Pediatric Primary Immunodeficiency Disease (PID) in China | NCT03252548

Studies
Pediatric Primary Immunodeficiency Disease (PID) in China

NCT03252548

Sponsor

Beijing Children's Hospital

Status

Unknown status

Last Update Posted

Jan 3, 2018Actual

Enrollment

400Estimated

Phase

Not provided

Conditions

Primary Immunodeficiency Disease

Interventions

Not provided

Countries

Not provided

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

May 2017

Overall Recruitment Status or Expanded Access Status

Unknown status

Last Known Status

Not yet recruiting

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jan 2018Estimated

Primary Completion Date

Aug 2022Estimated

Completion Date

Aug 2022Estimated

First Submitted Date

Aug 15, 2017

First Submission Date that Met QC Criteria

Aug 15, 2017

First Posted Date

Aug 17, 2017Actual

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 1, 2018

Last Update Posted Date

Jan 3, 2018Actual

Sponsor/Collaborators Module

Responsible Party, by Official Title

Kunling Shen, Chief of China National Clinical Research Center for Respiratory Diseases, Beijing Children's HospitalPrincipal Investigator

Lead Sponsor

Beijing Children's HospitalOTHER

Collaborators

Name	Class
Capital Institute of Pediatrics, China	OTHER
Shenzhen Children's Hospital	OTHER_GOV

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

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 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.

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

No data available

No data is available for this block.

Enrollment

400Estimated

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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	60 months

Secondary Outcomes

Measure	Description	Time Frame
Life quality of children with PID	Life Quality Questionnaire, once for every year	60 months
Complications of children with PID		60 months

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Age: from neonates to 18 years old
Fulfilling the one of the followings:
1. Severe infection after vaccination (BCG, polio arthritis pill)
2. Chronic/recurrent ear infection, sinus infections and respiratory tract infection
3. Recurrent, deep skin or deep-seated infections
4. Boys with early period and refractory thrombocytopenia
5. Infants with obviously decreased absolute number of lymphocytes
6. Severe allergy with infection
7. Infant diabetes with severe diarrhea
8. Boys with severe Epstein-Barr virus infection
9. Infants with hepatosplenomegaly and generalized lymphadenopathy
10. Infantile hemophagocytic syndrome
11. Failure of an infant to gain weight or grow normally
12. Persistent thrush in mouth or fungalinfection on skin
13. Family history of primary immunodeficiency
14. Invasive pneumococcal infection
15. Erythrodermic psoriasis
Gene test confirmed PID pediatric patients

Exclusion Criteria:

Subject will be excluded if she or he would not enroll into this study

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

1 Day

Maximum Age

18 Years

Standard Ages

ChildAdult

Study Population

Children who was confirmed diagnosis as PID at the certain hospitals

Sampling Method

Probability Sample

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
baoping xu, MD,PhD	Contact	861059616308		xubaopingbch@163.com

Overall Officials

Name	Affiliation	Role
kunling shen, MD,PhD	Beijing Children's Hospital, Capital Medical University, China	Study Director
jianxin he, MD,PhD	Beijing Children's Hospital, Capital Medical University, China	Principal Investigator

Locations

Not provided

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D000081207	Primary Immunodeficiency Diseases

Ancestor Terms

ID	Term
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D007153	Immunologic Deficiency Syndromes
D007154	Immune System Diseases

Browse Leaves

Not provided

Browse Branches

Not provided