Friedreich AtaxiaPhase 2NCT06447025
An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia
A Phase 2 study testing CTI-1601 in people with Friedreich Ataxia, run by Larimar Therapeutics, Inc. at 11 sites in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 2 to 60 years
- Conditions the study is for
- Friedreich Ataxia
- What is being tested?
- CTI-1601
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 85 participants
- Where
- 11 sitesUnited States
- Study dates
- 25 Jan 2024to Jan 2027
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
11 sites in 1 country. Contact the study team or a site near you to ask about taking part.
11 sites in 1 country
United States11 sites, 8 recruiting
- Recruiting
Uncommon Cures
Chevy Chase, Maryland, 20815
Rachael Batabyal, MD, 240-858-4938, Rbatabyal@uncommoncures.com
Tam Roshan Lal, MB, ChB, 240-858-4912, troshanlal@uncommoncures.com
- Active, not recruiting
Ohio State University Wexner Medical Center
Columbus, Ohio, 43210
- Recruiting
Clinilabs Drug Development, Corp.
Eatontown, New Jersey, 07724
Recruiting Department, (212) 994-4567
- Active, not recruiting
Fixel Institute for Neurological Disease, University of Florida Health
Gainesville, Florida, 32608
- Recruiting
University of Iowa Hospitals & Clinics
Iowa City, Iowa, 52242
Corey McDaniel, BS, 319-335-7498, corey-mcdaniel@uiowa.edu
- Active, not recruiting
University of California Los Angeles
Los Angeles, California, 90095
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
David Lynch, MD, PhD, 215-590-2242, lynchd@pennmedicine.upenn.edu
McKenzie Wells, MS, 267-426-9608, wellsm@chop.edu
- Recruiting
ICON
Salt Lake City, Utah, 84124
801-269-8200
- Recruiting
University of California, San Franciso Medical Center
San Francisco, California, 94143
Ria Guerrero, (415) 514-4832, maria.guerrero2@ucsf.edu
- Not yet recruiting
University of Washington
Seattle, Washington, 98195
- Recruiting
Morsani Center for Advanced Health Care, University of South Florida Health
Tampa, Florida, 33612
Kayla Zayas, 813-974-5909, Kaylazayas@usf.edu
Lucretia Campbell, 813-974-5909, lcampbel@usf.edu
About this study
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
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.
Read the 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
- Being tested
- CTI-1601
- Medical subject headings
- Friedreich Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CTI-1601Drug
Also called Nomlabofusp
CTI-1601 is a recombinant fusion protein and is intended to deliver human frataxin, the protein deficient in patients with Friedreich's ataxia
| Study group | Role | What they receive |
|---|---|---|
| CTI-1601 | Experimental | 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 ≥ 2 to 17 years of age. Receives: Drug: CTI-1601 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of subjects with treatment-emergent adverse events (TEAEs) by System Organ Class (SOC), Preferred Term (PT) and Maximum Severity
Number of subjects
Measured: 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
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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
Measured: Up to 24 months
Change from baseline in motor function as assessed by 9-hole peg test (9-HPT)
Measured: Up to 24 months
Change from baseline in motor function as assessed by the timed 25-foot walk test (T25-FW)
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Up to 24 months
Change from baseline in the assessment of disease as assessed by the Functional Staging for Ataxia
Measured: 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.
Measured: 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.
Measured: Up to 24 months
Area under the concentration-time curve for the dosing interval (AUC0-tau)
Measured: 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)
Measured: 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)
Measured: 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)
Measured: 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)
Measured: 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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jun 2024
- Study start25 Jan 2024
- Primary completionJan 2027 (estimated)
- Study completionJan 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (10)Papers the sponsor has linked to this study.
- Other trials for Friedreich AtaxiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 85 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Larimar Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Larimar Therapeutics, Inc., Study chair, Larimar Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06447025
- Sponsor's protocol ID
- CLIN-1601-201
- Status last verified
- Oct 2026
- First submitted
- 15 Feb 2024
- Last updated
- 9 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Delatycki MB, Corben LA. Clinical features of Friedreich ataxia. J Child Neurol. 2012 Sep;27(9):1133-7. doi: 10.1177/0883073812448230. Epub 2012 Jun 29.
PubMed 22752493Deutsch EC, Santani AB, Perlman SL, Farmer JM, Stolle CA, Marusich MF, Lynch DR. A rapid, noninvasive immunoassay for frataxin: utility in assessment of Friedreich ataxia. Mol Genet Metab. 2010 Oct-Nov;101(2-3):238-45. doi: 10.1016/j.ymgme.2010.07.001. Epub 2010 Jul 8.
PubMed 20675166Fahey MC, Corben L, Collins V, Churchyard AJ, Delatycki MB. How is disease progress in Friedreich's ataxia best measured? A study of four rating scales. J Neurol Neurosurg Psychiatry. 2007 Apr;78(4):411-3. doi: 10.1136/jnnp.2006.096008. Epub 2006 Oct 20.
PubMed 17056635Fisk JD, Ritvo PG, Ross L, Haase DA, Marrie TJ, Schlech WF. Measuring the functional impact of fatigue: initial validation of the fatigue impact scale. Clin Infect Dis. 1994 Jan;18 Suppl 1:S79-83. doi: 10.1093/clinids/18.supplement_1.s79.
PubMed 8148458Goodkin DE, Hertsgaard D, Seminary J. Upper extremity function in multiple sclerosis: improving assessment sensitivity with box-and-block and nine-hole peg tests. Arch Phys Med Rehabil. 1988 Oct;69(10):850-4.
PubMed 3178453Koeppen AH. Friedreich's ataxia: pathology, pathogenesis, and molecular genetics. J Neurol Sci. 2011 Apr 15;303(1-2):1-12. doi: 10.1016/j.jns.2011.01.010.
PubMed 21315377Guidelines MSCfCP. Fatigue and multiple sclerosis: evidence-based management strategies for fatigue in multiple sclerosis. 1998.
Lazaropoulos M, Dong Y, Clark E, Greeley NR, Seyer LA, Brigatti KW, Christie C, Perlman SL, Wilmot GR, Gomez CM, Mathews KD, Yoon G, Zesiewicz T, Hoyle C, Subramony SH, Brocht AF, Farmer JM, Wilson RB, Deutsch EC, Lynch DR. Frataxin levels in peripheral tissue in Friedreich ataxia. Ann Clin Transl Neurol. 2015 Aug;2(8):831-42. doi: 10.1002/acn3.225. Epub 2015 Jul 1.
PubMed 26339677Pandolfo M. Friedreich ataxia. Arch Neurol. 2008 Oct;65(10):1296-303. doi: 10.1001/archneur.65.10.1296.
PubMed 18852343Rudick R, Antel J, Confavreux C, Cutter G, Ellison G, Fischer J, Lublin F, Miller A, Petkau J, Rao S, Reingold S, Syndulko K, Thompson A, Wallenberg J, Weinshenker B, Willoughby E. Clinical outcomes assessment in multiple sclerosis. Ann Neurol. 1996 Sep;40(3):469-79. doi: 10.1002/ana.410400321.
PubMed 8797541