RTA 408 Capsules in Patients With Friedreich's Ataxia - MOXIe
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ClinicalTrials.gov Identifier: NCT02255435 |
Recruitment Status :
Active, not recruiting
First Posted : October 2, 2014
Results First Posted : November 29, 2022
Last Update Posted : February 5, 2024
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Tracking Information | |||||||
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First Submitted Date ICMJE | September 30, 2014 | ||||||
First Posted Date ICMJE | October 2, 2014 | ||||||
Results First Submitted Date ICMJE | September 30, 2022 | ||||||
Results First Posted Date ICMJE | November 29, 2022 | ||||||
Last Update Posted Date | February 5, 2024 | ||||||
Actual Study Start Date ICMJE | January 31, 2015 | ||||||
Actual Primary Completion Date | October 31, 2019 (Final data collection date for primary outcome measure) | ||||||
Current Primary Outcome Measures ICMJE |
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Original Primary Outcome Measures ICMJE |
Measure the change of peak workload (in watts/kg) during exercise testing [ Time Frame: 12 weeks ] | ||||||
Change History | |||||||
Current Secondary Outcome Measures ICMJE |
Change in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 12 in Part 1 [ Time Frame: 12 weeks after participant receives the first dose in Part 1 ] The mFARS includes 4 of the 5 sections of the Friedreich's Ataxia Rating Scale (FARS): bulbar (score 0 to 11), upper limb coordination (score 0 to 36), lower limb coordination (score 0 to 16), and upright stability (score 0 to 36). The minimum score is 0 and the maximum score is 99. A lower score indicates better neurological function.
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Original Secondary Outcome Measures ICMJE |
Measure the change in the modified Friedreich's ataxia rating scale (FARS) [ Time Frame: 12 weeks ] | ||||||
Current Other Pre-specified Outcome Measures | Not Provided | ||||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||||
Descriptive Information | |||||||
Brief Title ICMJE | RTA 408 Capsules in Patients With Friedreich's Ataxia - MOXIe | ||||||
Official Title ICMJE | A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Friedreich's Ataxia (MOXIe) | ||||||
Brief Summary | 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. |
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Detailed Description | Study Sponsor, originally Reata Pharmaceuticals, Inc., is now Reata Pharmaceuticals, Inc., a wholly owned subsidiary of Biogen. | ||||||
Study Type ICMJE | Interventional | ||||||
Study Phase ICMJE | Phase 2 | ||||||
Study Design ICMJE | Allocation: Randomized Intervention Model: Parallel Assignment Masking: Double (Participant, Investigator) Primary Purpose: Treatment |
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Condition ICMJE | Friedreich Ataxia | ||||||
Intervention ICMJE |
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Study Arms ICMJE |
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Publications * |
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||||
Recruitment Status ICMJE | Active, not recruiting | ||||||
Actual Enrollment ICMJE |
172 | ||||||
Original Estimated Enrollment ICMJE |
52 | ||||||
Estimated Study Completion Date ICMJE | December 1, 2024 | ||||||
Actual Primary Completion Date | October 31, 2019 (Final data collection date for primary outcome measure) | ||||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 16 Years to 40 Years (Child, Adult) | ||||||
Accepts Healthy Volunteers ICMJE | No | ||||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||||
Listed Location Countries ICMJE | Australia, Austria, Italy, United Kingdom, United States | ||||||
Removed Location Countries | Brazil | ||||||
Administrative Information | |||||||
NCT Number ICMJE | NCT02255435 | ||||||
Other Study ID Numbers ICMJE | RTA 408-C-1402 | ||||||
Has Data Monitoring Committee | Yes | ||||||
U.S. FDA-regulated Product | Not Provided | ||||||
IPD Sharing Statement ICMJE |
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Current Responsible Party | Biogen ( Reata, a wholly owned subsidiary of Biogen ) | ||||||
Original Responsible Party | Reata Pharmaceuticals, Inc. | ||||||
Current Study Sponsor ICMJE | Reata, a wholly owned subsidiary of Biogen | ||||||
Original Study Sponsor ICMJE | Reata Pharmaceuticals, Inc. | ||||||
Collaborators ICMJE |
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Investigators ICMJE | Not Provided | ||||||
PRS Account | Biogen | ||||||
Verification Date | February 2024 | ||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |