NCT07778836 · Larimar Therapeutics, Inc.
A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia
(FORWARD-FA)
What this study is about
To evaluate the effectiveness and safety of injected under the skin nomlabofusp in adult and pediatric subjects with Friedreich's ataxia
View original scientific description
To evaluate the efficacy and safety of subcutaneous nomlabofusp in adult and pediatric subjects with Friedreich's ataxia
Interventions
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.
DRUG
Placebo
The placebo is a sterile, preservative-free, clear liquid for subcutaneous injection.
Primary outcome measures
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.
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.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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)
- 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. Key
Exclusion criteria
- 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.
- 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.
Where
- Chicago, Illinois
- Eatontown, New Jersey
- Dallas, Texas
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Aug 21, 2026 · Source of record for eligibility and locations