Duchenne Muscular Dystrophy MedDRA version: 20.1 Level: PT Classification code 10052655 Term: Duchenne muscular dystrophy gene carrier System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: delandistrogene moxeparvovec
Product Code: RO7494222 (SRP-9001)
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: delandistrogene moxeparvovec
CAS Number: 2305040-16-6
Curr
Sponsors
F. Hoffmann-La Roche Ltd
Eligibility
Sex/Gender
Male
Inclusion criteria
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 =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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 •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 the opinion of the investigator, might compromise the participant’s ability to comply with the protocol-required testing or procedures, or compromise the participant’s well-being or safety, or clinical interpretability. •Left ventricular ejection fraction <50% on the screening echocardiogram (ECHO) or clinical signs and/or symptoms of cardiomyopathy •Known contact with acute or active hepatitis within 12 weeks or known contact with an infected person (e.g., suspected Epstein-Barr virus [EBV], Varicella zoster virus [VZV], parvovirus B19, human herpes virus 6, and cytomegalovirus [CMV]) within 6 weeks prior to Day 1 •Symptomatic infection (e.g., upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks prior to Day 1. •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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety of delandistrogene moxeparvovec;Secondary Objective: To evaluate SRP-9001-dystrophin expression from delandistrogene moxeparvovec at 12 weeks as measured by Western blot of biopsied muscle tissue;Primary end point(s): •Incidence of treatment-emergent adverse events •Incidence of serious adverse events •Incidence of adverse events of special interest •Clinically significant changes in vital signs and physical examination findings •Clinically significant changes in safety laboratory assessments, ECGs, and ECHOs ;Timepoint(s) of evaluation of this end point: Throughout the study | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Change in quantity of SRP-9001-dystrophin protein expression from baseline to Week 12 as measured by Western blot;Timepoint(s) of evaluation of this end point: At baseline and at Week 12 | — |
Countries
Belgium, France, Germany, Italy, Spain, United Kingdom
Contacts
Public ContactTrial Information Support Line-TISL
F. Hoffmann-La Roche Ltd
Outcome results
None listed