Antisense Oligonucleotide for Spinal Muscular Atrophy | NCT05187260

Studies
Antisense Oligonucleotide for Spinal Muscular Atrophy

NCT05187260

Sponsor

First Affiliated Hospital of Fujian Medical University

Status

Unknown status

Last Update Posted

Jan 26, 2022Actual

Enrollment

1,000Estimated

Phase

Not provided

Conditions

Spinal Muscular Atrophy

Interventions

Nusinersen

Countries

China

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Jan 2022

Overall Recruitment Status or Expanded Access Status

Unknown status

Last Known Status

Recruiting

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jan 1, 2022Actual

Primary Completion Date

Jan 31, 2024Estimated

Completion Date

Jan 31, 2024Estimated

First Submitted Date

Dec 25, 2021

First Submission Date that Met QC Criteria

Dec 25, 2021

First Posted Date

Jan 11, 2022Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jan 11, 2022

Last Update Posted Date

Jan 26, 2022Actual

Sponsor/Collaborators Module

Responsible Party, by Official Title

Wan-Jin Chen, The Director for the Department of Neurology, First Affiliated Hospital of Fujian Medical UniversityPrincipal Investigator

Lead Sponsor

First Affiliated Hospital of Fujian Medical UniversityOTHER

Collaborators

Name	Class
Fujian Medical University Union Hospital	OTHER
Peking Union Medical College Hospital	OTHER

Oversight Module

Has Data Monitoring Committee (DMC)

Not provided

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

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.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

Retention

Enrollment

1,000Estimated

Arms/Interventions Module

Arm Groups

Label	Description	Intervention Names
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

Name	Type	Description	Arm Group Labels	Other Names
Nusinersen	Drug	SPINRAZA® (nusinersen) prescribed as part of standard of care	5q SMA type I 5q SMA type II 5q SMA type III

Outcomes Module

Primary Outcomes

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

Secondary Outcomes

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

Other Outcomes

Not provided

Eligibility Module

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

Accepts Healthy Volunteers

Yes

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

1 Week

Maximum Age

50 Years

Standard Ages

ChildAdult

Study Population

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

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
Yi Lin, MD, PhD	Contact	13615039153		linyi7811@163.com

Overall Officials

Name	Affiliation	Role
Wan-Jin Chen	First Affiliated Hospital Fujian Medical University	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Department of Neurology, Peking Union Medical College Hospital	Recruiting	Beijing	Beijing Municipality	100010	China

Condition Browse Module

MeSH Terms

ID	Term
D009134	Muscular Atrophy, Spinal
C537987	Charcot-Marie-Tooth disease, Type 1F

Ancestor Terms

ID	Term
D013118	Spinal Cord Diseases
D002493	Central Nervous System Diseases
D009422	Nervous System Diseases
D016472	Motor Neuron Disease

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
C000590926	nusinersen

Ancestor Terms

Not provided

Browse Leaves

Not provided

Browse Branches

Not provided

Description

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.