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Vitamin D Supplementation in Children With Sickle Cell Disease

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ClinicalTrials.gov Identifier: NCT03417947
Recruitment Status : Recruiting
First Posted : January 31, 2018
Last Update Posted : April 4, 2019
Sponsor:
Collaborator:
Euro-Pharm
Information provided by (Responsible Party):
Genevieve Mailhot, St. Justine's Hospital

Tracking Information
First Submitted Date  ICMJE January 11, 2018
First Posted Date  ICMJE January 31, 2018
Last Update Posted Date April 4, 2019
Actual Study Start Date  ICMJE November 30, 2018
Estimated Primary Completion Date October 31, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 30, 2018)
Mean change in total serum 25-hydroxyvitamin D levels [ Time Frame: 3 months ]
Group difference in the mean change in total serum 25OHD from baseline to 3 months.
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03417947 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: January 30, 2018)
Vitamin D sufficiency [ Time Frame: 3 months ]
Difference in the proportion of children with serum 25-hydroxyvitamin D ≥75nmol/L at 3 months
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures
 (submitted: January 30, 2018)
  • Hypercalciuria [ Time Frame: 7 days post-intervention ]
    Number of patients with urinary calcium to creatinine ratio above normal reference range for age
  • Hypercalcemia [ Time Frame: 3 months ]
    Number of patients with serum calcium above normal reference range for age
  • Serum 25-hydroxyvitamin D levels [ Time Frame: 3 months ]
    Number of patients with serum 25-hydroxyvitamin D levels >250 nmol/L
  • Mean change in weight [ Time Frame: 3 months ]
    Group difference in the mean change of weight (kg) from baseline to 3 months.
  • Mean change in height [ Time Frame: 3 months ]
    Group difference in the mean change of height (kg) from baseline to 3 months.
  • Mean change in hemoglobin [ Time Frame: 3 months ]
    Group difference in the mean change of circulating hemoglobin from baseline to 3 months.
  • Mean change in fetal hemoglobin [ Time Frame: 3 months ]
    Group difference in the mean change of circulating fetal hemoglobin from baseline to 3 months.
  • Mean change in leucocyte counts [ Time Frame: 3 months ]
    Group difference in the mean change of blood leucocyte counts from baseline to 3 months.
  • Mean change in platelet counts [ Time Frame: 3 months ]
    Group difference in the mean change of blood platelet counts from baseline to 3 months.
  • Mean change in reticulocyte counts [ Time Frame: 3 months ]
    Group difference in the mean change of blood reticulocyte counts from baseline to 3 months
  • Mean change in neutrophil counts [ Time Frame: 3 months ]
    Group difference in the mean change of blood neutrophil counts from baseline to 3 months
  • Mean change in mean corpuscular volume [ Time Frame: 3 months ]
    Group difference in the mean change of blood mean corpuscular volume from baseline to 3 months
  • Mean change in serum creatinine [ Time Frame: 3 months ]
    Group difference in the mean change of serum creatinine from baseline to 3 months.
  • Mean change in serum bilirubin [ Time Frame: 3 months ]
    Group difference in mean change of serum bilirubin from baseline to 3 months.
  • Mean change in serum parathyroid hormone [ Time Frame: 3 months ]
    Group difference in mean change of serum parathyroid hormone from baseline to 3 months
  • Mean change in serum P1NP [ Time Frame: 3 months ]
    Group difference in mean change of serum amino-terminal propeptide of type I collagen (P1NP) from baseline to 3 months
  • Mean change in serum C-telopeptides [ Time Frame: 3 months ]
    Group difference in mean change of serum C-telopeptides from baseline to 3 months
  • Mean change in musculoskeletal pain scores [ Time Frame: 3 months ]
    Musculoskeletal pain will be assessed with the Brief Pain Inventory (BPI). Group difference in the mean change in BPI scores.
  • Mean change in quality of life scores [ Time Frame: 3 months ]
    Health-related quality of life will be assessed through the Pediatric Quality of life (PedQoL) inventory. Group difference in the mean change in PedQoL scores.
  • Sickle cell disease-related complications [ Time Frame: 3 months ]
    Occurrence of sickle cell disease complications affecting bone, the kidneys, the retina, blood vessels, the heart, the lungs, the spleen, the liver and gallbladder during the study period
  • Participant recruitment [ Time Frame: 3 months ]
    Percentage of patients recruited from those screened
  • Participant retention [ Time Frame: 3 months ]
    Percentage of patients retained for the entire study duration
  • Participant compliance [ Time Frame: 3 months ]
    Percentage of patients who comply with the study protocol
Original Other Pre-specified Outcome Measures Same as current
 
