A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Participants With Superoxide Dismutase 1 (SOD-1) Amyotrophic Lateral Sclerosis (ALS)

Официальное название

An Observational Registry-Based Study to Evaluate the Long-Term Safety of Tofersen in People With SOD1-ALS

Обзор

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.

Подробное описание

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.

Вмешательства

• Препарат Tofersen
  Administered as specified in the treatment arm.

Первичные конечные точки

• Baseline Demographic: Age [Срок оценки: At Baseline]
• Baseline Demographic: Participant Sex [Срок оценки: At Baseline]
• Baseline Demographic: Race/Ethnicity [Срок оценки: At Baseline]
• Baseline Demographic: Weight [Срок оценки: At Baseline]
• Baseline Demographic: Height [Срок оценки: At Baseline]
• Baseline Demographic: Body Mass Index (BMI) [Срок оценки: At Baseline]
• Baseline Demographic: Family History of Amyotrophic Lateral Sclerosis (ALS) [Срок оценки: At Baseline]
• Clinical Characteristics: Age at Diagnosis and Symptom Onset [Срок оценки: At Baseline]
• Clinical Characteristics: Revised El Escorial Classification [Срок оценки: At Baseline]
• Clinical Characteristics: Classification of SOD1-ALS Clinical Phenotypes [Срок оценки: At Baseline]

Вторичные конечные точки (4)

• Number of Participants With New Comorbid Conditions [Срок оценки: Up to 7 years]
• Number of Participants With Pregnancy and Pregnancy Outcomes [Срок оценки: Up to 7 years]
• Number of Participants With Reported Treatment Discontinuation [Срок оценки: Up to 7 years]
• Number of Participants With Reported Reasons for Treatment Discontinuation [Срок оценки: Up to 7 years]

Критерии участия

Критерии включения

• Participants with an ALS diagnosis and a confirmed SOD1 mutation from contributing registry networks will be considered for inclusion in the study.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

США · 1 центр

• Mass General Hospital -MGH — Boston

Идентификаторы

NCT: NCT07259980 · 233AS401

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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