Ignite Creation Date:
2025-12-24 @ 4:41 PM
Ignite Modification Date:
2025-12-24 @ 4:41 PM
Study NCT ID:
NCT06681766
Status:
RECRUITING
Last Update Posted:
2025-02-21
First Post:
2024-11-06
Is Possible Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Study to Assess Nomlabofusp in Adolescents and Children with Friedreich's Ataxia
Sponsor:
Larimar Therapeutics, Inc.
Organization:
Study Overview
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
Status:
RECRUITING
Status Verified Date:
2025-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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).
Detailed 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.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?: