NCT05579691

A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's Ataxia

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Dose Exploration Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CTI-1601 in Adult Subjects With Friedreich's Ataxia

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 28 (actual)
• Sponsor: Larimar Therapeutics, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

To evaluate the safety and tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of subcutaneous (SC) administration of CTI-1601 over 28 days in subjects with Friedreich's ataxia (FRDA).

Detailed description

This is a double-blind, placebo-controlled, study evaluating two doses (25 mg and 50 mg) of CTI-1601. This study will consist of at least 2 cohorts with 12 to 15 subjects participating in each cohort. Subjects will be dosed once daily (QD) for 14 days followed by dosing every other day (QOD) through Day 28.

Conditions

• Friedreich Ataxia

Interventions

Type	Name	Description
BIOLOGICAL	CTI-1601	CTI-1601 is a recombinant fusion protein and is intended to deliver human frataxin, the protein deficient in Friedreich's ataxia
OTHER	Placebo	Placebo Comparator

Timeline

• Start date: 2022-09-21
• Primary completion: 2023-12-04
• Completion: 2023-12-04
• First posted: 2022-10-14
• Last updated: 2024-11-29

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study