DFI17808 - BrAAVe
Research type
Research Study
Full title
A Phase 1/Phase 2 open-label single arm study with dose escalation (Part A), and dose expansion (Part B) parts to evaluate the safety, tolerability, and efficacy of SAR446268, an adeno-associated viral vector-mediated gene therapy in participants 10 to 50 years old with non-congenital myotonic dystrophy type 1
IRAS ID
1011635
Contact name
Patrick Maury
Contact email
Sponsor organisation
Sanofi-Aventis Recherche & Développement
Research summary
The DFI17808 study is a clinical trial designed to evaluate the safety, tolerability, and effectiveness of SAR446268, an experimental gene therapy, in treating individuals with non-congenital Myotonic Dystrophy Type 1 (DM1). DM1 is a rare genetic disorder caused by a mutation in the DMPK gene, leading to progressive muscle weakness, myotonia (delayed muscle relaxation), and other systemic complications.
SAR446268 is a gene therapy that uses an adeno-associated viral (AAV) vector to deliver a healthy copy of the DMPK gene to muscle cells. This approach aims to reduce the effects of the defective gene, restore normal protein function, and improve muscle and organ function in DM1 patients.
The study is structured in two parts:
* **Part A (Dose Escalation):** Participants receive increasing doses of SAR446268 to determine the highest dose that can be safely administered.
* **Part B (Dose Expansion):** Once a safe dose is identified, additional participants receive this dose to further assess its safety and effectiveness.
The trial is open-label and single-arm, meaning all participants receive the treatment without a placebo group. It is being conducted at multiple centers and involves participants aged 10 to 50 years with a confirmed genetic diagnosis of non-congenital DM1.
The results from this study could provide valuable insights into the potential of gene therapy as a treatment for DM1, a condition for which there are currently no effective therapies.
REC name
South Central - Oxford A Research Ethics Committee
REC reference
25/SC/0213
Date of REC Opinion
26 Sep 2025
REC opinion
Further Information Favourable Opinion