Mass General Hospital -MGH
Boston, Massachusetts, 02114, United States
Location status: Recruiting
NCT Number: NCT07259980
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.
Interested in participating?
Request Info18 year and older
All sexes
Observational
Boston, Massachusetts, 02114, United States
Location status: Recruiting
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.
Healthy volunteers accepted: No
Only the study team can determine whether someone qualifies for participation.
Key Inclusion Criteria:
Key Exclusion Criteria:
NOTE: Other protocol- defined Inclusion/Exclusion criteria may apply.
Administered as specified in the treatment arm.
Other names: BIIB067, Qalsody
Time frame: At Baseline
Time frame: At Baseline
Time frame: At Baseline
Time frame: At Baseline
Time frame: At Baseline
Time frame: At Baseline
Time frame: At Baseline
Time frame: At Baseline
Time frame: At Baseline
Time frame: At Baseline
Time frame: At Baseline
Time frame: At Baseline
Time frame: At Baseline
Time frame: At Baseline
Time frame: At Baseline
Time frame: Up to 7 years
Time frame: Up to 7 years
Time frame: Up to 7 years
Time frame: Up to 7 years
Time frame: Up to 7 years
Contact information is provided by the study sponsor or research team.
Global Biogen Clinical Trial Center
CONTACT
Study US Biogen Clinical Trial Center
CONTACT
Biogen
Industry
An Observational Registry-Based Study to Evaluate the Long-Term Safety of Tofersen in People With SOD1-ALS
OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.
View the official ClinicalTrials.gov record (opens in a new tab)This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.
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