Friedreich’s Ataxia Research

Exploring Innovative Treatments

Friedreich’s Ataxia Research Study (Nomlabofusp)

Study Drug:

Nomlabofusp

Age Group:

2 to 17 years old

Study Duration:

Approximately 72 days

Visits:

Remote screening, on-site visits, and follow-up with home care

Location:

Uncommon Cures, Chevy Chase, MD

Compensation:

Travel reimbursement and participant compensation available

Study Overview

Uncommon Cures is conducting a clinical trial for children and adolescents (ages 2-17) with Friedreich’s ataxia (FA), a rare genetic condition affecting the nervous system. This study is designed to test the safety and efficacy of an investigational drug, Nomlabofusp, developed by Larimar Therapeutics, Inc. The trial takes place at our Chevy Chase, MD location, and participants will be reimbursed for travel and compensated for their time.
Eligibility Criteria:
  • Children aged 2-17 years with genetically confirmed Friedreich’s ataxia (GAA repeat expansions).
  • Must not have used omaveloxolone in the 30 days leading up to, or during, the study.
Study Process
  • Screening (Up to 35 Days):
    Screening is conducted remotely, followed by an at-home nurse visit for confirmation.

  • Main Study Period (Up to 10 Days):
    Participants and their caregivers will stay at a nearby hotel for on-site visits and treatment.

  • Follow-Up (30 Days):
    Two follow-up visits with an at-home visiting nurse to ensure safety and monitor progress.

Advance Rare Treatments.

Have a Question?

FAQ

We understand that participating in a clinical trial can raise a lot of questions. Here, you’ll find answers to some of the most common questions about our process, what to expect, and how we support our patients. If you don’t see the information you’re looking for, feel free to contact us directly for more details.

Nomlabofusp is an investigational drug developed by Larimar Therapeutics. The study aims to evaluate its potential to slow or stop the progression of Friedreich’s ataxia in pediatric patients.

Yes, during the main study period, participants and their caregivers will stay at a nearby hotel for up to 10 days for closer monitoring and treatment.

After the study drug is administered, two follow-up visits will be conducted by an at-home visiting nurse over a 30-day period to ensure safety and monitor the child’s progress.

Patient safety is our top priority. The study follows a rigorous protocol approved by the local medicines regulatory authorities and an Ethics Committee to protect participants’ well-being.

Absolutely! We encourage parents to consult with their child’s physician before enrolling in the trial to ensure that it aligns with their ongoing medical care.

Clinical Trial Interest Form

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