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Trial record 1 of 1 for:    Reata | omaveloxolone and Friedreich's ataxia
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RTA 408 Capsules in Patients With Friedreich's Ataxia - MOXIe

This study is currently recruiting participants.
Verified November 2017 by Reata Pharmaceuticals, Inc.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02255435
First Posted: October 2, 2014
Last Update Posted: November 6, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborators:
AbbVie
Friedreich's Ataxia Research Alliance
Information provided by (Responsible Party):
Reata Pharmaceuticals, Inc.
  Purpose

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 frataxin gene, leading to impaired transcription of frataxin. The pathological consequences of frataxin deficiency include a severe disruption of iron-sulfur cluster biosynthesis, mitochondrial iron overload coupled to cellular iron dysregulation, and an increased sensitivity to oxidative stress.

A hallmark of Friedreich's ataxia is impairment of antioxidative defense mechanisms, which play a major role in disease progression. Studies have demonstrated that nuclear factor erythroid-derived 2-related factor 2 (Nrf2) signaling is grossly impaired in patients with Friedreich's ataxia. Therefore, the ability of omaveloxolone (RTA 408) to activate Nrf2 and induce antioxidant target genes is hypothesized to be therapeutic in patients with Friedreich's ataxia.

This 2-part study will evaluate the efficacy, safety, and pharmacodynamics of omaveloxolone (RTA 408) in the treatment of patients with Friedreich's ataxia.

Part 1: The first part of this study will be a randomized, placebo-controlled, double-blind, dose-escalation study to evaluate the safety of omaveloxolone (RTA 408) at various doses in patients with Friedreich's ataxia.

Part 2: The second part of this study is a randomized, placebo-controlled, double-blind, parallel-group study to evaluate the safety and efficacy of omaveloxolone (RTA 408) 150 mg in patients with Friedreich's ataxia. Patients enrolled in Part 2 will be randomized 1:1 to receive omaveloxolone (RTA 408) 150 mg or placebo.

Extension: The extension will assess long-term safety and tolerability of omaveloxolone (RTA 408) in qualified patients with Friedreich's ataxia following completion of Part 1 or Part 2. Patients will not be unblinded to study treatment in Part 1 or Part 2 upon entering the extension study. Patients will receive open-label omaveloxolone (RTA 408) at 150 mg once daily.


Condition Intervention Phase
Friedreich Ataxia 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 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Friedreich's Ataxia (MOXIe)

Resource links provided by NLM:


Further study details as provided by Reata Pharmaceuticals, Inc.:

Primary Outcome Measures:
  • Measure the change in the modified Friedreich's ataxia rating scale (FARS) [ Time Frame: 24 weeks ]

Secondary Outcome Measures:
  • Measure the change of peak workload (in watts/kg) during exercise testing [ Time Frame: 24 weeks ]

Estimated Enrollment: 172
Study Start Date: January 2015
Estimated Study Completion Date: December 2022
Estimated Primary Completion Date: January 2020 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Omaveloxolone Capsules 2.5 and 5 mg
omaveloxolone (RTA 408) Capsules, 2.5 mg taken orally one daily for 2 weeks, then 5 mg taken orally once daily for 10 weeks
Drug: Omaveloxolone Capsules, 2.5 mg
Other Name: RTA 408 Capsules 2.5 mg
Drug: Omaveloxolone Capsules, 5 mg
Other Name: RTA 408 capsules, 5 mg
Experimental: Omaveloxolone Capsules 10 mg
omaveloxolone (RTA 408) Capsules, 10 mg taken orally once daily for 12 weeks
Drug: Omaveloxolone Capsules, 10 mg
Other Name: RTA 408 capsules, 10 mg
Placebo Comparator: Placebo Capsules
Placebo capsules taken orally once daily for 12 weeks
Drug: Placebo
Experimental: Omaveloxolone Capsules 20 mg
omaveloxolone (RTA 408) Capsules, 20 mg taken orally once daily for 12 weeks
Drug: Omaveloxolone Capsules, 20 mg
Other Name: RTA 408 capsules, 20 mg
Experimental: Omaveloxolone Capsules 40 mg
omaveloxolone (RTA 408) Capsules, 40 mg taken orally once daily for 12 weeks
Drug: Omaveloxolone Capsules, 40 mg
Other Name: RTA 408 capsules, 40 mg
Experimental: Omaveloxolone Capsules 80 mg
omaveloxolone (RTA 408) Capsules, 80 mg taken orally once daily for 12 weeks
Drug: Omaveloxolone Capsules, 80 mg
Other Name: RTA 408 capsules, 48 mg
Experimental: Omaveloxolone Capsules 160 mg
omaveloxolone (RTA 408) Capsules, 160 mg taken orally once daily for 12 weeks
Drug: Omaveloxolone Capsules, 160 mg
Other Name: RTA 408 capsules, 160 mg
Experimental: Omaveloxolone Capsules 300 mg
omaveloxolone (RTA 408) Capsules, 300 mg taken orally once daily for 12 weeks
Drug: Omaveloxolone Capsules, 300 mg
Other Name: RTA 408 capsules, 300 mg
Experimental: Omaveloxolone Capsules 150 mg
omaveloxolone (RTA 408) Capsules, 150 mg taken orally once daily for 24 weeks
Drug: Omaveloxolone Capsules, 150 mg
Other Name: RTA 408 capsules, 150 mg

