Omaveloxolone

Generic Name: Omaveloxolone

Brand Names: Skyclarys

Drug Type: Small Molecule

Chemical Formula: C_₃₃H_₄₄F_₂N_₂O_₃

CAS Number: 1474034-05-3

Unique Ingredient Identifier: G69Z98951Q

Background: Omaveloxolone (RTA-408) is a semisynthetic oleanane triterpenoid with antioxidant and anti-inflammatory properties. Omaveloxolone acts as an activator of nuclear factor (erythroid-derived 2)-like 2 (Nrf2), a transcription factor that mitigates oxidative stress. In patients with Friedreich's ataxia, a genetic disease involving mitochondrial dysfunction, the Nrf2 pathway is impaired, and Nrf2 activity is lower. Therefore, the use of Nrf2 activators such as omaveloxolone represents a therapeutic advantage in this group of patients. In February 2023, omaveloxolone was approved by the FDA for the treatment of Friedreich's ataxia in adults and adolescents aged 16 years and older. The use of omaveloxolone for the treatment of conditions involving mitochondrial dysfunction and oxidative stress has also been evaluated.

Indication: Omaveloxolone is indicated for the treatment of Friedreich's ataxia in adults and adolescents aged 16 years and older.

Associated Conditions: Friedreich's Ataxia

Clinical Trials

Expanded Access to Omaveloxolone for Melanoma for Patients Previously Enrolled in 408-C-1401

Conditions: Melanoma

First Posted Date: 2018-07-20

Last Posted Date: 2024-02-02

Lead Sponsor: Reata, a wholly owned subsidiary of Biogen

Registration Number: NCT03593499

RTA 408 Capsules in Patients With Melanoma - REVEAL

Phase 1

Completed

Conditions: Melanoma
Unresectable (Stage III) Melanoma
Metastatic (Stage IV) Melanoma

Interventions: Drug: Omaveloxolone Capsules (2.5 mg/capsule)
Drug: Nivolumab (240 mg)
Drug: Ipilimumab (3 mg/kg)
Drug: Omaveloxolone Capsules (50 mg/capsule)
Drug: Omaveloxolone Capsules (10 mg/capsule)

First Posted Date: 2014-10-08

Last Posted Date: 2024-02-02

Lead Sponsor: Reata, a wholly owned subsidiary of Biogen

Target Recruit Count: 41

Registration Number: NCT02259231

Locations: 🇺🇸
University of Colorado Cancer Center, Anschutz Cancer Pavilion, Aurora, Colorado, United States
🇺🇸
Christiana Hospital Helen F. Graham Cancer Center, Newark, Delaware, United States
🇺🇸
Highlands Oncology Group, Fayetteville, Arkansas, United States

and more 7 locations

RTA 408 Capsules in Patients With Friedreich's Ataxia - MOXIe

Phase 2

Active, not recruiting

Conditions: Friedreich Ataxia

Interventions: Drug: Omaveloxolone Capsules, 80 mg
Drug: Omaveloxolone Capsules, 160 mg
Drug: Omaveloxolone Capsules, 300 mg
Drug: Placebo
Drug: Omaveloxolone Capsules, 150 mg
Drug: Omaveloxolone Capsules, 2.5 mg
Drug: Omaveloxolone Capsules, 10 mg
Drug: Omaveloxolone Capsules, 20 mg
Drug: Omaveloxolone Capsules, 40 mg
Drug: Omaveloxolone Capsules, 5 mg

First Posted Date: 2014-10-02

Last Posted Date: 2025-04-18

Lead Sponsor: Reata, a wholly owned subsidiary of Biogen

Target Recruit Count: 172

Registration Number: NCT02255435

Locations: 🇦🇹
Medical University Innsbruck, Innsbruck, Austria
🇺🇸
UCLA, Los Angeles, California, United States
🇬🇧
University College of London, London, United Kingdom

and more 8 locations

RTA 408 Capsules in Patients With Mitochondrial Myopathy - MOTOR

Phase 2

Completed

Conditions: MItochondrial Myopathies

Interventions: Drug: omaveloxolone capsules, 40 mg
Drug: omaveloxolone capsules, 80 mg
Drug: omaveloxolone capsules, 10 mg
Drug: Placebo capsules
Drug: Omaveloxolone capsules, 2.5 mg
Drug: omaveloxolone capsules, 20 mg
Drug: omaveloxolone capsules, 160 mg
Drug: omaveloxolone capsules, 5 mg

First Posted Date: 2014-10-02

Last Posted Date: 2024-02-05

Lead Sponsor: Reata, a wholly owned subsidiary of Biogen

Target Recruit Count: 53

Registration Number: NCT02255422

Locations: 🇺🇸
Mass General Hospital, Boston, Massachusetts, United States
🇺🇸
Baylor College of Medicine, Houston, Texas, United States
🇺🇸
UCLA, Los Angeles, California, United States

and more 6 locations

RTA 408 Lotion in Patients at Risk for Radiation Dermatitis - PRIMROSE

Phase 2

Completed

Conditions: Breast Cancer

Interventions: Drug: Omaveloxolone Lotion 0.5%
Drug: Omaveloxolone Lotion 3%
Drug: Vehicle Lotion
Radiation: 3D conformal radiation therapy

First Posted Date: 2014-05-20

Last Posted Date: 2024-02-05

Lead Sponsor: Reata, a wholly owned subsidiary of Biogen

Target Recruit Count: 187

Registration Number: NCT02142959

Locations: 🇺🇸
University of Colorado Hospital, Dept. of Radiation Oncology, Aurora, Colorado, United States
🇺🇸
Arizona Cancer Center, Scottsdale, Arizona, United States
🇺🇸
Lakeland Regional Cancer Center, Lakeland, Florida, United States

and more 24 locations

RTA 408 Ophthalmic Suspension for the Prevention of Corneal Endothelial Cell Loss Following Cataract Surgery - GUARD

Phase 2

Completed

Conditions: Ocular Pain
Ocular Inflammation
Corneal Endothelial Cell Loss
Cataract Surgery

Interventions: Drug: Vehicle Ophthalmic Solution
Drug: Omaveloxolone Ophthalmic Suspension 0.5%
Drug: Omaveloxolone Ophthalmic Suspension 1%

First Posted Date: 2014-05-01

Last Posted Date: 2024-02-05

Lead Sponsor: Reata, a wholly owned subsidiary of Biogen

Target Recruit Count: 307

Registration Number: NCT02128113

Locations: 🇺🇸
JacksonEye, Lake Villa, Illinois, United States
🇺🇸
Comprehensive Eye Care, Washington, Missouri, United States
🇺🇸
Levenson Eye Associates, Jacksonville, Florida, United States

and more 12 locations