Friedreich Ataxia, Friedreich's Ataxia
Conditions
Keywords
FA, FRDA
Brief summary
To evaluate the efficacy and safety of subcutaneous nomlabofusp in adult and pediatric subjects with Friedreich's ataxia
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
Interventions
DRUGNomlabofusp
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.
DRUGPlacebo
The placebo is a sterile, preservative-free, clear liquid for subcutaneous injection.
Sponsors
Larimar Therapeutics, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
12 Years to 40 Years
Healthy volunteers
No
Inclusion criteria
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline in Upright Stability Score (USS) subscale E of modified Friedreich's Ataxia Rating Scale (mFARS) at Week 72 (Region-specific) | 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) | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline in the Clinical Global Impression-Severity (CGI-S) score at Week 72 | 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. |
Countries
United States
Contacts
CONTACTLarimar Therapeutics, Inc.
Outcome results
None listed