Clinical features of Friedreich ataxia.
Martin B Delatycki, Louise A Corben
https://pubmed.ncbi.nlm.nih.gov/22752493Actively Recruiting
Phase 2
Age: 2Years - 60Years
All Genders
ID06447025
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
Led by Larimar Therapeutics, Inc. · Updated on 2026-05-13
85
Participants Needed
8
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the long-term safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and clinical effects of CTI-1601, a recombinant fusion protein designed to deliver human frataxin, in people with Friedreich's ataxia (FRDA). The study includes children aged 2 years and older, adolescents, and adults with FRDA, including those who have or have not participated in previous CTI-1601 studies. This open-label Phase 2 study aims to assess tissue frataxin levels, clinical evaluations, gene expression, and select lipids related to FRDA. Participants receive daily subcutaneous injections of CTI-1601. Adults 18 years and older receive a fixed 50 mg dose, while children and adolescents aged 2 to 17 years receive a weight-based dose of 0.8 mg/kg, up to a maximum of 50 mg. The study includes long-term treatment administration and monitoring to evaluate safety and clinical effects. During the study, participants will undergo various assessments including motor function tests, neurological evaluations using the modified Friedreich's Ataxia Rating Scale (mFARS), fatigue scales, daily living activities, and safety measures like electrocardiograms and echocardiograms. Researchers will also measure frataxin concentrations from cheek swabs and skin biopsies. The study will monitor treatment-emergent adverse events and other clinical outcomes over a period of up to 24 months.
CONDITIONS
Brief Title
An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia
Who Can Participate
Age: 2Years - 60Years
All Genders
Eligibility Criteria
You may qualify if you...
- Subjects with Friedreich's ataxia who have or have not completed a prior CTI-1601 study, unless they had serious or significant adverse events or withdrew from the previous study
- HbA1c less than or equal to 7.0%
- Ability to prepare and self-administer daily subcutaneous CTI-1601 injections or have a committed caregiver to do so
- Stable doses of permitted medications for at least 28 days before screening; niacin and resveratrol stable for 90 days
- If receiving omaveloxolone, must have used CTI-1601 or omaveloxolone alone for at least 3 months before starting the other
You will not qualify if you...
- Confirmed compound heterozygous status for Friedreich's ataxia (GAA repeat on only one allele)
- Any condition or disease, including heart conditions, that could affect study results or pose risk
- Use of investigational drugs or devices other than CTI-1601 within 90 days before screening
- Requirement for amiodarone use
- Use of erythropoietin, etravirine, or gamma interferon within 90 days before screening
- Biotin supplementation exceeding 30 mcg/day within 7 days before first dose; stable low-dose biotin allowed
- Use of more than 3 grams of acetaminophen daily
- Use of medications requiring subcutaneous injection in abdomen or thigh
- Unstable medication doses within 28 days before screening
- Screening echocardiogram showing left ventricular ejection fraction less than 45%
- Prolonged QTcF interval on ECG (above 450 ms for males, above 470 ms for females)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - Up to 24 months
Participants receive daily subcutaneous injections of CTI-1601 to evaluate long-term safety, tolerability, and clinical effects.
Regular visits for drug administration and assessments throughout treatment
Trial Site Locations
Total: 8 locations
1
University of California Los Angeles
Los Angeles, California, United States, 90095
Actively Recruiting
2
Fixel Institute for Neurological Disease, University of Florida Health
Gainesville, Florida, United States, 32608
Active, Not Recruiting
3
Morsani Center for Advanced Health Care, University of South Florida Health
Tampa, Florida, United States, 33612
Actively Recruiting
4
University of Iowa
Iowa City, Iowa, United States, 52242
Actively Recruiting
5
Uncommon Cures
Chevy Chase, Maryland, United States, 20815
Actively Recruiting
6
Clinilabs Drug Development, Corp.
Eatontown, New Jersey, United States, 07724
Actively Recruiting
7
Ohio State University United States
Columbus, Ohio, United States, 43210
Active, Not Recruiting
8
Children's Hospital of the University of Pennsylvania (CHOP)
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
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Research Team
L
Larimar Therapeutics, Inc.
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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