Study to assess the safety and expression of RO7494222 (SRP-9001) in participants under the age of 4 with Duchenne muscular dystrophy

Recruitment & Eligibility

Status: ot Recruiting

Sex: Male

Target Recruitment: 17

Inclusion Criteria

Signed Informed Consent Form, Signed Assent Form when appropriate, as determined by patient's age and individual site and country standards, Male at birth, Meets the following age requirements at the time of study drug infusion: – For Cohort A: 3 years of age – For Cohort B: 2 years of age – For Cohort C: >6 months to <2 years of age – For Cohort D: =6 months of age Note: To ensure that cohort-specific age criteria are met at the time of dosing, at the time of signing the Informed Consent Form participants should be approximately 1 month younger than the maximum age to qualify for a cohort that is actively enrolling., Has a definitive diagnosis of DMD prior to screening based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test. Genetic report must describe a frameshift deletion, frameshift duplication, premature stop (nonsense), canonical splice site mutation, or other pathogenic variant in the DMD gene fully contained between exons 18 to 79 (inclusive) that is expected to lead to absence of dystrophin protein. – Mutations between or including exons 1-17 are not eligible. – In-frame deletions, in-frame duplications, and variants of uncertain significance (VUS) are not eligible., Able to cooperate with age-appropriate motor assessment testing in the opinion of the investigator., Has (a) parent(s) or legal guardian(s) who is (are) able to understand and comply with the study visit schedule and all protocol requirements.

Exclusion Criteria

Has elevated anti rAAVrh74 antibody titers as determined by an investigational Elecsys anti-rAAVrh74 antibody assay obtained within 31 days of the infusion day, Positive COVID-19 test (antigen or PCR) on Day 1 prior to infusion, Cohort A and B: Positive serology testing for HIV 1 and/or 2, hepatitis C, or hepatitis B, Cohort C and D: – Born prematurely (before completion of gestation at 37 weeks) or relevant pregnancy complications in the opinion of the investigator – Participant's mother: Serological evidence of current, chronic, or active HIV (1 and/or 2), hepatitis B, or hepatitis C infection – Participant's mother if breastfeeding: o Clinical signs of acute CMV infection with confirmation by CMV PCR (urine) o Clinically significant abnormal liver function (GGT, AST, ALT, ALP, total bilirubin, GLDH) indicative of infectious hepatitis o Clinically significant illness or acute infection indicative of hepatotropic virus infection (e.g., CMV, EBV, VZV, etc.) within 6 weeks prior to Day 1 o Known contact with an infected person with acute or active hepatitis within 12 weeks prior to Day 1, Demonstrates cognitive delay or impairment that could confound motor development in the opinion of the investigator., Treatment with any of the following therapies during the specified time periods: – Any time: o Gene therapy o Cell-based therapy (e.g., stem cell transplantation) o CRISPR/Cas9, or any other form of gene editing – Within 12 weeks of Day 1 and any time during the study: o Use of human growth factor or vamorolone – Within 6 months of Day 1 and any time during the study: o Any investigational medication o Any treatment designed to increase dystrophin expression (e.g., Translarna™, EXONDYS 51™, VILTEPSO™), Has received a live virus vaccine or mRNA vaccine within 4 weeks or inactive vaccine within 2 weeks of the Day 1 visit, or expects to receive a vaccination that cannot be reasonably delayed to accommodate concomitant corticosteroid administration during the first 3 months after Day 1., Has abnormal laboratory values considered clinically significant including but not limited to: – GGT >2 x upper limit of normal (ULN) – GLDH >ULN – Total bilirubin >ULN. o Elevations in total bilirubin confirmed to be due to Gilbert's syndrome are not exclusionary. – White blood cell count >18,500 per µl – Platelets = 150,000 per µl, In the opinion of the investigator, the participant is not likely to be compliant with the study protocol., Family does not want to disclose participant's study participation with general practitioner or primary care physician and other medical providers., Poor peripheral venous access, which, in the opinion of the investigator, will lead to difficulty in venipuncture for the purposes of protocol-mandated procedures., Receiving regular oral corticosteroids as a treatment for DMD or planning to receive oral corticosteroids as a treatment for DMD within 1 year of baseline., Major surgery within 3 months prior to Day 1 or planned surgery during Part 1 of the study., Any other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or infection or malignancy or concomitant illness or requirement for chronic drug treatment that creates unnecessary risks to participate in the study in the opinion of the investigator., Known hypersensitivity to delandistrogene moxeparvovec or any excipients of the formulation., Medical condition or extenuating circumstance that, in

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

Related Trials