The primary objective of this study is to evaluate acute liver injury (ALI) rates associated with ELEVIDYS with the addition of sirolimus as an adjunct prophylactic immunosuppression agent.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
20
Administered via an intravenous infusion.
Administered orally.
Administered orally.
Baylor College of Medicine
Houston, Texas, United States
Cohort 1: Number of Participants with ALI
Time frame: 12 weeks
Cohort 1: Number of Participants with Treatment-emergent Adverse Events (TEAEs), Adverse Events of Special Interest (AESIs) and Serious Adverse Events (SAEs)
Time frame: Day 1 up to Week 24
Cohort 1: Number of Participants with Infections, Edema, Wound-healing Complications, Hyperlipidemia, Angioedema, and Interstitial Lung Disease/Non-infectious Pneumonitis
Time frame: Day 1 up to Week 24
Cohort 1: Number of Participants with Hepatic Adverse Events, Hepatic Biomarkers, and Laboratory Assessments Indicative of Either Acute Hepatocellular Injury or Acute Liver Dysfunction
Time frame: Day 1 up to Week 24
Cohort 1: Number of Participants with Severe ALI
Time frame: Day 1 up to Week 24
Cohort 1: Number of Participants with ALI
Time frame: Day 1 up to Week 24
Cohort 1: Duration of ALI
Time frame: Day 1 up to Week 24
Cohort 1: Amount of Steroid Use
Time frame: Day 1 up to Week 24
Cohort 1: Duration of Steroid Use
Time frame: Day 1 up to Week 24
Cohort 1: Quantity of ELEVIDYS Dystrophin Protein Expression as Measured by Western Blot
Time frame: Week 12
Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4,
CONTACT
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Administered orally.