NCT06681766

A Study to Assess Nomlabofusp in Adolescents and Children With Friedreich's Ataxia

A Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Nomlabofusp in Adolescents and Children With Friedreich's Ataxia

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 18 (actual)
• Sponsor: Larimar Therapeutics, Inc. · Industry
• Sex: All
• Age: 2 Years – 17 Years
• Healthy volunteers: Not accepted

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).

Detailed description

This is a randomized, double-blind, placebo-controlled study evaluating a weight-based dose of nomlabofusp versus placebo in adolescents and children with FRDA. The study will consist of at least two cohorts with at least 12 to 15 participants in each cohort. Participants will be dosed once daily (QD) for 7 days.

Conditions

• Friedreich Ataxia

Interventions

Type	Name	Description
DRUG	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.
DRUG	Placebo	The placebo is a sterile, preservative-free, clear liquid for subcutaneous injection.

Timeline

• Start date: 2024-12-06
• Primary completion: 2025-04-28
• Completion: 2025-04-28
• First posted: 2024-11-08
• Last updated: 2026-01-28

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study