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

Last updated: February 20, 2025
Sponsor: Larimar Therapeutics, Inc.
Overall Status: Active - Recruiting

Phase

1

Condition

Spinocerebellar Disorders

Friedreich's Ataxia

Dyskinesias

Treatment

Nomlabofusp

Placebo

Clinical Study ID

NCT06681766
CLIN-1601-103
  • Ages 2-17
  • All Genders

Study Summary

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).

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Subject has genetically confirmed diagnosis of FRDA manifested by homozygous GAArepeat expansions, with repeat sizing (if available) included on the diagnosisreport.

  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 someassistive device (e.g., cane, walker, crutches, self-propelled wheelchair) and meetthe following requirements:

  5. Be able to sit upright with thighs together and arms crossed without requiringsupport on more than 2 sides;

  6. Be able to transfer from bed to chair independently or with assistance if, inthe opinion of the investigator, the degree of physical disability does notresult in undue risk to the subject while participating in the study; and

  7. Perform basic age-appropriate daily care, such as feeding themselves andpersonal hygiene, with minimal assistance.

Exclusion

Exclusion Criteria:

  1. Subjects who are confirmed as compound heterozygous (GAA repeat expansion on only 1allele) for FRDA.

  2. Subject has any condition, disease, or situation, including a cardiac condition ordisease, that in the opinion of the investigator could confound the results of thestudy or put the subject at undue risk, making participation inadvisable.

  3. Subjects currently receiving or having received omaveloxolone within 30 days priorto Screening.

Study Design

Total Participants: 30
Treatment Group(s): 2
Primary Treatment: Nomlabofusp
Phase: 1
Study Start date:
December 06, 2024
Estimated Completion Date:
August 31, 2025

Study Description

This is a randomized, double-blind, placebo-controlled study evaluating a weight-based dose of nomlabofusp versus placebo in adolescents and children with FRDA. The study will consist of at least two cohorts with at least 12 to 15 participants in each cohort. Participants will be dosed once daily (QD) for 7 days.

Connect with a study center

  • Uncommon Cures

    Chevy Chase, Maryland 20815
    United States

    Active - Recruiting

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