Vitamin D Deficiency
Conditions
Brief summary
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.
Detailed description
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.
Interventions
DRUGTreatment
Sponsors
Johns Hopkins All Children's Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
36 Months to 18 Years
Healthy volunteers
Yes
Inclusion criteria
* Children with Cystic Fibrosis \>36 months of age * Serum/blood 25OHD level \< 30 ng/dL * Ability to provide valid informed consent to be a part of the study
Exclusion criteria
* Any history of kidney disease, kidney stones or on dialysis * Any history of hypercalcemia * Any history of hypercalciuria * Pregnancy at time of enrollment * Any history of parathyroid disorders * Inability to swallow pills by mouth
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety of a Single High-dose of Oral Cholecalciferol to Treat a Vitamin D Deficiency in Children With Cystic Fibrosis | 1 week, 3 months | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Efficacy of a Single High-dose of Oral Cholecalciferol in Treating a Vitamin D Insufficiency/Deficiency in Children With Cystic Fibrosis | 3 months, 6 months and 12 months | 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. |
| Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire | 1 week | 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. |
Countries
United States
Participant flow
Recruitment details
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.
Participants by arm
| Arm | Count |
|---|---|
| Treatment All patients with a 25OHD level \<30 ng/dL & given 250,000 IU D3 (cholecalciferol). | 26 |
| Total | 26 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Death | 1 |
| Overall Study | Lost to Follow-up | 1 |
Baseline characteristics
| Characteristic | Treatment |
|---|---|
| 25-hydroxycholecalciferol (25OHD) level | 22.69 ng/mL STANDARD_DEVIATION 4.75 |
| Age, Categorical <=18 years | 26 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Age, Continuous | 13.46 years STANDARD_DEVIATION 3.18 |
| BMI Percentile | 56.22 percentile STANDARD_DEVIATION 24.21 |
| Body Mass Index (BMI) | 20.09 kg/m^2 STANDARD_DEVIATION 3.25 |
| Pancreatic Sufficient No | 23 Participants |
| Pancreatic Sufficient Yes | 3 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 26 Participants |
| Race (NIH/OMB) White | 0 Participants |
| Region of Enrollment United States | 26 Participants |
| Sex: Female, Male Female | 15 Participants |
| Sex: Female, Male Male | 11 Participants |
| Taking additional vitamin D at enrollment No | 8 Participants |
| Taking additional vitamin D at enrollment Yes | 18 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 1 / 26 |
| other Total, other adverse events | 15 / 26 |
| serious Total, serious adverse events | 4 / 26 |
Outcome results
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.
Time frame: 1 week, 3 months
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Treatment With High-Dose D3 | Safety of a Single High-dose of Oral Cholecalciferol to Treat a Vitamin D Deficiency in Children With Cystic Fibrosis | Serum Calcium 1-week | 9.57 mg/dL | Standard Deviation 0.32 |
| Treatment With High-Dose D3 | Safety of a Single High-dose of Oral Cholecalciferol to Treat a Vitamin D Deficiency in Children With Cystic Fibrosis | Serum Calcium 3-month | 9.47 mg/dL | Standard Deviation 0.41 |
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.
Time frame: 3 months, 6 months and 12 months
Population: 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.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Treatment With High-Dose D3 | Efficacy of a Single High-dose of Oral Cholecalciferol in Treating a Vitamin D Insufficiency/Deficiency in Children With Cystic Fibrosis | 25OHD level at 3 months | 26 ng/dL |
| Treatment With High-Dose D3 | Efficacy of a Single High-dose of Oral Cholecalciferol in Treating a Vitamin D Insufficiency/Deficiency in Children With Cystic Fibrosis | 25OHD level at 6 months | 30 ng/dL |
| Treatment With High-Dose D3 | Efficacy of a Single High-dose of Oral Cholecalciferol in Treating a Vitamin D Insufficiency/Deficiency in Children With Cystic Fibrosis | 25OHD level at 12 months | 27 ng/dL |
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.
Time frame: 1 week
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Treatment With High-Dose D3 | Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire | Increased frequency of emesis (vomiting) | No | 26 Participants |
| Treatment With High-Dose D3 | Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire | Increased frequency of emesis (vomiting) | Yes | 0 Participants |
| Treatment With High-Dose D3 | Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire | Increased amount of diarrhea? | No | 23 Participants |
| Treatment With High-Dose D3 | Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire | Increased amount of diarrhea? | Yes | 3 Participants |
| Treatment With High-Dose D3 | Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire | Any constipation? | No | 25 Participants |
| Treatment With High-Dose D3 | Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire | Any constipation? | Yes | 1 Participants |
| Treatment With High-Dose D3 | Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire | Increased gas production, such as burping or passing gas? | No | 24 Participants |
| Treatment With High-Dose D3 | Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire | Increased amount of nausea | No | 24 Participants |
| Treatment With High-Dose D3 | Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire | Increased amount of nausea | Yes | 2 Participants |
| Treatment With High-Dose D3 | Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire | Increased gas production, such as burping or passing gas? | Yes | 2 Participants |
| Treatment With High-Dose D3 | Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire | Increased amount of abdominal pain/stomach aches? | No | 23 Participants |
| Treatment With High-Dose D3 | Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire | Increased amount of abdominal pain/stomach aches? | Yes | 3 Participants |
| Treatment With High-Dose D3 | Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire | Incred in heart burn or reflux? | No | 24 Participants |
| Treatment With High-Dose D3 | Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire | Incred in heart burn or reflux? | Yes | 2 Participants |
| Treatment With High-Dose D3 | Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire | Easy to take? | No | 0 Participants |
| Treatment With High-Dose D3 | Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire | Easy to take? | Yes | 26 Participants |
| Treatment With High-Dose D3 | Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire | Something you would do next year if you had another low vitamin D level? | No | 0 Participants |
| Treatment With High-Dose D3 | Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire | Something you would do next year if you had another low vitamin D level? | Yes | 26 Participants |
| Treatment With High-Dose D3 | Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire | Prefer taking a one-time dose of vitamin D instead of a daily vitamin D? | No | 0 Participants |
| Treatment With High-Dose D3 | Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire | Prefer taking a one-time dose of vitamin D instead of a daily vitamin D? | Yes | 26 Participants |