255 Participants Needed

Omaveloxolone for Friedreich's Ataxia

Recruiting at 26 trial locations
UB
GB
Overseen ByGlobal Biogen Clinical Trial Center
Age: < 18
Sex: Any
Trial Phase: Phase 3
Sponsor: Biogen
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.

What data supports the effectiveness of the drug Omaveloxolone for Friedreich's Ataxia?

Omaveloxolone has been shown to improve mitochondrial function, restore redox balance (a state of balance between antioxidants and free radicals), and reduce inflammation in models of Friedreich's Ataxia. It was approved in the USA in February 2023 for treating Friedreich's Ataxia in adults and adolescents aged 16 years and older, indicating its effectiveness.12345

Is omaveloxolone safe for humans?

Omaveloxolone has been studied for safety in people with Friedreich's ataxia, and it has been approved in the USA for this condition. The studies focused on its safety and how it works in the body, showing it can help reduce inflammation and improve cell function.12367

How is the drug omaveloxolone unique for treating Friedreich's ataxia?

Omaveloxolone is unique because it is the first approved drug specifically for Friedreich's ataxia, working by activating the Nrf2 pathway to increase antioxidant activity, improve mitochondrial function, and reduce inflammation, which are not targeted by other treatments.12378

What is the purpose of this trial?

In this study, researchers will learn more about the effects and safety of BIIB141, also known as omaveloxolone or SKYCLARYS®. This drug has been approved, or made available for doctors to prescribe, for people with Friedreich's Ataxia (FA) who are at least 16 years old. But, it is not yet available for children and teens with FA who are younger than 16 years old. The main objective of this study is to learn how BIIB141 works in the body and about its safety in children and teens who are 2 to 15 years old.The main questions researchers want to answer in this study are:* How does BIIB141 affect the participants' FA symptoms balance and stability?* How many participants have medical problems during the study?* Are there any changes in the participants' overall health during the study?* Are there any changes in the participants' heart health?* Are there any changes in how the participants move through puberty? Puberty is the time in someone's life when their body changes from a child to an adult.Researchers will also learn more about:- How the body processes BIIB141 in children and teensThis study will be done as follows:* Participants will be screened to check if they can join the study. The screening period will be up to 28 days, after which participants will check into their study research center.* There are 2 parts in this study. During Part 1, participants will take either BIIB141 or a placebo once a day.* In Part 1, participants will take BIIB141 or the placebo in a study research center on Day 1, and then at in-person visits at Week 4, Week 12, Week 26, and Week 52. On all other days, they will take BIIB141 or the placebo at home. Part 1 lasts up to 52 weeks.* During Part 2, participants from Part 1 will either continue taking BIIB141 or start it if they were taking the placebo. Part 2 will last up to 104 weeks.* In Part 1, participants will have up to 10 visits to their study research center and a phone call at Week 2. In Part 2, participants will have visits at Weeks 4, 8,12, 26, and every 26 weeks after that until they leave the study, and a phone call at Week 2. There will be a final phone call to check on the participants' health 31 days after their last dose.* Each participant will be in the study for up to about 3 years

Research Team

MD

Medical Director

Principal Investigator

Biogen

Eligibility Criteria

This trial is for children and teens aged 2 to 15 with Friedreich's Ataxia. It aims to understand the effects and safety of BIIB141 (Omaveloxolone) in this age group, as it's currently approved only for those over 16. Participants will be monitored for changes in symptoms, overall health, heart health, and puberty development.

Inclusion Criteria

My child is between 7 and 16 years old with a specific balance score.
Participants have completed Part 1 RCT of the study and no discontinuation criteria have been met
Safety and tolerability data from Part 1 RCT are supportive of continuation in the judgement of the investigator
See 2 more

Exclusion Criteria

If BNP is > 200 pg/mL at the previous visit assessment, Part 2 Day 1 should be delayed until BNP is < 200 pg/mL
If any other clinically significant laboratory abnormalities are present based on the previous visit assessments, Part 2 Day 1 should be delayed until the abnormalities are resolved
Glycosylated hemoglobin A1C (HbA1c) > 11%
See 5 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

