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

NCT06447025 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 85

Last updated 2026-05-13

No results posted yet for this study

Summary

An open label study designed to evaluate the safety, PK, PD, and clinical effects of long-term daily administration of CTI-1601 enrolling adolescent and adult patients with FRDA who have participated in a prior clinical study of CTI-1601 as well as children (age 2 years and older), adolescents and adults with FRDA who have not participated in a prior clinical study of CTI-1601.

Conditions

  • Friedreich Ataxia

Interventions

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

Sponsors & Collaborators

  • Larimar Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Larimar Therapeutics, Inc. · Larimar Therapeutics, Inc.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
60 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-01-25
Primary Completion
2027-01-31
Completion
2027-01-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06447025 on ClinicalTrials.gov