A Phase 2 interventional study of Treatment in Vitamin D Deficiency, sponsored by Johns Hopkins All Children's Hospital. Completed at 1 site in United States. Open to participants aged 36 Months to 18 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2021-02-03.
Sponsored by Johns Hopkins All Children's Hospital · Phase 2, Interventional, and Treatment
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.
1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.
This study's enrollment of 26 is below the median of 36 across 1,034 interventional studies indexed under Cystic Fibrosis.
Browse Cystic Fibrosis studies →Johns Hopkins All Children's Hospital is the lead sponsor of 25 studies on the registry; 6 are open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 5 (100%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
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.
Drug: Treatment
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
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
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
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.
| Milestone | Treatment With High-Dose D3 |
|---|---|
| Started | 26 |
| Safety endpoint 1 | 26 |
| Feasibility questionnaire | 26 |
| Safety endpoint 2 | 26 |
| Efficacy endpoint 1 | 25 |
| Efficacy timepoint 2 | 25 |
| Efficacy timepoint 3 | 24 |
| Completed | 24 |
| Not completed | 2 |
| Withdrew: Death | 1 |
| Withdrew: Lost to follow-up | 1 |
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.
| mg/dL | Treatment With High-Dose D3 |
|---|---|
| Serum Calcium 1-week | 9.57 ± 0.32 |
| Serum Calcium 3-month | 9.47 ± 0.41 |
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.
| ng/dL | Treatment |
|---|---|
| 25OHD level at 3 months | 26 (24 to 31) |
| 25OHD level at 6 months | 30 (24 to 32) |
| 25OHD level at 12 months | 27 (22.5 to 30.2) |
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.
| Participants | Treatment With High-Dose D3 |
|---|---|
| Increased amount of nausea — No | 24 |
| Increased amount of nausea — Yes | 2 |
| Increased frequency of emesis (vomiting) — No | 26 |
| Increased frequency of emesis (vomiting) — Yes | 0 |
| Increased amount of diarrhea? — No | 23 |
| Increased amount of diarrhea? — Yes | 3 |
| Any constipation? — No | 25 |
| Any constipation? — Yes | 1 |
| Increased gas production, such as burping or passing gas? — No | 24 |
| Increased gas production, such as burping or passing gas? — Yes | 2 |
| Increased amount of abdominal pain/stomach aches? — No | 23 |
| Increased amount of abdominal pain/stomach aches? — Yes | 3 |
| Incred in heart burn or reflux? — No | 24 |
| Incred in heart burn or reflux? — Yes | 2 |
| Easy to take? — No | 0 |
| Easy to take? — Yes | 26 |
| Something you would do next year if you had another low vitamin D level? — No | 0 |
| Something you would do next year if you had another low vitamin D level? — Yes | 26 |
| Prefer taking a one-time dose of vitamin D instead of a daily vitamin D? — No | 0 |
| Prefer taking a one-time dose of vitamin D instead of a daily vitamin D? — Yes | 26 |
Collected over 12 months. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Treatment | 1/26 (3.8%) | 4/26 (15.4%) | 15/26 (57.7%) |
| Event | Treatment |
|---|---|
| Decreased lung function and weight lossRespiratory, thoracic and mediastinal disorders | 2/26 |
| Lung infectionRespiratory, thoracic and mediastinal disorders | 1/26 |
| Bronchopneumonia exacerbationRespiratory, thoracic and mediastinal disorders | 1/26 |
| Event | Treatment |
|---|---|
| Elevated phosphorus levelGeneral disorders | 4/26 |
| DiarrheaGastrointestinal disorders | 3/26 |
| Stomach acheGastrointestinal disorders | 3/26 |
| Increased burpingGastrointestinal disorders | 2/26 |
| NauseaGastrointestinal disorders | 2/26 |
| Heart burn/RefluxGastrointestinal disorders | 2/26 |
| Lung infectionRespiratory, thoracic and mediastinal disorders | 2/26 |
| ConstipationGastrointestinal disorders | 1/26 |
| HypoxemiaRespiratory, thoracic and mediastinal disorders | 1/26 |
| Sinus InfectionRespiratory, thoracic and mediastinal disorders | 1/26 |
| Age, Categorical(Participants) | Treatment |
|---|---|
| <=18 years | 26 |
| Between 18 and 65 years | 0 |
| >=65 years | 0 |
| Age, Continuous(years) | Treatment |
|---|---|
| Mean | 13.46 ± 3.18 |
| Sex: Female, Male(Participants) | Treatment |
|---|---|
| Female | 15 |
| Male | 11 |
| Race (NIH/OMB)(Participants) | Treatment |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 0 |
| More than one race | 0 |
| Unknown or Not Reported | 26 |
| Region of Enrollment(Participants) | Treatment |
|---|---|
| United States | 26 |
| 25-hydroxycholecalciferol (25OHD) level(ng/mL) | Treatment |
|---|---|
| Mean | 22.69 ± 4.75 |
| Pancreatic Sufficient(Participants) | Treatment |
|---|---|
| Yes | 3 |
| No | 23 |
| Taking additional vitamin D at enrollment(Participants) | Treatment |
|---|---|
| Yes | 18 |
| No | 8 |
2 further baseline measures are reported on the registry.
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Plan to share: No
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