Spinal muscular atrophy MedDRA version: 20.1 Level: PT Classification code 10041582 Term: Spinal muscular atrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: BIIB115
Pharmaceutical Form: Solution for injection
INN or Proposed INN: BIIB115
Current Sponsor code: BIIB115
Other descriptive name: BIIB115
Concentration unit: mg/ml milligram(s)/mill
Sponsors
Biogen Idec Research Limited
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - 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 - Potential for improvement due to suboptimal clinical status secondary to SMA, as determined by the Investigator Are the trial subjects under 18? yes Number of subjects for this age range: 24 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: - Severe or serious AEs related to onasemnogene abeparvovec therapy that are ongoing during Screening - Interval of <180 days between onasemnogene abeparvovec therapy and first BIIB115 dose - Ongoing steroid treatment following onasemnogene abeparvovec at time of screening - History of drug induced liver injury or liver failure per Hy's law definition - History of thrombotic micrangiopathy - Treatment with any SMN2-splicing modifier (nusinersen or risdiplam) after receiving onasemnogene abeparvovec. Treatment with nusinersen <12 months from the first dose of BIIB115. - Any reason, anatomical or otherwise (including abnormal hematology/coagulation),that presents increase of risk of complication from the LP procedures, CSF circulation, or safety assessments, including a history of hydrocephalus or implanted shunt for CSF drainage - Permanent ventilation, defined as tracheostomy or =16 hours ventilation/day continuously for =21 days in the absence of an acute reversible event NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety and tolerability of multiple ascending doses of BIIB115 administered via IT bolus injection participants with SMA who previously received onasemnogene abeparvovec;Secondary Objective: To evaluate the PK of multiple ascending doses of BIIB115 administered via IT bolus injection to pediatric SMA participants who previously received onasemnogene abeparvovec;Primary end point(s): Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs);Timepoint(s) of evaluation of this end point: Up to Day 720 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Concentration of BIIB115 in Cerebral Spinal Fluid (CSF) 2. Concentration of BIIB115 in Serum 3. Terminal Elimination Half-Life (t½) of BIIB115 in Serum 4. Area Under the Concentration-Time Curve from Time 0 to Last Measurable Concentration (AUC0-last) of BIIB115 in Serum 5. Area Under the Concentration-Time Curve from Time 0 to Infinity (AUCinf) of BIIB115 in Serum 6. Maximum Observed Concentration (Cmax) of BIIB115 in Serum 7. Time to Reach Maximum Observed Concentration (Tmax) of BIIB115 in Serum;Timepoint(s) of evaluation of this end point: 1. Days 1 and 360 2. Day 1 to Day 720 3. Day 1 to Day 720 4. Day 1 to Day 720 5. Day 1 to Day 720 6. Day 1 to Day 720 7. Day 1 to Day 720 | — |
Countries
Australia, Belgium, Canada, France, Germany, Israel, Italy, Netherlands, Poland, Portugal, Spain, United Kingdom
Contacts
Public ContactSponsor
Biogen Idec Research Limited
Outcome results
None listed