Trial Outcomes & Findings for A Study to Evaluate Vonoprazan in Children Who Have Symptomatic Gastroesophageal Reflux Disease (NCT NCT06106022)

Results Overview

Plasma pharmacokinetic (PK) parameters were estimated using a non-linear mixed effects model and were determined from the concentration-time data for all evaluable participants. Actual sampling times, rather than scheduled or nominal sampling times, were used in all computations using sampling time. Data presented based on collections on both Day 7 and Day 14.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

22 participants

Primary outcome timeframe

Day 7: pre-dose, 0.5 to 1.5 hour and 2.5 to 3.5 hours post dose; Day 14: pre-dose, 1 to 2 hours and 3 to 4 hours post dose

Results posted on

2024-12-19

Participant Flow

A total of 22 participants were enrolled at 6 study sites in the United States between November 2023 and April 2024.

Participant milestones

Participant milestones
Measure	Vonoprazan 10 mg Participants received vonoprazan 10 mg once daily (QD) for 14 days.	Vonoprazan 20 mg Participants received vonoprazan 20 mg QD for 14 days.
Overall Study STARTED	11	11
Overall Study COMPLETED	10	9
Overall Study NOT COMPLETED	1	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Vonoprazan 10 mg Participants received vonoprazan 10 mg once daily (QD) for 14 days.	Vonoprazan 20 mg Participants received vonoprazan 20 mg QD for 14 days.
Overall Study Lost to Follow-up	1	0
Overall Study Voluntary Withdrawal	0	2

Baseline Characteristics

A Study to Evaluate Vonoprazan in Children Who Have Symptomatic Gastroesophageal Reflux Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Vonoprazan 10 mg n=11 Participants Participants received vonoprazan 10 mg QD for 14 days.	Vonoprazan 20 mg n=11 Participants Participants received vonoprazan 20 mg QD for 14 days.	Total n=22 Participants Total of all reporting groups
Age, Continuous	9.1 years STANDARD_DEVIATION 1.92 • n=5 Participants	8.6 years STANDARD_DEVIATION 1.69 • n=7 Participants	8.9 years STANDARD_DEVIATION 1.78 • n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants	5 Participants n=7 Participants	12 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants	6 Participants n=7 Participants	10 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	6 Participants n=5 Participants	9 Participants n=7 Participants	15 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	5 Participants n=5 Participants	2 Participants n=7 Participants	7 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race/Ethnicity, Customized White	10 Participants n=5 Participants	10 Participants n=7 Participants	20 Participants n=5 Participants
Race/Ethnicity, Customized Black or African American	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants

PRIMARY outcome

Timeframe: Day 7: pre-dose, 0.5 to 1.5 hour and 2.5 to 3.5 hours post dose; Day 14: pre-dose, 1 to 2 hours and 3 to 4 hours post dose

Population: PK Set: includes all participants who received at least 1 dose of study drug and had sufficient concentration data to support accurate estimation of at least 1 PK parameter.

Plasma pharmacokinetic (PK) parameters were estimated using a non-linear mixed effects model and were determined from the concentration-time data for all evaluable participants. Actual sampling times, rather than scheduled or nominal sampling times, were used in all computations using sampling time. Data presented based on collections on both Day 7 and Day 14.

Outcome measures

Outcome measures
Measure	Vonoprazan 10 mg n=10 Participants Participants received vonoprazan 10 mg QD for 14 days.	Vonoprazan 20 mg n=11 Participants Participants received vonoprazan 20 mg QD for 14 days.
Maximum Drug Concentration at Steady-state (Cmax,ss) of Vonoprazan	16.2 ng/mL Interval 9.31 to 27.1	42.1 ng/mL Interval 10.1 to 72.7

PRIMARY outcome

Timeframe: Day 7: pre-dose, 0.5 to 1.5 hour and 2.5 to 3.5 hours post dose; Day 14: pre-dose, 1 to 2 hours and 3 to 4 hours post dose

Population: PK Set: includes all participants who received at least 1 dose of study drug and had sufficient concentration data to support accurate estimation of at least 1 PK parameter.

Plasma PK parameters were estimated using a non-linear mixed effects model and were determined from the concentration-time data for all evaluable participants. Actual sampling times, rather than scheduled or nominal sampling times, were used in all computations using sampling time. Data presented based on collections on both Day 7 and Day 14.

Outcome measures

Outcome measures
Measure	Vonoprazan 10 mg n=10 Participants Participants received vonoprazan 10 mg QD for 14 days.	Vonoprazan 20 mg n=11 Participants Participants received vonoprazan 20 mg QD for 14 days.
Area Under the Plasma Concentration-time Curve During the Dosing Interval τ at Steady State (AUCτ,ss) of Vonoprazan	88.3 h*ng/mL Interval 51.7 to 121.0	251 h*ng/mL Interval 72.1 to 549.0

PRIMARY outcome

Timeframe: Day 7: pre-dose, 0.5 to 1.5 hour and 2.5 to 3.5 hours post dose; Day 14: pre-dose, 1 to 2 hours and 3 to 4 hours post dose

Population: PK Set: includes all participants who received at least 1 dose of study drug and had sufficient concentration data to support accurate estimation of at least 1 PK parameter.

Oral PK parameters were estimated using a non-linear mixed effects model and were determined from the concentration-time data for all evaluable participants. Actual sampling times, rather than scheduled or nominal sampling times, were used in all computations using sampling time. Data presented based on collections on both Day 7 and Day 14.

Outcome measures

Outcome measures
Measure	Vonoprazan 10 mg n=10 Participants Participants received vonoprazan 10 mg QD for 14 days.	Vonoprazan 20 mg n=11 Participants Participants received vonoprazan 20 mg QD for 14 days.
Apparent Oral Clearance (CL/F) at Steady State of Vonoprazan	125 L/h Interval 82.9 to 193.0	118 L/h Interval 36.4 to 278.0

PRIMARY outcome

Timeframe: Day 7: pre-dose, 0.5 to 1.5 hour and 2.5 to 3.5 hours post dose; Day 14: pre-dose, 1 to 2 hours and 3 to 4 hours post dose

Population: PK Set: includes all participants who received at least 1 dose of study drug and had sufficient concentration data to support accurate estimation of at least 1 PK parameter.

Plasma PK parameters were estimated using a non-linear mixed effects model and were determined from the concentration-time data for all evaluable participants. Actual sampling times, rather than scheduled or nominal sampling times, were used in all computations using sampling time. Data presented based on collections on both Day 7 and Day 14.

Outcome measures

Outcome measures
Measure	Vonoprazan 10 mg n=10 Participants Participants received vonoprazan 10 mg QD for 14 days.	Vonoprazan 20 mg n=11 Participants Participants received vonoprazan 20 mg QD for 14 days.
Apparent Central Volume of Distribution (Vz/F) at Steady State of Vonoprazan	491 liters Interval 227.0 to 828.0	444 liters Interval 164.0 to 1310.0

Adverse Events

Vonoprazan 10 mg

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Vonoprazan 20 mg

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Vonoprazan 10 mg n=11 participants at risk Participants received vonoprazan 10 mg QD for 14 days.	Vonoprazan 20 mg n=11 participants at risk Participants received vonoprazan 20 mg QD for 14 days.
Ear and labyrinth disorders Tympanic membrane perforation	9.1% 1/11 • Number of events 1 • Up to Day 28 Safety Set: includes all participants who received at least 1 dose of study drug.	0.00% 0/11 • Up to Day 28 Safety Set: includes all participants who received at least 1 dose of study drug.
Gastrointestinal disorders Vomiting	9.1% 1/11 • Number of events 1 • Up to Day 28 Safety Set: includes all participants who received at least 1 dose of study drug.	0.00% 0/11 • Up to Day 28 Safety Set: includes all participants who received at least 1 dose of study drug.
Infections and infestations Otitis media	9.1% 1/11 • Number of events 1 • Up to Day 28 Safety Set: includes all participants who received at least 1 dose of study drug.	0.00% 0/11 • Up to Day 28 Safety Set: includes all participants who received at least 1 dose of study drug.
Investigations Electrocardiogram QT prolonged	9.1% 1/11 • Number of events 1 • Up to Day 28 Safety Set: includes all participants who received at least 1 dose of study drug.	0.00% 0/11 • Up to Day 28 Safety Set: includes all participants who received at least 1 dose of study drug.

Additional Information

Phathom Medical Information

Phathom Pharmaceuticals, Inc.

Phone: 1-888-775-7428

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Principal investigators (PIs) are not permitted to publish the data. Data may be considered for reporting at a scientific meeting or for publication in a scientific journal. In these cases, the sponsor will be responsible for these activities and will work with the PIs to determine how the manuscript is written and edited, the number and order of authors, the publication to which it will be submitted, and any other related issues. The sponsor has final approval authority over all such issues.
Publication restrictions are in place

Restriction type: OTHER