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| ID | Type | Description | Link |
|---|---|---|---|
| R01FD003894 | U.S. FDA Grant/Contract | View source |
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This study aims to answer the question whether oral vitamin D supplementation can decrease lung complications in children and adolescents with sickle cell disease. Lung complications are the leading causes of morbidity and of death in sickle cell disease. Infections and increased inflammation play important roles in the development of the lung problems in sickle cell disease. Emerging evidence shows that vitamin D helps the immune system to fight infection and to control inflammation and could potentially help prevent respiratory complications in patients with sickle cell disease. The investigators hypothesize that oral vitamin D3, 100,000 IU (2.5 mg), given once a month to a group of children and adolescents with sickle cell disease, will reduce the rate of respiratory events (infection, asthma exacerbation and acute chest syndrome) compared to the rate in a group given standard dose oral vitamin D3, 12,000 IU (0.3 mg) given once a month.
Funding Source - U.S. Food & Drug Administration, Office of Orphan Products Development
This study will be a Phase 2 double-blind randomized clinical trial in 80 patients with sickle cell disease, ages 3 to 20 years-old, comparing a 2-year monthly oral dose of vitamin D3, 100,000 IU (equivalent to 3,300 IU/day) to a standard monthly dose, 12,000 IU (400 IU/day) in reducing the rate of respiratory events (defined as respiratory infections, acute asthma exacerbation, and the acute chest syndrome) in children and adolescents with sickle cell disease in comparison with the rates of respiratory events over a baseline period of one year.
Eligible participants (130 patients) will initially be screened to determine their blood vitamin D levels (serum 25-hydroxyvitamin D). Those with 25-hydroxyvitamin D levels between 5 and 60 ng/mL will be eligible for randomization. At study entry, blood and urine samples will be collected for routine and special blood tests including tests on immune function, inflammation, and bone function. Children above 5 years old will also have lung function and muscle strength tests. Participants will be followed once a month to administer the study medication (oral vitamin D3) and to monitor any side effects from the study medication by history, examination and blood and urine tests. After 12 and 24 months of therapy, the same study procedures at study entry will be repeated.
This study could help establish oral vitamin D3 as a simple, low cost treatment to reduce respiratory complications in children and adolescents with sickle cell disease.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Vitamin D3 100,000 IU | Experimental | Oral vitamin D3, 100,000 IU [2.5 mg] given once a month |
|
| Vitamin D3 12,000 IU | Active Comparator | Standard dose oral vitamin D3 12,000 IU [0.3 mg] given once a month |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Experimental: Vitamin D3 100,000 IU | Drug | Oral vitamin D3, 100,000 IU [2.5 mg] given once a month |
|
| Measure | Description | Time Frame |
|---|---|---|
| Mean Annual Rate of Respiratory Events | Defined as respiratory infection, acute asthma exacerbation, and acute chest syndrome. | Up to 2 years |
| Measure | Description | Time Frame |
|---|---|---|
| Mean 25-Hydroxyvitamin D (25-OHD) | The overall mean serum 25-OHD concentration will be measured for both groups. | 2 years |
| Forced Vital Capacity (FVC) | Percent predicted forced vital capacity will be calculated for both groups. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Gary Brittenham, MD | Columbia University | Principal Investigator |
| Margaret T. Lee, MD | Columbia University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Columbia University Irving Medical Center | New York | New York | 10032 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 29712666 | Derived | Lee MT, Kattan M, Fennoy I, Arpadi SM, Miller RL, Cremers S, McMahon DJ, Nieves JW, Brittenham GM. Randomized phase 2 trial of monthly vitamin D to prevent respiratory complications in children with sickle cell disease. Blood Adv. 2018 May 8;2(9):969-978. doi: 10.1182/bloodadvances.2017013979. |
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Only participants who were randomized (62) were included in the analysis of data.
Participants were consented and then randomized. Only individuals who were randomized were included in the data analysis, so demographics are only available for the 62 randomized.
