Vitamin D3 For CGD Patients With BCGosis/Itis

Fudan University

Status and phase: Completed; Phase 3; Phase 2

Conditions: Vitamin D3; Chronic-granulomatous Disease; BCG

Treatments: Drug: Traditional treatment of CGD and TB; Drug: Vitamin D3

Study type: Interventional

Funder types: Other

Identifiers

NCT03984890

VDBCG

Details and patient eligibility

About

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.

Full 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.

Enrollment: 50 patients

Sex: All

Ages: Under 18 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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50 participants in 2 patient groups

Vitamin D Group

Experimental group

Description:

Vitamin D3 Supplementation plus traditional treatment of CGD and TB

Treatment:

Drug: Vitamin D3

Drug: Traditional treatment of CGD and TB

Control Group

Other group

Description:

Traditional treatment of CGD and TB without Vitamin D Supplementation

Treatment:

Drug: Traditional treatment of CGD and TB