30 Participants Needed

Nomlabofusp for Friedreich's Ataxia

TR
KG
Overseen ByKerri Gallagher, RN, B
Age: < 18
Sex: Any
Trial Phase: Phase 1
Sponsor: Larimar Therapeutics, Inc.

What You Need to Know Before You Apply

What is the purpose of this trial?

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).

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot have taken omaveloxolone within 30 days before the trial starts.

How does the drug Nomlabofusp differ from other treatments for Friedreich's Ataxia?

Nomlabofusp may offer a novel approach to treating Friedreich's Ataxia by potentially addressing the processing of frataxin protein, which is not effectively increased by current treatments. This could provide a new way to modify the disease, especially for patients with specific mutations that affect frataxin processing.12345

Who Is on the Research Team?

LT

Larimar Therapeutics, Inc.

Principal Investigator

Larimar Therapeutics, Inc.

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

I can walk 25 feet with some help and can sit up, move from bed to chair, and take care of myself.
I weigh at least 10 kilograms.
I am between 2 and 17 years old.
See 1 more

Exclusion Criteria

I do not have any health conditions that could risk my safety in the study.
I have taken omaveloxolone within the last 30 days.
I have been diagnosed with Friedreich's ataxia with a specific genetic feature.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a subcutaneous injection of nomlabofusp or placebo once daily for 7 days

1 week
7 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

up to 72 days

What Are the Treatments Tested in This Trial?

Interventions

  • Nomlabofusp
Trial Overview 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.
How Is the Trial Designed?
2Treatment groups
Experimental Treatment
Placebo Group
Group I: NomlabofuspExperimental Treatment1 Intervention
Group II: PlaceboPlacebo Group1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Larimar Therapeutics, Inc.

Lead Sponsor

Trials
7
Recruited
270+

Published Research Related to This Trial

Friedreich ataxia is a genetic degenerative disease that mainly impacts the nervous system and heart, and its full understanding has evolved since the introduction of genetic testing in 1996.
Research into the complex causes of Friedreich ataxia is aiding the development of new treatments, highlighting the importance of genetic insights in addressing this condition.
Friedreich ataxia.Pandolfo, M.[2008]
Friedreich's ataxia is linked to a wide range of GAA trinucleotide repeat expansions, with patients showing between 66 to 1360 repeats, and the number of repeats correlates with disease onset and progression.
The study suggests that molecular genetic testing is essential for diagnosing Friedreich's ataxia, as the phenotypic presentation can vary significantly, and some patients may not show the typical symptoms associated with the disorder.
Friedreich's ataxia. Revision of the phenotype according to molecular genetics.Schรถls, L., Amoiridis, G., Przuntek, H., et al.[2023]
A study involving 11 patients with Friedreich's ataxia (FA) revealed that traditional clinical tests for joint position sense (JPS) and vibration sense (VS) may underestimate proprioceptive deficits, as all patients showed impairments in a more sensitive spatial position sense (SPS) test.
The SPS test not only detected deficits earlier than standard tests but also correlated positively with the severity of ataxia, suggesting that proprioceptive issues in FA patients are more severe and start earlier than previously recognized.
Routine clinical testing underestimates proprioceptive deficits in Friedreich's ataxia.Borchers, S., Synofzik, M., Kiely, E., et al.[2021]

Citations

Friedreich ataxia. [2008]
Friedreich's ataxia. Revision of the phenotype according to molecular genetics. [2023]
Routine clinical testing underestimates proprioceptive deficits in Friedreich's ataxia. [2021]
Identification of a novel missense mutation in Friedreich's ataxia -FXNW 168R. [2023]
Biological and clinical characteristics of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS) cohort: a cross-sectional analysis of baseline data. [2022]
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