A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy Clinical Trial

— ENVOL  

Official title:

A Two-Part, Open-Label Systemic Gene Delivery Study to Evaluate the Safety and Expression of RO7494222 (SRP-9001) in Subjects Under the Age of Four With Duchenne Muscular Dystrophy

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06128564
• Other study ID #: BN43881
• Secondary ID: 2022-000691-19
• Status: Recruiting
• Phase: Phase 2
• Start date: November 29, 2023
• Est. completion date: November 30, 2032

Study information

• Verified date: May 2024
• Source: Hoffmann-La Roche
• Contact: Reference Study ID Number: BN43881 https://forpatients.roche.com
• Phone: 888-662-6728 (U.S. and Canada)
• Email: global-roche-genentech-trials[@]gene.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 21
• Est. completion date: November 30, 2032
• Est. primary completion date: November 30, 2032
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A to 3 Years

Eligibility

Inclusion Criteria:

• Cohort A: >=3 years of age to <4 years of age

• Cohort B: >=2 years of age to <3 years of age

• Cohort C: >6 months to <2 years of age

• Cohort D: <=6 months of age

• 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

• Able to cooperate with age-appropriate motor assessment testing

• A pathogenic frameshift mutation or premature stop codon contained between exons 18 and 79 (inclusive)

Exclusion Criteria:

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

• Recombinant Adeno-Associated Virus Serotype rh74 (rAArh74) antibody titers are elevated, as per protocol-specified criteria

• 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

• Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer

• 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

Other inclusion or exclusion criteria could apply

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Genetic:
• delandistrogene moxeparvovec
Single IV infusion of delandistrogene moxeparvovec

Locations

Country	Name	City	State
Germany	Universitätsklinikum Essen	Essen
Spain	Hospital Sant Joan De Deu	Esplugues De Llobregas	Barcelona

Sponsors (2)

Lead Sponsor	Collaborator
Hoffmann-La Roche	Sarepta Therapeutics, Inc.

Countries where clinical trial is conducted

Germany,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percentage of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)		Baseline up to Week 260
Secondary	Change in Quantity of Delandistrogene Moxeparvovec Dystrophin as Measured by Western Blot		Baseline, Week 12