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

Phase 1

Recruiting

• Conditions: Friedreich Ataxia
• Interventions: Drug: Placebo; Drug: Nomlabofusp

• Registration Number: NCT06681766
• Lead Sponsor: Larimar Therapeutics, Inc.

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 Timeline

• Study First Submitted: 2024-11-06
• Study First Submitted QC: 2024-11-07
• Study First Posted: 2024-11-08
• Status Verified: 2024-12
• Study Start: 2024-12-06
• Last Update Submitted: 2024-12-20
• Last Update Posted: 2024-12-27
• Primary Completion: 2025-08
• Study Completion: 2025-08

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 30

Inclusion 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.

Read More

Exclusion Criteria

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.

Read More

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	Subcutaneous injection once daily for 7 days
Nomlabofusp	Nomlabofusp	Subcutaneous injection of 0.8 mg/kg, with a maximum dose of 50 mg, once daily for 7 days

Primary Outcome Measures

Name	Time	Method
Number of subjects with suicidal ideation, suicidal behavior, and suicidal ideation or behavior based on the Columbia Suicide Severity Rating Scale (C-SSRS)	Up to 72 days (including screening)	Number
Change from baseline in clinical laboratory assessment - GGT	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - ALP	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - total bilirubin	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - uric acid	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - HbA1c	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - hemoglobin	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - hematocrit	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - RBC count	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - RDW	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - MCV	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - MCH	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - platelet count	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - WBC count	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - ANC	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - Eosinophils	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - Monocytes	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - Basophils	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - Lymphocytes	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - Bands	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - Neutrophils	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - Cholesterol	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - HDL cholesterol	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - LDL cholesterol	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - Triglycerides	Baseline, Day 7	Number
Number of subjects who discontinue treatment and/or study	Up to 37 days	Number
Change from baseline in clinical laboratory assessment - very low-density lipoprotein cholesterol	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - pH	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - protein	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - blood	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - ketones	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - bilirubin	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - urobilinogen	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - nitrites	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - leukocyte esterase	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - color	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - specific gravity	Baseline, Day 7	Number
Change from baseline in pulse rate	Baseline, Day 7	Number
Number of subjects with abnormal physical examinations	Up to 72 days (including screening)	Number
Change from baseline in clinical laboratory assessment - creatinine	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - calcium, chloride	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - phosphorus	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - total protein	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - total CO2	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - albumin	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - AST	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - ALT	Baseline, Day 7	Number
Change from baseline in echocardiogram (ECHO) parameter - left ventricular posterior wall thickness	Baseline, Day 7	Number
Change from baseline in echocardiogram (ECHO) parameter - septal wall thickness	Baseline, Day 7	Number
Change from baseline in echocardiogram (ECHO) parameter - mitral valve inflow Doppler	Baseline, Day 7	Number
Change from baseline in echocardiogram (ECHO) parameter - tissue Doppler	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - sodium	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - potassium	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - glucose	Baseline, Day 7	Number
Change from baseline in clinical laboratory assessment - blood urea nitrogen	Baseline, Day 7	Number
Number of AEs, TEAEs, related TEAEs, Grade 3/4 TEAEs, and SAEs	Up to 72 days (including screening)	Number
Number of subjects with ISRs	Up to 37 days	Number
Change from baseline in electrocardiogram (ECG) parameter - heart rate (HR)	Baseline, Day 7	Number
Change from baseline in electrocardiogram (ECG) parameter - PR interval	Baseline, Day 7	Number
Change from baseline in electrocardiogram (ECG) parameter - QRS complex	Baseline, Day 7	Number
Change from baseline in electrocardiogram (ECG) parameter - QT interval	Baseline, Day 7	Number
Change from baseline in electrocardiogram (ECG) parameter - QTcF (Corrected QT Interval)	Baseline, Day 7	Number
Change from baseline in echocardiogram (ECHO) parameter - ejection fraction (EF)	Baseline, Day 7	Number
Change from baseline in echocardiogram (ECHO) parameter - left ventricular end-diastolic volume (LV EDV)	Baseline, Day 7	Number
Change from baseline in echocardiogram (ECHO) parameter - left ventricular end-systolic volume (LV ESV)	Baseline, Day 7	Number
Change from baseline in echocardiogram (ECHO) parameter - relative wall thickness (RWT)	Baseline, Day 7	Number
Change from baseline in echocardiogram (ECHO) parameter - left ventricular mass (LVM)	Baseline, Day 7	Number

Secondary Outcome Measures

Name	Time	Method
AUC0-last	Days 1, 7	Area under the concentration-time-curve to the last quantifiable timepoint
Cmax	Days 1, 7	Maximum observed concentration
Tmax	Days 1, 7	Time of maximum observed concentration
Ctrough	Days 1, 7	Predose concentration
Change from baseline in FXN concentrations normalized to total protein observed in buccal cells collected from cheek swabs	Baseline, Day 7	Number

Trial Locations

Locations (1)

Uncommon Cures; 🇺🇸 Chevy Chase, Maryland, United States