BIIB115 in Healthy Volunteers and Pediatric SMA Patients Previously Treated with Zolgensma
Research type
Research Study
Full title
A Randomized, Blinded, Placebo-Controlled, Phase 1 SingleAscending Dose Study in Healthy Adult Male Volunteers and an Open-Label Multiple Ascending Dose Study in Pediatric SMA Participants Previously Treated with Onasemnogene Abeparvovec (Zolgensma™) to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB115
IRAS ID
1007278
Contact name
Esha Dasgupta
Contact email
Sponsor organisation
Biogen Idec Research Limited
Eudract number
2022-000956-12
Clinicaltrials.gov Identifier
Research summary
This study is a first-in-human study in which it is proposed to evaluate the safety, tolerability and pharmacokinetics of BIIB115 multiple ascending dose (MAD) in paediatric participants with Spinal Muscular Atrophy (SMA) previously treated with onasemnogene abeparvovec (Zolgensma™). The study is being carried out at about multiple sites around the world. Across all sites, about 24 children will take part in Part B. The primary objective of Part B of the study is to evaluate the safety and tolerability of multiple ascending doses of BIIB115 administered via IT bolus injection to pediatric SMA participants previously treated with onasemnogene abeparvovec. If the participant is interested to take part in the study, the consent forms needs to be signed and the participant will take part in the study procedures as per study protocol. Participation in the study will last about 25 months. During this study, samples of blood, urine, and CSF will be taken as a part of study procedure. The study sponsor will use the results of the study to make decisions about further development and use of the study drug.
REC name
London - Hampstead Research Ethics Committee
REC reference
23/LO/0305
Date of REC Opinion
3 Jul 2023
REC opinion
Further Information Favourable Opinion