Clinical trial NCT06681766: A Study to Assess Nomlabofusp in Adolescents and Children with Friedreich's Ataxia
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
Conditions: Friedreich Ataxia
Lead sponsor: Larimar Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2024-11-08
Enrollment: 30
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Number of AEs, TEAEs, related TEAEs, Grade 3/4 TEAEs, and SAEs
timeFrame: Up to 72 days (including screening)
measure: Number of subjects with ISRs
timeFrame: Up to 37 days
measure: Change from baseline in electrocardiogram (ECG) parameter - heart rate (HR)
timeFrame: Baseline, Day 7
measure: Change from baseline in electrocardiogram (ECG) parameter - PR interval
timeFrame: Baseline, Day 7
measure: Change from baseline in electrocardiogram (ECG) parameter - QRS complex
timeFrame: Baseline, Day 7
measure: Change from baseline in electrocardiogram (ECG) parameter - QT interval
timeFrame: Baseline, Day 7
measure: Change from baseline in electrocardiogram (ECG) parameter - QTcF (Corrected QT Interval)
timeFrame: Baseline, Day 7
measure: Change from baseline in echocardiogram (ECHO) parameter - ejection fraction (EF)
timeFrame: Baseline, Day 7
measure: Change from baseline in echocardiogram (ECHO) parameter - left ventricular end-diastolic volume (LV EDV)
timeFrame: Baseline, Day 7
measure: Change from baseline in echocardiogram (ECHO) parameter - left ventricular end-systolic volume (LV ESV)
timeFrame: Baseline, Day 7
measure: Change from baseline in echocardiogram (ECHO) parameter - relative wall thickness (RWT)
timeFrame: Baseline, Day 7
measure: Change from baseline in echocardiogram (ECHO) parameter - left ventricular mass (LVM)
timeFrame: Baseline, Day 7
measure: Change from baseline in echocardiogram (ECHO) parameter - left ventricular posterior wall thickness
timeFrame: Baseline, Day 7
measure: Change from baseline in echocardiogram (ECHO) parameter - septal wall thickness
timeFrame: Baseline, Day 7
measure: Change from baseline in echocardiogram (ECHO) parameter - mitral valve inflow Doppler
timeFrame: Baseline, Day 7
measure: Change from baseline in echocardiogram (ECHO) parameter - tissue Doppler
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - sodium
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - potassium
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - glucose
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - blood urea nitrogen
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - creatinine
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - calcium, chloride
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - phosphorus
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - total protein
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - total CO2
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - albumin
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - AST
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - ALT
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - GGT
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - ALP
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - total bilirubin
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - uric acid
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - HbA1c
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - hemoglobin
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - hematocrit
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - RBC count
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - RDW
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - MCV
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - MCH
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - platelet count
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - WBC count
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - ANC
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - Eosinophils
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - Monocytes
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - Basophils
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - Lymphocytes
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - Bands
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - Neutrophils
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - Cholesterol
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - HDL cholesterol
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - LDL cholesterol
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - Triglycerides
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - very low-density lipoprotein cholesterol
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - pH
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - protein
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - blood
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - ketones
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - bilirubin
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - urobilinogen
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - nitrites
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - leukocyte esterase
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - color
timeFrame: Baseline, Day 7
measure: Change from baseline in clinical laboratory assessment - specific gravity
timeFrame: Baseline, Day 7
measure: Change from baseline in pulse rate
timeFrame: Baseline, Day 7
measure: Number of subjects with abnormal physical examinations
timeFrame: Up to 72 days (including screening)
measure: Number of subjects with suicidal ideation, suicidal behavior, and suicidal ideation or behavior based on the Columbia Suicide Severity Rating Scale (C-SSRS)
timeFrame: Up to 72 days (including screening)
measure: Number of subjects who discontinue treatment and/or study
timeFrame: Up to 37 days
Secondary outcomes:
measure: AUC0-last
timeFrame: Days 1, 7
measure: Cmax
timeFrame: Days 1, 7
measure: Tmax
timeFrame: Days 1, 7
measure: Ctrough
timeFrame: Days 1, 7
measure: Change from baseline in FXN concentrations normalized to total protein observed in buccal cells collected from cheek swabs
timeFrame: Baseline, Day 7
Other outcomes:
Locations:
city: Chevy Chase
country: United States
facility: Uncommon Cures
state: Maryland
Study officials:
affiliation: Larimar Therapeutics, Inc.
name: Larimar Therapeutics, Inc.
role: STUDY_CHAIR
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