Trial Radar AI | ||
|---|---|---|
Clinical Trial NCT06447025 for Friedreich Ataxia is recruiting. See the Trial Radar Card View and AI discovery tools for all the details. Or ask anything here. | ||
One study matched filter criteria
Card View
Back to Search
Larimar Therapeutics, Inc.An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia Phase 2 85 Adolescent Open-Label
Clinical Trial NCT06447025 is designed to study Treatment for Friedreich Ataxia. It is a Phase 2 interventional study that is recruiting, having started on 25 January 2024, with plans to enroll 85 participants. Led by Larimar Therapeutics, Inc., it is expected to complete by 1 January 2027. The latest data from ClinicalTrials.gov was last updated on 24 March 2026.
Brief 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 ev...
Official Title
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
Conditions
Friedreich AtaxiaPublications
Scientific articles and research papers published about this clinical trial:Other Study IDs
- CLIN-1601-201
NCT ID Number
Start Date (Actual)
2024-01-25
Last Update Posted
2026-03-24
Completion Date (Estimated)
2027-01
Enrollment (Estimated)
85
Study Type
Interventional
PHASE
Phase 2
Status
Recruiting
Primary Purpose
Treatment
Design Allocation
N/A
Interventional Model
Single Group
Masking
None (Open Label)
Arms / Interventions
| Participant Group/Arm | Intervention/Treatment |
|---|---|
ExperimentalCTI-1601 Once daily subcutaneous injection of 50 mg CTI-1601 in subjects ≥ 18 years of age or a weight-based dose of 0.8 mg/kg up to a maximum of 50 mg in subjects ≥ 12 to 17 years of age. | 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 |
Primary Outcome Measures
| Outcome Measure | Measure Description | Time Frame |
|---|---|---|
Number of subjects with treatment-emergent adverse events (TEAEs) by System Organ Class (SOC), Preferred Term (PT) and Maximum Severity | Number of subjects | Up to 24 months |
Change from baseline in electrocardiogram (ECG) parameters including, but not limited to, HR, RR interval, PR interval, QRS duration, QT interval, and QTcF interval | Number change in ECG parameters | Up to 24 months |
Change from baseline in left ventricular ejection fraction (LVEF) | LVEF indicates the percentage of change in LV volume from diastole to systole that measures how well the left ventricle of the heart pumps blood. | Up to 24 months |
Change from baseline in left ventricular end-diastolic volume (LVEDV) | LVEDV is the amount of blood, measured in milliliters (mL), in the heart's left ventricle just before the heart contracts. | Up to 24 months |
Number of subjects with any suicidal ideation or behavior (Categories 1-10) of the Columbia Suicide Severity Rating Scale (C-SSRS) | The Columbia Suicide Severity Rating Scale (C-SSRS) is a tool used to assess the occurrence, severity, and frequency of suicidal thoughts and behaviors. A higher score on the C-SSRS generally indicate a worse outcome, as they signify a higher level of suicidal ideation or behavior. | Up to 24 months |
Change from baseline at each collection timepoint in tissue frataxin concentrations normalized to total protein observed in buccal cells collected from cheek swabs and skin cells collected from skin punch biopsies | Up to 24 months | |
Change from baseline in motor function as assessed by 9-hole peg test (9-HPT) | Up to 24 months | |
Change from baseline in motor function as assessed by the timed 25-foot walk test (T25-FW) | Up to 24 months | |
Change from baseline in neurologic function as assessed by the modified Friedreich's Ataxia Rating Scale (mFARS) total score | The Modified Friedreich's Ataxia Rating Scale (mFARS) is a modified neurologic scale involving direct subject participation and targets specific areas impacted by Friedreich's ataxia (bulbar, upper limb, lower limb, and upright stability), with scores ranging from 0-67 points, with higher scores indicating a greater level of disability. | Up to 24 months |
Change from baseline in neurologic function as assessed by the upright stability subscale examination of the mFARS | The Upright Stability Subscale is an assessment of an individual's ability to maintain balance and stability while standing upright. It has a minimum value of 0 and a maximum value of 36. A higher score indicates a better outcome, reflecting greater stability and balance abilities while standing upright. | Through study completion, up to 24 months |
Change in activities of daily living (ADLs) as assessed by the Friedreich's Ataxia Rating Scale Activities of Daily Living (FARS_ADL) | The FARS\_ADL, scored 0 to 36, is a subscale of FARS assessing a subject's ability to complete activities of daily living. A higher score indicates a greater level of disability. The FARS\_ADL questionnaire will be performed at the timepoints indicated in protocol. | Up to 24 months |