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| ID | Title | Description |
|---|---|---|
| FG000 | Vitamin D3 100,000 IU | Oral vitamin D3, 100,000 IU [2.5 mg] given once a month Experimental: Vitamin D3 100,000 IU: Oral vitamin D3, 100,000 IU [2.5 mg] given once a month |
| FG001 | Vitamin D3 12,000 IU | Standard dose oral vitamin D3 12,000 IU [0.3 mg] given once a month Active Comparator: Vitamin D3 12,000 IU: Standard dose oral vitamin D3 12,000 IU [0.3 mg] given once a month |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Vitamin D3 100,000 IU | Oral vitamin D3, 100,000 IU [2.5 mg] given once a month Experimental: Vitamin D3 100,000 IU: Oral vitamin D3, 100,000 IU [2.5 mg] given once a month |
| BG001 | Vitamin D3 12,000 IU |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Mean Annual Rate of Respiratory Events | Defined as respiratory infection, acute asthma exacerbation, and acute chest syndrome. | Posted | Mean | Standard Deviation | events per year | Up to 2 years |
|
Up to 2 years
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Vitamin D3 100,000 IU | Oral vitamin D3, 100,000 IU [2.5 mg] given once a month Experimental: Vitamin D3 100,000 IU: Oral vitamin D3, 100,000 IU [2.5 mg] given once a month |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Acute Chest Syndrome | General disorders | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anemia | Blood and lymphatic system disorders | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Gary Brittenham, MD | Columbia University | 212-305-7005 | gmb31@cumc.columbia.edu |
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| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| D014808 | Vitamin D Deficiency |
| D056586 | Acute Chest Syndrome |
| D001249 | Asthma |
| D012141 | Respiratory Tract Infections |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
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| ID | Term |
|---|---|
| D002762 | Cholecalciferol |
| ID | Term |
|---|---|
| D002782 | Cholestenes |
| D002776 | Cholestanes |
| D013256 | Steroids |
| D000072473 | Fused-Ring Compounds |
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| Active Comparator: Vitamin D3 12,000 IU | Drug | Standard dose oral vitamin D3 12,000 IU [0.3 mg] given once a month |
|
|
| Up to 2 years |
| Forced Expiratory Volume (FEV) in 1 Second (FEV1) | Percent predicted forced expiratory volume in 1 second will be calculated for both groups. | Up to 2 years |
| FEV1/FVC Ratio | The ratio of FEV1 to FVC will be calculated (in percentage). | Up to 2 years |
| FEF 25-75 | Percent predicted forced expiratory flow (FEF) during expiration of 25 to 75% of the FVC will be calculated. | Up to 2 years |
| RV/TLC Ratio | The ratio of residual volume (RV) to total lung capacity (TLC) will be calculated (in percentage). | Up to 2 years |
| DLCO | Percent predicted of the diffusing capacity for carbon monoxide in the lungs (DLCO) will be measured. | Up to 2 years |
| FeNO | Fractional exhaled nitric oxide (FeNO) will be measured in parts per billion (ppb). | Up to 2 years |
| MIP | Maximum inspiratory pressure (MIP) will be measured. | Up to 2 years |
| MEP | Maximum expiratory pressure (MEP) will be measured. | Up to 2 years |
| Hand-grip, Right | Muscle strength was measured by looking at hand-grip strength in the right hand. | Up to 2 years |
| Hand-grip, Left | Muscle strength was measured by looking at hand-grip strength in the left hand. | Up to 2 years |
| Hand-grip, Dominant | Muscle strength was measured by looking at hand-grip strength in the dominant hand. | Up to 2 years |
| Adverse Event |
|
Standard dose oral vitamin D3 12,000 IU [0.3 mg] given once a month
Active Comparator: Vitamin D3 12,000 IU: Standard dose oral vitamin D3 12,000 IU [0.3 mg] given once a month
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Sickle-cell Type | Count of Participants | Participants |
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Secondary | Mean 25-Hydroxyvitamin D (25-OHD) | The overall mean serum 25-OHD concentration will be measured for both groups. | Posted | Mean | Full Range | ng/mL | 2 years |
|
|
|
| Secondary | Forced Vital Capacity (FVC) | Percent predicted forced vital capacity will be calculated for both groups. | Only includes participants who completed the assessment. | Posted | Mean | Standard Deviation | Percentage of predicted FVC | Up to 2 years |
|
|
|
| Secondary | Forced Expiratory Volume (FEV) in 1 Second (FEV1) | Percent predicted forced expiratory volume in 1 second will be calculated for both groups. | Only includes participants who completed the assessment. | Posted | Mean | Standard Deviation | Percentage of predicted FEV1 | Up to 2 years |
|
|
|
| Secondary | FEV1/FVC Ratio | The ratio of FEV1 to FVC will be calculated (in percentage). | Only includes participants who completed the assessment. | Posted | Mean | Standard Deviation | ratio | Up to 2 years |
|
|
|
| Secondary | FEF 25-75 | Percent predicted forced expiratory flow (FEF) during expiration of 25 to 75% of the FVC will be calculated. | Only includes participants who completed the assessment. | Posted | Mean | Standard Deviation | Percentage of predicted FEF | Up to 2 years |
