An Observational Registry-Based Study to Evaluate the Long-Term Safety of Tofersen in People With SOD1-ALS
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- Biogen
- 入组人数
- 125
- 试验地点
- 1
- 主要终点
- Baseline Demographic: Age
研究概览
简要总结
In this study, researchers will learn more about the safety of tofersen, also known as Qalsody®. This is a drug available for doctors to prescribe for participant with a certain type of amyotrophic lateral sclerosis, also known as ALS. This type is in participant who have a mutation in the superoxide dismutase 1 gene, also known as SOD-1.
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 2 different groups of study research centers that help provide clinical care for participant with ALS. These groups are in Europe and the United States and are called:
- the Precision-ALS programme
- the ALS/Motor Neuron Disease (MND) Natural History Consortium (NHC)
The main goal of this study is to collect safety information in participants with SOD-1 ALS who were in either of the groups.
The main question researchers want to answer in this study is:
- What are the characteristics of the participants in this study?
- How many participants had serious adverse events (SAEs), including ones that affect the brain, spinal cord, or nerves?
An adverse event is a health problem that may or may not be caused by a drug during the study. An adverse event is considered serious when it results in death, is life-threatening, causes lasting problems, or requires hospital care.
Researchers will also learn more about:
- How many participants develop other health conditions or become pregnant, including how the pregnancy turned out
- Why and when participants stopped treatment
This study will be done as follows:
- Participants will be screened to check if they can join the study.
- Data from the participants' regular visits to their clinic will be collected based on which study research center they are in.
- Each participant will be in the study until they decide to leave or until death. Currently, the study is planned to last at least 7 years.
详细描述
The primary objectives of this study are to describe demographic and clinical characteristics of participants with superoxide dismutase 1-amyotropic lateral sclerosis (SOD1-ALS); to describe the frequency of SAEs among participants with SOD1-ALS, including serious neurologic events previously reported in clinical trial participants (e.g., myelitis, radiculitis, aseptic meningitis, increased intracranial pressure, and/or papilloedema).
The secondary objectives of this study are to describe the frequency of new comorbid conditions, pregnancy and pregnancy outcome among participants with SOD1-ALS; to describe the frequency of treatment discontinuation among participants with SOD1-ALS treated with tofersen.
NHC registry has started the data collection while the Treatment Research Initiative to Cure ALS (TRICALS) is yet to start.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants with an ALS diagnosis and a confirmed SOD1 mutation from contributing registry networks will be considered for inclusion in the study.
排除标准
- •Data collected while a person with SOD1-ALS is participating in an interventional clinical trial (with tofersen or any other investigational medicinal product) will be excluded.
- •NOTE: Other protocol- defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
Tofersen
Data for participants with SOD1-ALS will be collected via ALS disease registries, the TRICALS network's Precision-ALS programme and ALS/MND NHC.
干预措施: Tofersen (Drug)
结局指标
主要结局
Baseline Demographic: Age
时间窗: At Baseline
Baseline Demographic: Participant Sex
时间窗: At Baseline
Clinical Characteristics: Disease History
时间窗: At Baseline
Baseline Demographic: Race/Ethnicity
时间窗: At Baseline
Baseline Demographic: Weight
时间窗: At Baseline
Baseline Demographic: Family History of Amyotrophic Lateral Sclerosis (ALS)
时间窗: At Baseline
Clinical Characteristics: SOD1 Mutation Type
时间窗: At Baseline
Clinical Characteristics: Pregnancy Status
时间窗: At Baseline
Number of Participants With Serious Adverse Events
时间窗: Up to 7 years
Baseline Demographic: Height
时间窗: At Baseline
Baseline Demographic: Body Mass Index (BMI)
时间窗: At Baseline
Clinical Characteristics: Classification of SOD1-ALS Clinical Phenotypes
时间窗: At Baseline
Clinical Characteristics: Age at Diagnosis and Symptom Onset
时间窗: At Baseline
Clinical Characteristics: Revised El Escorial Classification
时间窗: At Baseline
Clinical Characteristics: Concomitant Medications
时间窗: At Baseline
Clinical Characteristics: Medical History
时间窗: At Baseline
次要结局
- Number of Participants With New Comorbid Conditions(Up to 7 years)
- Number of Participants With Pregnancy and Pregnancy Outcomes(Up to 7 years)
- Number of Participants With Reported Treatment Discontinuation(Up to 7 years)
- Number of Participants With Reported Reasons for Treatment Discontinuation(Up to 7 years)