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

NCT06106022 | Completed Phase 1 Results FDA Drug

• 2 other identifiers: VPED-1032022-003228-42
• Study Type: interventional
• Target: 22
• Locations: 1 country
• Sites: 13

Brief Summary

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).

Conditions

Gastroesophageal Reflux Disease

Keywords

Gastrology; Vonoprazan; Gastroesophageal Reflux Disease; Children; GERD; Pharmacokinetics

Outcome Measures

Primary Outcomes (4)

• Maximum 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. 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

  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

  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

  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

Study Arms (2)

Vonoprazan 10mg

EXPERIMENTAL

Participants will receive vonoprazan 10mg QD for 14 days.

Drug: Vonoprazan

Vonoprazan 20mg

EXPERIMENTAL

Participants will receive vonoprazan 20mg QD for 14 days.

Drug: Vonoprazan

Interventions

Vonoprazan DRUG

Administered orally

Vonoprazan 10mg; Vonoprazan 20mg

Eligibility Criteria

• Age: 6 Years - 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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 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).
• The participant has at least one moderate GERD symptom based on the GERD Symptom Assessment Investigator scale performed at screening.
• The participant must be able to swallow study drug tablet with water.
• 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.
• Female participants who have experienced menarche must have a negative pregnancy test and will be counseled on pregnancy avoidance.

You may not qualify if:

• 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.
• 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 participant 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.
• The participant has any abnormal laboratory test values that are considered clinically significant in the opinion of the investigator during the Screening Period.
• 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).
• 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.
• 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.
• The participant has positive results at screening for human immunodeficiency virus, hepatitis B virus, or hepatitis C virus (HCV).
• The participant has severe renal impairment (estimated glomerular filtration rate \< 30 mL/min).
• The participant has moderate to severe hepatic impairment (Child-Pugh Class B and Child-Pugh Class C).

Sponsors & Collaborators

• Phathom Pharmaceuticals, Inc.: lead

Study Sites (13)

Strada Patient Care Center

Mobile, Alabama, 36604, United States

Location

Preferred Research Partners, Inc.

Little Rock, Arkansas, 72211, United States

Location

Med Research Associates, Inc

Hollywood, Florida, 33024, United States

Location

Avanza Medical Research Center

Pensacola, Florida, 32503, United States

Location

International Center for Research

Tampa, Florida, 33614, United States

Location

Children's Center for Digestive Health Care, LLC

Atlanta, Georgia, 30342, United States

Location

Riley Hospital for Children at IU Health

Indianapolis, Indiana, 46202, United States

Location

Tandem Clinical Research GI, LLC

Marrero, Louisiana, 70072, United States

Location

Advantage Clinical Trials

The Bronx, New York, 10467, United States

Location

PriMED Clinical Research

Dayton, Ohio, 45429, United States

Location

Cyn3rgy Research

Gresham, Oregon, 97030, United States

Location

Maspons Pediatric Gastro

El Paso, Texas, 79902, United States

Location

Stryde Research - NxT Step Pediatrics

Frisco, Texas, 75033, 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

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: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 24, 2023
• First Posted: October 30, 2023
• Study Start: November 1, 2023
• Primary Completion: April 16, 2024
• Study Completion: April 29, 2024
• Last Updated: December 19, 2024
• Results First Posted: December 19, 2024

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (13)