|
|
|
| Secondary | RV/TLC Ratio | The ratio of residual volume (RV) to total lung capacity (TLC) will be calculated (in percentage). | Only includes participants who completed the assessment. | Posted | Mean | Standard Deviation | Percent ratio | Up to 2 years |
|
|
|
| Secondary | DLCO | Percent predicted of the diffusing capacity for carbon monoxide in the lungs (DLCO) will be measured. | Only includes participants who completed the assessment. | Posted | Mean | Standard Deviation | Percentage predicted DLCO | Up to 2 years |
|
|
|
| Secondary | FeNO | Fractional exhaled nitric oxide (FeNO) will be measured in parts per billion (ppb). | Only includes participants who completed the assessment. | Posted | Mean | Standard Deviation | parts per billion | Up to 2 years |
|
|
|
| Secondary | MIP | Maximum inspiratory pressure (MIP) will be measured. | Only includes participants who completed the assessment. | Posted | Mean | Standard Deviation | cm of water | Up to 2 years |
|
|
|
| Secondary | MEP | Maximum expiratory pressure (MEP) will be measured. | Only includes participants who completed the assessment. | Posted | Mean | Standard Deviation | cm of water | Up to 2 years |
|
|
|
| Secondary | Hand-grip, Right | Muscle strength was measured by looking at hand-grip strength in the right hand. | Only includes participants who completed the assessment. | Posted | Mean | Standard Deviation | pounds of force | Up to 2 years |
|
|
|
| Secondary | Hand-grip, Left | Muscle strength was measured by looking at hand-grip strength in the left hand. | Only includes participants who completed the assessment. | Posted | Mean | Standard Deviation | pounds of force | Up to 2 years |
|
|
|
| Secondary | Hand-grip, Dominant | Muscle strength was measured by looking at hand-grip strength in the dominant hand. | Only includes participants who completed the assessment. | Posted | Mean | Standard Deviation | pounds of force | Up to 2 years |
|
|
|
| 11 |
| 31 |
| 26 |
| 31 |
| EG001 | Vitamin D3 12,000 IU | Standard dose oral vitamin D3 12,000 IU [0.3 mg] given once a month Active Comparator: Vitamin D3 12,000 IU: Standard dose oral vitamin D3 12,000 IU [0.3 mg] given once a month | 6 | 31 | 24 | 31 |
| Pain Syndrome (Grade 3 & 4) | General disorders | Non-systematic Assessment |
|
| Mucosal Infection (Herpetic Gingivostomatitis) | Infections and infestations | Non-systematic Assessment |
|
| Stroke | Nervous system disorders | Non-systematic Assessment |
|
| Cholecystitis | Gastrointestinal disorders | Non-systematic Assessment |
|
| Asthma Exacerbation | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
|
| Cholecystitis | Gastrointestinal disorders | Non-systematic Assessment |
|
| Hypoxemia | Blood and lymphatic system disorders | Non-systematic Assessment |
|
| Pneumonia | General disorders | Non-systematic Assessment |
|
| Sepsis | Blood and lymphatic system disorders | Non-systematic Assessment |
|
| Varicella Zoster | Infections and infestations | Non-systematic Assessment |
|
| Cervical Lymphadenitis | Blood and lymphatic system disorders | Non-systematic Assessment |
|
| Pain Syndrome (Grade 1 or 2) | General disorders | Non-systematic Assessment |
|
| Constipation | Gastrointestinal disorders | Non-systematic Assessment |
|
| Blood Bilirubin Increased | Blood and lymphatic system disorders | Non-systematic Assessment |
|
| Leukocytosis | Blood and lymphatic system disorders | Non-systematic Assessment |
|
| Acute Otitis Media | Infections and infestations | Non-systematic Assessment |
|
| Injury | Injury, poisoning and procedural complications | Non-systematic Assessment |
|
| Asthma Exacerbation | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
|
| Upper Respiratory Infection | Infections and infestations | Non-systematic Assessment |
|
| Streptococcal Pharyngitis | Infections and infestations | Non-systematic Assessment |
|
| Fever | General disorders | Non-systematic Assessment |
|
| Acute Chest Syndrome (Grade 1 & 2) | General disorders | Non-systematic Assessment |
|
| Influenza A/B | Infections and infestations | Non-systematic Assessment |
|
| Acute Pharyngitis | General disorders | Non-systematic Assessment |
|
| Seizure | General disorders | Non-systematic Assessment |
|
| Urinary Tract Infection | Renal and urinary disorders | Non-systematic Assessment |
|
| Headache | General disorders | Non-systematic Assessment |
|
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| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D001361 | Avitaminosis |
| D003677 | Deficiency Diseases |
| D044342 | Malnutrition |
| D009748 | Nutrition Disorders |
| D009750 | Nutritional and Metabolic Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D012120 | Respiration Disorders |
| D001982 | Bronchial Diseases |
| D008173 | Lung Diseases, Obstructive |
| D012130 | Respiratory Hypersensitivity |
| D006969 | Hypersensitivity, Immediate |
| D006967 | Hypersensitivity |
| D007154 | Immune System Diseases |
| D007239 | Infections |
| D011083 |
| Polycyclic Compounds |
| D013261 | Sterols |
| D014807 | Vitamin D |
| D012632 | Secosteroids |
| D008563 | Membrane Lipids |
| D008055 | Lipids |
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