Sickle Cell DiseasePhase 3NCT03417947
Vitamin D Supplementation in Children With Sickle Cell Disease
A Phase 3 study testing Vitamin D bolus in people with Sickle Cell Disease, run by St. Justine's Hospital at 1 site in Canada.
Trial 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. Read more about this study
- Who is it for?
- Ages 5 to 17 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Vitamin D bolus
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 42 participants
- Where
- 1 siteCanada
- Study dates
- 30 Nov 2018to 30 Sep 2019
- Phase
- Phase 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 5 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Children aged between 5 and 17 years old who are followed up at the SCD Clinic, CHU Sainte-Justine, Montreal, Canada.
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Vitamin D Intervention in Children With Sickle Cell Disease: A Pilot Randomized Controlled Trial
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.
Read the 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
- Being tested
- Vitamin D bolus, Placebo
- Keywords
- Bolus supplementation, Pilot randomised controlled trial, Vitamin D
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Vitamin D bolusDietary supplement
Also called Cholecalciferol
One single oral liquid vitamin D3 supplement of 300 000 IU
PlaceboDietary supplement
Placebo identical in taste and appearance to the vitamin D bolus
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | 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. Receives: Dietary Supplement: Placebo |
| Vitamin D bolus | Experimental | 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. Receives: Dietary Supplement: Vitamin D bolus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean change in total serum 25-hydroxyvitamin D levels
Group difference in the mean change in total serum 25OHD from baseline to 3 months.
Measured: 3 months
Secondary outcomes (1)
Vitamin D sufficiency
Difference in the proportion of children with serum 25-hydroxyvitamin D ≥75nmol/L at 3 months
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jan 2018
- Study start30 Nov 2018
- Primary completion30 Sep 2019
- Study completion30 Sep 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 3
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- This is a randomised, quadruble-blind, placebo-controlled, parallel-group trial of vitamin D3 bolus supplementation.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 42 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Justine's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Euro-Pharm
- Responsible party
- Principal investigator, Genevieve Mailhot, St. Justine's Hospital
- Investigators
- Genevieve Mailhot, PhD, Principal investigator, St. Justine's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03417947
- Sponsor's protocol ID
- ND
- Status last verified
- Mar 2020
- First submitted
- 11 Jan 2018
- Last updated
- 23 Mar 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
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.
PubMed 32462740
Study at a glance
From the registry record, last updated by the sponsor on 23 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Vitamin D Intervention in Children With Sickle Cell Disease: A Pilot Randomized Controlled Trial
- ClinicalTrials.gov ID
- NCT03417947
- Acronym / sponsor ID
- ND
- Conditions
- Sickle Cell Disease
- Keywords
- Bolus supplementation, Pilot randomised controlled trial, Vitamin D
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 42 participants (actual)
- Lead sponsor
- St. Justine's HospitalUniversity, hospital or other
- Collaborators
- Euro-Pharm
- Study start
- 30 Nov 2018 (actual)
- Primary completion
- 30 Sep 2019 (actual)
- Study completion
- 30 Sep 2019 (actual)
- First posted
- 31 Jan 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- This is a randomised, quadruble-blind, placebo-controlled, parallel-group trial of vitamin D3 bolus supplementation.
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Dietary Supplement: PlaceboPlacebo 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. |
| Vitamin D bolus | Experimental | Dietary Supplement: Vitamin D bolusThe 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. |
Who may qualify
- Ages
- 5 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- Children aged between 5 and 17 years old who are followed up at the SCD Clinic, CHU Sainte-Justine, Montreal, Canada.
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Vitamin D bolusAlso: CholecalciferolOne single oral liquid vitamin D3 supplement of 300 000 IU | Dietary supplement | Vitamin D bolus |
| PlaceboPlacebo identical in taste and appearance to the vitamin D bolus | Dietary supplement | Placebo |
Primary outcome
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.
1 secondary outcome
Vitamin D sufficiency
Time frame: 3 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Mar 2020.
- Derived from this study: PubMed 32462740Soe 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.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Genevieve Mailhot, PhD principal investigator
St. Justine's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Vitamin D bolus in people with Sickle Cell Disease, run by St. Justine's Hospital at 1 site in Canada.
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 1 inclusion and 9 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 23 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
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?
Mean change in total serum 25-hydroxyvitamin D levels (time frame: 3 months). 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 NCT03417947, which also lists 1 linked publication.
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 23 Mar 2020.