Vitamin D Supplementation in Children With Sickle Cell Disease

NCT ID: NCT03417947

Last Updated: 2020-03-23

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 42 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-11-30
• Study Completion Date: 2019-09-30

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).

Conditions

Sickle Cell Disease

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: OTHER
• Blinding Strategy: QUADRUPLE

Study Groups

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.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DIETARY_SUPPLEMENT

Placebo identical in taste and appearance to the vitamin D bolus

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.

Group Type: EXPERIMENTAL

Vitamin D bolus

Intervention Type: DIETARY_SUPPLEMENT

One single oral liquid vitamin D3 supplement of 300 000 IU

Interventions

Vitamin D bolus

One single oral liquid vitamin D3 supplement of 300 000 IU

Intervention Type: DIETARY_SUPPLEMENT

Placebo

Placebo identical in taste and appearance to the vitamin D bolus

Intervention Type: DIETARY_SUPPLEMENT

Other Intervention Names

Cholecalciferol

Eligibility Criteria

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

• Minimum Eligible Age: 5 Years
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Euro-Pharm

UNKNOWN

Sponsor Role: collaborator

St. Justine's Hospital

OTHER

Sponsor Role: lead

Responsible Party

Genevieve Mailhot

Researcher

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Genevieve Mailhot, PhD

Role: PRINCIPAL_INVESTIGATOR

St. Justine's Hospital

Locations

CHU Sainte-Justine

Montreal, Quebec, Canada

Countries

Canada

References

Soe HHK, Abas AB, Than NN, Ni H, Singh J, Said ARBM, Osunkwo I. Vitamin D supplementation for sickle cell disease. Cochrane Database Syst Rev. 2020 May 28;5(5):CD010858. doi: 10.1002/14651858.CD010858.pub3.

Reference Type: DERIVED

PMID: 32462740 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Other Identifiers

ND

Identifier Type: -

Identifier Source: org_study_id