Trial Outcomes & Findings for Safety, Efficacy, and Feasibility of High-dose Cholecalciferol in Pediatric Patients With Cystic Fibrosis (NCT NCT02613884)
NCT ID: NCT02613884
Last Updated: 2021-02-03
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
26 participants
Primary outcome timeframe
1 week, 3 months
Results posted on
2021-02-03
Participant Flow
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.
Participant milestones
| Measure |
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.
|
|---|---|
|
Overall Study
STARTED
|
26
|
|
Overall Study
Safety Endpoint 1
|
26
|
|
Overall Study
Feasibility Questionnaire
|
26
|
|
Overall Study
Safety Endpoint 2
|
26
|
|
Overall Study
Efficacy Endpoint 1
|
25
|
|
Overall Study
Efficacy Timepoint 2
|
25
|
|
Overall Study
Efficacy Timepoint 3
|
24
|
|
Overall Study
COMPLETED
|
24
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
| Measure |
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.
|
|---|---|
|
Overall Study
Death
|
1
|
|
Overall Study
Lost to Follow-up
|
1
|
Baseline Characteristics
Safety, Efficacy, and Feasibility of High-dose Cholecalciferol in Pediatric Patients With Cystic Fibrosis
Baseline characteristics by cohort
| Measure |
Treatment
n=26 Participants
All patients with a 25OHD level \<30 ng/dL \& given 250,000 IU D3 (cholecalciferol).
|
|---|---|
|
Age, Categorical
<=18 years
|
26 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
|
Age, Continuous
|
13.46 years
STANDARD_DEVIATION 3.18 • n=99 Participants
|
|
Sex: Female, Male
Female
|
15 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
26 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
26 Participants
n=99 Participants
|
|
25-hydroxycholecalciferol (25OHD) level
|
22.69 ng/mL
STANDARD_DEVIATION 4.75 • n=99 Participants
|
|
Pancreatic Sufficient
Yes
|
3 Participants
n=99 Participants
|
|
Pancreatic Sufficient
No
|
23 Participants
n=99 Participants
|
|
Taking additional vitamin D at enrollment
Yes
|
18 Participants
n=99 Participants
|
|
Taking additional vitamin D at enrollment
No
|
8 Participants
n=99 Participants
|
|
Body Mass Index (BMI)
|
20.09 kg/m^2
STANDARD_DEVIATION 3.25 • n=99 Participants
|
|
BMI Percentile
|
56.22 percentile
STANDARD_DEVIATION 24.21 • n=99 Participants
|
PRIMARY outcome
Timeframe: 1 week, 3 monthsThe 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.
Outcome measures
| Measure |
Treatment With High-Dose D3
n=26 Participants
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.
|
|---|---|
|
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
|
|
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 outcome
Timeframe: 3 months, 6 months and 12 monthsPopulation: 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.
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.
Outcome measures
| Measure |
Treatment With High-Dose D3
n=25 Participants
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.
|
|---|---|
|
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
Interval 24.0 to 31.0
|
|
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
Interval 24.0 to 32.0
|
|
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
Interval 22.5 to 30.2
|
SECONDARY outcome
Timeframe: 1 weekCF 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.
Outcome measures
| Measure |
Treatment With High-Dose D3
n=26 Participants
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.
|
|---|---|
|
Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire
Increased amount of nausea · Yes
|
2 Participants
|
|
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
|
|
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
|
|
Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire
Increased amount of diarrhea? · No
|
23 Participants
|
|
Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire
Increased amount of diarrhea? · Yes
|
3 Participants
|
|
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
|
|
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
|
|
Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire
Easy to take? · No
|
0 Participants
|
|
Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire
Easy to take? · Yes
|
26 Participants
|
|
Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire
Increased amount of nausea · No
|
24 Participants
|
|
Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire
Any constipation? · No
|
25 Participants
|
|
Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire
Any constipation? · Yes
|
1 Participants
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
Adverse Events
Treatment
Serious events: 4 serious events
Other events: 15 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Treatment
n=26 participants at risk
All patients with a 25OHD level \<30 ng/dL \& given 250,000 IU D3 (cholecalciferol).
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Lung infection
|
3.8%
1/26 • Number of events 1 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Decreased lung function and weight loss
|
7.7%
2/26 • Number of events 2 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Bronchopneumonia exacerbation
|
3.8%
1/26 • Number of events 1 • 12 months
|
Other adverse events
| Measure |
Treatment
n=26 participants at risk
All patients with a 25OHD level \<30 ng/dL \& given 250,000 IU D3 (cholecalciferol).
|
|---|---|
|
Gastrointestinal disorders
Diarrhea
|
11.5%
3/26 • Number of events 3 • 12 months
|
|
Gastrointestinal disorders
Stomach ache
|
11.5%
3/26 • Number of events 3 • 12 months
|
|
Gastrointestinal disorders
Increased burping
|
7.7%
2/26 • Number of events 2 • 12 months
|
|
Gastrointestinal disorders
Nausea
|
7.7%
2/26 • Number of events 2 • 12 months
|
|
Gastrointestinal disorders
Heart burn/Reflux
|
7.7%
2/26 • Number of events 2 • 12 months
|
|
Gastrointestinal disorders
Constipation
|
3.8%
1/26 • Number of events 1 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Lung infection
|
7.7%
2/26 • Number of events 2 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxemia
|
3.8%
1/26 • Number of events 1 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Sinus Infection
|
3.8%
1/26 • Number of events 1 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Increased cough
|
3.8%
1/26 • Number of events 1 • 12 months
|
|
General disorders
Elevated phosphorus level
|
15.4%
4/26 • Number of events 4 • 12 months
|
|
General disorders
Elevated PTH
|
3.8%
1/26 • Number of events 1 • 12 months
|
|
General disorders
Elevated calcium level
|
3.8%
1/26 • Number of events 1 • 12 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place