Treatment Part 1

Participants receive either BIIB141 or placebo once a day for up to 52 weeks

52 weeks
10 visits (in-person), 1 phone call at Week 2

Open-label Extension Part 2

Participants continue or start BIIB141 for up to 104 weeks

104 weeks
Visits at Weeks 4, 8, 12, 26, and every 26 weeks thereafter, 1 phone call at Week 2

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks
1 final phone call 31 days after last dose

Treatment Details

Interventions

  • BIIB141
Trial Overview The study tests Omaveloxolone against a placebo over approximately three years. Part one involves daily doses at home with periodic research center visits; participants then continue or switch to Omaveloxolone in part two. The focus is on symptom management, long-term safety, and how the body processes the drug.
Participant Groups
3Treatment groups
Experimental Treatment
Placebo Group
Group I: Part 2 OLE: OmaveloxoloneExperimental Treatment1 Intervention
Participants who complete Part 1 of the study and are eligible will receive a single oral dose of omaveloxolone, QD for up to 104 weeks in the Part 2 OLE study.
Group II: Part 1 RCT: OmaveloxoloneExperimental Treatment1 Intervention
Participants will receive a single oral dose of omaveloxolone once a day (QD) for up to 52 weeks in Part 1 of the study.
Group III: Part 1 RCT: PlaceboPlacebo Group1 Intervention
Participants will receive placebo, orally, QD for up to 52 weeks in Part 1 of the study.

Find a Clinic Near You

Who Is Running the Clinical Trial?

Biogen

Lead Sponsor

Trials
655
Recruited
468,000+
Daniel Quirk profile image

Daniel Quirk

Biogen

Chief Medical Officer

MD

Christopher A. Viehbacher profile image

Christopher A. Viehbacher

Biogen

Chief Executive Officer since 2022

Graduated from Queen's University, Kingston, Ontario, Canada

Findings from Research

Omaveloxolone (SKYCLARYS™) is an orally active drug that enhances antioxidant activity by activating the Nrf2 pathway, which is crucial for combating oxidative stress and mitochondrial dysfunction in patients with Friedreich's ataxia.
Approved in February 2023, omaveloxolone represents a significant advancement in treating Friedreich's ataxia, particularly for adults and adolescents aged 16 and older, by targeting the underlying cellular damage associated with the disease.
Omaveloxolone: First Approval.Lee, A.[2023]
In a phase 2 trial involving 103 patients with Friedreich ataxia, omaveloxolone significantly improved neurological function compared to placebo, with a notable difference in modified Friedreich's Ataxia Rating Scale scores after 48 weeks.
Omaveloxolone was generally safe and well tolerated, although some patients experienced transient increases in liver enzymes and common side effects like headache, nausea, and fatigue.
Safety and Efficacy of Omaveloxolone in Friedreich Ataxia (MOXIe Study).Lynch, DR., Chin, MP., Delatycki, MB., et al.[2023]
In a Phase 2 study involving 69 patients with Friedreich ataxia, omaveloxolone was found to be well tolerated with mostly mild adverse effects, indicating a favorable safety profile.
At a dose of 160 mg/day, omaveloxolone significantly improved neurological function as measured by the modified Friedreich Ataxia Rating Scale (mFARS), particularly in patients without musculoskeletal foot deformity, suggesting its potential efficacy in treating this condition.
Safety, pharmacodynamics, and potential benefit of omaveloxolone in Friedreich ataxia.Lynch, DR., Farmer, J., Hauser, L., et al.[2022]

References

Open-label pilot study of oral methylprednisolone for the treatment of patients with friedreich ataxia. [2020]
Omaveloxolone: First Approval. [2023]
Safety and Efficacy of Omaveloxolone in Friedreich Ataxia (MOXIe Study). [2023]
A phase 3, double-blind, placebo-controlled trial of idebenone in friedreich ataxia. [2022]
Triple therapy with deferiprone, idebenone and riboflavin in Friedreich's ataxia - open-label trial. [2018]
Safety, pharmacodynamics, and potential benefit of omaveloxolone in Friedreich ataxia. [2022]
Pharmacokinetics and pharmacodynamics of the novel Nrf2 activator omaveloxolone in primates. [2022]
Efficacy of Omaveloxolone in Friedreich's Ataxia: Delayed-Start Analysis of the MOXIe Extension. [2023]
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