An Open Label Individual Patient (OLIP)-02 Study of Nusinersen (BIIB058)

NCT07846696 | Not Yet Recruiting Phase 3

• 1 other identifier: Nusinersen OLIP-02
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief Summary

An Open Label Individual Patient (OLIP) Study of Nusinersen (BIIB058) in a Participant with Spinal Muscular Atrophy Who Previously Participated in an Investigational Study with Nusinersen

Conditions

SMA - Spinal Muscular Atrophy

Outcome Measures

Primary Outcomes (1)

• Long-term safety and tolerability of Nusinersen 28mg dosing through adverse events experienced

  From enrollment to the end of treatment, one year

Study Arms (1)

Open Label

EXPERIMENTAL

Drug: Nusinersen Injectable Product

Interventions

Nusinersen Injectable Product DRUG

Nusinersen intrathecal injection

Open Label

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Signed informed consent of parent(s) or legal guardian(s) and signed informed assent of the participant, if indicated per participant's capacity to assent and institutional guidelines.
• Completion of Study 232SM302.
• Ability to complete all study procedures, measurements, and visits, and parent(s) or legal guardian(s)/participant has adequately supportive psychosocial circumstances, in the opinion of the Investigator.
• Meets age-appropriate institutional criteria for the use of anesthesia/sedation, if use is planned for study procedures (as assessed by the Investigator and either anesthesiologist or pulmonologist).
• All female participants of childbearing potential (defined as any female physiologically capable of becoming pregnant) and all male participants of reproductive age must practice effective contraception.

You may not qualify if:

• Treatment with another investigational therapy or enrollment in another interventional clinical study after the final dose in Study 232SM302.
• Treatment with an approved therapy for SMA after the final dose in Study 232SM302.
• History of systemic hypersensitivity reaction to nusinersen, the excipients contained in the formulation, and if appropriate, any diagnostic agents to be administered during the study.
• Participants who are pregnant or currently breastfeeding and those intending to become pregnant during the study.
• The participant's parent(s) or legal guardian(s) is not willing or able to meet guidelines in the consensus statement for standard of care in SMA \[Finkel 2018; Mercuri 2018\] or provide nutritional and respiratory support throughout the study, per the Investigator's judgment.
• Other unspecified reasons that, in the opinion of the Investigator, make the participant unsuitable for enrollment.

Sponsors & Collaborators

• London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's: lead

Study Sites (1)

Children's Hospital - London Health Sciences Centre

London, Ontario, N6A 5W9, Canada

Location

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; Neuromuscular Diseases

Study Officials

• Craig Campbell, MD, FRCPC

  Children's Hospital, London Health Sciences Centre

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Cara Grobbecker

CONTACT

519-685-8500; cara.grobbecker@lhsc.on.ca

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Paediatric Neurologist

Study Record Dates

• First Submitted: September 10, 2026
• First Posted: September 29, 2026
• Study Start: September 17, 2026
• Primary Completion (Estimated): December 31, 2028
• Study Completion (Estimated): December 31, 2028
• Last Updated: September 29, 2026

Record last verified: 2026-09

Locations

CA (1)