This is a global, multi-center, randomized, placebo-controlled study to investigate the efficacy and safety of nomlabofusp in subjects with Friedreich’s ataxia. Nomlabofusp, previously called CTI-1601, is a recombinant fusion protein administered through subcutaneous (under the skin) injections intended to deliver human frataxin, the protein deficient in FA. Participants in this study will receive daily doses of nomlabofusp or placebo for 72 weeks.
The main objectives of the study are to evaluate:
- The efficacy of subcutaneous (SC) administration of nomlabofusp compared to placebo at Week 72 on
- 1. the Upright Stability Score (USS) Subscale 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
Anyone considering participating in a clinical trial should discuss the matter with their physician. FARA does not endorse or recommend any particular studies.
Study Details:
Key Inclusion Criteria:
- Genetically confirmed FA diagnosis with two GAA repeat expansions
- Ability to walk 25 feet within three minutes with or without an assistive device including a cane, crutches, walker, rollator, or canine assistance
- Able to self-administer injections of the study drug or has an identified caregiver to administer injections. Participants and caregivers will be trained to administer injections
- HbA1c less than or equal to 7.0%
- Has been taking a stable dose of permitted medications which includes a stable dose and frequency of omaveloxolone for at least one year prior to initiating screening
- Meets other study specific eligibility criteria as assessed by a study doctor
Key Exclusion Criteria:
- FA caused by compound heterozygous mutations (only one GAA repeat expansion)
- Previous participation in a nomlabofusp trial
- Use of investigational drug or device within 90 days of screening
- Previously received gene therapy
- Left ventricular fraction of less than 45% as measured by echocardiogram (ultrasound of the heart) at screening
- Other criteria assessed by a study doctor
Additional inclusion and exclusion criteria apply and will be evaluated by a study doctor.