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Vitamin D3Phase 2/3NCT03984890

Vitamin D3 For CGD Patients With BCGosis/Itis

A Phase 2/3 study testing Vitamin D3 and Traditional treatment of CGD and TB in people with Vitamin D3 and Chronic-granulomatous Disease, run by Children's Hospital of Fudan University at 2 sites in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages Up to 18 years
Conditions the study is for
Vitamin D3, Chronic-granulomatous Disease, BCG
What is being tested?
Vitamin D3 and Traditional treatment of CGD and TB
Could I receive a placebo?
No, everyone receives an active treatment
How many people
50 participants
Where
2 sitesChina
Study dates
1 Aug 2019to 31 Dec 2023
Phase
Phase 2/3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Up to 18 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • less than 18 years' old
  • Diagnosed with CGD
  • Got BCG infection after vaccination

Who cannot join

  • Serum 25-(OH)-vit D >75 nmol/L (30 ng/mL)
  • Hyperphosphatemia
  • Hypercalcemia
  • Acute or chronic renal failure
  • Acute or chronic cardiac failure
  • Kidney stone

Trial locations and contacts

2 sites in 1 country.

China2 sites

  • Children's Hospital of Fudan University

    Shanghai, Shanghai Municipality, 201102

  • Children's Hospital of Fudan University

    Shanghai, Shanghai Municipality

Other trials in China

About this study

Official title: Effect of Vitamin D3 Supplementation on Chronic Granulomatous Disease Patients With BCGosis/Itis

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.

Read the 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.

Being tested
Vitamin D3, Traditional treatment of CGD and TB
Medical subject headings
Granulomatous Disease, Chronic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Vitamin D3Drug

    Vitamin D3 drops 800IU/d for 3 months

  • Traditional treatment of CGD and TBDrug

    Anti-tuberculosis drugs, interferon-gamma

Study groups in NCT03984890
Study groupRoleWhat they receive
Vitamin D GroupExperimentalVitamin D3 Supplementation plus traditional treatment of CGD and TB
Receives: Drug: Vitamin D3; Drug: Traditional treatment of CGD and TB
Control GroupOtherTraditional treatment of CGD and TB without Vitamin D Supplementation
Receives: Drug: Traditional treatment of CGD and TB

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Mortality

    Death rate among patients

    Measured: 8 weeks

  2. Rate of Sputum Culture acid-fast bacilli microscopy Conversion

    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

    Measured: 8 weeks

  3. Duration of Fever

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

    Measured: 8 weeks

  4. Number of Anti-tuberculosis Drugs Used in the Treatment

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

    Measured: 8 weeks

  5. Urine Protein

    Urine protein quantitation

    Measured: 8 weeks

  6. Urine Calcium

    Concentration of calcium in urine

    Measured: 8 weeks

  7. Serum Levels of 25-OH Vitamin D3

    Concentration of 25-OH Vitamin D3 in serum

    Measured: 8 weeks

  8. Serum Levels of Calcium

    Concentration of calcium in serum

    Measured: 8 weeks

Secondary outcomes (2)

  1. Change in BMI

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

    Measured: 1 year

  2. Frequency of Recurrent Infections

    Use frequency of recurrent infections to evaluate long-term benefits

    Measured: 1 year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted13 Jun 2019
  2. Study start1 Aug 2019
  3. Primary completion31 Dec 2021
  4. Study completion31 Dec 2023

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2/3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
Parallel Assignment
Masking
None (open label)
Enrolment
50 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03984890
Sponsor's protocol ID
VDBCG
Status last verified
Jul 2024
First submitted
5 Dec 2018
Last updated
9 Jul 2024
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Martineau AR, Wilkinson KA, Newton SM, Floto RA, Norman AW, Skolimowska K, Davidson RN, Sorensen OE, Kampmann B, Griffiths CJ, Wilkinson RJ. IFN-gamma- and TNF-independent vitamin D-inducible human suppression of mycobacteria: the role of cathelicidin LL-37. J Immunol. 2007 Jun 1;178(11):7190-8. doi: 10.4049/jimmunol.178.11.7190.

    PubMed 17513768

  • Rockett KA, Brookes R, Udalova I, Vidal V, Hill AV, Kwiatkowski D. 1,25-Dihydroxyvitamin D3 induces nitric oxide synthase and suppresses growth of Mycobacterium tuberculosis in a human macrophage-like cell line. Infect Immun. 1998 Nov;66(11):5314-21. doi: 10.1128/IAI.66.11.5314-5321.1998.

    PubMed 9784538

  • Martineau AR, Timms PM, Bothamley GH, Hanifa Y, Islam K, Claxton AP, Packe GE, Moore-Gillon JC, Darmalingam M, Davidson RN, Milburn HJ, Baker LV, Barker RD, Woodward NJ, Venton TR, Barnes KE, Mullett CJ, Coussens AK, Rutterford CM, Mein CA, Davies GR, Wilkinson RJ, Nikolayevskyy V, Drobniewski FA, Eldridge SM, Griffiths CJ. High-dose vitamin D(3) during intensive-phase antimicrobial treatment of pulmonary tuberculosis: a double-blind randomised controlled trial. Lancet. 2011 Jan 15;377(9761):242-50. doi: 10.1016/S0140-6736(10)61889-2. Epub 2011 Jan 5.

    PubMed 21215445

  • Liu PT, Stenger S, Li H, Wenzel L, Tan BH, Krutzik SR, Ochoa MT, Schauber J, Wu K, Meinken C, Kamen DL, Wagner M, Bals R, Steinmeyer A, Zugel U, Gallo RL, Eisenberg D, Hewison M, Hollis BW, Adams JS, Bloom BR, Modlin RL. Toll-like receptor triggering of a vitamin D-mediated human antimicrobial response. Science. 2006 Mar 24;311(5768):1770-3. doi: 10.1126/science.1123933. Epub 2006 Feb 23.

    PubMed 16497887

  • 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, Frechet-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-Laborin 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.

    PubMed 30215381

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Vitamin D3 and Traditional treatment of CGD and TB in people with Vitamin D3 and Chronic-granulomatous Disease, run by Children's Hospital of Fudan University at 2 sites in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 9 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Mortality (time frame: 8 weeks). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03984890, which also lists 5 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Jul 2024.

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