Antisense Oligonucleotide for Spinal Muscular Atrophy

Last updated: January 11, 2022
Sponsor: First Affiliated Hospital of Fujian Medical University
Overall Status: Active - Recruiting

Phase

N/A

Condition

Myasthenia Gravis (Chronic Weakness)

Muscular Dystrophy

Spinal Muscular Atrophy

Treatment

N/A

Clinical Study ID

NCT05187260
MRCTA,ECFAH of FMU [2021]489
  • Ages < 50
  • All Genders
  • Accepts Healthy Volunteers

Study 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 months.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Patients genetically confirmed 5q SMA including types I, II and III, who are planningto 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 andcarriers, and patients undergoing clinical standard lumbar puncture
  • Participants or Parent(s)/legal guardian(s) willing and able to complete the informedconsent process

Exclusion

Exclusion Criteria:

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

Study Design

Total Participants: 1000
Study Start date:
January 01, 2022
Estimated Completion Date:
January 31, 2024

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.

Connect with a study center

  • Department of Neurology, Peking Union Medical College Hospital

    Beijing, Beijing 100010
    China

    Active - Recruiting

  • Department of Neurology, First Affiliated Hospital Fujian Medical University

    Fuzhou, Fujian 350005
    China

    Site Not Available

  • Department of Pediatrics, Fujian Medical University Union Hospital

    Fuzhou, Fujian 350001
    China

    Active - Recruiting

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