SMA - Spinal Muscular AtrophyPhase 3NCT07846696
An Open Label Individual Patient (OLIP)-02 Study of Nusinersen (BIIB058)
A Phase 3 study testing Nusinersen Injectable Product in people with SMA - Spinal Muscular Atrophy, run by London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's at 1 site in Canada.
Trial 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 Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- SMA - Spinal Muscular Atrophy
- What is being tested?
- Nusinersen Injectable Product
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteCanada
- Study dates
- 17 Sep 2026to 31 Dec 2028
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- Any new condition or worsening of an existing condition that, according to the Investigator, would make the participant unsuitable for inclusion, could interfere with the assessment of safety, or would compromise the ability of the participant to undergo study procedures.
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Canada1 site
Children's Hospital - London Health Sciences Centre
London, Ontario, N6A 5W9
Rhiannon Hicks, rhiannon.hicks@lhsc.on.ca
Craig Campbell, MD, FRCPC
About this study
Official title: 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
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
Read the detailed description
This is a Single Patient Study (SPS)/Open Label Individual Patient (OLIP) long-term extension study to evaluate the safety, tolerability, and efficacy of nusinersen administered by intrathecal injection at a higher dose (28 mg maintenance) for up to 5 years to a participant who has previously participated in Biogen Study 232SM302.
- Conditions
- SMA - Spinal Muscular Atrophy
- Being tested
- Nusinersen Injectable Product
- Medical subject headings
- Muscular Atrophy, Spinal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Nusinersen Injectable ProductDrug
Nusinersen intrathecal injection
| Study group | Role | What they receive |
|---|---|---|
| Open Label | Experimental | Not described Receives: Drug: Nusinersen Injectable Product |
What is being measured
The primary outcome is the main question the study is designed to answer.
Long-term safety and tolerability of Nusinersen 28mg dosing through adverse events experienced
Measured: From enrollment to the end of treatment, one year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Sep 2026
- Study start17 Sep 2026 (estimated)
- Primary completion31 Dec 2028 (estimated)
- Study completion31 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for SMA - Spinal Muscular AtrophyStudies of the same condition, open and under way.
- More trials run by London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph'sThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (estimated)
Sponsor and investigators
- Lead sponsor
- London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Craig Campbell, London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's
- Investigators
- Craig Campbell, MD, FRCPC, Principal investigator, Children's Hospital, London Health Sciences Centre
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07846696
- Sponsor's protocol ID
- Nusinersen OLIP-02
- Status last verified
- Sep 2026
- First submitted
- 10 Sep 2026
- Last updated
- 29 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: We have no plans to publish this data.
Study at a glance
From the registry record, last updated by the sponsor on 29 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07846696
- Acronym / sponsor ID
- Nusinersen OLIP-02
- Conditions
- SMA - Spinal Muscular Atrophy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 1 participants (estimated)
- Lead sponsor
- London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph'sUniversity, hospital or other
- Study start
- 17 Sep 2026 (estimated)
- Primary completion
- 31 Dec 2028 (estimated)
- Study completion
- 31 Dec 2028 (estimated)
- First posted
- 29 Sep 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Detailed description
This is a Single Patient Study (SPS)/Open Label Individual Patient (OLIP) long-term extension study to evaluate the safety, tolerability, and efficacy of nusinersen administered by intrathecal injection at a higher dose (28 mg maintenance) for up to 5 years to a participant who has previously participated in Biogen Study 232SM302.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Open Label | Experimental | Drug: Nusinersen Injectable Product |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (7 listed)
- Any new condition or worsening of an existing condition that, according to the Investigator, would make the participant unsuitable for inclusion, could interfere with the assessment of safety, or would compromise the ability of the participant to undergo study procedures.
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Nusinersen Injectable ProductNusinersen intrathecal injection | Drug | Open Label |
Primary outcome
Long-term safety and tolerability of Nusinersen 28mg dosing through adverse events experienced
Time frame: From enrollment to the end of treatment, one year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Sep 2026.
- Individual participant dataSharing planned: Undecided. We have no plans to publish this data.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Craig Campbell, MD, FRCPC principal investigator
Children's Hospital, London Health Sciences Centre
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Nusinersen Injectable Product in people with SMA - Spinal Muscular Atrophy, run by London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 29 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Cara Grobbecker (519-685-8500 ext. 56216, cara.grobbecker@lhsc.on.ca), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Long-term safety and tolerability of Nusinersen 28mg dosing through adverse events experienced (time frame: From enrollment to the end of treatment, one year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Sep 2026.