BIIB115 in Healthy Volunteers and Pediatric SMA Patients Previously Treated with Zolgensma
- Conditions
- Spinal muscular atrophyMedDRA version: 20.1Level: PTClassification code 10041582Term: Spinal muscular atrophySystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2022-000956-12-BE
- Lead Sponsor
- Biogen Idec Research Limited
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 24
- 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
- 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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method Secondary end point(s): 1. Concentration of BIIB115 in Cerebral Spinal Fluid (CSF)<br>2. Concentration of BIIB115 in Serum<br>3. Terminal Elimination Half-Life (t½) of BIIB115 in Serum<br>4. Area Under the Concentration-Time Curve from Time 0 to Last Measurable Concentration (AUC0-last) of BIIB115 in Serum<br>5. Area Under the Concentration-Time Curve from Time 0 to Infinity (AUCinf) of BIIB115 in Serum<br>6. Maximum Observed Concentration (Cmax) of BIIB115 in Serum<br>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<br>2. Day 1 to Day 720<br>3. Day 1 to Day 720<br>4. Day 1 to Day 720<br>5. Day 1 to Day 720<br>6. Day 1 to Day 720<br>7. Day 1 to Day 720