A Registered Cohort Study on SMA

NCT ID: NCT04010604

Last Updated: 2019-09-23

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Total Enrollment

2000 participants

Study Classification

OBSERVATIONAL

Study Start Date

2019-07-01

Study Completion Date

2049-12-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

Spinal muscular atrophy (SMA) is an autosomal recessive disease that causes progressive muscle wasting and weakness due to loss of motor neurons in the spinal cord. This is a registered cohort of spinal muscular atrophy (SMA) type I,II and III in China. This study will provide further insights into the clinical course of SMA including overall survival, demographic characteristics, motor function, respiratory support, feeding and nutritional support, growth and development. The correlation of genotype and phenotype will be conducted.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Spinal Muscular Atrophy

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

SMA type I

No interventions assigned to this group

SMA type II

No interventions assigned to this group

SMA type III

No interventions assigned to this group

Asymptomatic carriers of SMA

No interventions assigned to this group

Relatives of SMA patients and carriers

No interventions assigned to this group

Unrelated healthy controls

No interventions assigned to this group

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Patients with SMA types I, II and III
* Asymptomatic SMA carriers
* Relatives of SMA patients or carriers
* Unrelated healthy controls
* Participants or Parent(s)/legal guardian(s) willing and able to complete the informed consent process

Exclusion Criteria

\* Participants are unable to comply with trial procedures and visit schedule
Minimum Eligible Age

1 Week

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Wan-Jin Chen

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Wan-Jin Chen

The Vice-Director for the Department of Neurology

Responsibility Role SPONSOR_INVESTIGATOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Department of Neurology, First Affiliated Hospital Fujian Medical University

Fuzhou, Fujian, China

Site Status RECRUITING

Countries

Review the countries where the study has at least one active or historical site.

China

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Yi Lin, PhD

Role: CONTACT

86-0591-87982772

Wan-Jian Chen, PhD

Role: CONTACT

86-0591-87982772

Facility Contacts

Find local site contact details for specific facilities participating in the trial.

Wan-Jin Chen, PhD

Role: primary

86-0591-87982772 ext. +1386061359

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

MRCTA,ECFAHOFFMU[2019]196

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.