Clinical trial NCT03984890: Vitamin D3 For CGD Patients With BCGosis/Itis
Official title: Effect of Vitamin D3 Supplementation on Chronic Granulomatous Disease Patients With BCGosis/Itis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Vitamin D3
Chronic-granulomatous Disease
BCG
Lead sponsor: Children's Hospital of Fudan University
Sponsor class: OTHER
First posted: 2019-06-13
Enrollment: 50
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Parallel Assignment
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Mortality
timeFrame: 8 weeks
measure: Rate of Sputum Culture acid-fast bacilli microscopy Conversion
timeFrame: 8 weeks
measure: Duration of Fever
timeFrame: 8 weeks
measure: Number of Anti-tuberculosis Drugs Used in the Treatment
timeFrame: 8 weeks
measure: Urine Protein
timeFrame: 8 weeks
measure: Urine Calcium
timeFrame: 8 weeks
measure: Serum Levels of 25-OH Vitamin D3
timeFrame: 8 weeks
measure: Serum Levels of Calcium
timeFrame: 8 weeks
Secondary outcomes:
measure: Change in BMI
timeFrame: 1 year
measure: Frequency of Recurrent Infections
timeFrame: 1 year
Other outcomes:
Locations:
city: Shanghai
country: China
facility: Children's Hospital of Fudan University
state: Shanghai
city: Shanghai
country: China
facility: Children's Hospital of Fudan University
state: Shanghai
Study officials:
affiliation: Children's Hospital of Fudan University
name: Weili Yan, Ph.D
role: STUDY_DIRECTOR
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