Friedreich AtaxiaPhase 3FORWARD-FANCT07778836
A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia
A Phase 3 study testing Nomlabofusp in people with Friedreich Ataxia and Friedreich's Ataxia, run by Larimar Therapeutics, Inc. at 4 sites in United States.
Trial summary
To evaluate the efficacy and safety of subcutaneous nomlabofusp in adult and pediatric subjects with Friedreich's ataxia Read more about this study
- Who is it for?
- Ages 12 to 40 years
- Conditions the study is for
- Friedreich Ataxia, Friedreich's Ataxia
- What is being tested?
- Nomlabofusp
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 150 participants
- Where
- 4 sitesUnited States
- Study dates
- 26 Oct 2026to Dec 2029
- Phase
- Phase 3
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 12 to 40 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Subjects who meet all of the following criteria are potentially eligible for study participation:
- Subject must provide genetically confirmed FRDA diagnosis report and is homozygous for GAA repeat expansions documented on the genetic diagnostic report, with repeat sizing (if available).
- Subject must complete 1 trial at Screening and 1 trial at Day -1 of the T25-FW test, using their customary assistive device (e.g., cane, 2 canes/crutches [Canadian crutches], wheeled walker/rollator or canine assistance) if needed. Each trial must be completed within 3 minutes.
- Subject must have the following at Screening and Day -1 per the following upright stability items from Module E of the mFARS:
- Item #E1, Sitting Posture - no more than a maximum score of 2
- Item #E2A, Stance Feet Apart - no more than a maximum score of 2 (average of 3 attempts)
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- Subject must have an mFARS score ≥ 20 and < 60 at Screening and Day -1.
- Subject must have a Functional Staging for Ataxia score of 4 or less at Screening.
- Subject demonstrates sufficient dexterity and visual acuity to prepare and self-administer SC injections of study drug daily (QD) or has an identified caregiver who will be trained and committed to prepare and administer the injections.
- Subject has a Screening HbA1c ≤ 7.0%.
- If the 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 initiation of Screening. Subjects taking niacin and resveratrol must have been on a stable dose and frequency for 90 days prior to initiation of Screening and subjects taking omaveloxolone must have been on a stable dose and frequency for 1 years prior to initiation of Screening.
Who cannot join
- Subjects are excluded from the study if any of the following exclusion criteria are met:
- Subject who is confirmed as compound heterozygous (GAA repeat expansion on only 1 allele) for FRDA.
- Subject previously participated in a clinical trial involving nomlabofusp. Participation is defined as the subject having signed the informed consent for the study and received at least 1 dose of study drug (nomlabofusp or placebo).
- The subject has any condition, disease, or situation that could confound the results of the study or put the subject at undue risk, making participation inadvisable in the opinion of the PI.
- Women of childbearing potential who are pregnant (have a positive pregnancy test at Screening or Day -1), lactating, or planning to attempt to become pregnant during this study or within 90 days after the last dose of study drug (this includes male subjects with partners of childbearing potential who are attempting to become pregnant).
- Subject used any investigational drug or device within 90 days prior to the initiation of Screening.
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- Subject previously received a gene therapy (investigational or approved) at any time in the past.
- Subject requires use of amiodarone.
- Subject used erythropoietin, etravirine, or gamma interferon within 90 days prior to the initiation of Screening.
- Subject's use of biotin supplementation exceeds 30 μg/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 μg/day is permitted if taken at a stable dose and frequency for at least 28 days prior to the initiation of Screening and there is a commitment from the subject to maintain the biotin dose throughout the study (due to interference with assays).
- Subject receives medication that requires SC injection in the abdomen or thigh.
- Subject has a Screening ECHO left ventricular ejection fraction < 45%.
- Subject has a QTcF on an ECG as specified below:
- For subjects ≥ 12 and < 18 years of age, a male or female subject with a QTcF > 460 ms.
- For subjects ≥ 18 years of age, a male subject with a QTcF > 450 ms or a female subject has a QTcF > 470 ms.
- Subject has suicidal ideation as determined by a "yes" to item #2 on the C-SSRS at Screening (within the last 28 days) or at Day -1.
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States4 sites, 4 recruiting
- Recruiting
Northwestern Medical Group, Department of Neurology
Chicago, Illinois, 60611
Rizwan S Akhtar, MD
Cynthia Poon, PhD, (312) 503-8216, cynthia.poon@northwestern.edu
- Recruiting
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
Vikram Shakkottai, MD, PhD
Sharon Primeaux, (214) 645-0563, Sharon.Primeaux@UTSouthwestern.edu
- Recruiting
Clinilabs
Eatontown, New Jersey, 07724
Magdy Shenouda, MD, (646) 215-6400, mshenouda@clinilabs.com
- Not yet recruiting
Morsani Center for Advanced Health Care, University of South Florida Health
Tampa, Florida, 33612
Samuel Garside, samuelg@usf.edu
About this study
Official title: A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia
To evaluate the efficacy and safety of subcutaneous nomlabofusp in adult and pediatric subjects with Friedreich's ataxia
Read the detailed description
This is a global, randomized, multi-center, placebo-controlled study to investigate the efficacy and safety of nomlabofusp in subjects with Friedreich's ataxia
The main objectives of the study are to evaluate:
- The efficacy of subcutaneous (SC) administration of nomlabofusp compared to placebo at Week 72; 1) on the Upright Stability Score (USS) Subscale E of the Modified Friedreich's Ataxia Ratings Scale (mFARS), or 2) mFARS total score (Region specific objective)
- The efficacy of SC administration of nomlabofusp compared to placebo at Week 72 on the Clinical Global Impression of Severity (CGI-S) score
- The safety and tolerability of SC administration of nomlabofusp
- Conditions
- Friedreich Ataxia, Friedreich's Ataxia
- Being tested
- Nomlabofusp, Placebo
- Keywords
- FA, FRDA
- Medical subject headings
- Friedreich Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NomlabofuspDrug
Also called CTI-1601
Nomlabofusp is a recombinant fusion protein provided in a sterile, preservative-free buffered solution for subcutaneous injection intended to deliver human frataxin, the protein deficient in Friedreich's ataxia.
