Gastroesophageal RefluxPhase 4PGXNCT02794844
Implementation of PPI Medication PGX Testing
A Phase 4 study testing Genotype Guided PPI Dosing in people with Gastroesophageal Reflux, run by Nemours Children's Clinic at 1 site in United States.
Trial summary
Using genetic information about the individual to pick the right drug for the right disease at the right dose defines personalized medicine. This pilot study seeks to institute pharmacogenomic testing, that is identifying genetic variation that influences patient response to drugs, into the Nemours Children's Health… Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Gastroesophageal Reflux
- What is being tested?
- Genotype Guided PPI Dosing
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 28 participants
- Where
- 1 siteUnited States
- Study dates
- 21 Jul 2016to 31 Aug 2018
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Children diagnosed with Gastroesophageal Reflux Disease (GERD) or a stomach acid mediated condition for which a PPI is prescribed.
- Currently taking or will be prescribed Proton Pump Inhibitor (PPI) medication
- Parents/legal guardians and or child must also have access to the internet and a valid email address to complete weekly required forms.
Who cannot join
- Children who have had peptic ulcer surgery;
- with a history of PKU
- with a history of previous adverse effects from PPI treatment or a sensitivity to aspartame (NutraSweet, Equal);
- who are non-adherent including inability or unwillingness of the legal guardian to provide consent of unwillingness of the child to provide assent;
- who are unable to take study medications;
- who are unable to communicate via telephone or other device;
- who do not have access to a computer with internet access
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Implementation of Pharmacogenomic Testing in Nemours Children's Health System
Using genetic information about the individual to pick the right drug for the right disease at the right dose defines personalized medicine. This pilot study seeks to institute pharmacogenomic testing, that is identifying genetic variation that influences patient response to drugs, into the Nemours Children's Health system. We propose to initiate the study by identifying genetic differences in cyp2c19, a gene that is responsible for a certain enzyme in the liver that metabolizes many drugs including a class of drugs called proton pump inhibitors (ppi; Prevacid, Nexium). PPIs are used to treat heartburn and other symptoms of gastroesophageal reflux disease (gerd) and are extensively used in pediatrics. Chronic use of PPIs can cause serious side effects including cold, pneumonia and stomach infections, which gets worse at higher doses. Children who poorly metabolize drugs because of genetic variation in cyp2c19 should get lower doses of PPIs than children who metabolize PPIs normally. Our pilot study will genotype children with gerd or other stomach acid mediated conditions for which a PPI is prescribed using a sample of spit to determine which dose of PPI they get based on the form of the cyp2c19 gene they have. We will study 120 children 2-17 yo diagnosed with gastroesophageal reflux disease (gerd) or other stomach acid mediated conditions for which a ppi is prescribed . Genetic results are available in < 60 minutes, and their doses are determined by their doctor based on genetic results. This study will allow us to gain valuable experience that will be used to expand our genetic program to other genes and drugs.
Read the detailed description
The long-term objective of this research is to implement pharmacogenomic (PGX) testing of approved gene-drug pairs to maximize the efficacy and minimize adverse events for drugs that are used to treat childhood disease. Personalized medicine, that is, the use of the patient's own genetic information to predict the right dose of the right drug, has been a goal of medicine's since the publication of the human genome and HAPMAP projects. However, the adoption of PGX testing has been slow owing to several barriers and challenges. Recently President Obama announced the precision medicine initiative, which includes PGX testing, and has earmarked $ 1 b to implement it. The precision medicine initiative is expected to move personalized medicine forward so that most patients including pediatric patients will benefit. The Nemours Children's Health System is uniquely positioned to lead the effort to personalize medicine among pediatric patients. The goal of the proposed 1-year pilot project is to implement PGX testing in the Nemours children's health system. The experience, knowledge and skills gained in this pilot project will position Nemours to help lead precision medicine initiatives including PGX testing among pediatric populations. To implement PGX testing in Nemours we propose to begin testing the cyp2c19 - proton pump inhibitor gene-drug pair in the division of gastroenterology, Nemours children's hospital. A total of 120 children 2-17 yo who have been diagnosed with gastroesophageal reflux disease (GERD) or other stomach acid mediated conditions for which a PPI is prescribed will be recruited into the study by pediatric gastroenterologists (aim 1a). Drs. Franciosi and Lima will educate staff in the division of PGX testing and of the study. GI pediatricians are not required to participate in PGX testing. After obtaining consent, the study coordinator will obtain and bar code samples of saliva from the patient. Samples will be genotyped by Spartan Rx, a point-of-care genotyping platform, which are housed (2 of them) in the division of pathology. Loss-of-function alleles (*2, *3) and the gain-of-function allele (*17) are identified in < 60 minutes. The result of genotyping will be checked by dr. Badizadegan and entered into the patient's EMR. Clinical Decision Support (CDS) tools will advise Drs. Badizadegan and Franciosi of the patient's metabolic phenotype based on cyp2c19 genotype. Dr. Franciosi (or other GI pediatricians) will decide the dose of PPI to use based CDS tools carrying algorithms for conventional dosing and for genotype-guided dosing of PPIs. The electronic prescription for the PPI with genotype-guided dosing will be called in. PPI efficacy and safety for each study participant will be monitored weekly (aim 1B) by collecting and recording scores on validated GERD and adverse reaction questionnaires. Efficacy and safety scores will be communicated by each participant (or caregiver) using mobile devices (iPhone; computer) and recorded using redcap. Dr. Blake will supervise this phase of the study. Outcome metrics for aim 1A include: % of patients agreeing to volunteer for the study; % of patients reporting efficacy and toxicity data; % of providers agreeing to participate in study; and % of participants agreeing to future use of DNA. Outcome metrics for aim B will be judgments by Dr. Franciosi and his staff regarding success based on scores from GERD and adverse event questionnaires.
- Conditions
- Gastroesophageal Reflux
- Being tested
- Genotype Guided PPI Dosing
- Medical subject headings
- Gastroesophageal Reflux
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Genotype Guided PPI DosingDrug
Also called Prilosec, Nexium, Prevacid, Proton Pump Inhibitor
Dosing of PPIs such as Prevacid and Nexium will be recommended based on CYP2C19 genotype information.
| Study group | Role | What they receive |
|---|---|---|
| Genotype Guided PPI Dosing | Experimental | Genotype Guided PPI Dosing: PI type and dosing will be recommended in real time based on patients' CYP2C19 genotype / metabolizer phenotype.
No other ARM will be studied. Receives: Drug: Genotype Guided PPI Dosing |
What is being measured
The primary outcome is the main question the study is designed to answer.
Count of Patients Agreeing to Volunteer for the Study
Outcomes for evaluating the success of PGX implementation
Measured: Through 12 months study
Count of Patients Reporting Efficacy and Toxicity Data
Outcomes for evaluating the success of PGX implementation
Measured: Throughout 12 month study
Count of Providers Agreeing to Participate in Study
Outcomes for evaluating the success of PGX implementation
Measured: Throughout 12 month study
Count of Participants Agreeing to Future Use of DNA
Outcomes for evaluating the success of PGX implementation
Measured: Throughout 12 month study
Adverse Effects After Genotype-guided PPI Therapy
Count of participants reporting adverse effects after genotype-guided PPI therapy.
Measured: Throughout 12 month study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jun 2016 (estimated)
- Study start21 Jul 2016
- Primary completion23 Jan 2017
- Study completion31 Aug 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Gastroesophageal RefluxStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nemours Children's Clinic
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, James Franciosi, Nemours Children's Clinic
- Investigators
- James P Franciosi, MD, Principal investigator, Nemours Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02794844
- Sponsor's protocol ID
- 744230
- Acronym
- PGX
- Status last verified
- Oct 2018
- First submitted
- 27 Apr 2016
- Last updated
- 21 Nov 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Nov 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Implementation of Pharmacogenomic Testing in Nemours Children's Health System
- ClinicalTrials.gov ID
- NCT02794844
- Acronym / sponsor ID
- PGX / 744230
- Conditions
- Gastroesophageal Reflux
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 28 participants (actual)
- Lead sponsor
- Nemours Children's ClinicUniversity, hospital or other
- Study start
- 21 Jul 2016 (actual)
- Primary completion
- 23 Jan 2017 (actual)
- Study completion
- 31 Aug 2018 (actual)
- First posted
- 9 Jun 2016 (estimated)
- Results
- Posted 9 May 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Using genetic information about the individual to pick the right drug for the right disease at the right dose defines personalized medicine. This pilot study seeks to institute pharmacogenomic testing, that is identifying genetic variation that influences patient response to drugs, into the Nemours Children's Health system. We propose to initiate the study by identifying genetic differences in cyp2c19, a gene that is responsible for a certain enzyme in the liver that metabolizes many drugs including a class of drugs called proton pump inhibitors (ppi; Prevacid, Nexium). PPIs are used to treat heartburn and other symptoms of gastroesophageal reflux disease (gerd) and are extensively used in pediatrics. Chronic use of PPIs can cause serious side effects including cold, pneumonia and stomach infections, which gets worse at higher doses. Children who poorly metabolize drugs because of genetic variation in cyp2c19 should get lower doses of PPIs than children who metabolize PPIs normally. Our pilot study will genotype children with gerd or other stomach acid mediated conditions for which a PPI is prescribed using a sample of spit to determine which dose of PPI they get based on the form of the cyp2c19 gene they have. We will study 120 children 2-17 yo diagnosed with gastroesophageal reflux disease (gerd) or other stomach acid mediated conditions for which a ppi is prescribed . Genetic results are available in < 60 minutes, and their doses are determined by their doctor based on genetic results. This study will allow us to gain valuable experience that will be used to expand our genetic program to other genes and drugs.
