Nomlabofusp for Friedreich's Ataxia
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called nomlabofusp to determine its safety and tolerability in children and teens with Friedreich's ataxia, a rare genetic disease affecting movement and coordination. Participants will receive either the treatment or a placebo (a harmless substance resembling the treatment) through daily injections for a week. Ideal candidates are those diagnosed with Friedreich's ataxia, who can move at least 25 feet with or without assistance, and manage basic self-care tasks with minimal help. As a Phase 1 trial, this research aims to understand how the treatment works in people, offering participants the opportunity to be among the first to receive it.
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.
Is there any evidence suggesting that nomlabofusp is likely to be safe for humans?
Research has shown that nomlabofusp, a treatment for Friedreich's ataxia, has undergone safety evaluation. In earlier studies, patients generally tolerated nomlabofusp well, with no major safety issues identified, though some side effects may occur, as with any medication. The FDA recommends a safety database with at least 30 participants using the treatment continuously for six months. This requirement indicates that the treatment has been assessed sufficiently for more detailed study. While research continues, these findings offer a positive indication of nomlabofusp's safety in humans.12345
Why do researchers think this study treatment might be promising for Friedreich's ataxia?
Nomlabofusp is unique because it offers a new approach to treating Friedreich's Ataxia through subcutaneous injection, potentially providing more precise dosing and ease of use compared to oral medications. Unlike standard treatments that often focus on managing symptoms, Nomlabofusp may target the underlying causes of the condition more directly. Researchers are excited because this treatment could offer faster relief, with results potentially visible in just seven days, which is quicker than many existing options.
What evidence suggests that nomlabofusp might be an effective treatment for Friedreich's ataxia?
Research shows that nomlabofusp, which participants in this trial may receive, might help treat Friedreich's ataxia by boosting levels of frataxin, a protein typically low in individuals with this condition. Studies have found that after 14 days of treatment, patients experienced a noticeable increase in frataxin levels. Long-term evidence suggests that these protein levels continue to improve. This rise in frataxin is promising because it could address some of the core problems in Friedreich's ataxia.12467
Who Is on the Research Team?
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
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a subcutaneous injection of nomlabofusp or placebo once daily for 7 days
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Nomlabofusp
Find a Clinic Near You
Who Is Running the Clinical Trial?
Larimar Therapeutics, Inc.
Lead Sponsor