History of Changes for Study: NCT06128564

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) (ENVOL)

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Study Record Versions

Version	Submitted Date	Changes
1	November 8, 2023	None (earliest Version on record)
2	December 11, 2023	Recruitment Status, Study Status and Contacts/Locations
3	January 10, 2024	Study Status
4	February 6, 2024	Contacts/Locations and Study Status
5	March 5, 2024	Study Status
6	April 3, 2024	Study Status
7	April 30, 2024	Contacts/Locations and Study Status
8	May 28, 2024	Study Status

Comparison Format:

Merged
Side-by-Side

Study Identification


Unique Protocol ID:	BN43881
Brief Title:	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) (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
Secondary IDs:	2022-000691-19 [EudraCT Number]

Study Status


Record Verification:	November 2023
Overall Status:	Not yet recruiting
Study Start:	November 16, 2023
Primary Completion:	November 30, 2032 [Anticipated]
Study Completion:	November 30, 2032 [Anticipated]

First Submitted:	November 8, 2023
First Submitted that Met QC Criteria:	November 8, 2023
First Posted:	November 13, 2023 [Actual]

Last Update Submitted that Met QC Criteria:	November 8, 2023
Last Update Posted:	November 13, 2023 [Actual]

Sponsor/Collaborators


Sponsor:	Hoffmann-La Roche
Responsible Party:	Sponsor
Collaborators:	Sarepta Therapeutics, Inc.

Oversight


U.S. FDA-regulated Drug:	Yes
U.S. FDA-regulated Device:	No
Data Monitoring:	Yes

Study Description


Brief 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.
Detailed Description:

Conditions


Conditions:	Duchenne Muscular Dystrophy
Keywords:	DMD Duchenne Dystrophin Gene-Delivery Pediatric

Study Design


Study Type:	Interventional
Primary Purpose:	Treatment
Study Phase:	Phase 2
Interventional Study Model:	Single Group Assignment
Number of Arms:	1
Masking:	None (Open Label)
Allocation:	N/A
Enrollment:	21 [Anticipated]

Arms and Interventions

Arms	Assigned Interventions
Experimental: Delandistrogene Moxeparvovec Participants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1.	Genetic: delandistrogene moxeparvovec Single IV infusion of delandistrogene moxeparvovec Other Names: SRP-9001 ELEVIDYS RO7494222 delandistrogene moxeparvovec-rokl

Outcome Measures


Primary Outcome Measures:
1.	Percentage of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI) [ Time Frame: Baseline up to Week 260 ]
Secondary Outcome Measures:
1.	Change in Quantity of Delandistrogene Moxeparvovec Dystrophin as Measured by Western Blot [ Time Frame: Baseline, Week 12 ]

Eligibility


Minimum Age:
Maximum Age:	3 Years
Sex:	Male
Gender Based:
Accepts Healthy Volunteers:	No
Criteria:	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

Contacts/Locations


Central Contact Person:	Reference Study ID Number: BN43881 https://forpatients.roche.com/ Telephone: 888-662-6728 (U.S. and Canada) Email: global-roche-genentech-trials@gene.com
Study Officials:	Clinical Trials Study Director Hoffmann-La Roche
Locations:

IPDSharing


Plan to Share IPD:	Yes Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/). For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/innovation/process/clinical-trials/data-sharing/).
	Supporting Information:
	Time Frame:
	Access Criteria:
	URL:

References


Citations:
Links:
Available IPD/Information: