A Study to Evaluate the Pharmacokinetics, Pharmacodynamics and Safety of Vonoprazan in Adolescents With Symptomatic Gastroesophageal Reflux Disease

NCT05343364 | Completed Phase 1 Results FDA Drug

• 1 other identifier: VPED-102
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 10

Brief Summary

The primary objective of this study is to evaluate the pharmacokinetic profile of vonoprazan in adolescent participants with symptomatic gastroesophageal reflux disease (GERD).

Conditions

Gastroesophageal Reflux

Keywords

Vonoprazan; Gastroesophageal Reflux; GERD; Adolescents; Pharmacokinetics

Outcome Measures

Primary Outcomes (4)

• Maximum Observed Drug Concentration at Steady State (Cmax-ss) of Vonoprazan

  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.

  Blood samples were collected predose, once between 0.5 and 2 hours, and once between 2.5 and 4 hours post-dose on Days 7 and 14

• Area Under the Plasma Concentration-time Curve During the Dosing Interval τ (AUCτ) of Vonoprazan

  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.

  Blood samples were collected predose, once between 0.5 and 2 hours, and once between 2.5 and 4 hours post-dose on Days 7 and 14

• Apparent Oral Clearance (CL/F) of Vonoprazan

  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.

  Blood samples were collected predose, once between 0.5 and 2 hours, and once between 2.5 and 4 hours post-dose on Days 7 and 14

• Apparent Central Volume of Distribution (Vc/F) of Vonoprazan

  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.

  Blood samples were collected predose, once between 0.5 and 2 hours, and once between 2.5 and 4 hours post-dose on Days 7 and 14

Secondary Outcomes (1)

• Number of Participants Experiencing Adverse Events (AEs)

  Up to Day 28

Study Arms (2)

Vonoprazan 10 mg

EXPERIMENTAL

Participants will receive vonoprazan 10 mg once daily for 14 days.

Drug: Vonoprazan

Vonoprazan 20 mg

EXPERIMENTAL

Participants will receive vonoprazan 20 mg once daily for 14 days.

Drug: Vonoprazan

Interventions

Vonoprazan DRUG

Oral Tablet

Vonoprazan 10 mg; Vonoprazan 20 mg

Eligibility Criteria

• Age: 12 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• The participant is 12 to 17 years of age, inclusive, at the time of informed consent signing and throughout study participation.
• The participant has a body weight within the 5th through 95th percentile by age, inclusive, as determined by the National Center for Health Statistics.
• The participant has a medical history of 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.
• The participant has symptoms of at least moderate heartburn severity based on the GERD Symptom Assessment-Investigator scale performed at screening.
• The participant must be able to swallow study drug.
• Parent or legal guardian (ie, legally authorized representative \[LAR\]) is willing and able to complete the informed consent process and comply with study procedures and visit schedule. The participant will provide assent as applicable.
• A female participant of childbearing potential who is or may be sexually active with a nonsterilized male partner agrees to routinely use adequate contraception from the signing of informed consent until 2 weeks after the last dose of study drug.

You may not qualify if:

• The participant has used prescription or non-prescription proton pump inhibitors (PPIs) or histamine-2 receptor antagonists (H2RAs) within 7 days prior to randomization or requires their use during the Treatment Period.
• The participant has used sucralfate or antacids within 1 day prior to randomization or requires their use during the Treatment Period.
• 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.
• 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.
• The participant has received any investigational compound (including vonoprazan) within 30 days prior to the start of the Screening Period.
• 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 who may have consented under duress.
• 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.
• The participant has undergone prior gastrointestinal surgeries such as fundoplication.
• The participant has any abnormal laboratory test values at the start of the Screening Period.
• The participant has a history of hypersensitivity or allergies to vonoprazan (including the formulation excipients: D-mannitol, microcrystalline cellulose, hydroxypropyl cellulose, fumaric acid, croscarmellose sodium, magnesium stearate, hypromellose, macrogol 8000, and titanium oxide, or red or yellow ferric oxide).
• 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.
• Female participant has a positive pregnancy test at screening or check in or is lactating.
• The participant has a positive urine drug or alcohol result at screening.
• The participant has positive results at screening for human immunodeficiency virus, hepatitis B surface antigen, or hepatitis C virus.
• In the opinion of the investigator, the participant is not suitable for entry into the study.

Sponsors & Collaborators

• Phathom Pharmaceuticals, Inc.: lead

Study Sites (10)

University of South Alabama (USA) Physicians Group

Mobile, Alabama, 36604-1541, United States

Location

Preferred Research Partners, Inc.

Little Rock, Arkansas, 72211, United States

Location

Advanced Research Center - Elligo

Anaheim, California, 92805, United States

Location

Infinite Clinical Trials

Morrow, Georgia, 30260-2342, United States

Location

Legacy Clinical Solutions: Tandem Clinical Research, LLC

Marrero, Louisiana, 70072-3151, United States

Location

Boston Specialists

Boston, Massachusetts, 02111, United States

Location

University of Rochester Medical Center

Rochester, New York, 14642, United States

Location

PriMED Clinical Research

Dayton, Ohio, 45429, United States

Location

ARC Clinical Research at Four Points

Austin, Texas, 78726, United States

Location

Pediatric Gastro

El Paso, Texas, 79902, United States

Location

MeSH Terms

Conditions

Gastroesophageal Reflux

Interventions

1-(5-(2-fluorophenyl)-1-(pyridin-3-ylsulfonyl)-1H-pyrrol-3-yl)-N-methylmethanamine

Condition Hierarchy (Ancestors)

Esophageal Motility Disorders; Deglutition Disorders; Esophageal Diseases; Gastrointestinal Diseases; Digestive System Diseases

Results Point of Contact

• Title: Phathom Medical Information
• Organization: Phathom Pharmaceuticals, Inc.

medicalinformation@phathompharma.com

1-888-775-7428

Study Officials

• Medical Director

  Phathom Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 18, 2022
• First Posted: April 25, 2022
• Study Start: May 9, 2022
• Primary Completion: May 30, 2023
• Study Completion: June 13, 2023
• Last Updated: April 23, 2025
• Results First Posted: June 3, 2024

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (10)