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A Study to Learn About the Safety of BIIB115 Injections and How BIIB115 is Processed in the Bodies of Healthy Adult Male Volunteers and of Pediatric Participants With Spinal Muscular Atrophy Who Previously Took Onasemnogene Abeparvovec
Sponsor: Biogen
Summary
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: * After screening, healthy volunteers will be randomly placed into 1 of 4 groups to receive either BIIB115 or a placebo. A placebo looks like the study drug but contains no real medicine. * Participants will receive a single dose of either BIIB115 or the placebo as an injection directly into the spinal canal on Day 1. * Neither the researchers nor the participants will know if the participants will receive BIIB115 or the placebo. * The Part A treatment and follow-up period will last for 13 months. * Participants will have up to 6 clinic visits and 4 phone calls. During Part B: * After screening, children with SMA will be placed into 1 of 2 groups to receive BIIB115. * The doses of each group will be decided based on the results of Part A. * Both researchers and participants will know they are receiving BIIB115. * Participants will first receive 2 total doses of BIIB115 given at 2 different times. * The Part B treatment and follow-up period will last for 24 months. * Participants will have up to 14 clinic visits and 6 phone calls. Part B Long-Term Extension: * After completing the 25 months in Part B, participants may move onto the long-term extension (LTE). * They will receive 5 more doses of BIIB115 at different times. * The Part B LTE treatment and follow-up will last for 60 months. * Participants will have up to 12 more clinic visits and 19 phone calls. In both Part A and Part B, participants will stay in the clinic for 24 hours after each dose so that researchers can check on their health. This 24-hour stay will not be required for the Part B LTE period.
Official title: A Randomized, Blinded, Placebo-Controlled, Phase 1 Single Ascending Dose Study in Healthy Adult Male Volunteers and an Open-Label Multiple Ascending Dose Study With Long-Term Extension in Pediatric SMA Participants Previously Treated With Onasemnogene Abeparvovec (Zolgensma™) to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB115
Key Details
Gender
All
Age Range
6 Months - 55 Years
Study Type
INTERVENTIONAL
Enrollment
62
Start Date
2022-10-10
Completion Date
2031-11-14
Last Updated
2025-07-09
Healthy Volunteers
Yes
Interventions
BIIB115
Administered as specified in the treatment arm
BIIB115-Matching Placebo
Administered as specified in the treatment arm
Locations (17)
Universitair Ziekenhuis Gent
Ghent, Belgium
Children's Hospital of Eastern Ontario
Ontario, Canada
Hôpital Armand Trousseau
Paris, France
Universitatsklinikum Essen
Essen, Germany
Universitaetsklinikum Freiburg
Freiburg im Breisgau, Germany
Universitaetsklinikum Heidelberg
Heidelberg, Germany
Fondazione Serena Onlus - Centro Clinico Nemo
Milan, Italy
Pediatric Neurology Unit, Catholic University
Rome, Italy
Centre For Human Drug Research
Leiden, Netherlands
UMC Utrecht
Utrecht, Netherlands
Instytut Centrum Zdrowia Matki Polki Dept of Neurology
Lodz, Poland
PRATIA S.A. MTZ Clinical Research Powered by Pratia
Warsaw, Poland
Instytut "Pomnik - Centrum Zdrowia Dziecka
Warsaw, Poland
Kyungpook National University Hospital
Daegu, South Korea
Seoul National University Hospital
Seoul, South Korea
Great Ormond Street Hospital for Children
Bloomsbury, United Kingdom
Sheffield Childrens Hospital
Sheffield, United Kingdom