BIIB115 in Healthy Volunteers and Pediatric SMA Patients Previously Treated with Zolgensma

Phase 1

• Conditions: Spinal muscular atrophy; MedDRA version: 20.1Level: PTClassification code 10041582Term: Spinal muscular atrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2022-000956-12-DE
• Lead Sponsor: Biogen Idec Research Limited

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2023-01-23
• Last Update Posted: 8 January 2024

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 24

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

- 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