A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia (FORWARD-FA)

August 27, 2026 updated by: Larimar Therapeutics, Inc.

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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

Study Type

Interventional

Enrollment (Estimated)

150

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Recruiting
        • Northwestern Medical Group, Department of Neurology
        • Contact:
          • Rizwan S Akhtar, MD
        • Contact:
    • New Jersey
      • Eatontown, New Jersey, United States, 07724
    • Texas
      • Dallas, Texas, United States, 75390
        • Recruiting
        • University of Texas Southwestern Medical Center
        • Contact:
          • Vikram Shakkottai, MD, PhD
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

Subjects who meet all of the following criteria are potentially eligible for study participation:

  1. 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).
  2. 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.

  3. Subject must have the following at Screening and Day -1 per the following upright stability items from Module E of the mFARS:

    1. Item #E1, Sitting Posture - no more than a maximum score of 2
    2. Item #E2A, Stance Feet Apart - no more than a maximum score of 2 (average of 3 attempts)
  4. Subject must have an mFARS score ≥ 20 and < 60 at Screening and Day -1.
  5. Subject must have a Functional Staging for Ataxia score of 4 or less at Screening.
  6. 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.
  7. Subject has a Screening HbA1c ≤ 7.0%.
  8. 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.

Key Exclusion Criteria

Subjects are excluded from the study if any of the following exclusion criteria are met:

  1. Subject who is confirmed as compound heterozygous (GAA repeat expansion on only 1 allele) for FRDA.
  2. 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).
  3. 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.
  4. 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).
  5. Subject used any investigational drug or device within 90 days prior to the initiation of Screening.
  6. Subject previously received a gene therapy (investigational or approved) at any time in the past.
  7. Subject requires use of amiodarone.
  8. Subject used erythropoietin, etravirine, or gamma interferon within 90 days prior to the initiation of Screening.
  9. 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).
  10. Subject receives medication that requires SC injection in the abdomen or thigh.
  11. Subject has a Screening ECHO left ventricular ejection fraction < 45%.

  12. Subject has a QTcF on an ECG as specified below:

    1. For subjects ≥ 12 and < 18 years of age, a male or female subject with a QTcF > 460 ms.
    2. For subjects ≥ 18 years of age, a male subject with a QTcF > 450 ms or a female subject has a QTcF > 470 ms.
  13. 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Nomlabofusp
Daily subcutaneous injection of nomlabofusp for 72 weeks
Drug: Nomlabofusp
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.
Other Names:
  • CTI-1601
Placebo Comparator: Placebo
Daily subcutaneous injection of placebo for 72 weeks
Drug: Placebo
The placebo is a sterile, preservative-free, clear liquid for subcutaneous injection.
Other Names:
  • CTI-1601 Placebo, Nomlabofusp Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from baseline in Upright Stability Score (USS) subscale E of modified Friedreich's Ataxia Rating Scale (mFARS) at Week 72 (Region-specific)
Time Frame: Baseline, Weeks 12, 24, 36, 48, 72
Number - The USS has a score range from 0 to 36. Lower scores mean less impairment and higher scores mean greater neurological impairment.
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)
Time Frame: Baseline, Weeks 12, 24, 36, 48, 72
Number - The mFARS has a total score range from 0 to 93. Lower scores mean less impairment and higher scores mean greater neurological impairment.
Baseline, Weeks 12, 24, 36, 48, 72

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from baseline in the Clinical Global Impression-Severity (CGI-S) score at Week 72
Time Frame: Baseline, Weeks 12, 24, 36, 48, 72
Number - The CGI-S is a 7-point scale. Lower scores mean less impairment and higher scores mean greater neurological impairment.
Baseline, Weeks 12, 24, 36, 48, 72

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Larimar Therapeutics, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

August 5, 2026

Primary Completion (Estimated)

July 1, 2029

Study Completion (Estimated)

December 1, 2029

Study Registration Dates

First Submitted

August 17, 2026

First Submitted That Met QC Criteria

August 18, 2026

First Posted (Actual)

August 21, 2026

Study Record Updates

Last Update Posted (Actual)

August 31, 2026

Last Update Submitted That Met QC Criteria

August 27, 2026

Last Verified

August 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CLIN-1601-301
  • 2025 (U.S. NIH Grant/Contract: Faculty of Social Sciences Scientific Grant at the University of Gdańsk)
  • 2025-521628-31-00 (Ctis)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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