Spinal Muscular AtrophyNCT04010604
A Registered Cohort Study on SMA
An observational study of Spinal Muscular Atrophy, run by Wan-Jin Chen at 1 site in China.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 1 week to 70 yearsHealthy volunteers can take part
- Conditions the study is for
- Spinal Muscular Atrophy
- How many people
- 2,000 participants
- Where
- 1 siteChina
- Study dates
- 1 Jul 2019to 31 Dec 2049
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 week to 70 years
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: SMA patients are diagnosed with SMN1 gene deletion or mutation.
Who may be able to join
- 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
Who cannot join
- Participants are unable to comply with trial procedures and visit schedule
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Department of Neurology, First Affiliated Hospital Fujian Medical University
Fuzhou, Fujian, 350005
Wan-Jin Chen, PhD, 86-0591-87982772 ext. +1386061359, wanjinchen75@fjmu.edu.cn
Wan-Jin Chen, MD,PhD
About this study
Official title: A Registered Cohort Study on Spinal Muscular Atrophy
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.
- Conditions
- Spinal Muscular Atrophy
- Medical subject headings
- Muscular Atrophy, Spinal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| SMA type I | Not described | |
| SMA type II | Not described | |
| SMA type III | Not described | |
| Asymptomatic carriers of SMA | Not described | |
| Relatives of SMA patients and carriers | Not described | |
| Unrelated healthy controls | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
The time to death
Measured: From date of enrollment until the date of death from any cause, assessed up to 20years
The correlation of genotype and phenotype
Genotype is defined by survival motor neuron (SMN) 2 copy number(s) and phenotype is defined by clinical types and characteristics.
Measured: From date of enrollment until the date of death from any cause, assessed up to 20years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jul 2019
- Study start1 Jul 2019
- Primary completion31 Dec 2039 (estimated)
- Study completion31 Dec 2049 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinal Muscular AtrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 2,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Wan-Jin Chen
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Wan-Jin Chen, First Affiliated Hospital of Fujian Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04010604
- Sponsor's protocol ID
- MRCTA,ECFAHOFFMU[2019]196
- Status last verified
- Sep 2019
- First submitted
- 1 Jul 2019
- Last updated
- 23 Sep 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Registered Cohort Study on Spinal Muscular Atrophy
- ClinicalTrials.gov ID
- NCT04010604
- Acronym / sponsor ID
- MRCTA,ECFAHOFFMU[2019]196
- Conditions
- Spinal Muscular Atrophy
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 2,000 participants (estimated)
- Lead sponsor
- Wan-Jin ChenUniversity, hospital or other
- Study start
- 1 Jul 2019 (actual)
- Primary completion
- 31 Dec 2039 (estimated)
- Study completion
- 31 Dec 2049 (estimated)
- First posted
- 8 Jul 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| SMA type I | – | – |
| SMA type II | – | – |
| SMA type III | – | – |
| Asymptomatic carriers of SMA | – | – |
| Relatives of SMA patients and carriers | – | – |
| Unrelated healthy controls | – | – |
Who may qualify
- Ages
- 1 week to 70 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- SMA patients are diagnosed with SMN1 gene deletion or mutation.
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (1 listed)
- Participants are unable to comply with trial procedures and visit schedule
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
The time to death
Time frame: From date of enrollment until the date of death from any cause, assessed up to 20years
The correlation of genotype and phenotype
Time frame: From date of enrollment until the date of death from any cause, assessed up to 20years
Genotype is defined by survival motor neuron (SMN) 2 copy number(s) and phenotype is defined by clinical types and characteristics.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Sep 2019.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Spinal Muscular Atrophy, run by Wan-Jin Chen at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 23 Sep 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Yi Lin, PhD (86-0591-87982772, linyi7811@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The time to death (time frame: From date of enrollment until the date of death from any cause, assessed up to 20years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Sep 2019.