Trial Outcomes & Findings for Clearance of 25-hydroxyvitamin D in Cystic Fibrosis (NCT NCT03104855)

Results Overview

Metabolic clearance is calculated as the administered dose of 25(OH)D3 divided by the area under the plasma concentration-time curve (AUC). AUC is calculated using the linear trapezoidal method.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

10 participants

Primary outcome timeframe

8 weeks

Results posted on

2023-10-12

Participant Flow

Participant milestones

Participant milestones
Measure	CF Group Participants with cystic fibrosis	Healthy Controls Participants without cystic fibrosis
Overall Study STARTED	5	5
Overall Study COMPLETED	5	5
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Clearance of 25-hydroxyvitamin D in Cystic Fibrosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	CF Group n=5 Participants Participants with cystic fibrosis	Healthy Controls n=5 Participants Participants without cystic fibrosis	Total n=10 Participants Total of all reporting groups
Age, Continuous	38.8 years STANDARD_DEVIATION 14.3 • n=5 Participants	24.8 years STANDARD_DEVIATION 3.3 • n=7 Participants	31.8 years STANDARD_DEVIATION 12.3 • n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	4 Participants n=7 Participants	5 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants	1 Participants n=7 Participants	5 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	5 Participants n=5 Participants	5 Participants n=7 Participants	10 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: 8 weeks

Metabolic clearance is calculated as the administered dose of 25(OH)D3 divided by the area under the plasma concentration-time curve (AUC). AUC is calculated using the linear trapezoidal method.

Outcome measures

Outcome measures
Measure	CF Group n=5 Participants Participants with cystic fibrosis	Healthy Controls n=5 Participants Participants without cystic fibrosis
Metabolic Clearance of D6-25(OH)D3	397 mL/day Standard Deviation 73	342 mL/day Standard Deviation 41

SECONDARY outcome

Timeframe: 8 weeks

AUC is calculated using the linear trapezoidal method.

Outcome measures

Outcome measures
Measure	CF Group n=5 Participants Participants with cystic fibrosis	Healthy Controls n=5 Participants Participants without cystic fibrosis
AUC of D6-25(OH)D3	58.3 ng x day/mL Standard Deviation 9.7	67.2 ng x day/mL Standard Deviation 8.3

SECONDARY outcome

Timeframe: 8 weeks

Terminal half-life is equal to ln2/k, where k is the slope of the terminal regression line estimated using ≥3 plasma concentrations.

Outcome measures

Outcome measures
Measure	CF Group n=5 Participants Participants with cystic fibrosis	Healthy Controls n=5 Participants Participants without cystic fibrosis
Terminal Half-life of D6-25(OH)D3	16.2 day Standard Deviation 3.3	15.8 day Standard Deviation 1.6

SECONDARY outcome

Timeframe: 8 weeks

Volume of distribution in the central compartment is calculated as dose/C0, where dose is the administered dose of 25(OH)D3 and C0 is the initial (estimated) concentration of drug in plasma.

Outcome measures

Outcome measures
Measure	CF Group n=5 Participants Participants with cystic fibrosis	Healthy Controls n=5 Participants Participants without cystic fibrosis
Volume of Distribution of D6-25(OH)D3	8.4 Liters Standard Deviation 1.4	7.2 Liters Standard Deviation 1.1

OTHER_PRE_SPECIFIED outcome

Timeframe: 8 weeks

Metabolic formation clearance is calculated as the daughter metabolite plasma AUC divided by the AUC of D6-25(OH)D3 (metabolite/parent AUC ratio). AUC is calculated using the linear trapezoidal method.

Outcome measures

Outcome measures
Measure	CF Group n=5 Participants Participants with cystic fibrosis	Healthy Controls n=5 Participants Participants without cystic fibrosis
Metabolic Formation Clearance of D6-25(OH)D3 Metabolites	0.10 ratio Standard Deviation 0.02	0.08 ratio Standard Deviation 0.01

OTHER_PRE_SPECIFIED outcome

Timeframe: 7 days

Change in the serum concentration of calcium from baseline to 7 days after 25(OH)D3

Outcome measures

Outcome measures
Measure	CF Group n=5 Participants Participants with cystic fibrosis	Healthy Controls n=5 Participants Participants without cystic fibrosis
Serum Concentration of Calcium	0.00 mg/dL Standard Deviation 0.37	0.14 mg/dL Standard Deviation 0.27

OTHER_PRE_SPECIFIED outcome

Timeframe: 7 days

Change in the serum concentration of creatinine from baseline to 7 days after 25(OH)D3

Outcome measures

Outcome measures
Measure	CF Group n=5 Participants Participants with cystic fibrosis	Healthy Controls n=5 Participants Participants without cystic fibrosis
Serum Concentration of Creatinine	0.00 mg/dL Standard Deviation 0.03	0.00 mg/dL Standard Deviation 0.07

OTHER_PRE_SPECIFIED outcome

Timeframe: 7 days

Change in the serum concentration of AST from baseline to 7 days after 25(OH)D3

Outcome measures

Outcome measures
Measure	CF Group n=5 Participants Participants with cystic fibrosis	Healthy Controls n=5 Participants Participants without cystic fibrosis
Serum Concentration of AST	0.60 Units/L Standard Deviation 5.03	-1.00 Units/L Standard Deviation 2.55

OTHER_PRE_SPECIFIED outcome

Timeframe: 7 days

Change in the serum concentration of ALT from baseline to 7 days after 25(OH)D3

Outcome measures

Outcome measures
Measure	CF Group n=5 Participants Participants with cystic fibrosis	Healthy Controls n=5 Participants Participants without cystic fibrosis
Serum Concentration of ALT	0.00 Units/L Standard Deviation 0.00	0.00 Units/L Standard Deviation 0.00

Adverse Events

CF Group

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Healthy Controls

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	CF Group n=5 participants at risk Participants with cystic fibrosis	Healthy Controls n=5 participants at risk Participants without cystic fibrosis
General disorders Pain or sensation in arm during infusion	40.0% 2/5 • Number of events 2 • 8 weeks	0.00% 0/5 • 8 weeks
Blood and lymphatic system disorders Bruising after blood draw	0.00% 0/5 • 8 weeks	20.0% 1/5 • Number of events 1 • 8 weeks

Additional Information

Dr. Ian de Boer

University of Washington

Phone: 206-616-5403

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place