Antisense Oligonucleotide for Spinal Muscular Atrophy
Launched by FIRST AFFILIATED HOSPITAL OF FUJIAN MEDICAL UNIVERSITY · Dec 25, 2021
Trial Information
Current as of July 23, 2025
Unknown status
Keywords
ClinConnect Summary
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 treatment of SMA in 2016, was approved by the Chinese National Medical Products Administration in 2019.
This is a prospective, longitudinal, multi-center, observational study designed to evaluate the efficacy, safety, tolerability and of nusinersen in patients genetically confirmed chromosome 5q SMA in China. Subjects with SMA I/II/III who are planning to initiate treatment with nusinersen will be en...
Gender
ALL
Eligibility criteria
- Inclusion Criteria:
- • 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 Criteria:
- • Contraindication for lumbar puncture
- • Inability to access intrathecal space for nusinersen injection
About First Affiliated Hospital Of Fujian Medical University
The First Affiliated Hospital of Fujian Medical University is a leading medical institution dedicated to advancing healthcare through innovative clinical research and education. As a premier sponsor of clinical trials, the hospital is committed to improving patient outcomes and contributing to medical knowledge across various specialties. With a robust infrastructure and a multidisciplinary team of experienced researchers and healthcare professionals, the hospital ensures the ethical conduct and scientific rigor of its trials, fostering collaboration with global research communities. Its mission is to enhance the quality of care through evidence-based practices and to support the development of novel therapies that address unmet medical needs.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Fuzhou, Fujian, China
Beijing, Beijing, China
Fuzhou, Fujian, China
Patients applied
Trial Officials
Wan-Jin Chen
Principal Investigator
First Affiliated Hospital Fujian Medical University
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported
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