Phase 2 study for people with Muscular Dystrophy

Reproduced exactly, unedited. Written for researchers rather than for patients.

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.