Pediatric Primary Immunodeficiency Disease (PID) in China

Primary Immunodeficiency Disease Clinical Trial

Official title:

Clinical Characteristics and Prognosis of Primary Immunodeficiency Disease (PID) in Chinese Children

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT03252548
• Other study ID #: BCHlung006
• Status: Not yet recruiting
• Phase: N/A
• First received: August 15, 2017
• Last updated: January 1, 2018
• Start date: January 2018
• Est. completion date: August 2022

Study information

• Verified date: May 2017
• Source: Beijing Children's Hospital
• Contact: baoping xu, MD,PhD
• Phone: 861059616308
• Email: xubaopingbch[@]163.com
• Is FDA regulated: No
• Study type: Observational

Clinical 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.

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 400
• Est. completion date: August 2022
• Est. primary completion date: August 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 18 Years

Eligibility

Inclusion Criteria:

1. Age: from neonates to 18 years old

2. 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

3. Gene test confirmed PID pediatric patients

Exclusion Criteria:

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

Related Conditions & MeSH terms

• Immunologic Deficiency Syndromes
• Primary Immunodeficiency Disease

Locations

Country	Name	City	State
n/a

Sponsors (3)

Lead Sponsor	Collaborator
Beijing Children's Hospital	Capital Institute of Pediatrics, China, Shenzhen Children's Hospital

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Life quality of children with PID	Life Quality Questionnaire, once for every year	60 months
Secondary	Complications of children with PID		60 months
Secondary	Long-term changes on lung function of children with PID		60 months