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Children with Cystic Fibrosis, ages greater than and equal to 36 months of age and less than or equal to 18 years of age, with a 25OHD level less than 30 ng/dL will be asked to participate in this study. Upon consent, they will be given oral cholecalciferol supplementation of 250,000 IU during their next CF clinic visit. The safety will be assessed by measuring a serum calcium level within 1 week of supplementation. Efficacy will be assessed using repeated 25OHD levels throughout the course of 12 months. Feasibility will be assessed with the previous two measures as well as a brief questionnaire administered via telephone within 1 week of supplementation.
Children and adults with Cystic Fibrosis (CF) are at risk of developing a vitamin D deficiency or insufficiency, defined as a 2,25-hydroxyvitamin D (25OHD) serum blood level <30 ng/dL. Greater than 85% of people with CF have pancreatic insufficiency, which contributes to poor absorption of fat soluble vitamins in addition to dietary fat. A 25OHD level greater than 30 ng/dL has been described as providing potential improvements to markers of inflammation in adults with CF and is known to improve bone mineral density and prevent bone fractures in all populations, including CF. This study will assess the safety of a one-time high dose of cholecalciferol or vitamin D3 along with the efficacy and feasibility.
Children between the ages of 3 years and 18 years (inclusive), with a 25OHD level <30 ng/dL will be provided with a vitamin D3 supplement of 250,000 international units (IU) observed in our CF clinic. We hypothesize that this one-time, oral, high dose of vitamin D3 will safely and effectively raise the 25OHD level to above 30 ng/dL.
Safety will be monitored with serum calcium levels 1 week following the dosage, since 25OHD has a half-life of 2-3 weeks and this serum calcium level measurement time-point would be in or near the middle of this range. Feasibility will be measured using a 5-question phone survey 1 week following the dosage (see appendix A - attached). Efficacy will be measured by the 25OHD level itself; if 25OHD levels are found to be between 30-100 ng/dL over the course of the study, the dose will have demonstrated effective in achieving the study's goal.
The purpose of this study is to show that 25OHD levels can be safely corrected with a one-time dose of vitamin D3 that can be safely and feasibly provided in the outpatient setting to children with CF.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment | Experimental | All patients with a 25OHD level <30 ng/dL will be given 250,000 IU D3 (cholecalciferol) orally at one point in time and during CF clinic. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Treatment | Drug |
|
| Measure | Description | Time Frame |
|---|---|---|
| Safety of a Single High-dose of Oral Cholecalciferol to Treat a Vitamin D Deficiency in Children With Cystic Fibrosis | The safety of a single high-dose of oral cholecalciferol will be assessed using a serum calcium measurement after administration of treatment. Instances of hypercalcemia will be assessed at 1 week and at 3 months post-treatment. The treatment will be considered to be safe if the serum calcium level does not exceed 14 mg/dL. | 1 week, 3 months |
| Measure | Description | Time Frame |
|---|---|---|
| Efficacy of a Single High-dose of Oral Cholecalciferol in Treating a Vitamin D Insufficiency/Deficiency in Children With Cystic Fibrosis | The efficacy of this treatment will be assessed by the 25OHD level measured after treatment at 3 months, 6 months, and 12 months. The treatment will be considered to be efficacious if the 25OHD level is greater than or equal to 30 ng/dL. | 3 months, 6 months and 12 months |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Deanna Green, MD | Johns Hopkins All Children's Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Johns Hopkins All Children's Hospital | St. Petersburg | Florida | 33701 | United States |
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Pediatric patients with Cystic fibrosis (CF) and greater than or equal to 36 months of age with a serum/blood 25OHD level < 30 ng/mL were recruited to participate in the study.
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| ID | Title | Description |
|---|---|---|
| FG000 | Treatment With High-Dose D3 | All patients with a 25OHD level <30 ng/dL will be given 250,000 IU D3 (cholecalciferol) orally at one point in time and during CF clinic. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Treatment | All patients with a 25OHD level <30 ng/dL & given 250,000 IU D3 (cholecalciferol). |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| 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 | Safety of a Single High-dose of Oral Cholecalciferol to Treat a Vitamin D Deficiency in Children With Cystic Fibrosis | The safety of a single high-dose of oral cholecalciferol will be assessed using a serum calcium measurement after administration of treatment. Instances of hypercalcemia will be assessed at 1 week and at 3 months post-treatment. The treatment will be considered to be safe if the serum calcium level does not exceed 14 mg/dL. | Posted | Mean | Standard Deviation | mg/dL | 1 week, 3 months |
|
|
12 months
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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 | Treatment | All patients with a 25OHD level <30 ng/dL & given 250,000 IU D3 (cholecalciferol). |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Lung infection | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhea | Gastrointestinal disorders | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Vanessa Millovich | Kate Farms | 3522634409 | vannanole@gmail.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Dec 20, 2016 | Dec 28, 2020 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D014808 | Vitamin D Deficiency |
| ID | Term |
|---|---|
| D001361 | Avitaminosis |
| D003677 | Deficiency Diseases |
| D044342 | Malnutrition |
| D009748 | Nutrition Disorders |
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| ID | Term |
|---|---|
| D013812 | Therapeutics |
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| Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire | CF patients with a 25OHD level <30 ng/dL who were given 250,000 IU D3. Feasibility will be measured using a 5-item Yes/No questionnaire where item 1 contains 5 sub-item yes/no questions which all relate to the experience of gastrointestinal symptoms. This was administered via telephone 1-week after administration of the treatment. | 1 week |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | Participants |
|
| 25-hydroxycholecalciferol (25OHD) level | The normal range for 25OHD is greater than or equal to 30 ng/mL. | Mean | Standard Deviation | ng/mL |
|
| Pancreatic Sufficient | Pancreatic sufficiency is defined as a patient having enough exocrine pancreatic function to allow normal digestion without pancreatic enzyme supplementation. | Count of Participants | Participants |
|
| Taking additional vitamin D at enrollment | Count of Participants | Participants |
|
| Body Mass Index (BMI) | Mean | Standard Deviation | kg/m^2 |
|
| BMI Percentile | Mean | Standard Deviation | percentile |
|
| Counts |
|---|
| Participants |
|
|
| Secondary | Efficacy of a Single High-dose of Oral Cholecalciferol in Treating a Vitamin D Insufficiency/Deficiency in Children With Cystic Fibrosis | The efficacy of this treatment will be assessed by the 25OHD level measured after treatment at 3 months, 6 months, and 12 months. The treatment will be considered to be efficacious if the 25OHD level is greater than or equal to 30 ng/dL. | 26 participants were enrolled and given the treatment (250,000 IU D3) and assessed for the primary outcome measure. At the 3-month outcome measure, one participant had passed away, so 25 participants were included in the analysis. At the 12-month outcome measure, one additional participant was lost to follow-up, so 24 participants were included for analysis. | Posted | Mean | Inter-Quartile Range | ng/dL | 3 months, 6 months and 12 months |
|
|
|
| Secondary | Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire | CF patients with a 25OHD level <30 ng/dL who were given 250,000 IU D3. Feasibility will be measured using a 5-item Yes/No questionnaire where item 1 contains 5 sub-item yes/no questions which all relate to the experience of gastrointestinal symptoms. This was administered via telephone 1-week after administration of the treatment. | Posted | Count of Participants | Participants | 1 week |
|
|
|
| 1 |
| 26 |
| 4 |
| 26 |
| 15 |
| 26 |
| Decreased lung function and weight loss | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Bronchopneumonia exacerbation | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Stomach ache | Gastrointestinal disorders | Systematic Assessment |
|
| Increased burping | Gastrointestinal disorders | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | Systematic Assessment |
|
| Heart burn/Reflux | Gastrointestinal disorders | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | Systematic Assessment |
|
| Lung infection | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Hypoxemia | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Sinus Infection | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Increased cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Elevated phosphorus level | General disorders | Systematic Assessment |
|
| Elevated PTH | General disorders | Systematic Assessment |
|
| Elevated calcium level | General disorders | Systematic Assessment |
|
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| D009750 |
| Nutritional and Metabolic Diseases |
|
| 25OHD level at 12 months |
|
|
| Yes |
|
| Increased amount of diarrhea? |
|
| Any constipation? |
|
| Increased gas production, such as burping or passing gas? |
|
| Increased amount of abdominal pain/stomach aches? |
|
| Incred in heart burn or reflux? |
|
| Easy to take? |
|
| Something you would do next year if you had another low vitamin D level? |
|
| Prefer taking a one-time dose of vitamin D instead of a daily vitamin D? |
|