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Tofersen
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)
Ensayo de BIOGEN INC. en Esclerosis lateral amiotrofica.
Más ensayos de: Esclerosis lateral amiotrófica.
- Fase
- no aplica
- Estado
- Reclutando pacientes
- Participantes
- 125
- Centros
- 1
- Fin del objetivo primario
- dic 2033
Qué significa cada fase y cada estado.
Estudio observacional. Comenzó en ene 2026.
Qué mide
Demografia basal: Edad (en el inicio)
Qué se prueba
Se recoge información de seguridad, de forma observacional, sobre el uso de tofersen (Qalsody) en participantes con ELA asociada a mutaciones en el gen SOD-1.
Lo que dice el documentoThe main goal of this study is to collect safety information in participants with SOD-1 ALS who were in either of the groups.
Traducción automática (claude-sonnet-5). Lo comprobado es la cita: está literal en el documento y sus cifras cuadran con las de la frase. Todavía no la ha leído una persona.
Cómo lo describe el promotor
En el documentoEn inglés, del registroIn 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.
Más ensayos de BIIB
| Ensayo | Fase | Estado | Lectura de resultados |
|---|---|---|---|
| Diranersen · CELIA | Fase 2 | En marcha, sin reclutar | mar 2026 (real) |
| BIIB142 | Fase 1 | Reclutando pacientes | ago 2026 (prevista, ya pasada: sin actualizar en el registro) |
| Litifilimab · TOPAZ-2 | Fase 3 | En marcha, sin reclutar | sep 2026 (prevista, ya pasada: sin actualizar en el registro) |
| Litifilimab · TOPAZ-1 | Fase 3 | En marcha, sin reclutar | sep 2026 (prevista, ya pasada: sin actualizar en el registro) |
| Litifilimab · AMETHYST | Fase 2/3 | En marcha, sin reclutar | oct 2026 (prevista) |
| Natalizumab | no aplica | En marcha, sin reclutar | oct 2026 (prevista) |
Todos los de BIIB, en su ficha.
Ficha completa en ClinicalTrials.gov (NCT07259980), actualizada en abr 2026.