Descriptive Information
Brief Title  ICMJE Vitamin D Supplementation in Children With Sickle Cell Disease
Official Title  ICMJE Vitamin D Intervention in Children With Sickle Cell Disease: A Pilot Randomized Controlled Trial
Brief Summary Sickle cell disease (SCD) is a genetic disease characterized by abnormal hemoglobin, the main constituent of red blood cells. People with SCD have nutritional deficiencies, and vitamin D deficiency is one of the most common. Symptoms of vitamin D deficiency are similar to those of SCD and include chronic pain and bone complications. Correcting vitamin D nutrition of children with SCD represents a treatment that will improve their health. A single oral high-dose of vitamin D3 will be given to SCD children during one of their follow-up visits at the SCD clinic of CHU Sainte-Justine, Montreal, Canada. This mode of administration was chosen to ensure a better adherence to the treatment. The investigators will determine whether this dose is safe and its administration feasible in clinic. The impact of this dose on blood vitamin D and calcium, urinary calcium, growth, inflammation, bone health, pain and quality of life will also be assessed. This study intends to propose a new intervention to improve the nutrition of children with this disease.
Detailed Description Vitamin D deficiency is one of the most common nutritional conditions among patients with sickle cell disease (SCD). Since vitamin D deficiency and SCD share common manifestations including chronic pain, poor bone health and chronic systemic inflammation, it is reasonable to postulate that vitamin D deficiency may contribute to these complications. Thus, optimizing vitamin D nutrition represents an inexpensive strategy that may improve vitamin D status and health outcomes in SCD children. The working hypothesis is that administration of a single oral bolus of 300,000 IU of vitamin D3 to SCD children will result in the attainment of vitamin D sufficiency (25OHD levels >75 nmol/L) in 80% of participants after 3 months. The primary objectives are to assess feasibility, acceptability, and safety of the vitamin D3 bolus while secondary objectives are related to the mean change in serum 25OHD from baseline to 3 months post-bolus and its clinical impact. Seventy-two SCD children (5-17 years, SS and SC genotypes) will be randomized to one bolus of 300,000 IU of vitamin D3 or identical placebo. Blood will be collected at baseline and 3-month post-bolus to measure serum 25OHD and calculate the change from baseline at 3 months (efficacy outcomes). Other outcomes include urinary calcium/creatinine ratio and serum calcium (safety), questionnaires (acceptability and musculoskeletal pain) and parameters related to growth, haematology, inflammation and bone health (exploratory outcomes).
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:
This is a randomised, quadruble-blind, placebo-controlled, parallel-group trial of vitamin D3 bolus supplementation.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description:

The Applied Clinical Research Unit of Sainte-Justine UHC will generate the randomisation scheme. Group allocation codes will be held in a secure location with a restricted access by the Central pharmacy (Sainte-Justine UHC). All participants and research personnel, including the nurse, research trainee and research team will be blinded to group assignment.

The supplier Euro-Pharm will provide the placebo and vitamin D3 preparations in coded bottles. Pharmacy will prepare the 6-mL bolus in coded syringes following the randomisation scheme.

Primary Purpose: Other
Condition  ICMJE Sickle Cell Disease
Intervention  ICMJE
  • Dietary Supplement: Vitamin D bolus
    One single oral liquid vitamin D3 supplement of 300 000 IU
    Other Name: Cholecalciferol
  • Dietary Supplement: Placebo
    Placebo identical in taste and appearance to the vitamin D bolus
Study Arms  ICMJE
  • Placebo Comparator: Placebo
    Placebo identical to the vitamin D bolus in taste and appearance. The placebo will be administered once, at the beginning of the study. The oral liquid placebo will be prepared at the Pharmacy in coded syringes and will be administered to the participants by a nurse at the sickle cell disease Clinic.
    Intervention: Dietary Supplement: Placebo
  • Experimental: Vitamin D bolus
    The vitamin D bolus is an oral liquid supplement that will be administered once, at the beginning of the study. The oral liquid vitamin D bolus will be prepared at the Pharmacy in coded syringes and will be administered to the participants by a nurse at the sickle cell disease Clinic.The dose of vitamin D3 contained in the bolus is 300 000 IU.
    Intervention: Dietary Supplement: Vitamin D bolus
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: January 30, 2018)
72
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE October 31, 2019
Estimated Primary Completion Date October 31, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Children aged between 5 and 17 years old who are followed up at the SCD Clinic, CHU Sainte-Justine, Montreal, Canada.

Exclusion Criteria:

  • Conditions or use of medications known to interfere with calcium or vitamin D absorption or metabolism
  • Known hypercalcemia
  • Conditions characterized by a hypersensitivity to vitamin D (e.g. granulomatous disorders)
  • Patients clinically diagnosed with rickets or other conditions requiring vitamin D therapy
  • History or presence of urolithiasis
  • Anticipated difficult follow up
  • Patients already enrolled in other investigational studies
  • Patients who have recently been hospitalized for severe pain crisis or acute sickle complication in the past 2 weeks
  • Patients with unresolved pain issues
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 5 Years to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Genevieve Mailhot, PhD 514-345-4931 ext 6200 genevieve.mailhot@umontreal.ca
Contact: Nathalie Alos, MD 514-345-4931 ext 2993 nathalie.alos@umontreal.ca
Listed Location Countries  ICMJE Canada
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03417947
Other Study ID Numbers  ICMJE ND
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party Genevieve Mailhot, St. Justine's Hospital
Study Sponsor  ICMJE St. Justine's Hospital
Collaborators  ICMJE Euro-Pharm
Investigators  ICMJE
Principal Investigator: Genevieve Mailhot, PhD St. Justine's Hospital
PRS Account St. Justine's Hospital
Verification Date April 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP