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)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Tofersen.
- Who it may be relevant to
- Registry conditions: Amyotrophic Lateral Sclerosis. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
An Observational Registry-Based Study to Evaluate the Long-Term Safety of Tofersen in People With SOD1-ALS
Overview
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.
Detailed description
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.
Interventions
- Drug Tofersen
Administered as specified in the treatment arm.
Primary outcome measures
- Baseline Demographic: Age [Time frame: At Baseline]
- Baseline Demographic: Participant Sex [Time frame: At Baseline]
- Baseline Demographic: Race/Ethnicity [Time frame: At Baseline]
- Baseline Demographic: Weight [Time frame: At Baseline]
- Baseline Demographic: Height [Time frame: At Baseline]
- Baseline Demographic: Body Mass Index (BMI) [Time frame: At Baseline]
- Baseline Demographic: Family History of Amyotrophic Lateral Sclerosis (ALS) [Time frame: At Baseline]
- Clinical Characteristics: Age at Diagnosis and Symptom Onset [Time frame: At Baseline]
- Clinical Characteristics: Revised El Escorial Classification [Time frame: At Baseline]
- Clinical Characteristics: Classification of SOD1-ALS Clinical Phenotypes [Time frame: At Baseline]
Secondary outcome measures (4)
- Number of Participants With New Comorbid Conditions [Time frame: Up to 7 years]
- Number of Participants With Pregnancy and Pregnancy Outcomes [Time frame: Up to 7 years]
- Number of Participants With Reported Treatment Discontinuation [Time frame: Up to 7 years]
- Number of Participants With Reported Reasons for Treatment Discontinuation [Time frame: Up to 7 years]
Eligibility criteria
Inclusion criteria
- Participants with an ALS diagnosis and a confirmed SOD1 mutation from contributing registry networks will be considered for inclusion in the study.
Exclusion criteria
- 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 1 center
- Mass General Hospital -MGH — Boston
Identifiers
NCT: NCT07259980 · 233AS401