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   16 Years to 40 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Have genetically confirmed Friedreich's ataxia
  2. Have a modified FARS score ≥20 and ≤80
  3. Be male or female and ≥16 years of age and ≤40 years of age
  4. 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
  5. Have the ability to complete maximal exercise testing
  6. Be able to swallow capsules

Exclusion Criteria:

  1. Have uncontrolled diabetes (HbA1c >11.0%)
  2. Have B-type natriuretic peptide value >200 pg/mL
  3. Have a history of clinically significant left-sided heart disease and/or clinically significant cardiac disease
  4. Have known active fungal, bacterial, and/or viral infection, including human immunodeficiency virus or hepatitis virus (B or C)
  5. Have known or suspected active drug or alcohol abuse
  6. 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
  7. 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
  8. 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:

    1. Sensitive substrates for cytochrome P450 2C8 or 3A4 (e.g., repaglinide, midazolam, sildenafil)
    2. Moderate or strong inhibitors or inducers of cytochrome P450 3A4 (e.g., carbamazepine, phenytoin, ciprofloxacin, grapefruit juice)
    3. Substrates for p-glycoprotein transporter (e.g., ambrisentan, digoxin)
  9. Have participated in any other interventional clinical study within 30 days prior to Study Day 1
  10. Have a cognitive impairment that may preclude ability to comply with study procedures
  11. Prior participation in a trial with omaveloxolone (RTA 408)
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02255435


Locations
United States, California
UCLA Recruiting
Los Angeles, California, United States, 90095
Contact: Aaron Fisher    310-206-8153    adfisher@mednet.ucla.edu   
Principal Investigator: Susan Perlman, MD         
United States, Florida
University of Florida - Neurology Recruiting
Gainesville, Florida, United States, 32610
Contact: Aika Konn    352-273-6003    aika.konn@neurology.ufl.edu   
Principal Investigator: SH Subramony, MD         
USF Ataxia Research Center Recruiting
Tampa, Florida, United States, 33612
Contact: Paola Fink-Irizarry       paolaf@health.usf.edu   
Contact: Jessica Shaw       jshaw@helath.usf.edu   
Principal Investigator: Theresa Zesiewicz, MD         
United States, Georgia
Emory University Hospital - Neurology Recruiting
Atlanta, Georgia, United States, 30329
Contact: Becky McMurray, RN    404-712-7013    rebecca.s.mcmurray@emory.edu   
Principal Investigator: George Wilmot, MD         
United States, Iowa
University of Iowa Stead Family Children's Hospital Not yet recruiting
Iowa City, Iowa, United States, 52242
Contact: Corey McDaniel       corey-mcdaniel@uiowa.edu   
Principal Investigator: Katherine Mathews, MD         
United States, Ohio
Ohio State University - Neurology Recruiting
Columbus, Ohio, United States, 43221
Contact: Louisa Mezache    614-685-3030    louisa.mezache@osumc.edu   
Principal Investigator: Chad Hoyle, MD         
United States, Pennsylvania
Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Lauren Hauser    267-426-9738    hauserl@email.chop.edu   
Principal Investigator: David Lynch, MD         
Australia, Victoria
Murdoch Childrens Research Institute Recruiting
Parkville, Victoria, Australia, 3052
Contact: Geneieve Tai    61 3 8341 6374    geneieve.tai@mcri.edu.au   
Principal Investigator: Martin Delatycki, MD         
Austria
Medical University Innsbruck Recruiting
Innsbruck, Austria, 6020
Contact: SN Sigrid Zimmermann       sigrid.zimmermann@tirol-kliniken.at   
Principal Investigator: Sylvia Boesch, MD         
United Kingdom
University College of London Not yet recruiting
London, United Kingdom, WC1E 6BT
Contact: Mila Resuello-Dauti    0203 4484531    Mila.Resuello-Dauti@nhs.net   
Principal Investigator: Paola Giunti, MD         
Sponsors and Collaborators
Reata Pharmaceuticals, Inc.
AbbVie
Friedreich's Ataxia Research Alliance
  More Information

Responsible Party: Reata Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02255435     History of Changes
Other Study ID Numbers: RTA 408-C-1402
First Submitted: September 30, 2014
First Posted: October 2, 2014
Last Update Posted: November 6, 2017
Last Verified: November 2017

Keywords provided by Reata Pharmaceuticals, Inc.:
RTA 408
RTA 408 Capsules
Oxidative Stress
Mitochondrial dysfunction
omaveloxolone

Additional relevant MeSH terms:
Ataxia
Cerebellar Ataxia
Friedreich Ataxia
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinocerebellar Degenerations
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases