Status:

ACTIVE_NOT_RECRUITING

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

Lead Sponsor:

Biogen

Collaborating Sponsors:

AbbVie

Friedreich's Ataxia Research Alliance

Conditions:

Friedreich Ataxia

Eligibility:

All Genders

16-40 years

Phase:

PHASE2

Brief 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 aff...

Detailed Description

Friedreich's ataxia is an autosomal recessive cerebellar ataxia caused by triplet-repeat expansions. The causative mutation is a trinucleotide (GAA) repeat expansion in the first intron of the frataxi...

Eligibility Criteria

Inclusion

  • Have genetically confirmed Friedreich's ataxia
  • Have a modified FARS score ≥20 and ≤80
  • Be male or female and ≥16 years of age and ≤40 years of age
  • Have no changes to exercise regimen within 30 days prior to Study Day 1 and be willing to remain on the same exercise regimen during the 16-week study period
  • Have the ability to complete maximal exercise testing
  • Be able to swallow capsules

Exclusion

  • Have uncontrolled diabetes (HbA1c \>11.0%)
  • Have B-type natriuretic peptide value \>200 pg/mL
  • Have a history of clinically significant left-sided heart disease and/or clinically significant cardiac disease
  • Have known active fungal, bacterial, and/or viral infection, including human immunodeficiency virus or hepatitis virus (B or C)
  • Have known or suspected active drug or alcohol abuse
  • Have clinically significant abnormalities of clinical hematology or biochemistry, including but not limited to elevations greater than 1.5 times the upper limit of normal of aspartate aminotransferase, or alanine aminotransferase
  • Have any abnormal laboratory test value or serious pre-existing medical condition that, in the opinion of the investigator, would put the patient at risk by study enrollment
  • Have taken any of the following drugs within 7 days prior to Study Day 1 or plan to take any of these drugs during the time of study participation:
  • Sensitive substrates for cytochrome P450 2C8 or 3A4 (e.g., repaglinide, midazolam, sildenafil)
  • Moderate or strong inhibitors or inducers of cytochrome P450 3A4 (e.g., carbamazepine, phenytoin, ciprofloxacin, grapefruit juice)
  • Substrates for p-glycoprotein transporter (e.g., ambrisentan, digoxin)
  • Have participated in any other interventional clinical study within 30 days prior to Study Day 1
  • Have a cognitive impairment that may preclude ability to comply with study procedures
  • Prior participation in a trial with omaveloxolone (RTA 408)

Key Trial Info

Start Date :

January 31 2015

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

December 31 2025

Estimated Enrollment :

172 Patients enrolled

Trial Details

Trial ID

NCT02255435

Start Date

January 31 2015

End Date

December 31 2025

Last Update

December 31 2025

Active Locations (11)

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Page 1 of 3 (11 locations)

1

UCLA

Los Angeles, California, United States, 90095

2

University of Florida - Neurology

Gainesville, Florida, United States, 32610

3

USF Ataxia Research Center

Tampa, Florida, United States, 33612

4

Emory University Hospital - Neurology

Atlanta, Georgia, United States, 30329