An Observational, Multinational, Post-Marketing Registry of Omaveloxolone-Treated Patients With Friedreich's Ataxia
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- Biogen
- 入组人数
- 300
- 试验地点
- 28
- 主要终点
- Omaveloxolone Naive Cohort: Number of Participants With Treatment-Emergent Serious Adverse Events (TESAEs)
研究概览
简要总结
In this study, researchers will learn more about the safety of BIIB141, also known as omaveloxolone or SKYCLARYS®. This is a drug available for doctors to prescribe for people with Friedreich's Ataxia, also known as FA. This is known as an "observational" study, which collects health information about study participants without changing their medical care. Participants for this study will be found using a group called the Friedreich's Ataxia Global Clinical Consortium (FA GCC) UNIFIED Natural History Study (UNIFAI). The FA-GCC is a group of study research centers that helps provide clinical care for FA patients and also helps researchers learn more about how FA affects patients over a long time.
The main objective of this study is to collect safety information in participants with FA from UNIFAI. Some of the participants in this study will be prescribed BIIB141 for the first time by their own doctors. Some of the participants will have started taking BIIB141 after joining UNIFAI, but less than 12 months before joining this study.
The main questions researchers want to answer in this study are:
- How many participants had serious adverse events (SAEs)? An adverse event is considered serious when it results in death, is life-threatening, causes lasting problems, or requires hospital care.
- How many participants had adverse events (AEs) related to heart failure or liver damage caused by the drug?
Researchers will also learn more about :
• Why and when participants stopped treatment, left the study, or took more of the drug than was prescribed
This study will be done as follows:
- Participants will be screened to check if they can join the study.
- After joining the study, the participants who had never started BIIB141 treatment before must start it within 6 months. Otherwise, all participants will take BIIB141 throughout this study as prescribed by their own doctor.
- During the study, each participant's doctor will decide how often the participant visits the study research center to check on their health. This will be based on the doctor's own clinical judgment and what is recommended by the drug's label.
- Data from the participants' regular visits to their doctor will be collected at 1 month, 2 months, 3 months, 6 months, 12 months, 24 months, 36 months, 48 months, and 60 months.
- Each participant will be in the study for up to 5 years.
详细描述
The primary objective of this study is to assess the long-term safety of omaveloxolone as prescribed to participants with FA in the real-world setting, including characterization of all drug-induced liver injury (DILI) and congestive heart failure (CHF) AEs. The secondary objective of this study is to capture the reasons and timing of omaveloxolone treatment interruptions, discontinuations, and drug overdose.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Documented diagnosis of FA, including confirmation via genetic testing.
- •Participants aged 16 years and older at initiation of omaveloxolone treatment.
- •For the omaveloxolone-naive cohort
- •- Initiating omaveloxolone treatment as per an approved label concurrent with enrolling in this study.
- •For the omaveloxolone-non-naive cohort
- •Initiated omaveloxolone treatment as per an approved label less than 12 months prior to enrollment in this study
- •Prior to enrollment, maintained omaveloxolone treatment with no discontinuation of more than 60 days
- •Actively on treatment at the time of enrollment in this study
- •Treating physician is the study site principal investigator or sub-investigator
- •Study site confirms ability to provide required baseline data through medical record review, UNIFAI database, or other site-collected data
- •Enrolled in the UNIFAI study prior to initiation of omaveloxolone treatment
排除标准
- •Received off-label prescription of omaveloxolone at any time.
- •Previously enrolled in a clinical trial of omaveloxolone.
- •Participating in a blinded interventional trial at the time of enrollment in the study; participants may participate in other clinical trials after baseline data are collected.
- •Note: Other protocol-defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
Omaveloxolone Naive Participants
Participants with FA who will initiate omaveloxolone treatment per its approved label will be followed prospectively for up to 5 years.
干预措施: Omaveloxolone (Drug)
Omaveloxolone Non-Naive Cohort
Participants with FA who initiated omaveloxolone treatment as per the approved label less than 12 months prior to enrollment in this study will be analyzed retrospectively (baseline data) followed by prospective analysis (post-baseline data) for up to 5 years.
干预措施: Omaveloxolone (Drug)
结局指标
主要结局
Omaveloxolone Naive Cohort: Number of Participants With Treatment-Emergent Serious Adverse Events (TESAEs)
时间窗: From the start of the treatment up to end of the study (up to 5 years)
Omaveloxolone Non-Naive Cohort: Number of Participants With Treatment-Emergent Serious Adverse Events (TESAEs)
时间窗: From enrolment in the current study up to end of the study (up to 5 years)
Omaveloxolone Naive Cohort: Number of Participants With DILI and CHF AEs
时间窗: From the start of the treatment up to end of the study (up to 5 years)
Omaveloxolone Non-Naive Cohort: Number of Participants With DILI and CHF AE
时间窗: From enrolment in the current study up to end of the study (up to 5 years)
次要结局
- Time to Omaveloxolone Treatment Interruption(Up to 5 years)
- Time to Omaveloxolone Treatment Discontinuation(Up to 5 years)
- Time to Omaveloxolone Drug Overdose(Up to 5 years)
- Number of Participants With Reasons for Omaveloxolone Treatment Interruption, Treatment Discontinuation and Overdose(Up to 5 years)