Monogenic DiseasesNCT03528525
Registry Study on Respiratory Monogenic Diseases in Chinese Children
An observational study of Monogenic Diseases, run by Kunling Shen at 1 site in China.
Trial summary
This study is a multicenter, prospective cohort study of patients diagnosed with respiratory monogenic diseases, the clinical information of recruited patients, including clinical manifestations, lung function, chest imaging, quality of life and other indicators, will be followed for 5 years. Read more about this study
- Who is it for?
- Ages 1 month to 18 years
- Conditions the study is for
- Monogenic Diseases
- How many people
- 200 participants
- Where
- 1 siteChina
- Study dates
- 1 Jan 2017to 30 Dec 2030
- How long will I take part?
- 5 Years
- 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 month to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Children who was confirmed diagnosis as respiratory monogenic diseases at the certain hospitals (sponsor and collaborators)
Who may be able to join
- Age: 0-18 years, male and female.
- Any organ system symptoms consistent with the diagnostic points of respiratory monogenic diseases.
- Agree to retain specimens related to disease research and agree to store the specimens in the sample bank.
- willing and able to cooperate with long-term follow-up.
- The guardian of the child has a full understanding of the purpose of the study, a basic understanding of the clinical research program, and voluntarily allows the child to participate in the study and sign an informed consent form.
Who cannot join
- It is unable to provide complete medical records or the current condition can not accept the diagnosis process
- She or he cannot agree to participate in the study.
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
Beijing Children's hospital
Beijing, Beijing Municipality, 100045
Kunling Shen, MD,PhD, 861059616308, kunlingshen1717@163.com
About this study
Official title: Beijing Children's Hospital
This study is a multicenter, prospective cohort study of patients diagnosed with respiratory monogenic diseases, the clinical information of recruited patients, including clinical manifestations, lung function, chest imaging, quality of life and other indicators, will be followed for 5 years.
Read the detailed description
All new cases of respiratory monogenic diseases was confirmed or diagnosed at each center from the beginning of the study are made the investigation of the clinical manifestations and specific tests by the standard diagnostic process.Then all the patients' following clinical data will be followed for 5 years: clinical manifestations, lung function, chest imaging (once per year), gene test, quality of life and other indicators.
- Conditions
- Monogenic Diseases
- Keywords
- Diseases, Children, Respiratory, Monogenic diseases, China
- Medical subject headings
- Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Other
observation
| Study group | Role | What they receive |
|---|---|---|
| Monogenic diseases cases | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in lung function on the spirometry
forced expiratory volume at one second (FEV1) in Liter
Measured: 1 year later
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 May 2018
- Study start1 Jan 2017
- Primary completion1 Jan 2018
- Study completion30 Dec 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Monogenic DiseasesStudies 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
- Case only
- Time perspective
- Prospective
- Enrolment
- 200 participants (estimated)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- Kunling Shen
- Sponsor type
- University, hospital or other
- Collaborators
- Capital Institute of Pediatrics, China, Shengjing Hospital, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine, The Children's Hospital of Zhejiang University School of Medicine, Children's Hospital of Chongqing Medical University, The first clinical college of Guangzhou medical university, Guangzhou Women and Children's Medical Center, Yuying Children's Hospital of Wenzhou Medical University
- Responsible party
- Sponsor-investigator, Kunling Shen, Beijing Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03528525
- Sponsor's protocol ID
- BCHlung 010
- Status last verified
- May 2018
- First submitted
- 6 May 2018
- Last updated
- 18 May 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 May 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Beijing Children's Hospital
- ClinicalTrials.gov ID
- NCT03528525
- Acronym / sponsor ID
- BCHlung 010
- Conditions
- Monogenic Diseases
- Keywords
- Diseases, Children, Respiratory, Monogenic diseases, China
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Kunling ShenUniversity, hospital or other
- Collaborators
- Capital Institute of Pediatrics, China, Shengjing Hospital, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine, The Children's Hospital of Zhejiang University School of Medicine, Children's Hospital of Chongqing Medical University, The first clinical college of Guangzhou medical university, Guangzhou Women and Children's Medical Center, Yuying Children's Hospital of Wenzhou Medical University
- Study start
- 1 Jan 2017 (actual)
- Primary completion
- 1 Jan 2018 (actual)
- Study completion
- 30 Dec 2030 (estimated)
- First posted
- 18 May 2018 (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.
This study is a multicenter, prospective cohort study of patients diagnosed with respiratory monogenic diseases, the clinical information of recruited patients, including clinical manifestations, lung function, chest imaging, quality of life and other indicators, will be followed for 5 years.
Detailed description
All new cases of respiratory monogenic diseases was confirmed or diagnosed at each center from the beginning of the study are made the investigation of the clinical manifestations and specific tests by the standard diagnostic process.Then all the patients' following clinical data will be followed for 5 years: clinical manifestations, lung function, chest imaging (once per year), gene test, quality of life and other indicators.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Target follow-up
- 5 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Monogenic diseases cases | – | – |
Who may qualify
- Ages
- 1 month to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Children who was confirmed diagnosis as respiratory monogenic diseases at the certain hospitals (sponsor and collaborators)
Key inclusion criteria (5 listed)
- Age: 0-18 years, male and female.
- Any organ system symptoms consistent with the diagnostic points of respiratory monogenic diseases.
- Agree to retain specimens related to disease research and agree to store the specimens in the sample bank.
- willing and able to cooperate with long-term follow-up.
- The guardian of the child has a full understanding of the purpose of the study, a basic understanding of the clinical research program, and voluntarily allows the child to participate in the study and sign an informed consent form.
Key exclusion criteria (2 listed)
- It is unable to provide complete medical records or the current condition can not accept the diagnosis process
- She or he cannot agree to participate in the study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| observation | Other | Monogenic diseases cases |
Primary outcome
Change from baseline in lung function on the spirometry
Time frame: 1 year later
forced expiratory volume at one second (FEV1) in Liter
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 18 May 2018.
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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 Monogenic Diseases, run by Kunling Shen 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 2 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 18 May 2018. 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 Kunling Shen, MD,PhD (861059616308, kunlingshen1717@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline in lung function on the spirometry (time frame: 1 year later). 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 18 May 2018.