Larimar Therapeutics, Inc.
To evaluate the efficacy and safety of subcutaneous nomlabofusp in adult and pediatric subjects with Friedreich's ataxia
This is a global, randomized, multi-center, placebo-controlled study to investigate the efficacy and safety of nomlabofusp in subjects with Friedreich's ataxia The main objectives of the study are to evaluate: * The efficacy of subcutaneous (SC) administration of nomlabofusp compared to placebo at Week 72; 1) on the Upright Stability Score (USS) Subscale E of the Modified Friedreich's Ataxia Ratings Scale (mFARS), or 2) mFARS total score (Region specific objective) * The efficacy of SC administration of nomlabofusp compared to placebo at Week 72 on the Clinical Global Impression of Severity…
Key Inclusion Criteria: Subjects who meet all of the following criteria are potentially eligible for study participation: 1. Subject must provide genetically confirmed FRDA diagnosis report and is homozygous for GAA repeat expansions documented on the genetic diagnostic report, with repeat sizing (if available). 2. Subject must complete 1 trial at Screening and 1 trial at Day -1 of the T25-FW test, using their customary assistive device (e.g., cane, 2 canes/crutches \[Canadian crutches\], wheeled walker/rollator or canine assistance) if needed. Each trial must be completed within 3 minutes.…
Nomlabofusp is a recombinant fusion protein provided in a sterile, preservative-free buffered solution for subcutaneous injection intended to deliver human frataxin, the protein deficient in Friedreich's ataxia.
The placebo is a sterile, preservative-free, clear liquid for subcutaneous injection.
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