Detailed description
The long-term objective of this research is to implement pharmacogenomic (PGX) testing of approved gene-drug pairs to maximize the efficacy and minimize adverse events for drugs that are used to treat childhood disease. Personalized medicine, that is, the use of the patient's own genetic information to predict the right dose of the right drug, has been a goal of medicine's since the publication of the human genome and HAPMAP projects. However, the adoption of PGX testing has been slow owing to several barriers and challenges. Recently President Obama announced the precision medicine initiative, which includes PGX testing, and has earmarked $ 1 b to implement it. The precision medicine initiative is expected to move personalized medicine forward so that most patients including pediatric patients will benefit. The Nemours Children's Health System is uniquely positioned to lead the effort to personalize medicine among pediatric patients. The goal of the proposed 1-year pilot project is to implement PGX testing in the Nemours children's health system. The experience, knowledge and skills gained in this pilot project will position Nemours to help lead precision medicine initiatives including PGX testing among pediatric populations. To implement PGX testing in Nemours we propose to begin testing the cyp2c19 - proton pump inhibitor gene-drug pair in the division of gastroenterology, Nemours children's hospital. A total of 120 children 2-17 yo who have been diagnosed with gastroesophageal reflux disease (GERD) or other stomach acid mediated conditions for which a PPI is prescribed will be recruited into the study by pediatric gastroenterologists (aim 1a). Drs. Franciosi and Lima will educate staff in the division of PGX testing and of the study. GI pediatricians are not required to participate in PGX testing. After obtaining consent, the study coordinator will obtain and bar code samples of saliva from the patient. Samples will be genotyped by Spartan Rx, a point-of-care genotyping platform, which are housed (2 of them) in the division of pathology. Loss-of-function alleles (*2, *3) and the gain-of-function allele (*17) are identified in < 60 minutes. The result of genotyping will be checked by dr. Badizadegan and entered into the patient's EMR. Clinical Decision Support (CDS) tools will advise Drs. Badizadegan and Franciosi of the patient's metabolic phenotype based on cyp2c19 genotype. Dr. Franciosi (or other GI pediatricians) will decide the dose of PPI to use based CDS tools carrying algorithms for conventional dosing and for genotype-guided dosing of PPIs. The electronic prescription for the PPI with genotype-guided dosing will be called in. PPI efficacy and safety for each study participant will be monitored weekly (aim 1B) by collecting and recording scores on validated GERD and adverse reaction questionnaires. Efficacy and safety scores will be communicated by each participant (or caregiver) using mobile devices (iPhone; computer) and recorded using redcap. Dr. Blake will supervise this phase of the study. Outcome metrics for aim 1A include: % of patients agreeing to volunteer for the study; % of patients reporting efficacy and toxicity data; % of providers agreeing to participate in study; and % of participants agreeing to future use of DNA. Outcome metrics for aim B will be judgments by Dr. Franciosi and his staff regarding success based on scores from GERD and adverse event questionnaires.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Genotype Guided PPI Dosing | Experimental | Drug: Genotype Guided PPI DosingGenotype Guided PPI Dosing: PI type and dosing will be recommended in real time based on patients' CYP2C19 genotype / metabolizer phenotype. No other ARM will be studied. |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Children diagnosed with Gastroesophageal Reflux Disease (GERD) or a stomach acid mediated condition for which a PPI is prescribed.
- Currently taking or will be prescribed Proton Pump Inhibitor (PPI) medication
- Parents/legal guardians and or child must also have access to the internet and a valid email address to complete weekly required forms.
Key exclusion criteria (7 listed)
- Children who have had peptic ulcer surgery;
- with a history of PKU
- with a history of previous adverse effects from PPI treatment or a sensitivity to aspartame (NutraSweet, Equal);
- who are non-adherent including inability or unwillingness of the legal guardian to provide consent of unwillingness of the child to provide assent;
- who are unable to take study medications;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Genotype Guided PPI DosingAlso: Prilosec, Nexium, Prevacid, Proton Pump InhibitorDosing of PPIs such as Prevacid and Nexium will be recommended based on CYP2C19 genotype information. | Drug | Genotype Guided PPI Dosing |
Primary outcome
Count of Patients Agreeing to Volunteer for the Study
Time frame: Through 12 months study
Outcomes for evaluating the success of PGX implementation
Count of Patients Reporting Efficacy and Toxicity Data
Time frame: Throughout 12 month study
Outcomes for evaluating the success of PGX implementation
Count of Providers Agreeing to Participate in Study
Time frame: Throughout 12 month study
Outcomes for evaluating the success of PGX implementation
Count of Participants Agreeing to Future Use of DNA
Time frame: Throughout 12 month study
Outcomes for evaluating the success of PGX implementation
Adverse Effects After Genotype-guided PPI Therapy
Time frame: Throughout 12 month study
Count of participants reporting adverse effects after genotype-guided PPI therapy.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Nov 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
James P Franciosi, MD principal investigator
Nemours Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Genotype Guided PPI Dosing in people with Gastroesophageal Reflux, run by Nemours Children's Clinic at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 21 Nov 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 9 May 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Count of Patients Agreeing to Volunteer for the Study (time frame: Through 12 months study). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Nov 2018.