Assessing nomlabofusp in children and adolescents with Friedreich's Ataxia

A Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Nomlabofusp in Adolescents and Children with Friedreich's Ataxia

This study is testing a new treatment called nomlabofusp to see if it is safe and helps children and teens with Friedreich's ataxia feel better.

Quick facts

• Phase: PHASE1
• Study type: Interventional
• Enrollment: 30 (estimated)
• Ages: 2 Years to 17 Years
• Sex: All
• Sponsor: Larimar Therapeutics, Inc. (industry)
• Locations: 1 site (Chevy Chase, Maryland)
• Trial ID: NCT06681766 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and tolerability of nomlabofusp (CTI-1601) in adolescents and children diagnosed with Friedreich's ataxia (FRDA). It is a randomized, double-blind, placebo-controlled study that will involve at least two cohorts, each consisting of 12 to 15 participants. Participants will receive either the treatment or a placebo once daily for 7 days, allowing researchers to assess the drug's effects compared to no treatment.

Who should consider this trial

Good fit: Ideal candidates for this study are children and adolescents aged 2 to 18 years with a genetically confirmed diagnosis of Friedreich's ataxia.

Not a fit: Patients who do not have a confirmed genetic diagnosis of Friedreich's ataxia or are outside the age range may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new therapeutic option for managing Friedreich's ataxia in younger patients.

How similar studies have performed: While this approach is novel in the context of Friedreich's ataxia, similar studies targeting genetic conditions have shown promise in other contexts.

Eligibility criteria

Inclusion Criteria:

1. Subject has genetically confirmed diagnosis of FRDA manifested by homozygous GAA repeat expansions, with repeat sizing (if available) included on the diagnosis report.
2. Male or female subjects ≥ 2 to \< 18 years of age at screening.
3. Subjects must weigh ≥ 10.0 kg.
4. Subject must be able to traverse a distance of 25 feet with or without some assistive device (e.g., cane, walker, crutches, self-propelled wheelchair) and meet the following requirements:

   1. Be able to sit upright with thighs together and arms crossed without requiring support on more than 2 sides;
   2. 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 subject while participating in the study; and
   3. Perform basic age-appropriate daily care, such as feeding themselves and personal hygiene, with minimal assistance.

Exclusion Criteria:

1. Subjects who are confirmed as compound heterozygous (GAA repeat expansion on only 1 allele) for FRDA.
2. Subject has any condition, disease, or situation, including a cardiac condition or disease, that in the opinion of the investigator could confound the results of the study or put the subject at undue risk, making participation inadvisable.
3. Subjects currently receiving or having received omaveloxolone within 30 days prior to Screening.

Where this trial is running

Chevy Chase, Maryland

• Uncommon Cures — Chevy Chase, Maryland, United States (RECRUITING)

Study contacts

• Study coordinator: Tamanna Roshan Lal, MB ChB

Conditions: Friedreich Ataxia