Change from baseline in total fatigue score and all the subscale scores as assessed by the Fatigue Impact Scale (MFIS) | The Modified Fatigue Impact Scale (MFIS) is a revised form of the Fatigue Impact Scale based on items derived from interviews with MS patients concerning how fatigue impacts their lives. This instrument provides an assessment of the effects of fatigue in terms of physical, cognitive, and psychosocial functioning. Participants rate on a 5-point scale, with 0 = 'Never' to 4 = 'Almost always' their agreement with 21 statements. Total score (0-84) and subscales for physical (0-36), cognitive (0-40) and psychosocial functioning (0-8). The 5-item version is scored (0-20). Higher numbers indicate greater fatigue. The MFIS will be performed at the timepoints indicated in protocol. | Up to 24 months |
Change from baseline in the assessment of disease as assessed by the Functional Staging for Ataxia | Up to 24 months | |
Overall impression of change as assessed by the patient using the Patient Global Impression of Change (PGI-C) Scale | The Patient Global Impression of Change (PGI-C) reflects a patient's assessment about the efficacy of treatment. PGIC is a 7 point scale depicting a patient's rating of overall improvement. Patients rate their change as "very much improved," "much improved," "minimally improved," "no change," "minimally worse," "much worse," or "very much worse." The PGI-C will be performed at the timepoints indicated in protocol. | Up to 24 months |
Overall impression of change assessed by a clinician using the Clinical Global Impression of Change (CGI-C) | The Clinical Global Impression of Change (CGI-C) is an assessment to measure change in clinical status (symptoms and functional ability) of the subject's condition from baseline with study drug. CGI-C scores range from 1 (very much improved) through to 7 (very much worse). The CGI-C will be performed at the timepoints indicated in protocol. | Up to 24 months |
Area under the concentration-time curve for the dosing interval (AUC0-tau) | Days 1, 30, 60, 90: pre-dose, 5, 15, 30 minutes after the dose, and 1, 2, 4, 6, 8 hours after the dose; Day 180: pre-dose and 5, 15 minutes after the dose; Days 270, 360, Q3M thereafter: pre-dose; through study completion, up to 24 months | |
Area under the concentration-time curve from time 0 to the time of last quantifiable concentration (AUC0-t) | Days 1, 30, 60, 90: pre-dose, 5, 15, 30 minutes after the dose, and 1, 2, 4, 6, 8 hours after the dose; Day 180: pre-dose and 5, 15 minutes after the dose; Days 270, 360, Q3M thereafter: pre-dose; through study completion, up to 24 months | |
Mean maximum observed concentration (Cmax) | Days 1, 30, 60, 90: pre-dose, 5, 15, 30 minutes after the dose, and 1, 2, 4, 6, 8 hours after the dose; Day 180: pre-dose and 5, 15 minutes after the dose; Days 270, 360, Q3M thereafter: pre-dose; through study completion, up to 24 months | |
Mean time of maximum observed concentration (Tmax) | Days 1, 30, 60, 90: pre-dose, 5, 15, 30 minutes after the dose, and 1, 2, 4, 6, 8 hours after the dose; Day 180: pre-dose and 5, 15 minutes after the dose; Days 270, 360, Q3M thereafter: pre-dose; through study completion, up to 24 months | |
Concentration reached immediately before the next dose is administered (Ctrough) | Days 1, 30, 60, 90: pre-dose, 5, 15, 30 minutes after the dose, and 1, 2, 4, 6, 8 hours after the dose; Day 180: pre-dose and 5, 15 minutes after the dose; Days 270, 360, Q3M thereafter: pre-dose; through study completion, up to 24 months |
Participation Assistant
Eligibility Criteria
Eligible Ages
Child, Adult
Minimum Age
12 Years
Eligible Sexes
All
- Subjects with FRDA who have or have not previously completed participation in a study of CTI-1601 are eligible to participate in this study unless the subject experienced one or more of the following in a previous CTI-1601 study: a) serious adverse event (SAE) related to study drug; b) significant AE, defined as Grade 3 or higher according to the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0 (or higher), related to study drug; c) some other event, related to participation in a previous study with CTI-1601, that supports the exclusion of the subject from participating in this study as determined by the Sponsor (i.e., an AE considered clinically significant by the Sponsor regardless of whether it met SAE criteria and regardless of CTCAE grade); d) Withdraw from participation in a previous study of CTI-1601 for any reason.
