A Randomized, Blinded, Placebo-Controlled, Phase 1 SingleAscending Dose Study in Healthy Adult Male Volunteers and an Open-LabelMultiple Ascending Dose Study in Pediatric SMA Participants PreviouslyTreated with Onasemnogene Abeparvovec (Zolgensma™) to Evaluate theSafety, Tolerability, and Pharmacokinetics of BIIB115

Biogen Idec Research Limited0 sites62 target enrollmentMarch 7, 2023

ConditionsSpinal muscular atrophy MedDRA version: 20.1Level: PTClassification code 10041582Term: Spinal muscular atrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Spinal muscular atrophy
Sponsor: Biogen Idec Research Limited
Enrollment: 62
Status: Active, not recruiting
Last Updated: last year

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

March 7, 2023

End Date

TBD

Last Updated

last year

Study Type

Interventional clinical trial of medicinal product

Sex

All

Investigators

Sponsor

Biogen Idec Research Limited

Eligibility Criteria

Inclusion Criteria

•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 between 18 and 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
•\- Weight \=7 kg at the time of informed consent
•\- Genetic diagnosis of SMA (5q SMA homozygous survival motor neuron 1 (SMN1\)gene deletion or mutation or compound heterozygous mutation)\- Survival motor neuron 2 (SMN2\) copy number \=1
•\- Must have received IV onasemnogene abeparvovec per the approved label or per guidelines including the steroid regimen and monitoring specified therein
•\- Treatment with onasemnogene abeparvovec \=180 days prior to first BIIB115 dose

Exclusion Criteria

•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 malignancy, carcinoma in situ, or high grade dysplasia (with the exception of no more than 1 basal cell carcinoma or squamous cell carcinoma that was completely excised and cured at least 12 months prior to randomization).
•5\.Systolic blood pressure \> 150 mmHg or \< 90 mmHg after resting in a supine or semi recumbent position for at least 5 minutes at screening or prior to dosing.
•6\.Clinically significant 12\-lead electrocardiogram abnormalities.
•7\.Confirmed demonstration of corrected QT interval, using Fridericia’s correction method, of \> 450 ms.
•8\.Plans to undergo elective procedures or surgeries at any time after signing the ICF through the follow\-up visit.
•9\.History of a suicide attempt within 5 years prior to screening or suicidal ideation in the past 6 months
•10\.History of, or positive test result at Screening for, human immunodeficiency virus.