NCT05187260

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 months.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
1,000

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Jan 2022

Typical duration for all trials

Geographic Reach
1 country

3 active sites

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

December 25, 2021

Completed
7 days until next milestone

Study Start

First participant enrolled

January 1, 2022

Completed
10 days until next milestone

First Posted

Study publicly available on registry

January 11, 2022

Completed
2.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 31, 2024

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 31, 2024

Completed
Last Updated

January 26, 2022

Status Verified

January 1, 2022

Enrollment Period

2.1 years

First QC Date

December 25, 2021

Last Update Submit

January 11, 2022

Conditions

Spinal Muscular Atrophy

Keywords

Spinal Muscular AtrophyAntisense oligonucleotideNeurofilament light chain

Outcome Measures

Primary Outcomes (1)

  • Change in CSF Neurofilament Light Chain levels since baseline

    Measured by Single-molecule Array

    up to 24months

Secondary Outcomes (1)

  • Change in serum Neurofilament Light Chain levels since baseline

    up to 24months

Study Arms (5)

5q SMA type I

Drug: Nusinersen

5q SMA type II

Drug: Nusinersen

5q SMA type III

Drug: Nusinersen

Non-5q SMA

Non-SMA subjects

Including asymptomatic carriers of SMA, relatives of SMA patients and carriers, and patients undergoing clinical standard lumbar puncture

Interventions

NusinersenDRUG

SPINRAZA® (nusinersen) prescribed as part of standard of care

5q SMA type I5q SMA type II5q SMA type III

Eligibility Criteria

Age1 Week - 50 Years
Sexall
Healthy VolunteersYes
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodNon-Probability Sample
Study Population

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

You may qualify if:

  • 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

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Department of Neurology, Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100010, China

RECRUITING

Department of Pediatrics, Fujian Medical University Union Hospital

Fuzhou, Fujian, 350001, China

RECRUITING

Department of Neurology, First Affiliated Hospital Fujian Medical University

Fuzhou, Fujian, 350005, China

NOT YET RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

5ml of CSF is collected and typically discarded as part of the typical clinical encounter when SPINRAZA® (nusinersen) is administered. This 5ml of CSF will be collected for research purposes at each dosing visit. Blood samples, including whole blood, serum, and plasma (less than 10 ml total) will be collected before intrathecal injection of SPINRAZA® (nusinersen) at each dosing visit.

MeSH Terms

Conditions

Muscular Atrophy, SpinalCharcot-Marie-Tooth disease, Type 1F

Interventions

nusinersen

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesNeuromuscular Diseases

Study Officials

  • Wan-Jin Chen

    First Affiliated Hospital Fujian Medical University

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Yi Lin, MD, PhD

CONTACT

13615039153linyi7811@163.com

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
The Director for the Department of Neurology

Study Record Dates

First Submitted

December 25, 2021

First Posted

January 11, 2022

Study Start

January 1, 2022

Primary Completion

January 31, 2024

Study Completion

January 31, 2024

Last Updated

January 26, 2022

Record last verified: 2022-01

Locations

CN(3)