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Vitamin D3 For CGD Patients With BCGosis/Itis

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ClinicalTrials.gov Identifier: NCT03984890
Recruitment Status : Not yet recruiting
First Posted : June 13, 2019
Last Update Posted : June 13, 2019
Sponsor:
Information provided by (Responsible Party):
Jinqiao Sun, 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.

Condition or disease Intervention/treatment Phase
Vitamin D3 Chronic-granulomatous Disease BCG Drug: Vitamin D3 Drug: Traditional treatment of CGD and TB Phase 2 Phase 3

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.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Effect of Vitamin D3 Supplementation on Chronic Granulomatous Disease Patients With BCGosis/Itis
Estimated Study Start Date : August 1, 2019
Estimated Primary Completion Date : December 31, 2020
Estimated Study Completion Date : December 31, 2022


Arm Intervention/treatment
Experimental: Vitamin D Group
Vitamin D3 Supplementation plus traditional treatment of CGD and TB
Drug: Vitamin D3
Vitamin D3 drops 800IU/d for 3 months

Drug: Traditional treatment of CGD and TB
Anti-tuberculosis drugs, interferon-gamma

Control Group
Traditional treatment of CGD and TB without Vitamin D Supplementation
Drug: Traditional treatment of CGD and TB
Anti-tuberculosis drugs, interferon-gamma




Primary Outcome Measures :
  1. Mortality [ Time Frame: 8 weeks ]
    Death rate among patients

  2. Rate of Sputum Culture acid-fast bacilli microscopy Conversion [ Time Frame: 8 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

  3. Duration of Fever [ Time Frame: 8 weeks ]
    Calculate the days suffer from fevers to show the severity of the infection and the efficacy of the treatment

  4. Number of Anti-tuberculosis Drugs Used in the Treatment [ Time Frame: 8 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

  5. Urine Protein [ Time Frame: 8 weeks ]
    Urine protein quantitation

  6. Urine Calcium [ Time Frame: 8 weeks ]
    Concentration of calcium in urine

  7. Serum Levels of 25-OH Vitamin D3 [ Time Frame: 8 weeks ]
    Concentration of 25-OH Vitamin D3 in serum

  8. Serum Levels of Calcium [ Time Frame: 8 weeks ]
    Concentration of calcium in serum


Secondary Outcome Measures :
  1. Change in BMI [ Time Frame: 1 year ]
    Evaluate the change in BMI by calculating weight(kg)/height(m)^2 before treatment and 1year after the treatment

  2. Frequency of Recurrent Infections [ Time Frame: 1 year ]
    Use frequency of recurrent infections to evaluate long-term benefits



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Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03984890


Contacts
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Contact: Jinqiao Sun, Ph.D.,M.D 86-21-64932909 jinqiaosun@sina.com

Locations
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China, Shanghai
Children's Hospital of Fudan University Not yet recruiting
Shanghai, Shanghai, China, 201102
Contact: Weili Yan, Ph.D       yanwl@fudan.edu.cn   
Children's Hospital of Fudan University Not yet recruiting
Shanghai, Shanghai, China
Contact: Jinqiao Sun, Ph.D.,M.D       jinqiaosun@sina.com   
Sponsors and Collaborators
Children's Hospital of Fudan University
Investigators
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Study Director: Weili Yan, Ph.D Children's Hospital of Fudan University

Publications:
Collaborative Group for the Meta-Analysis of Individual Patient Data in MDR-TB treatment–2017, Ahmad N, Ahuja SD, Akkerman OW, Alffenaar JC, Anderson LF, Baghaei P, Bang D, Barry PM, Bastos ML, Behera D, Benedetti A, Bisson GP, Boeree MJ, Bonnet M, Brode SK, Brust JCM, Cai Y, Caumes E, Cegielski JP, Centis R, Chan PC, Chan ED, Chang KC, Charles M, Cirule A, Dalcolmo MP, D'Ambrosio L, de Vries G, Dheda K, Esmail A, Flood J, Fox GJ, Fréchet-Jachym M, Fregona G, Gayoso R, Gegia M, Gler MT, Gu S, Guglielmetti L, Holtz TH, Hughes J, Isaakidis P, Jarlsberg L, Kempker RR, Keshavjee S, Khan FA, Kipiani M, Koenig SP, Koh WJ, Kritski A, Kuksa L, Kvasnovsky CL, Kwak N, Lan Z, Lange C, Laniado-Laborín R, Lee M, Leimane V, Leung CC, Leung EC, Li PZ, Lowenthal P, Maciel EL, Marks SM, Mase S, Mbuagbaw L, Migliori GB, Milanov V, Miller AC, Mitnick CD, Modongo C, Mohr E, Monedero I, Nahid P, Ndjeka N, O'Donnell MR, Padayatchi N, Palmero D, Pape JW, Podewils LJ, Reynolds I, Riekstina V, Robert J, Rodriguez M, Seaworth B, Seung KJ, Schnippel K, Shim TS, Singla R, Smith SE, Sotgiu G, Sukhbaatar G, Tabarsi P, Tiberi S, Trajman A, Trieu L, Udwadia ZF, van der Werf TS, Veziris N, Viiklepp P, Vilbrun SC, Walsh K, Westenhouse J, Yew WW, Yim JJ, Zetola NM, Zignol M, Menzies D. Treatment correlates of successful outcomes in pulmonary multidrug-resistant tuberculosis: an individual patient data meta-analysis. Lancet. 2018 Sep 8;392(10150):821-834. doi: 10.1016/S0140-6736(18)31644-1. Review.

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Responsible Party: Jinqiao Sun, Professor, Children's Hospital of Fudan University
ClinicalTrials.gov Identifier: NCT03984890     History of Changes
Other Study ID Numbers: VDBCG
First Posted: June 13, 2019    Key Record Dates
Last Update Posted: June 13, 2019
Last Verified: June 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Granuloma
Granulomatous Disease, Chronic
Lymphoproliferative Disorders
Lymphatic Diseases
Pathologic Processes
Phagocyte Bactericidal Dysfunction
Leukocyte Disorders
Hematologic Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Immunologic Deficiency Syndromes
Immune System Diseases
Vitamin D
Ergocalciferols
Cholecalciferol
Vitamins
Micronutrients
Nutrients
Growth Substances
Physiological Effects of Drugs
Bone Density Conservation Agents
Calcium-Regulating Hormones and Agents