Clinical trial 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)
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Duchenne Muscular Dystrophy
Keywords: DMD
Duchenne
Dystrophin
Gene-Delivery
Pediatric
Lead sponsor: Hoffmann-La Roche
Sponsor class: INDUSTRY
Collaborators: name: Sarepta Therapeutics, Inc.
First posted: 2023-11-13
Enrollment: 21
Sex: MALE
Maximum age (years): 3
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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

Primary outcomes:
measure: Percentage of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)
timeFrame: Baseline up to Week 260
Secondary outcomes:
measure: Change in Quantity of Delandistrogene Moxeparvovec Dystrophin as Measured by Western Blot
timeFrame: Baseline, Week 12
Other outcomes:
Locations:
city: Liege
country: Belgium
facility: Chr de La Citadelle
city: Paris
country: France
facility: Hôpital Necker-Enfants Malades
city: Essen
country: Germany
facility: Universitätsklinikum Essen
city: Roma
country: Italy
facility: PU A. Gemelli, Università Cattolica del Sacro Cuore
state: Lazio
city: Esplugues De Llobregas
country: Spain
facility: Hospital Sant Joan De Deu
state: Barcelona
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children
city: Oxford
country: United Kingdom
facility: John Radcliffe Hospital
Study officials:
affiliation: Hoffmann-La Roche
name: Clinical Trials
role: STUDY_DIRECTOR
References: