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Clinical Trials/EUCTR2022-000956-12-BE
EUCTR2022-000956-12-BE
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

Biogen Idec Research Limited0 sites24 target enrollmentJanuary 20, 2023
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ConditionsSpinal 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]

Overview

Phase
Phase 1
Intervention
Not specified
Conditions
Spinal muscular atrophy
Sponsor
Biogen Idec Research Limited
Enrollment
24
Status
Active, not recruiting
Last Updated
last year
OverviewInvestigatorsEligibility CriteriaOutcomesSimilar Trials

Overview

Brief Summary

No summary available.

Registry
who.int
Start Date
January 20, 2023
End Date
TBD
Last Updated
last year
Study Type
Interventional clinical trial of medicinal product
Sex
All

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

Similar Trials

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