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Nusinersen for Spinal Muscular Atrophy

CG
Overseen ByCara Grobbecker
Age: Any Age
Sex: Any
Trial Phase: Academic
Sponsor: London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This study is an open label individual patient study of the drug Nusinsersen.

Are You a Good Fit for This Trial?

This trial is for people with spinal muscular atrophy who have already taken part in a previous study using Nusinersen. Only those who meet specific medical requirements and do not have conditions that would make participation unsafe can join.

Inclusion Criteria

Completion of Study 232SM302
I am using effective birth control if I can have children.
I am able to follow all study procedures and have good support at home.
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Exclusion Criteria

Other reasons making the participant unsuitable for enrollment, per Investigator's opinion
Any new condition or worsening of an existing condition that would make the participant unsuitable for inclusion, interfere with safety assessment, or compromise ability to undergo study procedures, per Investigator
Participants who are pregnant, currently breastfeeding, or intending to become pregnant during the study
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What Are the Treatments Tested in This Trial?

Interventions

  • Nusinersen

Trial Overview

The study gives Nusinersen injections to one participant in an open-label format, meaning both doctors and the patient know what treatment is being given. The goal is to monitor safety and effects of continued use.

How Is the Trial Designed?

1

Treatment groups

Experimental Treatment

Group I: Open labelExperimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

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

Lead Sponsor

Trials
678
Recruited
421,000+

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