A Study to Assess Nomlabofusp in Adolescents and Children with Friedreich's Ataxia

Launched by LARIMAR THERAPEUTICS, INC. · Nov 7, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called nomlabofusp (also known as CTI-1601) for children and teenagers with Friedreich's ataxia (FRDA), a genetic condition that affects movement and coordination. The main goal of the trial is to find out if this treatment is safe and how well it can be tolerated by young participants. The trial is currently looking for participants who are between 2 and 17 years old and have a genetic confirmation of FRDA. To be eligible, they should be able to move a short distance, such as 25 feet, with or without help from devices like a cane or walker, and they need to weigh at least 10 kilograms.

If your child is eligible and chooses to participate, they will undergo tests to ensure their safety and will receive the treatment while being closely monitored by medical professionals. The trial aims to improve our understanding of this condition and explore potential new therapies. It's important to note that certain health conditions may prevent participation, so it's best to discuss any concerns with the medical team.

Gender

ALL

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.