barcelona, Spain

Site Status

Hospital Universitario y Politecnico La fe. Neurology Department

Valencia, , Spain

Site Status

Kaohsiung Medical University Chung-Ho Memorial Hospital

Kaohsiung City, , Taiwan

Site Status

National Taiwan University Hospital

Taipei, , Taiwan

Site Status

Great Ormond Street Hospital For Children NHS Foundation Trust

London, Greater London, United Kingdom

Site Status

Oxford University Hospitals NHS Foundation Trust

Oxford, Oxfordshire, United Kingdom

Site Status

Institute of Translational and Clinical Research

Newcastle upon Tyne, , United Kingdom

Site Status

Countries

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United States Belgium Germany Hong Kong Italy Japan Spain Taiwan United Kingdom

Other Identifiers

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2023-505043-39-00

Identifier Type: OTHER

Identifier Source: secondary_id

SRP-9001-305

Identifier Type: -

Identifier Source: org_study_id

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