Status:

COMPLETED

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

Lead Sponsor:

Phathom Pharmaceuticals, Inc.

Conditions:

Gastroesophageal Reflux Disease

Eligibility:

All Genders

6-11 years

Phase:

PHASE1

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 D...

Eligibility Criteria

Inclusion

  • 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.

Exclusion

  • 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).
  • The participant has any of the following abnormal laboratory test values at the start of the Screening Period:
  • Creatinine levels: \>0.8 mg/dL (\>70 μmol/L).
  • Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>2 × the upper limit of normal (ULN) or total bilirubin \>2 × ULN (except participants with Gilbert Syndrome).
  • In the opinion of the investigator, the participant is not suitable for entry into the study.

Key Trial Info

Start Date :

November 1 2023

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

April 29 2024

Estimated Enrollment :

22 Patients enrolled

Trial Details

Trial ID

NCT06106022

Start Date

November 1 2023

End Date

April 29 2024

Last Update

December 19 2024

Active Locations (13)

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Page 1 of 4 (13 locations)

1

Strada Patient Care Center

Mobile, Alabama, United States, 36604

2

Preferred Research Partners, Inc.

Little Rock, Arkansas, United States, 72211

3

Med Research Associates, Inc

Hollywood, Florida, United States, 33024

4

Avanza Medical Research Center

Pensacola, Florida, United States, 32503