RECRUITING

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

Conditions

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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).

Official Title

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

Quick Facts

Study Start:2024-12-06

Study Completion:2025-08

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06681766

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.

Ages Eligible for Study:2 Years to 17 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion 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.

Inclusion Criteria

Exclusion 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.

Contacts and Locations

Study Contact

Tamanna Roshan Lal, MB ChB

CONTACT

541-232-7818

troshanlal@UncommonCures.com

Kerri Gallagher, RN, B

CONTACT

267-234-2167

kgallagher@UncommonCures.com

Principal Investigator

Larimar Therapeutics, Inc.

STUDY_CHAIR

Larimar Therapeutics, Inc.

Study Locations (Sites)

Uncommon Cures

Chevy Chase, Maryland, 20815

United States

Collaborators and Investigators

Sponsor: Larimar Therapeutics, Inc.

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

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-12-06

Study Completion Date2025-08

Study Record Updates

Study Start Date2024-12-06

Study Completion Date2025-08

Terms related to this study

Additional Relevant MeSH Terms

Friedreich Ataxia