NCT06447025

An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia

An Open-Label Extension Study to Assess the Long-Term Safety, Efficacy, Pharmacodynamics, Pharmacokinetics, and Tolerability of Subcutaneous CTI-1601 in Subjects With Friedreich's Ataxia

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 85 (estimated)
• Sponsor: Larimar Therapeutics, Inc. · Industry
• Sex: All
• Age: 12 Years – 60 Years
• Healthy volunteers: Not accepted

Summary

An open label study designed to evaluate the safety, PK, PD, and clinical effects of long-term daily administration of CTI-1601 enrolling patients with FRDA who have participated in a prior clinical study of CTI-1601 as well as adolescents and adults with FRDA who have not participated in a prior clinical study of CTI-1601.

Detailed description

An open-label study designed to evaluate the long-term safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and clinical effects of subcutaneous (SC) administration of CTI-1601, also known as nomlabofusp, in subjects with Friedreich's ataxia (FRDA). The objectives of this study are: * To evaluate the safety and PK of long-term subcutaneous (SC) administration of CTI-1601 in subjects with FRDA * To evaluate the PD and clinical effects of long-term subcutaneous (SC) administration of CTI-1601 in subjects with FRDA on: * Tissue FXN concentrations * Clinical evaluations of FRDA * Gene expression and select lipids

Conditions

• Friedreich Ataxia

Interventions

Type	Name	Description
DRUG	CTI-1601	CTI-1601 is a recombinant fusion protein and is intended to deliver human frataxin, the protein deficient in patients with Friedreich's ataxia

Timeline

• Start date: 2024-01-25
• Primary completion: 2027-01-01
• Completion: 2027-01-01
• First posted: 2024-06-06
• Last updated: 2026-03-24

Locations

8 sites across 1 country: United States

Regulatory

• FDA-regulated drug study