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.

Conditions

Spinal muscular atrophy

Brief summary

Parts A, B and Long Term Extension (LTE): Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) [Time Frame: Part A: Up to Day 393, Part B: Up to Day 720; Part B LTE: Up to Day 2520]

Detailed description

Parts A,B and B LTE: Concentration of BIIB115 in Cerebral Spinal Fluid (CSF) [Time Frame: Part A: Day 1 to Day 180, Part B: Days 1 and 720; Part B LTE: Day 720 to Day 2520]., Part A: Terminal Elimination Half-Life (t½) of BIIB115 in CSF. [Time Frame: Day 1 to Day 180]., Parts A,B and B LTE: Concentration of BIIB115 in Serum [Time Frame: Part A: Day 1 to Day 180, Part B: Day 1 to 720; Part B LTE: Day 720 to Day 2520]., Parts A and B: Terminal Elimination Half-Life (t½) of BIIB115 in Serum. [Time Frame: Part A: Day 1 to Day 180, Part B: Day 1 to Day 720]., Parts A and B: Area Under the Concentration-Time Curve from Time 0 to Last Measurable Concentration (AUC0-last) of BIIB115 in Serum. [Time Frame: Part A: Day 1 to Day 180, Part B: Day 1 to Day 720]., Parts A and B: Area Under the Concentration-Time Curve from Time 0 to Infinity (AUCinf) of BIIB115 in Serum. [Time Frame: Part A: Day 1 to Day 180, Part B: Day 1 to Day 720]., Parts A and B: Maximum Observed Concentration (Cmax) of BIIB115 in Serum. [Time Frame: Part A: Day 1 to Day 180, Part B: Day 1 to Day 720]., Parts A and B: Time to Reach Maximum Observed Concentration (Tmax) of BIIB115 in Serum. [Time Frame: Part A: Day 1 to Day 180, Part B: Day 1 to Day 720].

Related papers

No related papers found yet.

Interventions

DRUGBIIB115

DRUGBiiB115 placebo is a sterile liquid for injection. the formulation composition of the placebo is identical to that of BIIB115 drug product minus the active ingredient

Eligibility

Sex/Gender

All

Age

0 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Parts A, B and Long Term Extension (LTE): Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) [Time Frame: Part A: Up to Day 393, Part B: Up to Day 720; Part B LTE: Up to Day 2520]	—

Secondary

Measure	Time frame
Parts A,B and B LTE: Concentration of BIIB115 in Cerebral Spinal Fluid (CSF) [Time Frame: Part A: Day 1 to Day 180, Part B: Days 1 and 720; Part B LTE: Day 720 to Day 2520]., Part A: Terminal Elimination Half-Life (t½) of BIIB115 in CSF. [Time Frame: Day 1 to Day 180]., Parts A,B and B LTE: Concentration of BIIB115 in Serum [Time Frame: Part A: Day 1 to Day 180, Part B: Day 1 to 720; Part B LTE: Day 720 to Day 2520]., Parts A and B: Terminal Elimination Half-Life (t½) of BIIB115 in Serum. [Time Frame: Part A: Day 1 to Day 180, Part B: Day 1 to Day 720]., Parts A and B: Area Under the Concentration-Time Curve from Time 0 to Last Measurable Concentration (AUC0-last) of BIIB115 in Serum. [Time Frame: Part A: Day 1 to Day 180, Part B: Day 1 to Day 720]., Parts A and B: Area Under the Concentration-Time Curve from Time 0 to Infinity (AUCinf) of BIIB115 in Serum. [Time Frame: Part A: Day 1 to Day 180, Part B: Day 1 to Day 720]., Parts A and B: Maximum Observed Concentration (Cmax) of B	—

Measure

Time frame

Parts A,B and B LTE: Concentration of BIIB115 in Cerebral Spinal Fluid (CSF) [Time Frame: Part A: Day 1 to Day 180, Part B: Days 1 and 720; Part B LTE: Day 720 to Day 2520]., Part A: Terminal Elimination Half-Life (t½) of BIIB115 in CSF. [Time Frame: Day 1 to Day 180]., Parts A,B and B LTE: Concentration of BIIB115 in Serum [Time Frame: Part A: Day 1 to Day 180, Part B: Day 1 to 720; Part B LTE: Day 720 to Day 2520]., Parts A and B: Terminal Elimination Half-Life (t½) of BIIB115 in Serum. [Time Frame: Part A: Day 1 to Day 180, Part B: Day 1 to Day 720]., Parts A and B: Area Under the Concentration-Time Curve from Time 0 to Last Measurable Concentration (AUC0-last) of BIIB115 in Serum. [Time Frame: Part A: Day 1 to Day 180, Part B: Day 1 to Day 720]., Parts A and B: Area Under the Concentration-Time Curve from Time 0 to Infinity (AUCinf) of BIIB115 in Serum. [Time Frame: Part A: Day 1 to Day 180, Part B: Day 1 to Day 720]., Parts A and B: Maximum Observed Concentration (Cmax) of B

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Countries

Belgium, France, Germany, Italy, Netherlands, Poland

Outcome results

None listed