Larimar Therapeutics, Inc., is sponsoring a clinical trial to learn about the safety and tolerability of an investigational drug called nomlabofusp (previously called CTI-1601) in children and adolescents with Friedreich’s ataxia (FA). Nomlabofusp is a recombinant fusion protein intended to deliver human frataxin into the mitochondria of patients with FA. Frataxin is the protein deficient in individuals with FA.
This study is currently recruiting participants ages 2 to 17 years old at Uncommon Cures in Chevy Chase, Maryland.
This study will be up to 75 days in duration. The study will require one remote visit, one home visit during the screening period (up to 35 days in duration), and daily treatment visits over a 7 day period at the study site, Uncommon Cures in Chevy Chase, MD followed by two home visits during the 30-day follow-up period. Study drug will be given by injection, and monitoring of response to study drug will include periodic blood draws.
Study Details:
Key Inclusion Criteria:
- Participant has confirmed diagnosis of Friedreich’s ataxia via genetic testing
- Male or female participants 2 to 17 years of age at screening and weighs ≥ 22.0 lbs
- Participant must be able to travel a distance of 25 feet with or without some assistive device (e.g., cane, walker, crutches, self-propelled wheelchair) and meet the following requirements:
- Be able to sit upright with thighs together and arms crossed without requiring support on more than 2 sides;
- Be able to transfer from bed to chair independently or with assistance if, in the opinion of the investigator, the degree of physical disability does not result in undue risk to the participant while participating in the study; and
- Perform basic age-appropriate daily care, such as feeding themselves and personal hygiene, with minimal assistance
Key Exclusion Criteria:
- Participants who are confirmed as compound heterozygous (GAA repeat expansion on only 1 allele) for Friedreich’s ataxia
- Participant used erythropoietin, etravirine, or gamma interferon within 90 days prior to screening
- Participant has a screening/baseline ECHO ejection fraction < 45%
- Participant requires use of amiodarone
- Participant used an investigational drug or device within 90 days of screening
- Participant used omaveloxolone for the last 30 days or plans to receive omaveloxolone treatment during the study