Status and phase
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Study type
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About
In this study, researchers will learn about a study drug called BIIB115 in healthy adult male volunteers and in participants with spinal muscular atrophy (SMA). This study will focus on children with SMA.
The main objective of the study is to learn about the safety of BIIB115 and how participants respond to different doses of BIIB115. The main question researchers want to answer is:
• How many participants have adverse events and serious adverse events during the study?
Adverse events are unwanted health problems that may or may not be caused by the study drug.
Researchers will also learn about how the body processes BIIB115. They will do this by measuring the levels of BIIB115 in both the blood and the cerebrospinal fluid, also known as the CSF. This is the fluid around the brain and spinal cord.
The study will be split into 2 parts - Part A and Part B.
During Part A:
During Part B:
Part B Long-Term Extension:
Full description
The primary objective of the study is to assess the safety and tolerability of BIIB115 administered via intrathecal (IT) bolus injection to healthy participants in Part A, pediatric participants with spinal muscular atrophy who have previously received onasemnogene abeparvovec in Part B, and to participants who complete Part B in Part B LTE. The secondary objectives of the study is to evaluate the pharmacokinetics (PK) of BIIB115 in Parts A, B, and B LTE.
Enrollment
Sex
Ages
Volunteers
Inclusion and exclusion criteria
Key Inclusion Criteria:
Part A:
Part B:
Part B LTE
Key Exclusion Criteria:
Part A:
Part B:
Part B LTE:
NOTE: Other protocol-defined Inclusion/Exclusion criteria may apply.
Primary purpose
Allocation
Interventional model
Masking
62 participants in 8 patient groups, including a placebo group
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Central trial contact
US Biogen Clinical Trial Center; Global Biogen Clinical Trial Center
Data sourced from clinicaltrials.gov
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