Friedreich Ataxia
Conditions
Brief summary
To evaluate the safety and tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of subcutaneous (SC) administration of CTI-1601 over 28 days in subjects with Friedreich's ataxia (FRDA).
Detailed description
This is a double-blind, placebo-controlled, study evaluating two doses (25 mg and 50 mg) of CTI-1601. This study will consist of at least 2 cohorts with 12 to 15 subjects participating in each cohort. Subjects will be dosed once daily (QD) for 14 days followed by dosing every other day (QOD) through Day 28.
Interventions
BIOLOGICALCTI-1601
CTI-1601 is a recombinant fusion protein and is intended to deliver human frataxin, the protein deficient in Friedreich's ataxia
OTHERPlacebo
Placebo Comparator
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
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Subject has a genetically confirmed diagnosis of FRDA manifested by homozygous GAA repeat expansions, with repeat sizing (if available) included on the diagnosis report. 2. Subject is biologically male or female, 18 years of age or older at screening. 3. Subject must have a mFARS score ≥ 20 and be able to traverse a distance of 25 feet with or without some assistive device (e.g., cane, walker, crutches, self-propelled wheelchair), and (a) be able to sit upright with thighs together and arms crossed without requiring support on more than two sides; (b) be able to transfer from bed to chair independently or with assistance if, in the opinion of the PI, the degree of physical disability does not result in undue risk to the subject while participating in the study; and (c) perform basic daily care, such as feeding themselves and personal hygiene, with minimal assistance. 4. Subject must weigh \> 40.0 kg.
Exclusion criteria
Subjects are excluded from the study if any of the following
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants with Treatment Emergent Adverse Events | Through study completion, an average of 93 days | Overall summary of Participants with Treatment Emergent Adverse Events |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Area under the concentration time curve (AUC) of CTI-1601 from time 0 through the last measurable time point | At baseline and up to 29 days | Summary assessment of changes in the AUC of CTI-1601 from time 0 to the last measurable time point and during the dosing interval |
| Time to maximum observed plasma concentration (tmax) of CTI-1601 after multiple doses | At baseline and up to 29 days | Summary assessment of the time to maximum observed plasma concentration (tmax) of CTI-1601 after multiple doses |
| Maximum observed plasma concentration (Cmax) of CTI-1601 after multiple doses | At baseline and up to 29 days | Summary assessment of changes in the maximum observed plasma concentration (Cmax) of CTI-1601 after multiple doses |
| Changes from baseline in frataxin levels in buccal cells | At baseline and up to 58 days | Summary assessment of changes in frataxin levels in buccal cells |
| Changes from baseline in frataxin levels in skin punch cells | At baseline and up to 29 days | Summary assessment of changes in frataxin levels in skin punch cells |
| Time to last observed plasma concentration (tlast) of CTI-1601 after multiple doses | At baseline and up to 29 days | Summary assessment of the time to last observed plasma concentration (tlast) of CTI-1601 after multiple doses |
Countries
United States
Outcome results
None listed