A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age12-40
SponsorLarimar Therapeutics, Inc.
About this trial
To evaluate the efficacy and safety of subcutaneous nomlabofusp in adult and pediatric subjects with Friedreich's ataxia
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
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)
Disqualifiers
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).
Trial design
Design model
Parallel
Treatments tested in this trial
Nomlabofusp
DrugNomlabofusp 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.
Placebo
DrugThe placebo is a sterile, preservative-free, clear liquid for subcutaneous injection.
Treatment groups
150 Participants
are divided into 2 treatment groupsGroup A: NomlabofuspExperimental treatment 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
Change 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, 722
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, 72Secondary outcomes
1
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, 72Sponsors and contacts
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Source ClinicalTrials.gov