Status:
UNKNOWN
Antisense Oligonucleotide for Spinal Muscular Atrophy
Lead Sponsor:
First Affiliated Hospital of Fujian Medical University
Collaborating Sponsors:
Fujian Medical University Union Hospital
Peking Union Medical College Hospital
Conditions:
Spinal Muscular Atrophy
Eligibility:
All Genders
1-50 years
Brief Summary
This is a longitudinal, multiple-center, observational study of patients genetically confirmed chromosome 5q SMA to monitor the efficacy, safety, tolerability of SPINRAZA® (nusinersen) for up to 24 mo...
Detailed Description
SPINRAZA® (nusinersen) is an antisense oligonucleotide (ASO) designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. Nusinersen, approved by the FDA for treatme...
Eligibility Criteria
Inclusion
- Patients genetically confirmed 5q SMA including types I, II and III, who are planning to initiate treatment with SPINRAZA® (nusinersen) as part of their clinical care plan.
- Non-5q SMA patients undergoing clinical standard lumbar puncture
- Non-SMA subjects including Asymptomatic carriers of SMA, relatives of SMA patients and carriers, and patients undergoing clinical standard lumbar puncture
- Participants or Parent(s)/legal guardian(s) willing and able to complete the informed consent process
Exclusion
- Contraindication for lumbar puncture
- Inability to access intrathecal space for nusinersen injection
Key Trial Info
Start Date :
January 1 2022
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
January 31 2024
Estimated Enrollment :
1000 Patients enrolled
Trial Details
Trial ID
NCT05187260
Start Date
January 1 2022
End Date
January 31 2024
Last Update
January 26 2022
Active Locations (3)
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1
Department of Neurology, Peking Union Medical College Hospital
Beijing, Beijing Municipality, China, 100010
2
Department of Pediatrics, Fujian Medical University Union Hospital
Fuzhou, Fujian, China, 350001
3
Department of Neurology, First Affiliated Hospital Fujian Medical University
Fuzhou, Fujian, China, 350005