NCT02255435Befejeződött2. fázis
A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia
A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Friedreich's Ataxia (MOXIe)
Forrás: ClinicalTrials.gov · Utoljára frissítve: 2026. szept. 3.
Rövid összefoglaló (regiszter, angolul)
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 assigned to take different doses of RTA 408 or a placebo by mouth once a day for 12 weeks. A placebo looks like the study drug but contains no real medicine. * Researchers will compare these doses to decide which one to use in Part 2. * In Part 2, a different group of participants will take either the chosen dose of RTA 408 (150 mg) or placebo once a day for 48 weeks. * Participants who complete Part 1 or Part 2 may be able to join an Extension period, where everyone receives RTA 408. * In the Extension period, participants will continue to receive RTA 408 until the drug becomes commercially available or until they leave the study * Participants in Part 1 will have up to 9 study visits and 2 phone calls. If they do not move onto the Extension period, they will stay in the study for up to 20 weeks. * Participants in Part 2 will have up to 10 study visits and 3 phone calls. If they do not move onto the Extension period, they will stay in the study for up to 61 weeks. * Participants in the Extension period will have 2 visits in the first month, followed by visits every 6 months.
- Állapot (regiszter)
- Befejeződött
- Fázis (regiszter)
- 2. fázis
- Vizsgálattípus
- Beavatkozással járó
- Résztvevők száma
- 172 (tényleges)
- Első benyújtás
- 2014. szept. 30.
- Kezdés
- 2015. jan. 31.
- Elsődleges befejezés
- 2019. okt. 31.
- Fő szponzor
- Biogen
- Betegségek
- Friedreich-ataxia
- NCT-szám
- NCT02255435
- EudraCT-szám
- 2024-517436-22
Mit vállalsz
- Vizsgálat típusa: Beavatkozással járó · 2. fázis
- Bevont résztvevők: 172
- 2015. jan. 31. és az elsődleges befejezés (2019. okt. 31.) között
- 11 helyszín, 5 országban
- Vizitek és vizsgálatok: kérdezd a vizsgálat csapatát, a regiszter nem sorolja fel őket
A(z) ClinicalTrials.gov regisztermezőiből összeállítva. A vizsgálat felépítését írja le.
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Korábbi kezelés
- Have known or suspected active drug or alcohol abuse
- 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
- Prior participation in a trial with omaveloxolone (RTA 408)
Más betegség
- 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 a cognitive impairment that may preclude ability to comply with study procedures
Laborértékek
- Have uncontrolled diabetes (HbA1c >11.0%)
- 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
Egyéb
- Have B-type natriuretic peptide value >200 pg/mL
- 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
Kiket keres a vizsgálat
- 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
Életkor a regiszterben: 16 év – 40 év · Nem: nők és férfiak · Egészséges önkénteseket nem fogad
Helyszínek (11)
Helyszínek a regiszter sorrendjében. Ha a profilodba mented a városodat, távolság szerint rendeződnek.
| Helyszín | Város | Ország | Helyszín állapota |
|---|---|---|---|
| Medical University Innsbruck | Innsbruck | Ausztria | Ismeretlen állapot |
| Murdoch Childrens Research Institute | Parkville | Ausztrália | Ismeretlen állapot |
| University College of London | London | Egyesült Királyság | Ismeretlen állapot |
| Neurological Institute Carlo Besta | Milan | Olaszország | Ismeretlen állapot |
| Emory University Hospital - Neurology | Atlanta | Egyesült Államok | Ismeretlen állapot |
| Ohio State University - Neurology | Columbus | Egyesült Államok | Ismeretlen állapot |
| University of Florida - Neurology | Gainesville | Egyesült Államok | Ismeretlen állapot |
| University of Iowa Stead Family Children's Hospital | Iowa City | Egyesült Államok | Ismeretlen állapot |
| UCLA | Los Angeles | Egyesült Államok | Ismeretlen állapot |
| Children's Hospital of Philadelphia | Philadelphia | Egyesült Államok | Ismeretlen állapot |
| USF Ataxia Research Center | Tampa | Egyesült Államok | Ismeretlen állapot |
Ez az oldal tájékoztatás, nem orvosi tanács. Bármely vizsgálatról az orvosoddal beszélj.