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Cancer Vaccine

Nomlabofusp for Friedreich's Ataxia

Phase 1
Waitlist Available
Research Sponsored by Larimar Therapeutics, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Subjects must weigh ≥ 10.0 kg
Subject has genetically confirmed diagnosis of FRDA manifested by homozygous GAA repeat expansions, with repeat sizing included on the diagnosis report
Must not have
Subjects who are confirmed as compound heterozygous (GAA repeat expansion on only 1 allele) for FRDA
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 72 days (including screening)

Summary

"This trial aims to test if nomlabofusp is safe and well-tolerated in adolescents and children with Friedreich's ataxia."

Who is the study for?
This trial is for children and adolescents aged 2 to less than 18 with genetically confirmed Friedreich's ataxia. They must be able to move 25 feet, perform basic self-care, and weigh at least 10 kg. Those with heart conditions or who have taken omaveloxolone recently cannot participate.
What is being tested?
The study tests the safety of a drug called Nomlabofusp (CTI-1601) against a placebo in young patients with Friedreich's Ataxia. It aims to see how well they tolerate the medication over time.
What are the potential side effects?
While specific side effects are not listed here, generally, clinical trials look out for any adverse reactions ranging from mild discomforts like headaches or nausea to more serious issues affecting organ function.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I weigh at least 10 kilograms.
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My condition, Friedreich's ataxia, is confirmed by genetic testing.
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I can walk 25 feet with some help and can sit up, move from bed to chair, and take care of myself.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I have been diagnosed with Friedreich's ataxia with a specific genetic feature.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 72 days (including screening)
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 72 days (including screening) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change from baseline in clinical laboratory assessment - ALP
Change from baseline in clinical laboratory assessment - ALT
Change from baseline in clinical laboratory assessment - ANC
+64 more
Secondary study objectives
AUC0-last
Change from baseline in FXN concentrations normalized to total protein observed in buccal cells collected from cheek swabs
Cmax
+2 more

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: NomlabofuspExperimental Treatment1 Intervention
Subcutaneous injection of 0.8 mg/kg, with a maximum dose of 50 mg, once daily for 7 days
Group II: PlaceboPlacebo Group1 Intervention
Subcutaneous injection once daily for 7 days

Find a Location

Who is running the clinical trial?

Larimar Therapeutics, Inc.Lead Sponsor
6 Previous Clinical Trials
238 Total Patients Enrolled
6 Trials studying Friedreich Ataxia
238 Patients Enrolled for Friedreich Ataxia
~20 spots leftby Aug 2025
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