A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 50 Years of Age With Non-congenital Myotonic Dystrophy Type 1
NCT ID: NCT06844214
Last Updated: 2026-02-02
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
32 participants
INTERVENTIONAL
2025-07-23
2029-02-28
Brief Summary
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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 4th 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 110 weeks (approximately 2 years) for each participant in Parts A and B respectively and includes a 6-week screening phase and a 104-week follow-up period post-SAR446268 administration.
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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SAR446268
Participants will receive a single dose of SAR446268 on Day 1
SAR446268
Pharmaceutical form: Solution for infusion; Route of administration: IV infusion
Interventions
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SAR446268
Pharmaceutical form: Solution for infusion; Route of administration: IV infusion
Eligibility Criteria
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Inclusion Criteria
* For Part B, participants must be as follows:
* 10 to 17 years of age inclusive, at the time of signing the informed consent or,
* 18 to 50 years of age inclusive, at the time of signing the informed consent.
* Participants with non-congenital onset DM1
* Participants presenting with signs of DM1 including myotonia and muscle weakness, as diagnosed previously by a clinician based on medical history.
* Participants with genetic diagnosis of DM1 \[cytosine-thymine-guanine (CTG) repeat length ≥50 in one allele from medical history\]
* Participants who can walk independently for at least 10 meters at screening (orthoses and ankle braces allowed).
* Participants who have been classified according to cardiac risk by the Investigator as:
* Moderate risk participants with pacemaker and/or implantable cardioverter-defibrillator (ICD) for Part A
* Low, moderate, or high cardiac risk for Part B
Exclusion Criteria
* Participants with left ventricular ejection fraction (LVEF) \<50%
* Participants with liver or biliary disease defined as having at least one of the following:
* Alanine aminotransferase (ALT) \>2 x ULN and aspartate aminotransferase (AST) \>2 x ULN
* Alkaline phosphatase \>2 x ULN
* Total bilirubin \>1.5 x ULN (unless has a genetically confirmed diagnosis of Gilbert's syndrome)
* Direct bilirubin ≥1.5 x ULN
* Participants with International normalized ratio \>1.5
* Participants with renal disease defined as:
• Serum creatinine \>1.5 x ULN and/or estimated glomerular filtration rate \<60 mL/min/1.73 m2 as determined by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) (2021) for those age ≥18 years and Bedside Schwartz Equation for those \<18 years
* Participants with chronic respiratory insufficiency and on non-invasive ventilatory support, nighttime ventilatory support or full-time ventilation.
* Participants with contraindication to corticosteroid or with conditions that could worsen in the presence of corticosteroids, as determined by the Investigator.
* Participants with active hepatitis B or C infection; HBsAg (+), or HCV RNA (+), or current antiviral therapy for either.
* Participants with HBcAb (+) who are not amenable for prophylactic anti-HBV therapy or pre-emptive therapy guided by serial HBV DNA monitoring during the corticosteroids therapy.
* Participants at high risk for tuberculosis reactivation during the corticosteroids therapy as determined by the Investigator.
* Participants with a known HIV infection
* Participants with serious intercurrent illness that, in the opinion of the Investigator, would preclude participation in the study or potentially decrease survival.
* Participants with recent history of or current drug or alcohol abuse in the past 12 months prior to screening.
* Participants with history of tibialis anterior biopsy within 12 weeks from Day 1 or planning to undergo tibialis anterior biopsies during the duration of this clinical trial.
* Participants with significant developmental delay, intellectual disability, or behavioral neuropsychiatric manifestations as determined by the Investigator.
* Participants with previous systemic corticosteroids treatment at doses of \>5 mg/day within 15 days of Day 1
* Participants with previous treatment with anti-myotonic medication within 15 days of Day 1
* Participants not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures.
The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
10 Years
50 Years
ALL
No
Sponsors
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Sanofi
INDUSTRY
Responsible Party
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Locations
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University of Florida, 2004 Mowry Road - Site Number: 8400005
Gainesville, Florida, United States
University of South Florida - Neuromuscular Research, 13330 USF Laurel Drive - Site Number: 8400001
Tampa, Florida, United States
Columbia University Medical Center - Neurological Institute, 710 W. 168th, 2nd floor, suite 204 - Site Number : 8400003
New York, New York, United States
Hospital Italiano de Buenos Aires, Juan Domingo Peron 4190 - Site Number: 0320001
Buenos Aires, , Argentina
Investigational Site Number : 0360001
Brisbane, Queensland, Australia
The Montreal Neurological Institute and Hospital, 3801 rue University - Site Number: 1240001
Montreal, Quebec, Canada
Investigational Site Number : 3760002
Ramat Gan, , Israel
Investigational Site Number : 8260002
Newcastle upon Tyne, , United Kingdom
Countries
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Central Contacts
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Trial Transparency email recommended (Toll free for US & Canada)
Role: CONTACT
Facility Contacts
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Role: primary
Related Links
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DFI17808 Plain Language Results Summary
Other Identifiers
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U1111-1290-9594
Identifier Type: REGISTRY
Identifier Source: secondary_id
DFI17808
Identifier Type: -
Identifier Source: org_study_id
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