A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia
2상
완료
NCT02255435
| 대상 질환 | Friedreich Ataxia |
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| 임상 단계 | 2상 |
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| 스폰서 | Biogen |
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| 상태 | 완료 |
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| 참여자 수 | 172 명 |
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| 중재(약물) | Omaveloxolone Capsules, 2.5 mg, Omaveloxolone Capsules, 5 mg, Omaveloxolone Capsules, 10 mg, Omaveloxolone Capsules, 20 mg, Omaveloxolone Capsules, 40 mg |
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| 시작일 | 2015년 1월 31일 |
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| 완료일 | 2019년 10월 31일 |
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| 결과 공개일 | 2022년 11월 29일 |
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요약
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 assig
Change From Baseline in Peak Work (in Watts/kg) During Exercise Testing at Week 12 in Part 1
Baseline through 12 weeks after participant receives the first dose in Part 1. · W/kg
| Part 1 Omaveloxolone Capsules 2.5 and 5 mg | 0.14 W/kg |
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| Part 1 Omaveloxolone Capsules 10 mg | 0.07 W/kg |
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| Part 1 Omaveloxolone Capsules 20 mg | -0.09 W/kg |
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| Part 1 Omaveloxolone Capsules 40 mg | 0.06 W/kg |
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| Part 1 Omaveloxolone Capsules 80 mg | 0 W/kg |
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| Part 1 Omaveloxolone Capsules 160 mg | 0.02 W/kg |
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| p-value | 0.1524 |
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| LS Mean difference (Net) | 0.10 (95% CI -0.04–0.23) |
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Change in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 48 in Part 2
48 weeks after participant receives the first dose in Part 2 · score on a scale
| Part 2 Placebo Capsules | 0.85 ± 0.640 score on a scale |
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| Part 2 Omaveloxolone Capsules 150 mg | -1.55 ± 0.689 score on a scale |
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| p-value | 0.0141 |
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| LS Mean difference (Net) | -2.40 (95% CI -4.31–-0.50) |
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