Antisense Oligonucleotide for Spinal Muscular Atrophy

study id #: NCT05187260

condition: Spinal Muscular Atrophy

status: Not yet recruiting

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 months.

intervention: Nusinersen

results: https://clinicaltrials.gov/ct2/show/results/NCT05187260

last updated: February 04, 2022

start date: January 1, 2022

estimated completion: January 31, 2024

last updated: January 11, 2022

size / enrollment: 1000

study 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 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 enrolled in this study. All patients will be treated by their physicians according to standard clinical practice. SPINRAZA® (nusinersen) is administered as an intrathecal injection. A total of 5ml of cerebrospinal fluid (CSF) will be removed prior to administration of SPINRAZA® (nusinersen), which will be collected by the study. Neurofilament light chain (NfL) in CSF and blood will be assessed for the efficacy of nusinersen, as well as motor and pulmonary function.

There will be a total of nine visits. All the patients with 5q SMA receiving nusinersen will be visited face to face at baseline, day 14, day 28 and day 63 after treatment initiation, and then 4-month intervals through month 24/22.

primary outcomes:

• Change in CSF Neurofilament Light Chain levels since baseline
  Measured by Single-molecule Array
• up to 24months
  

secondary outcomes:

• Change in serum Neurofilament Light Chain levels since baseline
  up to 24months

inclusion criteria:

exclusion criteria: Criteria:

Contraindication for lumbar puncture
Inability to access intrathecal space for nusinersen injection

sponsor: First Affiliated Hospital of Fujian Medical University

contacts: Yi Lin, MD, PhD, 13615039153, linyi7811@163.com

investigators: Wan-Jin Chen,First Affiliated Hospital Fujian Medical University

trial center locations: China

• Antisense Oligonucleotide for Spinal Muscular Atrophy
  Yi Lin, MD, PhD, 13615039153, linyi7811@163.com
  
  
• Antisense Oligonucleotide for Spinal Muscular Atrophy
  Yi Dai, MD,PhD, 13811177531, pumchdy@pumch.cn
  
  
• Antisense Oligonucleotide for Spinal Muscular Atrophy
  Jun Hu, MD, PhD, 18950472106, hujun2252@126.com