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ACTIVE NOT RECRUITING
NCT05575011
PHASE1

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

View on ClinicalTrials.gov

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

DRUG

BIIB115

Administered as specified in the treatment arm

DRUG

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