What side effects are associated with this type of intervention?

Common treatments for Friedreich's Ataxia that focus on Nrf2 activation and antioxidant gene induction, such as Omaveloxolone, generally aim to mitigate oxidative stress and improve mitochondrial function. Known side effects of these treatments can include gastrointestinal issues (nausea, diarrhea), liver enzyme elevations, and potential interactions with other medications. Patients may also experience fatigue and headaches. It is important to monitor these side effects closely and discuss them with a healthcare provider to manage and mitigate any adverse effects effectively.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for Friedreich's Ataxia. Omaveloxolone, which activates Nrf2 and induces antioxidant target genes, is currently being studied for its potential therapeutic effects in Friedreich's Ataxia. This approach aims to address the impaired antioxidative defense mechanisms characteristic of the disease. Other treatments under investigation include various antioxidants and agents targeting mitochondrial dysfunction, but none have received FDA approval yet.

What other types of research exist?

Currently, there are no specific alternatives to Omaveloxolone mentioned in the provided context for Friedreich's Ataxia. However, treatments that enhance antioxidant defense mechanisms or target oxidative stress pathways, such as N-acetylcysteine (NAC) and other Nrf2 activators, may be considered as potential alternatives. Consulting with a healthcare provider for the latest research and clinical trials is recommended.

← Back to Search

Nrf2 Activator

Omaveloxolone for Friedreich's Ataxia

Phase 2

Waitlist Available

Research Sponsored by Biogen

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Have genetically confirmed Friedreich's ataxia

Be male or female and ≥16 years of age and ≤40 years of age

Must not have

Have uncontrolled diabetes (HbA1c >11.0%)

Have known active fungal, bacterial, and/or viral infection, including human immunodeficiency virus or hepatitis virus (B or C)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 weeks after participant receives the first dose in part 1

Summary

This trial is testing omaveloxolone, a drug that may help people with Friedreich's ataxia by boosting their body's natural defenses against cell damage. The study focuses on patients with this genetic condition because they have weakened antioxidant systems. Omaveloxolone works by activating a protein that helps protect cells from damage. It aims to improve cell function and reduce harmful effects in people with Friedreich's ataxia.

Who is the study for?

This trial is for people aged 16-40 with genetically confirmed Friedreich's ataxia, who can swallow capsules and have a specific level of disease severity (modified FARS score between 20 and 80). Participants should be stable in their exercise routine and able to complete maximal exercise testing. They cannot join if they've used certain drugs recently, have significant heart disease or abnormal blood tests, uncontrolled diabetes, cognitive impairments, active infections, or substance abuse issues.

What is being tested?

The study examines omaveloxolone (RTA 408) capsules at various doses for treating Friedreich's ataxia. It has two parts: the first part tests safety across different doses; the second part assesses safety and effectiveness of a set dose (150 mg). An extension phase will look into long-term use. The drug aims to activate Nrf2 signaling impaired in this condition.

What are the potential side effects?

While not explicitly listed here, potential side effects may include reactions related to oxidative stress mitigation since omaveloxolone activates antioxidant pathways. These could involve changes in energy levels, gastrointestinal symptoms or possible liver enzyme alterations given its mechanism.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

My condition is genetically confirmed Friedreich's ataxia.

Select...

I am between 16 and 40 years old.

Select...

I can swallow pills.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

My diabetes is not under control (HbA1c >11.0%).

Select...

I do not have any active infections, including HIV or hepatitis.

Select...

I have previously participated in a trial with omaveloxolone.

Select...

I have a history of serious heart disease.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline through 12 weeks after participant receives the first dose in part 1.

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline through 12 weeks after participant receives the first dose in part 1.

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline through 12 weeks after participant receives the first dose in part 1. for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change From Baseline in Peak Work (in Watts/kg) During Exercise Testing at Week 12 in Part 1

Change in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 48 in Part 2

Secondary study objectives

Change in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 12 in Part 1

Trial Design

10Treatment groups

Experimental Treatment

Placebo Group

Group I: Part 2 Omaveloxolone Capsules 150 mgExperimental Treatment1 Intervention

Omaveloxolone (RTA 408) Capsules, 150 mg administered orally once daily for 48 weeks

Group II: Part 1 Omaveloxolone Capsules 80 mgExperimental Treatment1 Intervention

Omaveloxolone (RTA 408) Capsules, 80 mg administered orally once daily for 12 weeks

Group III: Part 1 Omaveloxolone Capsules 40 mgExperimental Treatment1 Intervention

Omaveloxolone (RTA 408) Capsules, 40 mg administered orally once daily for 12 weeks

Group IV: Part 1 Omaveloxolone Capsules 300 mgExperimental Treatment1 Intervention

Omaveloxolone (RTA 408) Capsules, 300 mg administered orally once daily for 12 weeks

Group V: Part 1 Omaveloxolone Capsules 20 mgExperimental Treatment1 Intervention

Omaveloxolone (RTA 408) Capsules, 20 mg administered orally once daily for 12 weeks

Group VI: Part 1 Omaveloxolone Capsules 2.5 and 5 mgExperimental Treatment2 Interventions

omaveloxolone (RTA 408) Capsules, 2.5 mg administered orally one daily for 2 weeks, then 5 mg taken orally once daily for 10 weeks

Group VII: Part 1 Omaveloxolone Capsules 160 mgExperimental Treatment1 Intervention

Omaveloxolone (RTA 408) Capsules, 160 mg administered orally once daily for 12 weeks

Group VIII: Part 1 Omaveloxolone Capsules 10 mgExperimental Treatment1 Intervention

omaveloxolone (RTA 408) Capsules, 10 mg administered orally once daily for 12 weeks

Group IX: Part 1 Placebo CapsulesPlacebo Group1 Intervention

Placebo capsules administered orally once daily for 12 weeks

Group X: Part 2 Placebo CapsulesPlacebo Group1 Intervention

Placebo capsules administered orally once daily for 48 weeks

0 / 7Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Friedreich's Ataxia often focus on addressing oxidative stress and mitochondrial dysfunction, which are central to the disease's pathology. Omaveloxolone, for example, activates the Nrf2 pathway, leading to the induction of antioxidant genes that help mitigate oxidative damage. This is crucial for Friedreich's Ataxia patients because their condition is characterized by impaired antioxidative defense mechanisms and mitochondrial iron overload, which contribute to cellular damage and disease progression. By enhancing the body's natural antioxidant defenses, treatments like Omaveloxolone aim to reduce oxidative stress and improve cellular function, potentially slowing disease progression and improving quality of life.

Myricetin alleviates cuprizone-induced behavioral dysfunction and demyelination in mice by Nrf2 pathway.