To evaluate the efficacy and safety of subcutaneous nomlabofusp in adult and pediatric subjects with Friedreich's ataxia
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
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
150
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.
The placebo is a sterile, preservative-free, clear liquid for subcutaneous injection.
Northwestern Medical Group, Department of Neurology
Chicago, Illinois, United States
RECRUITINGClinilabs
Eatontown, New Jersey, United States
RECRUITINGUniversity of Texas Southwestern Medical Center
Dallas, Texas, United States
RECRUITINGChange 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.
Time frame: 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.
Time frame: Baseline, Weeks 12, 24, 36, 48, 72
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.
Time frame: Baseline, Weeks 12, 24, 36, 48, 72
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