Testing SAR446268 for myotonic dystrophy

Exclusion

• −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 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 with a known HIV infection
• −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:
• −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 InvestigatorPrincipal investigatorThe doctor or researcher responsible for running the study at a site.Read more →.
• −Participants at high risk for tuberculosis reactivation during the corticosteroids therapy as determined by the Investigator.
• −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 screeningScreeningThe checks done before joining, to see whether a study fits.Read more →.
• −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 trialInterventional studyA study where participants are given something to see what happens.Read more →.
• −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.