Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

Phase 2

• Conditions: Spinal Muscular Atrophy; Muscular Atrophy Muscular Atrophy, Spinal Atrophy Pathological Conditions, Anatomical Neuromuscular Manifestations Neurologic Manifestations Nervous System Diseases Spinal Cord Diseases Central Nervous System Diseases Motor Neuron Disease Neurodegenerative Diseases Neuromuscular Diseases; G12.9

• Registration Number: LBCTR2021064778
• Lead Sponsor: Biogen Idec Research Limited

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2021-09-18
• Last Update Posted: 19 August 2024

Recruitment & Eligibility

• Status: Pending
• Sex: All
• Target Recruitment: 2

Inclusion Criteria

Part A, B and C:
- Genetic documentation of 5q SMA (homozygous gene deletion, mutation, or compound heterozygote)

Part A:
- Onset of clinical signs and symptoms consistent with SMA at > 6 months (> 180 days) of age (i.e., later-onset SMA)
- Age 2 to = 15 years, inclusive, at the time of informed consent

Part B:
- Participants with SMA symptom onset = 6 months (= 180 days) of age (infantile onset) should have age > 1 week to = 7 months (= 210 days) at the time of informed consent
- Participants with SMA symptom onset > 6 months (> 180 days) of age (later onset):
*Age 2 to < 10 years at the time of informed consent
*Can sit independently but has never had the ability to walk independently
*HFMSE score = 10 and = 54 at Screening

Part C:
- Participants = 18 years of age at Screening must be ambulatory
- Currently on nusinersen treatment at the time of Screening, with the first dose being at least 1 year prior to Screening

Exclusion Criteria

Part A, B and C:
- Presence of an untreated or inadequately treated active infection requiring systemic antiviral or antimicrobial therapy at any time during the Screening period
- Presence of an implanted shunt for the drainage of cerebrospinal fluid (CSF) or of an implanted central nervous system (CNS) catheter
- Hospitalization for surgery, pulmonary event, or nutritional support within 2 months prior to Screening or planned within 12 months after the participant's first dose.

Part A:
- Respiratory insufficiency, defined by the medical necessity for invasive or noninvasive ventilation for > 6 hours during a 24-hour period, at Screening
- Medical necessity for a gastric feeding tube
- Treatment with an investigational drug given for the treatment of SMA, biological agent, or device within 30 days or 5 half-lives of the agent, whichever is longer, prior to Screening or anytime during the study; any prior or current treatment with any survival motor neuron-2 (SMN2)-splicing modifier or gene therapy; or prior antisense oligonucleotide treatment, or cell transplantation.

Part B:
- Treatment with an investigational drug given for the treatment of SMA, biological agent, or device within 30 days or 5 half-lives of the agent, whichever is longer, prior to Screening or anytime during the study; any prior or current treatment with any SMN2-splicing modifier or gene therapy; or prior antisense oligonucleotide treatment, or cell transplantation
- Participants with SMA symptom onset > 6 months (> 180 days) of age (later onset):
*Respiratory insufficiency, defined by the medical necessity for invasive or noninvasive ventilation for > 6 hours during a 24-hour period, at Screening
*Medical necessity for a gastric feeding tube
*Participants with SMA symptom onset = 6 months (= 180 days) of age (infantile-onset): Signs or symptoms of SMA present at birth or within the first week after birth.

Part C:
- Concurrent or previous participation and/or administration of nusinersen in another clinical study

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Study & Design

• Study Type: Interventional
• Study Design: Not specified