Calendario›SNY›SAR446268 · BrAAVe
SAR446268 · BrAAVe
A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1
Ensayo de Sanofi en Myotonic Dystrophy (del registro, en inglés).
- Fase
- Fase 1/2
- Estado
- Reclutando pacientes
- Participantes
- 32
- Centros
- 9
- Fin del objetivo primario
- feb 2029
previstos
estimado por el promotor
Qué significa cada fase y cada estado.
Estudio de intervención, de un solo grupo, abierto. Comenzó en jul 2025.
Qué mide
Part A and Part B: Incidence of treatment-emergent adverse events (TEAEs) following SAR446268 administration (del registro, en inglés) (del inicio a la semana 52)
Cómo lo describe el promotor
En el documentoEn inglés, del registroThis is a Phase 1/Phase 2 open-label single arm, multicenter, and multinational study with SAR446268 for treatment of male and female participants 10 to 55 years old with non-congenital myotonic dystrophy (DM) type 1 (DM1). The purpose of this study is to evaluate the safety and efficacy of SAR446268 in knocking down dystrophia myotonica protein kinase (DMPK) messenger ribonucleic acid (mRNA) levels and improving neuromuscular function in DM1 participants receiving a single intravenous (IV) administration of SAR446268. The study consists of a dose escalation part (Part A) during which single ascending doses of SAR446268 will be evaluated in 3 distinct cohorts and an optional fourth dose cohort. Once a safe and effective dose is identified, additional participants will be treated in Part B, the dose expansion phase of the study. The study duration will be 112 weeks (approximately 2 years) for each participant in Parts A and B respectively and includes an optional pre-screening period, approximately 8-week screening phase and a 104-week follow-up period post-SAR446268 administration.
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Todos los de SNY, en su ficha.
Ficha completa en ClinicalTrials.gov (NCT06844214), actualizada en may 2026.