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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
Led by Sanofi · Updated on 2026-04-28
32
Participants Needed
9
Research Sites
352 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants aged 18 to 55 years for Part A
- Participants aged 10 to 17 years or 18 to 55 years for Part B
- Participants with non-congenital onset DM1
- Previous clinical diagnosis of DM1 with signs including myotonia and muscle weakness
- Genetic diagnosis of DM1 with CTG repeat length 50 in one allele
- Ability to walk independently for at least 10 meters (orthoses and ankle braces allowed)
You will not qualify if you...
- Presence of neutralizing antibodies against the AAV.SAN011 capsid
- Left ventricular ejection fraction below 50%
- Liver or biliary disease defined by elevated liver enzymes or bilirubin beyond specified limits
- International normalized ratio above 1.5
- Renal disease defined by elevated serum creatinine or reduced estimated glomerular filtration rate
- Chronic respiratory insufficiency requiring long-term ventilatory support at least 6 hours daily for 21 consecutive days
- Contraindication to corticosteroids or conditions worsening with corticosteroids
- Active hepatitis B or C infection or current antiviral therapy for these infections
- HBcAb positive without ability for prophylactic or pre-emptive anti-HBV therapy
- High risk for tuberculosis reactivation during corticosteroid therapy
- Known HIV infection
- Serious intercurrent illness that may limit participation or survival
- Recent history or current drug or alcohol abuse in past 12 months
- Tibialis anterior biopsy within 12 weeks prior to Day 1 or planned biopsy during the trial
- Significant developmental delay, intellectual disability, or behavioral neuropsychiatric issues
- Prior systemic corticosteroid treatment over 5 mg/day within 15 days before Day 1
- Prior anti-myotonic medication treatment within 15 days before Day 1
- Unsuitability for participation due to medical, clinical, or compliance reasons
- Classified as severe cardiac risk by the Investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 9 locations
1
University of Florida, 2004 Mowry Road - Site Number: 8400005
Gainesville, Florida, United States, 32601
Actively Recruiting
2
University of South Florida - Neuromuscular Research, 13330 USF Laurel Drive - Site Number: 8400001
Tampa, Florida, United States, 33612
Actively Recruiting
3
Columbia University Medical Center - Neurological Institute, 710 W. 168th, 2nd floor, suite 204 - Site Number : 8400003
New York, New York, United States, 10032
Actively Recruiting
4
Virginia Commonwealth University Medical Center- Site Number : 8400006
Richmond, Virginia, United States, 23219
Actively Recruiting
5
Hospital Italiano de Buenos Aires, Juan Domingo Peron 4190 - Site Number: 0320001
Buenos Aires, Argentina, 1181
Actively Recruiting
6
Investigational Site Number : 0360001
Brisbane, Queensland, Australia, 4029
Actively Recruiting
7
The Montreal Neurological Institute and Hospital, 3801 rue University - Site Number: 1240001
Montreal, Quebec, Canada, H3A 2B4
Actively Recruiting
8
Investigational Site Number : 3760002
Ramat Gan, Israel, 5262100
Actively Recruiting
9
Investigational Site Number : 8260002
Newcastle upon Tyne, United Kingdom, NE7 7DN
Actively Recruiting
Research Team
T
Trial Transparency email recommended (Toll free for US & Canada)
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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