PlaceboDrug
Also called CTI-1601 Placebo, Nomlabofusp Placebo
The placebo is a sterile, preservative-free, clear liquid for subcutaneous injection.
| Study group | Role | What they receive |
|---|---|---|
| Nomlabofusp | Experimental | Daily subcutaneous injection of nomlabofusp for 72 weeks Receives: Drug: Nomlabofusp |
| Placebo | Placebo comparator | Daily subcutaneous injection of placebo for 72 weeks Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in Upright Stability Score (USS) subscale E of modified Friedreich's Ataxia Rating Scale (mFARS) at Week 72 (Region-specific)
Number - The USS has a score range from 0 to 36. Lower scores mean less impairment and higher scores mean greater neurological impairment.
Measured: Baseline, Weeks 12, 24, 36, 48, 72
Change from baseline in the total modified Friedreich's Ataxia Rating Scale (mFARS) at Week 72 (Region-specific)
Number - The mFARS has a total score range from 0 to 93. Lower scores mean less impairment and higher scores mean greater neurological impairment.
Measured: Baseline, Weeks 12, 24, 36, 48, 72
Secondary outcomes (1)
Change from baseline in the Clinical Global Impression-Severity (CGI-S) score at Week 72
Number - The CGI-S is a 7-point scale. Lower scores mean less impairment and higher scores mean greater neurological impairment.
Measured: Baseline, Weeks 12, 24, 36, 48, 72
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Aug 2026
- Study start26 Oct 2026 (estimated)
- Primary completionJul 2029 (estimated)
- Study completionDec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (26)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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Larimar Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07778836
- Sponsor's protocol ID
- CLIN-1601-301
- Other IDs
- 2025-521628-31-00, 2025
- Acronym
- FORWARD-FA
- Status last verified
- Oct 2026
- First submitted
- 17 Aug 2026
- 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
Liu, C. (1993). "Bartlett's Decomposition of the Posterior Distribution of the Covariance for Normal Monotone Ignorable Missing Data." Journal of Multivariate Analysis 46:198-206.
Bauer P, Kohne K. Evaluation of experiments with adaptive interim analyses. Biometrics. 1994 Dec;50(4):1029-41.
PubMed 7786985Carpenter JR, Roger JH, Kenward MG. Analysis of longitudinal trials with protocol deviation: a framework for relevant, accessible assumptions, and inference via multiple imputation. J Biopharm Stat. 2013;23(6):1352-71. doi: 10.1080/10543406.2013.834911.
PubMed 24138436Delatycki 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 20675166Dmitrienko, A., Yuan, Y. (2017). Interim data monitoring. Analysis of Clinical Trials Using SAS: A Practical Guide (Second Edition). Dmitrienko, A., Koch, G.G. (editors). SAS Press: Cary, NC.
Fahey 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 8148458Gladman D, Nash P, Goto H, Birt JA, Lin CY, Orbai AM, Kvien TK. Fatigue numeric rating scale validity, discrimination and responder definition in patients with psoriatic arthritis. RMD Open. 2020 Jan;6(1):e000928. doi: 10.1136/rmdopen-2019-000928.
PubMed 31958274Goodkin 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 3178453Indelicato E, Nachbauer W, Eigentler A, Amprosi M, Matteucci Gothe R, Giunti P, Mariotti C, Arpa J, Durr A, Klopstock T, Schols L, Giordano I, Burk K, Pandolfo M, Didszdun C, Schulz JB, Boesch S; EFACTS (European Friedreich's Ataxia Consortium for Translational Studies). Onset features and time to diagnosis in Friedreich's Ataxia. Orphanet J Rare Dis. 2020 Aug 3;15(1):198. doi: 10.1186/s13023-020-01475-9.
PubMed 32746884Koeppen 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 21315377Lawerman TF, Brandsma R, Burger H, Burgerhof JGM, Sival DA; the Childhood Ataxia and Cerebellar Group of the European Pediatric Neurology Society. Age-related reference values for the pediatric Scale for Assessment and Rating of Ataxia: a multicentre study. Dev Med Child Neurol. 2017 Oct;59(10):1077-1082. doi: 10.1111/dmcn.13507. Epub 2017 Aug 17.
PubMed 28815574Lawrence, J. and Hung, H.M.J. (2003), Estimation and Confidence Intervals after Adjusting the Maximum Information. Biom. J., 45: 143-152. https://doi.org/10.1002/bimj.200390001
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 26339677