EUCTR2022-000956-12-PT
Active, not recruiting
Phase 1
A Randomized, Blinded, Placebo-Controlled, Phase 1 SingleAscending Dose Study in Healthy Adult Male Volunteers and an Open-Label Multiple Ascending Dose Study in Pediatric SMA Participants Previously Treated with Onasemnogene Abeparvovec (Zolgensma™) to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB115
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Spinal muscular atrophy
- Sponsor
- Biogen Idec Research Limited
- Enrollment
- 24
- Status
- Active, not recruiting
- Last Updated
- 2 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility 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
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.
Outcomes
Primary Outcomes
Not specified
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