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

December 17, 2024 updated by: Phathom Pharmaceuticals, Inc.

A Phase 1, Randomized, Parallel-group, Open-label, Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics and Safety of Vonoprazan (10 or 20 mg Once Daily) in Children Aged ≥ 6 to < 12 Years Who Have Symptomatic Gastroesophageal Reflux Disease

The aim of this study is to evaluate the pharmacokinetic (PK) profile of vonoprazan (10 or 20 mg once daily [QD]) in children ≥ 6 to < 12 years of age who have symptomatic Gastroesophageal Reflux Disease (GERD).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
22

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • United States
    • Alabama
      • Mobile, Alabama, United States, 36604
        • Strada Patient Care Center
    • Arkansas
      • Little Rock, Arkansas, United States, 72211
        • Preferred Research Partners, Inc.
    • Florida
      • Hollywood, Florida, United States, 33024
        • Med Research Associates, Inc
      • Pensacola, Florida, United States, 32503
        • Avanza Medical Research Center
      • Tampa, Florida, United States, 33614
        • International Center for Research
    • Georgia
      • Atlanta, Georgia, United States, 30342
        • Children's Center for Digestive Health Care, LLC
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Riley Hospital for Children at IU Health
    • Louisiana
      • Marrero, Louisiana, United States, 70072
        • Tandem Clinical Research GI, LLC
    • New York
      • Bronx, New York, United States, 10467
        • Advantage Clinical Trials
    • Ohio
      • Dayton, Ohio, United States, 45429
        • PriMED Clinical Research
    • Oregon
      • Gresham, Oregon, United States, 97030
        • Cyn3rgy Research
    • Texas
      • El Paso, Texas, United States, 79902
        • Maspons Pediatric Gastro
      • Frisco, Texas, United States, 75033
        • Stryde Research - NxT Step Pediatrics

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. The participant has a body weight within the 5th through 95th percentile by age, inclusive, as determined by the National Center for Health Statistics.
  2. The participant must have a diagnosis of GERD prior to randomization and medical history of signs or symptoms of GERD for at least 3 months prior to screening, based on physical examination, current symptoms (eg, heartburn), or diagnostic tests (eg, pH or endoscopy). Notes in the medical records and/or other source documents, such as prior endoscopies, can be used to support the diagnosis and will be recorded in the electronic case report form (eCRF).
  3. The participant has at least one moderate GERD symptom based on the GERD Symptom Assessment Investigator scale performed at screening.
  4. The participant must be able to swallow study drug tablet with water.
  5. Parent or legal guardian (ie, legally authorized representative [LAR]) is willing and able to complete the informed consent process and participants are able to comply with study procedures and visit schedule.
  6. Female participants who have experienced menarche must have a negative pregnancy test and will be counseled on pregnancy avoidance.

Exclusion Criteria:

  1. The participant has used prescription or non-prescription proton pump inhibitors (PPIs) or histamine-2 receptor antagonist (H2RAs) within 7 days prior to randomization or requires use during the Treatment Period.
  2. The participant has used sucralfate, or antacids within 1 day prior to randomization or requires their use during the Treatment Period.
  3. The participant has received other agents affecting digestive organs, including muscarinic antagonists (eg, hyoscyamine), prokinetics, oral anticholinergic agents, prostaglandins, bismuth from 30 days prior to Day 1 or requires their use during the course of the study.
  4. The participant has received atazanavir sulfate or rilpivirine hydrochloride from 5 days prior to Day 1 or requires their use during the course of the study.
  5. The participant has received any investigational compound (including vonoprazan) within 30 days prior to the start of the Screening Period.
  6. The participant is an immediate family member or is in a dependent relationship with a study site employee who is involved in the conduct of this study (eg, child, sibling) or participant may have consented under duress.
  7. The participant requires hospitalization or has surgery scheduled during the course of the study or has undergone major surgical procedures within 30 days prior to the Screening Period.
  8. The participant has undergone prior gastrointestinal surgeries.
  9. The participant has any abnormal laboratory test values that are considered clinically significant in the opinion of the investigator during the Screening Period.
  10. The participant has a history of hypersensitivity or allergies to vonoprazan (including the formulation excipients: D-mannitol, microcrystalline cellulose, hydroxypropylcellulose, fumaric acid, ascorbic acid, croscarmellose sodium, magnesium stearate, hypromellose, macrogol 8000, and titanium dioxide, or red or yellow ferric oxide).
  11. The participant has used any prescription or over-the-counter medications (including herbal or nutritional supplements), other than those already excluded in criteria 1 to 5 above, within 14 days before the first dose of study drug or throughout the study. That is, unless the medication(s) is permitted by the sponsor following a review of available data which confirms concomitant administration of the medication is unlikely to affect either the safety of the participant or the pharmacokinetics of vonoprazan.
  12. The participant has consumed grapefruit or grapefruit juice, Seville orange or Seville orange-containing products (eg, marmalade), or other food products that may be CYP3A4 inhibitors (eg, vegetables from the mustard green family [kale, broccoli, watercress, collard greens, kohlrabi, Brussels sprouts, mustard] and charbroiled meats) within 7 days (or 5 half-lives) before the first dose of study drug or throughout the study.
  13. The participant has positive results at screening for human immunodeficiency virus, hepatitis B virus, or hepatitis C virus (HCV).
  14. The participant has severe renal impairment (estimated glomerular filtration rate < 30 mL/min).
  15. The participant has moderate to severe hepatic impairment (Child-Pugh Class B and Child-Pugh Class C).

  16. The participant has any of the following abnormal laboratory test values at the start of the Screening Period:

    1. Creatinine levels: >0.8 mg/dL (>70 μmol/L).
    2. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2 × the upper limit of normal (ULN) or total bilirubin >2 × ULN (except participants with Gilbert Syndrome).
  17. In the opinion of the investigator, the participant is not suitable for entry into the study.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Vonoprazan 10mg
Participants will receive vonoprazan 10mg QD for 14 days.
Drug: Vonoprazan
Administered orally
Experimental: Vonoprazan 20mg
Participants will receive vonoprazan 20mg QD for 14 days.
Drug: Vonoprazan
Administered orally

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Maximum Drug Concentration at Steady-state (Cmax,ss) of Vonoprazan
Time Frame: 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
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.
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
Area Under the Plasma Concentration-time Curve During the Dosing Interval τ at Steady State (AUCτ,ss) of Vonoprazan
Time Frame: 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
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.
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
Apparent Oral Clearance (CL/F) at Steady State of Vonoprazan
Time Frame: 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
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.
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
Apparent Central Volume of Distribution (Vz/F) at Steady State of Vonoprazan
Time Frame: 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
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.
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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Phathom Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
November 1, 2023

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
April 16, 2024

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
April 29, 2024

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
October 24, 2023

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 24, 2023

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
October 30, 2023

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 25, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 17, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
December 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • VPED-103
  • 2022-003228-42 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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