Trial Outcomes & Findings for Open-label Vitamin D Trial for Patients With Cystic Fibrosis and Allergic Bronchopulmonary Aspergillosis (NCT NCT01222273)

Last Updated: 2018-03-29

Results Overview

To test the hypothesis that supplementation with Vitamin D in CF patients with ABPA will reduce Aspergillus induced IL-13 responses in peripheral CD4+ T-cells. Response confirmed for each individual patient and recorded as number of participants with response.

Recruitment status

COMPLETED

Study phase

Target enrollment

7 participants

Primary outcome timeframe

6 months

Results posted on

2018-03-29

Participant Flow

The CF Foundation patient registry will be queried for patients meeting inclusion/exclusion criteria. A recruitment letter will be sent to these patients. Additionally, patients/parents of patients will be approached regarding study participation by the investigator at routine clinic visits.

Participant milestones

Participant milestones
Measure	Cholecalciferol 2000 Units of cholecalciferol once daily cholecalciferol (Vitamin D3): 4,000 IU of cholecalciferol (Vitamin D3) orally every day for six months
Overall Study STARTED	7
Overall Study COMPLETED	7
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cholecalciferol n=7 Participants 2000 Units of cholecalciferol once daily cholecalciferol (Vitamin D3): 4,000 IU of cholecalciferol (Vitamin D3) orally every day for six months
Age, Categorical <=18 years	2 Participants n=7 Participants
Age, Categorical Between 18 and 65 years	5 Participants n=7 Participants
Age, Categorical >=65 years	0 Participants n=7 Participants
Age, Continuous	21.91 years STANDARD_DEVIATION 2.69 • n=7 Participants
Sex: Female, Male Female	4 Participants n=7 Participants
Sex: Female, Male Male	3 Participants n=7 Participants
Region of Enrollment United States	7 participants n=7 Participants
BMI	22.5 kg/m^2 STANDARD_DEVIATION 10.68 • n=7 Participants
IgE	344.6 IU/mL STANDARD_DEVIATION 284.9 • n=7 Participants
Asp IgE	18.4 kUA/I STANDARD_DEVIATION 14.7 • n=7 Participants
Genotype ΔF508/R553X	1 Participants n=7 Participants
Genotype ΔF508/612+ 1G-T	1 Participants n=7 Participants
Genotype neg/ΔF508	1 Participants n=7 Participants
Genotype ΔF508/ΔF508	1 Participants n=7 Participants
Genotype MEG/G542X	1 Participants n=7 Participants
Genotype ΔF508/1213delT	1 Participants n=7 Participants
Genotype ΔF508/R1162X	1 Participants n=7 Participants

PRIMARY outcome

Timeframe: 6 months

Outcome measures

Outcome measures
Measure	Cholecalciferol n=7 Participants 2000 Units of cholecalciferol once daily cholecalciferol (Vitamin D3): 4,000 IU of cholecalciferol (Vitamin D3) orally every day for six months
Number of Participants With Aspergillus Induced IL-13 Responses in CD4+ T-cells	7 Participants

SECONDARY outcome

Timeframe: 6 months

To test the hypothesis that supplementation with Vitamin D in CF patients with ABPA will reduce total IgE levels by the end of the 24-week period

Outcome measures

Outcome measures
Measure	Cholecalciferol n=7 Participants 2000 Units of cholecalciferol once daily cholecalciferol (Vitamin D3): 4,000 IU of cholecalciferol (Vitamin D3) orally every day for six months
Change in Patient Total IgE Levels	312.6 IU/mL Standard Deviation 77.66

SECONDARY outcome

Timeframe: 6 months

To test the hypothesis that supplementation with Vitamin D in CF patients with ABPA will reduce aspergillus specific IgE levels by the end of the 24-week period

Outcome measures

Outcome measures
Measure	Cholecalciferol n=7 Participants 2000 Units of cholecalciferol once daily cholecalciferol (Vitamin D3): 4,000 IU of cholecalciferol (Vitamin D3) orally every day for six months
Change in Patient Aspergillus Specific IgE Levels	11.73 kUA/I Standard Deviation 3.581

Adverse Events

Cholecalciferol

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Joseph M. Pilewski, MD

Children's Hospital of Pittsburgh of UPMC

Phone: (412) 692-2164

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place