Omaveloxolone for Friedreich's Ataxia
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to assess the safety of a drug called BIIB141 (omaveloxolone) for individuals with Friedreich's Ataxia (FA), a condition affecting movement and coordination. Researchers will monitor any serious side effects, particularly those related to heart or liver issues, in participants taking this drug. The study includes two groups: those starting BIIB141 for the first time and those who began the treatment less than 12 months ago. Individuals with FA confirmed by a genetic test and a prescription for BIIB141 from their doctor may be suitable candidates for this trial. As a Phase 2 trial, this research focuses on evaluating the treatment's effectiveness in an initial, smaller group, providing an opportunity to contribute to understanding its impact.
Do I have to stop taking my current medications to join the trial?
The trial does not specify if you need to stop taking your current medications. Since this is an observational study, it is likely that you can continue your current medications, but you should confirm with the study team or your doctor.
What is the safety track record for BIIB141 (omaveloxolone or SKYCLARYS®)?
Research has shown that omaveloxolone, also known as BIIB141, is generally well tolerated. In one study involving 137 patients who took 150 mg of omaveloxolone for 48 weeks or more, no deaths occurred, suggesting the drug is quite safe over a long period. While some studies mention side effects, they did not include serious issues like life-threatening conditions or those requiring hospital care.
Omaveloxolone is already approved for individuals aged 16 and older with Friedreich's Ataxia, indicating a good safety record. However, like any drug, risks exist, so monitoring for signs of heart or liver problems is important. Participants in this study will contribute to a better understanding of the long-term safety of this treatment.12345Why are researchers excited about this trial?
Omaveloxolone is unique because it targets mitochondrial dysfunction, which is a key issue in Friedreich's Ataxia (FA). Unlike other treatments that mainly focus on symptom management, omaveloxolone activates the Nrf2 pathway, which helps reduce oxidative stress and inflammation in cells. Researchers are excited about this treatment because it addresses the underlying cellular problems in FA, offering the potential for more effective long-term management of the disease.
What is the effectiveness track record for BIIB141 in treating Friedreich's Ataxia?
Studies have shown that omaveloxolone can help treat Friedreich's ataxia (FA). Research indicates it can lower mFARS scores, which measure disease severity, by 55%, suggesting milder symptoms for patients. Omaveloxolone aids cells affected by FA, potentially improving muscle control and coordination. In this trial, participants will be divided into two cohorts: the Omaveloxolone Naive Cohort, for those starting treatment according to its approved label, and the Omaveloxolone Non-Naive Cohort, for those who began treatment less than 12 months before enrollment. The drug has been approved for use, demonstrating effectiveness in some FA-related conditions.23678
Who Is on the Research Team?
Medical Director
Principal Investigator
Biogen
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants will take BIIB141 as prescribed by their own doctor, with initiation required within 6 months for those who have not started treatment before
Follow-up
Participants' health will be monitored through regular visits to their doctor, with data collected at specified intervals
What Are the Treatments Tested in This Trial?
Interventions
- Omaveloxolone
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
Participants with FA who initiated omaveloxolone treatment as per the approved label less than 12 months prior to enrollment in this study will be analyzed retrospectively (baseline data) followed by prospective analysis (post-baseline data) for up to 5 years.
Participants with FA who will initiate omaveloxolone treatment per its approved label will be followed prospectively for up to 5 years.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Reata, a wholly owned subsidiary of Biogen
Lead Sponsor
Biogen
Lead Sponsor
Daniel Quirk
Biogen
Chief Medical Officer
MD
Christopher A. Viehbacher
Biogen
Chief Executive Officer since 2022
Graduated from Queen's University, Kingston, Ontario, Canada
Citations
Safety and efficacy of omaveloxolone v/s placebo for the ...
Overall, in a research analysis performed by the same group of researchers on the MOXIe study demonstrated a reduction of mFARS by 55% in the ...
NCT02255435 | A Study to Learn About the Effects and ...
This 2-part study will evaluate the efficacy, safety, and pharmacodynamics of omaveloxolone (RTA 408) in the treatment of participants with Friedreich's ataxia.
216718Orig1s000 CLINICAL REVIEW(S) - accessdata.fda.gov
The evidence of effectiveness of omaveloxolone in the treatment of Friedreich's ataxia was based on a single Study 408-C-1402 Part 2 (MoXIe) ...
Omaveloxolone, But Not Dimethyl Fumarate, Improves ...
These data demonstrate that omaveloxolone significantly improved contractile function but not survival in FA hearts because cardiac fibrosis and ...
5.
clinicaltrials.uihealthcare.org
clinicaltrials.uihealthcare.org/studies/phase-2-study-safety-efficacy-and-pharmacodynamics-rta-408-treatment-friedreichs-ataxiaA PHASE 2 STUDY OF THE SAFETY, EFFICACY, AND ...
This 2-part study will evaluate the efficacy, safety, and pharmacodynamics of omaveloxolone (RTA 408) in the treatment of participants with Friedreich's ataxia.
216718Orig1s000 SUMMARY REVIEW - accessdata.fda.gov
A total of 137 patients provided long-term safety data of 150 mg omaveloxolone for ≥ 48 weeks of exposure. • There were no deaths in the FA ...
7.
alzdiscovery.org
alzdiscovery.org/uploads/cognitive_vitality_media/Omaveloxolone-Cognitive-Vitality-For-Researchers.pdfOmaveloxolone (RTA-408)
In addition, these reports include evaluation of safety data, from clinical trials if available, and from preclinical models. Omaveloxolone (RTA-408).
Omaveloxolone approved for patients aged 16 years and ...
Omaveloxolone approved for patients aged 16 years and older with Friedreich ataxia (FRDA): A therapeutics bulletin of the American College of ...
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