Study to Investigate Drug BIIB132 Administered to Adults With Spinocerebellar Ataxia 3

Study of a Study Medicine for Adults With Spinocerebellar Ataxia Type 3

Age
18 years - 70 years
Gender
All
participants needed
3
Location
1
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Brief description of study

The purpose of this research study is to learn more about a study drug that may affect patients with spinocerebellar ataxia type 3 (SCA3). This type of drug is called an antisense oligonucleotide. Eligible participants in this study will have the study drug injected into the fluid surrounding their spinal cord and brain through a fine needle placed in the spinal canal in the lower back. This procedure is called a lumbar puncture. After injection of the study drug, participants will be monitored in the hospital for 6 to 24 hours before going home. During the study, participants will receive study drug at up to 4 visits. Some of the assessments include: examinations of the brain and body, collection of blood, urine, and CSF (the fluid in the spine) samples, medical scans of the brain, and questionnaires about health and daily life.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: Machado-Joseph disease, Spinocerebellar Ataxia
  • Age: 18 years - 70 years
  • Gender: All

The purpose of this research study is to learn more about a study medicine that may affect adults with spinocerebellar ataxia type 3 (SCA3). The study medicine is an antisense oligonucleotide, which is a type of medicine designed to affect how the body makes certain proteins.

Participants will receive the study medicine as an injection into the fluid around the brain and spinal cord. This is done with a lumbar puncture, which uses a fine needle placed into the lower back. After each injection, participants will stay in the hospital for monitoring for 6 to 24 hours before going home. Participants will receive the study medicine at up to 4 visits.

During the study, participants will have checks of the brain and body. Participants will also provide blood, urine, and spinal fluid samples. Participants will have brain scans and will complete questionnaires about their health and daily life.

Who can participate:
Adults with spinocerebellar ataxia type 3 (SCA3) may be able to participate if they:

  • Have spinocerebellar ataxia type 3 (SCA3)

Updated on 04 Aug 2024. Study ID: 850137

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