A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia

Summary

In this study, researchers are learning more about RTA 408, also known as omaveloxolone, BIIB141, or SKYCLARYS®. The main goal of this study is to learn more about the safety of RTA 408 and how it affects physical effort, movement, coordination, and how participants feel in daily life.

The main questions researchers want to answer in this study are:

* How much physical effort can a participant produce during a cycling test after 12 weeks of treatment?
* How do scores on the modified Friedreich's Ataxia Rating Scale (mFARS) change after 48 weeks?

Researchers will use the modified Friedreich's Ataxia Rating Scale (mFARS) to measure how FA affects the nervous system. The mFARS looks at movement ability, balance, coordination, speech, and how well the arms and legs work.

They will also use a cycling test to measure physical effort, along with questionnaires to learn how participants feel and function in daily life.

Safety will also be tested using physical exams, vital sign checks, echocardiograms (ECHO), electrocardiograms (ECG), and blood and urine tests.

The study will be done in 2 main parts, followed by an optional Extension period:

* In Part 1, participants will be randomly assigned to take different doses of RTA 408 or a placebo by mouth once a day for 12 weeks. A placebo looks like the study drug but contains no real medicine.
* Researchers will compare these doses to decide which one to use in Part 2.
* In Part 2, a different group of participants will take either the chosen dose of RTA 408 (150 mg) or placebo once a day for 48 weeks.
* Participants who complete Part 1 or Part 2 may be able to join an Extension period, where everyone receives RTA 408.
* In the Extension period, participants will continue to receive RTA 408 until the drug becomes commercially available or until they leave the study
* Participants in Part 1 will have up to 9 study visits and 2 phone calls. If they do not move onto the Extension period, they will stay in the study for up to 20 weeks.
* Participants in Part 2 will have up to 10 study visits and 3 phone calls. If they do not move onto the Extension period, they will stay in the study for up to 61 weeks.
* Participants in the Extension period will have 2 visits in the first month, followed by visits every 6 months.

Conditions

• Friedreich Ataxia

Interventions

DRUG

Omaveloxolone Capsules, 2.5 mg

DRUG

Omaveloxolone Capsules, 5 mg

DRUG

Omaveloxolone Capsules, 10 mg

DRUG

Placebo

DRUG

Omaveloxolone Capsules, 20 mg

DRUG

Omaveloxolone Capsules, 40 mg

DRUG

Omaveloxolone Capsules, 80 mg

DRUG

Omaveloxolone Capsules, 160 mg

DRUG

Omaveloxolone Capsules, 300 mg

DRUG

Omaveloxolone Capsules, 150 mg

Sponsors & Collaborators

• AbbVie

  collaborator INDUSTRY

• Friedreich's Ataxia Research Alliance

  collaborator OTHER

• Biogen

  lead INDUSTRY

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Model

PARALLEL

Eligibility

Min Age

16 Years

Max Age

40 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2015-01-31

Primary Completion

2019-10-31

Completion

2025-12-19

Countries

• United States
• Australia
• Austria
• Italy
• United Kingdom

Study Locations