Clinical trial NCT06270719: An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy
Official title: A Long-term Multicenter Prospective Observational Study Evaluating the Comparative Effectiveness and Safety of Sarepta Gene Transfer Therapy vs. Standard of Care in Participants With Duchenne Muscular Dystrophy Under Conditions of Routine Clinical Practice
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Duchenne Muscular Dystrophy
Keywords: Duchenne Muscular Dystrophy
DMD
Observational
Standard of Care
Phase 4
Lead sponsor: Sarepta Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2024-02-21
Enrollment: 500
Sex: MALE
Minimum age (years): 4
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: DMD participants within each prescriber's practice who either (1) have been prescribed delandistrogene moxeparvovec or (2) are receiving or have been prescribed to begin chronic glucocorticoid therapy in routine clinical practice, and who meet the study eligibility criteria, will be invited to enroll into the study and will be followed according to the protocol.

Brief summary: This is a multicenter, prospective, observational Phase 4 study in the United States. The study is designed to collect both medical history and prospective data on Duchenne muscular dystrophy (DMD) treatment outcomes in participants receiving delandistrogene moxeparvovec as part of clinical care, compared to participants with DMD receiving or prescribed to start chronic glucocorticoid treatment at baseline in routine clinical practice.


Eligibility criteria: Inclusion Criteria:

* Has a definitive diagnosis of DMD prior to Screening based on documentation of clinical findings and confirmatory genetic testing.
* Is currently receiving or has been prescribed to start chronic glucocorticoid therapy at the time of this observational study enrollment.

A participant recruited to Cohorts 1a or 2:

* Is at least 4 years of age at the time of enrollment.
* Is ambulatory per protocol specified criteria.

A participant recruited to Cohort 1b:

- Is non-ambulatory per protocol-specified criteria.

For Delandistrogene Moxeparvovec-treated Participants:

- Will be initiating usual care treatment with delandistrogene moxeparvovec at the time of study enrollment.

For Comparators:

- Is unexposed to DMD gene therapy at the time of study enrollment.

Exclusion Criteria:

* Has any deletion of exon 8 and/or exon 9 in the DMD gene.
* Is currently participating in any DMD interventional study at the time of this observational study enrollment.
* Has a medical condition or confounding circumstances (for example, prior traumatic limitation for mobility or significant behavioral comorbidity) that, in the opinion of the Investigator, might compromise:

  * The participant's ability to comply with the protocol-required procedures,
  * The participant's wellbeing or safety, and/or
  * The clinical interpretability of the data collected from the participant.

Other inclusion/exclusion criteria may apply.

Primary outcomes:
measure: Mean Change From Baseline in Time to Walk/Run 10 Meters (10MWR) (Calculated Velocity) at Month 12
timeFrame: Baseline, Month 12
Secondary outcomes:
measure: Time to Rise From Floor (Supine to Stand)
timeFrame: Up to 10 years
measure: Loss of Ambulation (LOA)
timeFrame: Up to 10 years
measure: Performance of Upper Limb (PUL) Version 2.0 Entry Item A Score
timeFrame: Up to 10 years
measure: Patient-reported Outcomes Measurement Information (PROMIS) Domain Scores of Mobility, Upper Extremity and Fatigue
timeFrame: Up to 10 years
measure: Pulmonary Function, as Measured by Forced Vital Capacity (FVC) (% Predicted)
timeFrame: Up to 10 years
measure: Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF) as Measured by Echocardiogram (ECHO)
timeFrame: Up to 10 years
measure: Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs)
timeFrame: Up to 10 years
measure: Time to Walk/Run 10 Meters (Calculated Velocity)
timeFrame: Up to 10 years
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: Aurora
country: United States
facility: University of Colorado - PPDS
state: Colorado
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Miami
country: United States
facility: Nicklaus Children's Hospital
state: Florida
city: Orlando
country: United States
facility: Nemours Children's Hospital - Orlando
state: Florida
city: Chicago
country: United States
facility: Ann and Robert H Lurie Childrens Hospital of Chicago
state: Illinois
city: Indianapolis
country: United States
facility: Indiana Clinical and Translational Science Institute
state: Indiana
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Hershey
country: United States
facility: Penn State Health Milton S. Hershey Medical Center
state: Pennsylvania
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Fort Worth
country: United States
facility: Cook Children's Hospital
state: Texas
city: Norfolk
country: United States
facility: Children's Hospital of the King's Daughters
state: Virginia
city: Milwaukee
country: United States
facility: The Medical College of Wisconsin
state: Wisconsin
Study officials:
References: