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

Description

The goal of this clinical trial is to evaluate the safety and tolerability of nomlabofusp (CTI-1601) in adolescents and children with Friedreich's ataxia (FRDA).

Conditions

Friedreich Ataxia

Study Overview

Study Details

Study overview

The goal of this clinical trial is to evaluate the safety and tolerability of nomlabofusp (CTI-1601) in adolescents and children with Friedreich's ataxia (FRDA).

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

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

Condition
Friedreich Ataxia
Intervention / Treatment

-

Contacts and Locations

Chevy Chase

Uncommon Cures, Chevy Chase, Maryland, United States, 20815

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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.
  • 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.

Ages Eligible for Study

2 Years to 17 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Larimar Therapeutics, Inc.,

Larimar Therapeutics, Inc., STUDY_CHAIR, Larimar Therapeutics, Inc.

Study Record Dates

2025-08