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TERMINATED
NCT06681766
PHASE1

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

Sponsor: Larimar Therapeutics, Inc.

View on ClinicalTrials.gov

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).

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

Key Details

Gender

All

Age Range

2 Years - 17 Years

Study Type

INTERVENTIONAL

Enrollment

18

Start Date

2024-12-06

Completion Date

2025-04-27

Last Updated

2026-07-10

Healthy Volunteers

No

Interventions

DRUG

Nomlabofusp

Nomlabofusp is a recombinant fusion protein provided in a sterile, preservative-free buffered solution for subcutaneous injection intended to deliver human frataxin, the protein deficient in Friedreich's ataxia.

DRUG

Placebo

The placebo is a sterile, preservative-free, clear liquid for subcutaneous injection.

Locations (1)

Uncommon Cures

Chevy Chase, Maryland, United States