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A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort

ConditionDuchenne Muscular Dystrophy
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexMale
Age2+
SponsorSarepta Therapeutics, Inc.

About this trial

Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed.

This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with Duchenne Muscular Dystrophy (DMD). The maximum participant duration for this study is 156 weeks.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

For Cohorts 1-8: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.

Cohort 8: Non-ambulatory per protocol-specified criteria at the time of Screening, has a performance upper limb (PUL) entry item score ≥2 at the Screening visit and has a total PUL score of ≥12 and ≤40 at the time of Screening.

Cohorts 1, 2, 3, 5, 7 and 8 only: Stable dose equivalent of oral glucocorticoids for at least 12 weeks before screening and the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the first year of the study.

Cohort 1: Is ambulatory, and ≥4 to <8 years of age at the time of Screening.

Disqualifiers

Cohort 8: Any confounding factors that would prevent the use of oral sirolimus including a known hypersensitivity to sirolimus or any of its excipients.

Has a concomitant illness, autoimmune disease, chronic drug treatment, and/or cognitive delay/impairment that in the opinion of the Investigator creates unnecessary risks for gene transfer.

Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol-specified time limits.

Abnormality in protocol-specified diagnostic evaluations or laboratory tests.

Trial design

Design model

Single group

Treatments tested in this trial

  • delandistrogene moxeparvovec

    Genetic

    Single IV infusion of delandistrogene moxeparvovec

Treatment groups

83 Participants
are divided into 1 treatment group
Group A: Delandistrogene MoxeparvovecExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Part 1 (Cohorts 1 to 5): Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12, as Measured by Western Blot

Time frame
Baseline, Week 12
2

Part 1 (Cohorts 6 to 8): Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot

Time frame
Week 12
3

Cohort 8: Number of Participants with Acute Liver Injury (ALI)

Time frame
Baseline up to Week 72

Secondary outcomes

1

Vector Shedding, Measured in Urine, Saliva, and Stool Samples Post-Infusion

Time frame
Day 1 up to Week 104
2

Level of Antibody Titers to Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74)

Time frame
Day 2 up to Week 156
3

Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs)

Time frame
Baseline up to Week 156
4

Cohort 8: Number of Participants With Infections, Edema, Wound-healing Complications, Hyperlipidemia, Angioedema, and Intestinal Lung Disease/ Non-infectious Pneumonitis

Time frame
Baseline up to Week 72

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Sarepta Therapeutics, Inc.

Lead sponsor

Hoffmann-La Roche

Collaborator

Source ClinicalTrials.gov
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