Clinical trial NCT05967351: A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
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
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE3
Conditions: Duchenne Muscular Dystrophy
Keywords: Duchenne Muscular Dystrophy
DMD
Pediatric
Long-term
Follow-up
Safety
Efficacy
Lead sponsor: Sarepta Therapeutics, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Hoffmann-La Roche
First posted: 2023-08-01
Enrollment: 400
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

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.


Eligibility 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.

Primary outcomes:
measure: Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)
timeFrame: Up to 5 years after dosing
Secondary outcomes:
measure: Change in the North Star Ambulatory Assessment (NSAA) Total Score From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
timeFrame: Baseline, 5 years
measure: Change in Time to Rise From Floor From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
timeFrame: Baseline, 5 years
measure: Change in the Time of 10-meter Walk/Run (10MWR) From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
timeFrame: Baseline, 5 years
measure: Change in Performance of Upper Limb (PUL) (Version 2.0) Total Scores From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
timeFrame: Baseline, 5 years
measure: Change in PUL (Version 2.0) Domain Specific Scores From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
timeFrame: Baseline, 5 years
measure: Change in Forced Vital Capacity Percent (FVC%) Predicted From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
timeFrame: Baseline, 5 years
measure: Change in Peak Expiratory Flow Percent (PEF%) Predicted From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
timeFrame: Baseline, 5 years
measure: Change in Cardiac Magnetic Resonance Imaging (MRI) Findings From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
timeFrame: Baseline, 5 years
measure: Change in Musculoskeletal MRI Findings From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
timeFrame: Baseline, 5 years
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: Los Angeles
country: United States
facility: UCLA Medical Center
state: California
city: Palo Alto
country: United States
facility: Lucile Packard Children's Hospital Stanford (LPCH)
state: California
city: Sacramento
country: United States
facility: University of California, Davis
state: California
city: San Diego
country: United States
facility: Rady Children's Hospital
state: California
city: Aurora
country: United States
facility: Children's Hospital of Colorado
state: Colorado
city: Gainesville
country: United States
facility: University of Florida Clinical Research Center
state: Florida
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Iowa City
country: United States
facility: University of Iowa ICTS Clinical Research Unit
state: Iowa
city: Baltimore
country: United States
facility: The Johns Hopkins Hospital, Rubenstein Child Health Bldg
state: Maryland
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: New York
country: United States
facility: Columbia University Pediatric Neuromuscular Center
state: New York
city: Rochester
country: United States
facility: University of Rochester Medical Center
state: New York
city: Durham
country: United States
facility: Lenox Baker Children's Hospital (Duke University)
state: North Carolina
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
city: Philadelphia
country: United States
facility: Chiildren's Hospital of Philadelphia
state: Pennsylvania
city: Dallas
country: United States
facility: Children's Health Specialty Center
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah Health
state: Utah
city: Norfolk
country: United States
facility: Children's Hospital of The King's Daughters
state: Virginia
city: Milwaukee
country: United States
facility: Children's Hospital Wisconsin
state: Wisconsin
city: Gent
country: Belgium
facility: Universitair Ziekenhuis Gent, Neuromuscular Reference Centre (NMRC)
state: East Flanders
city: Essen
country: Germany
facility: Universitätsklinikum Essen - Klinik für Kinderheilkunde I
state: Nordrhein-Westfalen
city: Hong Kong
country: Hong Kong
facility: Hong Kong Children's Hospital
city: Genova
country: Italy
facility: UOC Neurologia Pediatrica e Malattie Muscolari, Istituto G. Gaslini, Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico
city: Milano
country: Italy
facility: UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
city: Roma
country: Italy
facility: UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gemelli IRCCS, Universita Cattoclica del Sacro Cuore
city: Kobe
country: Japan
facility: Kobe University Hospital
state: Hyogo
city: Kodaira
country: Japan
facility: National Center of Neurology and Psychiatry
state: Tokyo
city: Esplugues de Llobregat
country: Spain
facility: Hospital Sant Joan de Deu
state: Barcelona
city: Valencia
country: Spain
facility: Hospital Universitario y Politecnico La fe. Neurology Department
city: Kaohsiung
country: Taiwan
facility: Kaohsiung Medical University Chung-Ho Memorial Hospital
city: Taipei
country: Taiwan
facility: National Taiwan University Hospital
city: London
country: United Kingdom
facility: Great Ormond Street Hospital For Children NHS Foundation Trust
state: Greater London
city: Oxford
country: United Kingdom
facility: Oxford University Hospitals NHS Foundation Trust
state: Oxfordshire
city: Newcastle Upon Tyne
country: United Kingdom
facility: Institute of Translational and Clinical Research
Study officials:
References: