Primary Immunodeficiency DiseaseNCT03252548
Pediatric Primary Immunodeficiency Disease (PID) in China
An observational study of Primary Immunodeficiency Disease, run by Beijing Children's Hospital.
Trial summary
This study is a prospective cohort study of children diagnosed with primary immunodeficiency disease (PID). The aim is to investigate the clinical characteristics and prognosis of PID in Chinese children. Read more about this study
- Who is it for?
- Ages 1 day to 18 years
- Conditions the study is for
- Primary Immunodeficiency Disease
- How many people
- 400 participants
- Where
- No sites listed yet
- Study dates
- Jan 2018to Aug 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
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 day to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Children who was confirmed diagnosis as PID at the certain hospitals
Who may be able to join
- Age: from neonates to 18 years old
- Fulfilling the one of the followings:
- Severe infection after vaccination (BCG, polio arthritis pill)
- Chronic/recurrent ear infection, sinus infections and respiratory tract infection
- Recurrent, deep skin or deep-seated infections
- Boys with early period and refractory thrombocytopenia
Show all 18Show fewer
- Infants with obviously decreased absolute number of lymphocytes
- Severe allergy with infection
- Infant diabetes with severe diarrhea
- Boys with severe Epstein-Barr virus infection
- Infants with hepatosplenomegaly and generalized lymphadenopathy
- Infantile hemophagocytic syndrome
- Failure of an infant to gain weight or grow normally
- Persistent thrush in mouth or fungalinfection on skin
- Family history of primary immunodeficiency
- Invasive pneumococcal infection
- Erythrodermic psoriasis
- Gene test confirmed PID pediatric patients
Who cannot join
- Subject will be excluded if she or he would not enroll into this study
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Clinical Characteristics and Prognosis of Primary Immunodeficiency Disease (PID) in Chinese Children
This study is a prospective cohort study of children diagnosed with primary immunodeficiency disease (PID). The aim is to investigate the clinical characteristics and prognosis of PID in Chinese children.
Read the detailed description
Primary immunodeficiency disease (PID) is a group of rare and fatal disease. However, the research about clinical characteristics, treatment, management and prognosis of Chinese children with PID is still not perfect and there is no basic and large database. Therefore, this study is conducted, in order to set up a complete database and long term follow up of Chinese children with PID, and establish foundations for basic research and precise medicine.
This study is divided into two parts. In part one, pediatric patients with PID in the past 10 years will be collected retrospectively by review medical records from these centers, and investigate the clinical features and prognosis. In part two, all new cases with suspected PID will be collected. Clinical symptoms, physical examination, blood tests, and humoral and cellular immune functions will be detected. Gene tests would be done when necessary to confirm the diagnosis. Then follow up at 6-moth, 1-year, 2-year, 3-year, 4-year and 5-year respectively. The trial will be completed in 60 months.
- Conditions
- Primary Immunodeficiency Disease
- Medical subject headings
- Primary Immunodeficiency Diseases
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequencies of severe infection of children with PID
Times of severe infection attack will be recorded every year from baseline to 60 months, assessed by questionnaire
Measured: 60 months
Secondary outcomes (3)
Life quality of children with PID
Life Quality Questionnaire, once for every year
Measured: 60 months
Complications of children with PID
Measured: 60 months
Long-term changes on lung function of children with PID
Measured: 60 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Aug 2017
- Study startJan 2018 (estimated)
- Primary completionAug 2022 (estimated)
- Study completionAug 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Immunodeficiency DiseaseStudies 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
- 400 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Beijing Children's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Capital Institute of Pediatrics, China, Shenzhen Children's Hospital
- Responsible party
- Principal investigator, Kunling Shen, Beijing Children's Hospital
- Investigators
- kunling shen, MD,PhD, Study director, Beijing Children's Hospital, Capital Medical University, Chinajianxin he, MD,PhD, Principal investigator, Beijing Children's Hospital, Capital Medical University, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03252548
- Sponsor's protocol ID
- BCHlung006
- Status last verified
- May 2017
- First submitted
- 15 Aug 2017
- Last updated
- 3 Jan 2018
- 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 3 Jan 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Characteristics and Prognosis of Primary Immunodeficiency Disease (PID) in Chinese Children
- ClinicalTrials.gov ID
- NCT03252548
- Acronym / sponsor ID
- BCHlung006
- Conditions
- Primary Immunodeficiency Disease
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 400 participants (estimated)
- Lead sponsor
- Beijing Children's HospitalUniversity, hospital or other
- Collaborators
- Capital Institute of Pediatrics, China, Shenzhen Children's Hospital
- Study start
- Jan 2018 (estimated)
- Primary completion
- Aug 2022 (estimated)
- Study completion
- Aug 2022 (estimated)
- First posted
- 17 Aug 2017 (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 prospective cohort study of children diagnosed with primary immunodeficiency disease (PID). The aim is to investigate the clinical characteristics and prognosis of PID in Chinese children.
Detailed description
Primary immunodeficiency disease (PID) is a group of rare and fatal disease. However, the research about clinical characteristics, treatment, management and prognosis of Chinese children with PID is still not perfect and there is no basic and large database. Therefore, this study is conducted, in order to set up a complete database and long term follow up of Chinese children with PID, and establish foundations for basic research and precise medicine.
This study is divided into two parts. In part one, pediatric patients with PID in the past 10 years will be collected retrospectively by review medical records from these centers, and investigate the clinical features and prognosis. In part two, all new cases with suspected PID will be collected. Clinical symptoms, physical examination, blood tests, and humoral and cellular immune functions will be detected. Gene tests would be done when necessary to confirm the diagnosis. Then follow up at 6-moth, 1-year, 2-year, 3-year, 4-year and 5-year respectively. The trial will be completed in 60 months.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 1 day to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Children who was confirmed diagnosis as PID at the certain hospitals
Key inclusion criteria (18 listed)
- Age: from neonates to 18 years old
- Fulfilling the one of the followings:
- Gene test confirmed PID pediatric patients
Key exclusion criteria (1 listed)
- Subject will be excluded if she or he would not enroll into this study
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
Frequencies of severe infection of children with PID
Time frame: 60 months
Times of severe infection attack will be recorded every year from baseline to 60 months, assessed by questionnaire
3 secondary outcomes
Life quality of children with PID
Time frame: 60 months
Complications of children with PID
Time frame: 60 months
Long-term changes on lung function of children with PID
Time frame: 60 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jan 2018.
- 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. This study is not recruiting at the moment.
kunling shen, MD,PhD study director
Beijing Children's Hospital, Capital Medical University, China
jianxin he, MD,PhD principal investigator
Beijing Children's Hospital, Capital Medical University, China
Questions about this trial
What is this clinical trial studying?
An observational study of Primary Immunodeficiency Disease, run by Beijing Children's Hospital.
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 18 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: status not confirmed, as of the sponsor's last update on 3 Jan 2018. Status can change between updates, so confirm with the study contact before making plans.
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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact baoping xu, MD,PhD (861059616308, xubaopingbch@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Frequencies of severe infection of children with PID (time frame: 60 months). 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 3 Jan 2018.