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

Phase 1TerminatedNCT06681766

Larimar Therapeutics, Inc.18 enrolled

Overview

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).

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 Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Friedreich Ataxia

Interventions

NomlabofuspDRUG

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.

PlaceboDRUG

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

Eligibility

Sex: ALLMin age: 2 YearsMax age: 17 Years

Medical Language ↔ Plain English

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. 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.

Locations (1)

Uncommon Cures

Chevy Chase, Maryland, United States

Outcomes

Primary Outcomes

Number of AEs, TEAEs, related TEAEs, Grade 3/4 TEAEs, and SAEs

Number

Time frame: Up to 72 days (including screening)

Number of subjects with ISRs

Number

Time frame: Up to 37 days

Change from baseline in electrocardiogram (ECG) parameter - heart rate (HR)

Number

Time frame: Baseline, Day 7

Change from baseline in electrocardiogram (ECG) parameter - PR interval

Number

Time frame: Baseline, Day 7

Change from baseline in electrocardiogram (ECG) parameter - QRS complex

Number

Time frame: Baseline, Day 7

Change from baseline in electrocardiogram (ECG) parameter - QT interval

Number

Time frame: Baseline, Day 7

Change from baseline in electrocardiogram (ECG) parameter - QTcF (Corrected QT Interval)

Number

Time frame: Baseline, Day 7

Change from baseline in echocardiogram (ECHO) parameter - ejection fraction (EF)

Number

Time frame: Baseline, Day 7

Change from baseline in echocardiogram (ECHO) parameter - left ventricular end-diastolic volume (LV EDV)

Number

Time frame: Baseline, Day 7

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.

Change from baseline in echocardiogram (ECHO) parameter - left ventricular end-systolic volume (LV ESV)

Time frame: Baseline, Day 7

Change from baseline in echocardiogram (ECHO) parameter - relative wall thickness (RWT)

Number

Time frame: Baseline, Day 7

Change from baseline in echocardiogram (ECHO) parameter - left ventricular mass (LVM)

Number

Time frame: Baseline, Day 7

Change from baseline in echocardiogram (ECHO) parameter - left ventricular posterior wall thickness

Number

Time frame: Baseline, Day 7

Change from baseline in echocardiogram (ECHO) parameter - septal wall thickness

Number

Time frame: Baseline, Day 7

Change from baseline in echocardiogram (ECHO) parameter - mitral valve inflow Doppler

Number

Time frame: Baseline, Day 7

Change from baseline in echocardiogram (ECHO) parameter - tissue Doppler

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - sodium

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - potassium

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - glucose

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - blood urea nitrogen

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - creatinine

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - calcium, chloride

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - phosphorus

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - total protein

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - total CO2

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - albumin

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - AST

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - ALT

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - GGT

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - ALP

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - total bilirubin

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - uric acid

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - HbA1c

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - hemoglobin

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - hematocrit

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - RBC count

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - RDW

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - MCV

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - MCH

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - platelet count

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - WBC count

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - ANC

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - Eosinophils

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - Monocytes

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - Basophils

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - Lymphocytes

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - Bands

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - Neutrophils

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - Cholesterol

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - HDL cholesterol

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - LDL cholesterol

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - Triglycerides

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - very low-density lipoprotein cholesterol

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - pH

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - protein

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - blood

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - ketones

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - bilirubin

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - urobilinogen

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - nitrites

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - leukocyte esterase

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - color

Number

Time frame: Baseline, Day 7

Change from baseline in clinical laboratory assessment - specific gravity

Number

Time frame: Baseline, Day 7

Change from baseline in pulse rate

Number

Time frame: Baseline, Day 7

Number of subjects with abnormal physical examinations

Number

Time frame: Up to 72 days (including screening)

Number of subjects with suicidal ideation, suicidal behavior, and suicidal ideation or behavior based on the Columbia Suicide Severity Rating Scale (C-SSRS)

Number

Time frame: Up to 72 days (including screening)

Number of subjects who discontinue treatment and/or study

Number

Time frame: Up to 37 days

Secondary Outcomes

AUC0-last

Area under the concentration-time-curve to the last quantifiable timepoint

Time frame: Days 1, 7

Cmax

Maximum observed concentration

Time frame: Days 1, 7

Tmax

Time of maximum observed concentration

Time frame: Days 1, 7

Ctrough

Predose concentration

Time frame: Days 1, 7

Change from baseline in FXN concentrations normalized to total protein observed in buccal cells collected from cheek swabs

Number

Time frame: Baseline, Day 7