BIIB115 for Spinal Muscular Atrophy

Phase 1

Recruiting

Research Sponsored by Biogen

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up part a: up to day 393, part b: up to day 720

Summary

This trial aims to test the safety and tolerability of a new drug called BIIB115 given as a single dose to healthy male participants and as multiple doses to pediatric patients with Spinal Muscular At

Who is the study for?

This trial is for healthy males aged 18-55 and children aged 0.5 to 12 with Spinal Muscular Atrophy (SMA) who've been treated with onasemnogene abeparvovec. Adults must have a BMI of 18-30 kg/m^2 and be in good health, while children need to weigh at least 7 kg and may benefit from treatment due to SMA.

What is being tested?

The study tests the safety and tolerability of BIIB115, given through spinal injection, comparing it with a placebo. It's conducted in two parts: one-time dosing for healthy adults (Part A), and multiple doses for pediatric SMA patients previously treated with gene therapy (Part B).

What are the potential side effects?

While specific side effects are not listed here, the trial aims to assess how safe BIIB115 is when administered via spinal injection which could include typical drug administration reactions like pain or discomfort at the injection site.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ part a: up to day 393, part b: up to day 720

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~part a: up to day 393, part b: up to day 720

This trial's timeline: 3 weeks for screening, Varies for treatment, and part a: up to day 393, part b: up to day 720 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Parts A and B: Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)

Trial Design

7Treatment groups

Experimental Treatment

Placebo Group

Group I: Part B: Cohort 6: BIIB115 Dose 4Experimental Treatment1 Intervention

Pediatric SMA participants previously treated with onasemnogene abeparvovec will receive two doses of BIIB115, Dose 4, via IT bolus injectionat two separate time points.

Group II: Part B: Cohort 5: BIIB115 Dose 3Experimental Treatment1 Intervention

Pediatric SMA participants previously treated with onasemnogene abeparvovec will receive two doses of BIIB115, Dose 3, via IT bolus injection at two separate time points.

Group III: Part A: Cohort 4: BIIB115 Dose 4Experimental Treatment1 Intervention

Participants will receive a single dose of BIIB115, Dose 4, via IT bolus injection, on Day 1.

Group IV: Part A: Cohort 3: BIIB115 Dose 3Experimental Treatment1 Intervention

Participants will receive a single dose of BIIB115, Dose 3, via IT bolus injection, on Day 1.

Group V: Part A: Cohort 2: BIIB115 Dose 2Experimental Treatment1 Intervention

Participants will receive a single dose of BIIB115, Dose 2, via IT bolus injection, on Day 1.

Group VI: Part A: Cohort 1: BIIB115 Dose 1Experimental Treatment1 Intervention

Participants will receive a single dose of BIIB115, Dose 1, via IT bolus injection, on Day 1.

Group VII: Part A: Cohorts 1-4: BIIB115-Matching PlaceboPlacebo Group1 Intervention

Participants will receive a single dose of BIIB115-matching placebo, via IT bolus injection, on Day 1.

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