Antisense Oligonucleotide for Spinal Muscular Atrophy

January 11, 2022 updated by: Wan-Jin Chen, First Affiliated Hospital of Fujian Medical University

Antisense Oligonucleotide Therapy in Spinal Muscular Atrophy: An Observational Study in China

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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 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.

Study Type

Observational

Enrollment (Anticipated)

1000

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • China
    • Beijing
      • Beijing, Beijing, China, 100010
        • Recruiting
        • Department of Neurology, Peking Union Medical College Hospital
        • Contact:
        • Sub-Investigator:
          • Yi Dai, MD, PhD
    • Fujian
      • Fuzhou, Fujian, China, 350001
        • Recruiting
        • Department of Pediatrics, Fujian Medical University Union Hospital
        • Contact:
        • Sub-Investigator:
          • Jun Hu, MD, PhD
      • Fuzhou, Fujian, China, 350005
        • Not yet recruiting
        • Department of Neurology, First Affiliated Hospital Fujian Medical University
        • Principal Investigator:
          • Wan-Jin Chen, MD, PhD
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 week to 50 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Patients genetically confirmed 5q SMA including types I, II and III

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
5q SMA type I
Drug: Nusinersen
SPINRAZA® (nusinersen) prescribed as part of standard of care
5q SMA type II
Drug: Nusinersen
SPINRAZA® (nusinersen) prescribed as part of standard of care
5q SMA type III
Drug: Nusinersen
SPINRAZA® (nusinersen) prescribed as part of standard of care
Non-5q SMA
Non-SMA subjects
Including asymptomatic carriers of SMA, relatives of SMA patients and carriers, and patients undergoing clinical standard lumbar puncture

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in CSF Neurofilament Light Chain levels since baseline
Time Frame: up to 24months
Measured by Single-molecule Array
up to 24months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in serum Neurofilament Light Chain levels since baseline
Time Frame: up to 24months
Measured by Single-molecule Array
up to 24months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

First Affiliated Hospital of Fujian Medical University

Collaborators

Peking Union Medical College Hospital
Fujian Medical University Union Hospital

Investigators

  • Principal Investigator: Wan-Jin Chen, First Affiliated Hospital Fujian Medical University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 1, 2022

Primary Completion (Anticipated)

January 31, 2024

Study Completion (Anticipated)

January 31, 2024

Study Registration Dates

First Submitted

December 25, 2021

First Submitted That Met QC Criteria

December 25, 2021

First Posted (Actual)

January 11, 2022

Study Record Updates

Last Update Posted (Actual)

January 26, 2022

Last Update Submitted That Met QC Criteria

January 11, 2022

Last Verified

January 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MRCTA,ECFAH of FMU [2021]489

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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