A Randomized, Blinded, Placebo-Controlled, Phase 1 Single Ascending 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 - 277HV101

Biogen Idec Research Limited0 sites42 target enrollmentTBD

Overview

No summary available.

Registry

who.int

Start Date

TBD

End Date

TBD

Last Updated

2 years ago

Study Type

Interventional

Sponsor

•1\. Ability to understand the purpose and risks of the study and provide signed
•and dated informed consent and authorization to use confidential health
•information in accordance with applicable privacy regulations.
•2\. Males aged 18 to 55 years, inclusive, at the time of informed consent.
•3\. Have a body mass index of 18 to 30 kg/m2, inclusive.
•4\. All participants must practice highly effective contraception as described
•in Section 12\.5 of the protocol.
•5\. Must be in good health as determined by the Investigator, based on medical
•history and Screening evaluations.
•\- Age 0\.5 to 12 years old, inclusive, at the time of informed consent

•Part A: 1\. Any reason, anatomical or otherwise (including abnormal
•hematology/coagulation), that presents increase of risk of complication from
•multiple LP procedures required for dosing and CSF collection, per the
•investigator discretion. 2\. History of any clinically significant cardiac,
•endocrine, gastrointestinal, hematologic, hepatic, immunologic, metabolic,
•urologic, pulmonary, neurologic, dermatologic, psychiatric, or renal disease,
•or other major disease, as determined by the Investigator. 3\. History of severe
•allergic or anaphylactic reactions, or of any allergic reactions that in the
•opinion of the Investigator are likely to be exacerbated by any component of
•the study treatment, including LP procedures. 4\. History of, or ongoing