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Vitamin D3 For CGD Patients With BCGosis/Itis

Phase 2
Completed
Conditions
Vitamin D3
BCG
Chronic-granulomatous Disease
Interventions
Drug: Traditional treatment of CGD and TB
Registration Number
NCT03984890
Lead Sponsor
Children's Hospital of Fudan University
Brief Summary

When children with chronic granulomatous disease (CGD) got BCG infection the treatment would be a tough task. The goal of the proposed research is to observe weather vitamin D supplementation can help the CGD children get through this challenge.

Detailed Description

Chronic granulomatous disease (CGD) is one of primary immunodeficiency diseases. Due to the deficiency of the phagocyte nicotinamide adenine dinucleotide phosphate (NADPH) oxidase the respiratory burst of all types of phagocytic cells is badly impaired which lead to a susceptibility to infection among CGD patients.

BCG vaccine is wildly used in China to avoid severe tuberculosis infection. Children are supposed to get BCG vaccine injected within 24 hours after birth. When patients with CGD got the vaccination of BCG they will easily got infected. And due to the immunodeficiency of these children, the infection cannot be cure by normal treatment.

Vitamin D supplementation was used to treat tuberculosis in the pre-antibiotic era and is reported to have influence on immune system especially on monocytes and macrophages thus may help CGD children defend the BCG infection. In addition, studies show that 1,25-Dihydroxyvitamin D3 can induce nitric oxide synthase thus may up regulate NO production and help host defense against human tuberculosis without the help of NADPH oxidase. Other researches indicate that Vitamin D and the expression of vitamin D receptor may lead to induction of antimicrobial peptide such as LL-37 which help macrophages kill the intracellular Mycobacterium tuberculosis. These discoveries indicated that vitamin D may induce immune response against BCG in a nontraditional way. Therefore, when CGD patients face BCG infection, add vitamin D supplementation to the treatment may help them survive this challenge.

Since there have had clinical trials revealing that intermittent high dose vitamin D3 supplementation as 2.5mg per 14 days only receive positive effect on partial patients the investigators decide to choose a mild dose treatment as 800IU/d for 3 month to see if things get different in this way.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
50
Inclusion Criteria
  1. less than 18 years' old
  2. Diagnosed with CGD
  3. Got BCG infection after vaccination
Exclusion Criteria
  1. Serum 25-(OH)-vit D >75 nmol/L (30 ng/mL)
  2. Hyperphosphatemia
  3. Hypercalcemia
  4. Acute or chronic renal failure
  5. Acute or chronic cardiac failure
  6. Kidney stone

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Control GroupTraditional treatment of CGD and TBTraditional treatment of CGD and TB without Vitamin D Supplementation
Vitamin D GroupVitamin D3Vitamin D3 Supplementation plus traditional treatment of CGD and TB
Vitamin D GroupTraditional treatment of CGD and TBVitamin D3 Supplementation plus traditional treatment of CGD and TB
Primary Outcome Measures
NameTimeMethod
Mortality8 weeks

Death rate among patients

Duration of Fever8 weeks

Calculate the days suffer from fevers to show the severity of the infection and the efficacy of the treatment

Serum Levels of 25-OH Vitamin D38 weeks

Concentration of 25-OH Vitamin D3 in serum

Urine Protein8 weeks

Urine protein quantitation

Urine Calcium8 weeks

Concentration of calcium in urine

Rate of Sputum Culture acid-fast bacilli microscopy Conversion8 weeks

If sputum culture and acid-fast bacilli microscopy show positive results before treatments, compare the results before and after treatments to see if the rate that results changed from positive to negative differ between control group and vitamin D group

Serum Levels of Calcium8 weeks

Concentration of calcium in serum

Number of Anti-tuberculosis Drugs Used in the Treatment8 weeks

Calculate the number of anti-tuberculosis drugs used in the treatment to show the severity of the infection and the efficacy of the treatment

Secondary Outcome Measures
NameTimeMethod
Change in BMI1 year

Evaluate the change in BMI by calculating weight(kg)/height(m)\^2 before treatment and 1year after the treatment

Frequency of Recurrent Infections1 year

Use frequency of recurrent infections to evaluate long-term benefits

Trial Locations

Locations (1)

Children's Hospital of Fudan University

🇨🇳

Shanghai, Shanghai, China

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