Omaveloxolone for Healthy Adults

Overseen By US Biogen Clinical Trial Center

Age: 18 - 65

Sex: Any

Trial Phase: Phase 1

Sponsor: Biogen

Must not be taking: Prescription medications

Disqualifiers: Cardiac, Hepatic, HIV, Hepatitis, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 30 days

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to understand how the body processes the drug omaveloxolone, also known as BIIB141 or SKYCLARYS®, when taken in two ways: as a capsule or sprinkled over yogurt. Researchers seek to determine if yogurt can serve as a new method for administering this medication, which is already used for Friedrich's Ataxia, a rare genetic disease affecting movement. The trial will also monitor any health changes or reactions to the drug. Healthy adults without major health issues and with a BMI between 18 and 32 may be suitable for this study. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants a chance to contribute to groundbreaking medical research.

Will I have to stop taking my current medications?

Yes, you will need to stop taking prescription medications (except oral contraceptives and hormone replacement therapy), certain over-the-counter medications, and supplements at least 28 days before the study starts. Other over-the-counter medications, vitamins, and supplements should be stopped 14 days before the study.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that omaveloxolone, also known as BIIB141 or SKYCLARYS®, is safe and well-tolerated in adults with Friedreich's Ataxia (FA). In a study involving 137 FA patients over 48 weeks, no deaths occurred while taking 150 mg of the drug, indicating its safety.

Omaveloxolone has been tested in real-life situations and is generally safe for these patients. The FDA has approved the drug for treating FA in individuals aged 16 and older, based on strong evidence of its effectiveness and safety.

While researchers continue to study the drug for other uses, such as in the current trial, its existing approval and safety record in FA patients provide reassurance. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Omaveloxolone is unique because it offers a novel approach by targeting oxidative stress and mitochondrial dysfunction, which are underlying issues in many diseases. Unlike other treatments that may focus on symptom management, omaveloxolone works at a cellular level to potentially alter disease progression. Researchers are excited about its potential to provide a more effective and targeted treatment option, especially considering its unique mechanism of action that differs from standard therapies.

What is the effectiveness track record for omaveloxolone?

Research has shown that omaveloxolone can slow the progression of Friedreich's Ataxia (FA). Studies have found that it improved test scores measuring disease progression over four years. Specifically, one study showed that omaveloxolone lowered mFARS scores, helping patients maintain more of their daily functioning abilities. The treatment is generally safe and well-tolerated, with few side effects reported. This evidence supports its use for people with FA, although further research, such as the current trial, is underway to understand how the body processes it in different forms. Participants in this trial will receive omaveloxolone either as a capsule or sprinkled over yogurt to assess these variations. ^{1 4 6 7 8}

Who Is on the Research Team?

Medical Director

Principal Investigator

Biogen

Are You a Good Fit for This Trial?

This trial is for healthy adults aged 18 to 55 who are interested in helping researchers learn how the body processes a drug called BIIB141, or omaveloxolone. Participants must be able to stay at the research center for about a month and follow study procedures.

Inclusion Criteria

Body Mass Index (BMI) at screening between 18 and 32 kg/m^2, inclusive

Must be in good health as determined by the Investigator, based on medical history and Screening evaluations

Exclusion Criteria

Systolic blood pressure > 150 mmHg or < 90 mmHg after sitting for 5 minutes

I have no cancer history, except for treated skin cancer over a year ago.

Corrected QT interval > 450 ms for males and > 460 ms for females

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

Up to 4 weeks

Treatment

Participants receive a single dose of BIIB141 as a capsule or sprinkled over yogurt, followed by a 14-day break, then receive the alternate form

30 days

In-patient stay at research center

Follow-up

Participants are monitored for safety and effectiveness after treatment

Up to 3 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Omaveloxolone

Trial Overview The study tests if there's any difference in how omaveloxolone is processed when taken as capsules versus sprinkled over yogurt. It's a crossover trial where all participants try both methods, with a break in between, to see if yogurt could be an alternative way to take this medication.

How Is the Trial Designed?

Group I: Treatment Sequence BA Experimental Treatment 1 Intervention

Participants will receive treatment B (omaveloxolone capsule content sprinkled over low-fat, non-Greek yogurt on Day 1) followed by treatment A (omaveloxolone capsule orally on Day 15).

Group II: Treatment Sequence AB Experimental Treatment 1 Intervention

Participants will receive treatment A (omaveloxolone capsule orally on Day 1) followed by treatment B (omaveloxolone capsule content sprinkled over low-fat, non-Greek yogurt on Day 15).

Who Is Running the Clinical Trial?

Biogen

Lead Sponsor

Trials

655

Recruited

468,000+

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

1. clinicaltrials.gov clinicaltrials.gov/study/NCT03664453

A Pharmacokinetic Study of Omaveloxolone in Healthy ... This study will determine the effect of food on the pharmacokinetics of omaveloxolone (150 mg) in healthy adult subjects and will assess the safety, ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12579712/

Early experience on omaveloxolone in adult patients with ... Conclusions. Omaveloxolone seems to be safe and well-tolerated in adult FRDA patients in the real-life setting. No neurological progression was ...

3. ascpt.onlinelibrary.wiley.com ascpt.onlinelibrary.wiley.com/doi/full/10.1111/cts.70139

Effect of a Supratherapeutic Dose of Omaveloxolone on the ... Omaveloxolone has been shown to not increase blood pressure and has not been associated with any other AEs on ECG parameters, including ejection ...

4. neurologylive.com neurologylive.com/view/long-term-data-suggest-friedreich-ataxia-agent-omaveloxolone-slows-disease-progression-over-4-years

Long-Term Data Suggest Friedreich Ataxia Agent ... Omaveloxolone treatment in FA patients resulted in slower disease progression, with improved mFARS and FA-ADL scores over four years compared to ...

5. ojrd.biomedcentral.com ojrd.biomedcentral.com/articles/10.1186/s13023-024-03474-6

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 ...

6. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/nda/2023/216718Orig1s000SumR.pdf

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. link.springer.com link.springer.com/article/10.1007/s40120-025-00749-3

Safety Monitoring of Omaveloxolone in Friedreich Ataxia The drug was also safe and well tolerated in the MOXIe study, but its dosing is complicated by interactions with strong CYP4A3 inhibitors.

8. curefa.org curefa.org/clinical-studies/observational-multinational-post-marketing-registry-of-omaveloxolone-treated-patients-with-friedreichs-ataxia/

A Study to Learn More About the Long-Term Safety of ... In this study, researchers will learn more about the safety of BIIB141, also known as omaveloxolone or SKYCLARYS®, in people with Friedreich's ataxia (FA).