Trial Outcomes & Findings for Proton Pump Inhibitors (PPI) and Fat Absorption in Cystic Fibrosis (CF) and Exocrine Pancreatic Insufficiency (EPI) (NCT NCT03551691)

Results Overview

Gold standard measurement of fat malabsorption

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

19 participants

Primary outcome timeframe

After 28 days of treatment or placebo

Results posted on

2024-04-30

Participant Flow

This is a randomized, cross-over study for which 19 participants were consented/enrolled; not all participants initiated study drug/placebo. Participants were randomized to receive placebo or drug first.

19 subjects were randomized. There were 13 evaluable subjects who completed data collection at the end of the treatment period and 6 subjects did not complete data collection.

Participant milestones

Participant milestones
Measure	Placebo First, Then Study Drug Subjects will take a placebo daily for 28 days, then undergo assessments of fat absorption. Placebo oral capsule: Identically-appearing capsule to omeprazole for 28 days	Study Drug First, Then Placebo Subjects will take omeprazole 20mg daily for 28 days, then undergo assessments of fat absorption. Omeprazole 20mg Capsule: Omeprazole 20mg daily for 28 days
Overall Study STARTED	10	9
Overall Study COMPLETED	7	6
Overall Study NOT COMPLETED	3	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Placebo First, Then Study Drug Subjects will take a placebo daily for 28 days, then undergo assessments of fat absorption. Placebo oral capsule: Identically-appearing capsule to omeprazole for 28 days	Study Drug First, Then Placebo Subjects will take omeprazole 20mg daily for 28 days, then undergo assessments of fat absorption. Omeprazole 20mg Capsule: Omeprazole 20mg daily for 28 days
Overall Study Withdrawal by Subject	3	3

Baseline Characteristics

Proton Pump Inhibitors (PPI) and Fat Absorption in Cystic Fibrosis (CF) and Exocrine Pancreatic Insufficiency (EPI)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Study Drug First, Then Placebo n=6 Participants Subjects will take omeprazole 20mg daily for 28 days, then undergo assessments of fat absorption. Omeprazole 20mg Capsule: Omeprazole 20mg daily for 28 days	Placebo First, Then Study Drug n=7 Participants Subjects will take a placebo daily for 28 days, then undergo assessments of fat absorption. Placebo oral capsule: Identically-appearing capsule to omeprazole for 28 days	Total n=13 Participants Total of all reporting groups
Age, Continuous	19 years STANDARD_DEVIATION 4 • n=5 Participants	19 years STANDARD_DEVIATION 4 • n=7 Participants	19 years STANDARD_DEVIATION 4 • n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants	4 Participants n=7 Participants	7 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants	3 Participants n=7 Participants	6 Participants n=5 Participants
Race/Ethnicity, Customized White/Caucasian	6 Participants n=5 Participants	6 Participants n=7 Participants	12 Participants n=5 Participants
Race/Ethnicity, Customized African American	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: After 28 days of treatment or placebo

Population: The primary outcome measurement (CFA) was not available for 2 participants who got study drug first and 2 participants who got placebo first.

Gold standard measurement of fat malabsorption

Outcome measures

Outcome measures
Measure	Omeprazole n=7 Participants Subjects will take omeprazole 20mg daily for 28 days, then undergo assessments of fat absorption. Omeprazole 20mg Capsule: Omeprazole 20mg daily for 28 days	Placebo n=4 Participants Subjects will take a placebo daily for 28 days, then undergo assessments of fat absorption. Placebo oral capsule: Identically-appearing capsule to omeprazole for 28 days
Coefficient of Fat Absorption	80 % fat absorption Standard Deviation 20	77 % fat absorption Standard Deviation 16

SECONDARY outcome

Timeframe: After 28 days of treatment or placebo

Change in duodenal pH as measured by the motility testing system (SmartPill)

Outcome measures

Outcome measures
Measure	Omeprazole n=4 Participants Subjects will take omeprazole 20mg daily for 28 days, then undergo assessments of fat absorption. Omeprazole 20mg Capsule: Omeprazole 20mg daily for 28 days	Placebo n=4 Participants Subjects will take a placebo daily for 28 days, then undergo assessments of fat absorption. Placebo oral capsule: Identically-appearing capsule to omeprazole for 28 days
Duodenal pH	6.03 pH Standard Deviation 0.4	5.38 pH Standard Deviation 0.8

SECONDARY outcome

Timeframe: After 28 days of treatment or placebo

Measurement of serum pentadecanoic acid and heptadecanoic acid

Outcome measures

Outcome measures
Measure	Omeprazole n=9 Participants Subjects will take omeprazole 20mg daily for 28 days, then undergo assessments of fat absorption. Omeprazole 20mg Capsule: Omeprazole 20mg daily for 28 days	Placebo n=9 Participants Subjects will take a placebo daily for 28 days, then undergo assessments of fat absorption. Placebo oral capsule: Identically-appearing capsule to omeprazole for 28 days
Fat Absorption Via Malabsorption Blood Test	7.3 mg*h/dl Standard Deviation 2.1	9.6 mg*h/dl Standard Deviation 3.4

Adverse Events

Omeprazole

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Placebo

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

Dr. Virginia Stallings

Children's Hospital of Philadelphia

Phone: 267-425-1633

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place