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Gene Therapy Trial Report

Summary

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


NCTID NCT06844214 (View at clinicaltrials.gov)
Description πŸ”„
Development Status Active
Indication Myotonic Dystrophy
Disease Ontology Term DOID:11722
Drug Product Name SAR446268
Sponsor Sanofi
Funder Type Industry
Recruitment Status
Enrollment Count 32 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant DMPK
Therapeutic Modality Gene transfer
Therapy Route in vivo
Mechanism of Action Gene inactivation
Route of Administration Intravenous
Drug Product Type Viral vector
Gene Delivery System Type Viral transduction
Vector Type πŸ”„ Adeno-associated virus
Viral Vector Subtype AAV.SAN011
Dose 1 3 cohort dose escalation, undisclosed concentrations

Study Record Dates


Current Phase Phase1, Phase2
Submit Date 2025-02-19
Completion Date πŸ”„ 2032-04-27
Last Update πŸ”„ 2026-09-21

Participation Criteria


Eligible Age πŸ”„ 10 Years - 55 Years
Standard Ages Child, Adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: 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 informed consent. * 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 55 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). Exclusion Criteria: 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: * ALT \>3 x ULN and AST \>3 x ULN * Alkaline phosphatase \>3 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 (2021) for those age ≥18 years and Bedside Schwartz Equation for those \<18 years * Participants with chronic respiratory insufficiency and on long term/hull-time ventilatory assistance requiring at least 6 hours per day for at least 21 consecutive days. * 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. * Participants who have been classified as severe cardiac risk by the Investigator. The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites πŸ”„ 10
Locations Canada,Argentina,United States,United Kingdom,Israel,Australia

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation
Recent Updates

Resources/Links