At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- SAR446268 (biological)
- How long the study runs
- Study runs about 81 months (dates as stated)
- About the drug or intervention
- SAR446268 — biological: Pharmaceutical form: Solution for infusion; Route of administration: IV infusion
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Testing a treatment
- Ages
- 10 Years to 55 Years
- Who
- All
- Number of participants
- 32
- Started
- 2025-07-23
- Last checked
- 2026-09
Plain English Summary
What is this study?
- • Testing a new treatment for myotonic dystrophy
- • Phase1/Phase2 - 32 participants
- • This 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)
Who can take part?
- • Ages 10 Years to 55 Years
- • Diagnosed with myotonic dystrophy
Where?
- • Newcastle upon Tyne - Investigational Site Number : 8260002
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
This 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.
More detail
Each participant meeting the eligibility criteria for each of the study parts will receive a single dose administration of SAR446268.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 10 Years - 55 Years
- Who can join: All genders
What the study is looking for
- ✓Participants are eligible to be included in the study only if all of the following criteria apply:
- ✓For Part A, participants must be 18 to 55 years of age inclusive, at the time of signing the agreement to take part.
- ✓For Part B, participants must be as follows:
- ✓10 to 17 years of age inclusive, at the time of signing the agreement to take part or,
- ✓18 to 55 years of age inclusive, at the time of signing the agreement to take part.
Who cannot take part
- ✗Participants are excluded from the study if any of the following criteria apply:
- ✗Participants with neutralizing antibodies against the AAV.SAN011 capsid
- ✗Participants with left ventricular ejection fraction \<50%
- ✗Participants with liver or biliary disease defined as having at least one of the following:
- ✗liver enzymes \>3 x ULN and liver enzymes \>3 x ULN
See the full criteria
Where Is This Study? (1 UK site)
Investigational Site Number : 8260002
Newcastle upon Tyne NE7 7DN, United Kingdom
How to Get in Touch
Trial Transparency email recommended (Toll free for US & Canada)
Sponsor contactCONTACT