- Subject has a HbA1c less than or equal to 7.0%.
- Subject must demonstrate sufficient dexterity and visual acuity to prepare and self-administer SC injections of CTI-1601 QD or is able to identify a caregiver who will be trained and committed to prepare and administer the daily injections.
If subject is taking permitted concomitant medication(s), subject must have been on a stable dose and frequency of medication(s) over the past 28 days prior to the initiation of Screening; however, subjects taking niacin and resveratrol must have been on a stable dose and frequency for 90 days prior to the initiation of Screening
- Subjects who are currently receiving omaveloxolone or intend to receive omaveloxolone are permitted in the study but must either receive CTI-1601 for 3 months prior to their first dose of omaveloxolone or receive omaveloxolone for 3 months prior to their first dose of CTI-1601.
Subjects are excluded from the study if any of the following exclusion criteria are met:
- Subjects who are confirmed as compound heterozygous (GAA repeat expansion on only one allele) for FRDA.
- Subject has any condition, disease, or situation, including a cardiac condition or disease, that in the opinion of the PI, could confound the results of the study or put the subject at undue risk, making participation inadvisable.
- Subject used any investigational drug (other than CTI-1601) or device within 90 days prior to Screening.
- Subject requires use of amiodarone.
- Subject used erythropoietin, etravirine, or gamma interferon within 90 days prior to Screening.
- Subject use of biotin supplementation that exceeds 30 mcg/day, either as part of a multivitamin or as a standalone supplement, within 7 days prior to the first dose of study drug. Biotin supplementation ≤30 mcg/day is permitted if taken at a stable dose and frequency for at least 28 days prior to Screening and there is a commitment from the subject to maintain the biotin dose throughout the study (due to interference with assays).
- Subject uses more than 3 grams of acetaminophen daily.
- Subject receives medication that requires SC injection in the abdomen or thigh.
- Subject is unable to discontinue medications that have not been at a stable dose and frequency for at least 28 days prior to Screening.
- Subject has a Screening echocardiogram (ECHO) LVEF < 45%.
- Male subject has a QTcF > 450 milliseconds or female subject has a QTcF > 470 milliseconds on an ECG.
Larimar Therapeutics, Inc.1 active studies to exploreStudy Central Contact
Contact: Larimar Therapeutics, Inc., 844-511-9056, [email protected]
8 Study Locations in 1 Countries
California
University of California Los Angeles, Los Angeles, California, 90095, United States
Recruiting
Florida
Fixel Institute for Neurological Disease, University of Florida Health, Gainesville, Florida, 32608, United States
Active, not recruiting
Morsani Center for Advanced Health Care, University of South Florida Health, Tampa, Florida, 33612, United States
Recruiting
Iowa
University of Iowa, Iowa City, Iowa, 52242, United States
Recruiting
Maryland
Uncommon Cures, Chevy Chase, Maryland, 20815, United States
Recruiting
New Jersey
Clinilabs Drug Development, Corp., Eatontown, New Jersey, 07724, United States
Recruiting
Ohio
Ohio State University United States, Columbus, Ohio, 43210, United States
Active, not recruiting
Pennsylvania
Children's Hospital of the University of Pennsylvania (CHOP), Philadelphia, Pennsylvania, 19104, United States
Recruiting