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A Study to Assess Nomlabofusp in Adolescents and Children With Friedreich's Ataxia
Sponsor: Larimar Therapeutics, Inc.
Summary
The goal of this clinical trial is to evaluate the safety and tolerability of nomlabofusp (CTI-1601) in adolescents and children with Friedreich's ataxia (FRDA).
Official title: A Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Nomlabofusp in Adolescents and Children With Friedreich's Ataxia
Key Details
Gender
All
Age Range
2 Years - 17 Years
Study Type
INTERVENTIONAL
Enrollment
18
Start Date
2024-12-06
Completion Date
2025-04-27
Last Updated
2026-07-10
Healthy Volunteers
No
Conditions
Interventions
Nomlabofusp
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.
Placebo
The placebo is a sterile, preservative-free, clear liquid for subcutaneous injection.
Locations (1)
Uncommon Cures
Chevy Chase, Maryland, United States