Gastrointestinal CancerNCT04541381
The PhOCus Trial: Implementation of Pharmacogenomic Testing in Oncology Care
A study testing Availability of clinical decision support based on pharmacogenomic results. in people with Gastrointestinal Cancer and Head and Neck Cancer, run by University of Chicago at 1 site in United States.
Trial summary
Doctors leading this study hope to find out if giving study participants' genetic information to cancer care providers will help personalize chemotherapy dosing decisions and decrease common chemotherapy side effects. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Gastrointestinal Cancer, Head and Neck Cancer, Dihydropyrimidine Dehydrogenase Deficiency
- What is being tested?
- Availability of clinical decision support based on pharmacogenomic results.
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 860 participants
- Where
- 1 siteUnited States
- Study dates
- 7 Feb 2022to 31 Mar 2028
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
None listed.
Who cannot join
- Subjects who have previously been exposed to the planned chemotherapy agent at any time (fluoropyrimidine and/or irinotecan).
- Subjects enrolled in an investigational trial which would preclude dose modifications of fluoropyrimidine and/or irinotecan chemotherapies.
- Subjects who have undergone, or are being actively considered for, bone marrow, liver or kidney transplantation.
- Subjects with a history of or active blood cancer (e.g., leukemia).
- Chronic kidney disease, as defined by glomerular filtration rate (GFR) < 30/mL/min/1.73m2, due to the risk of decreased drug excretion.
- Liver dysfunction, as defined by the following laboratory values, due to the risk of decreased drug metabolism: Total bilirubin more than 1.5 mg/dL, aspartate Aminotransferase (AST) and alanine transaminase (ALT) more than 2.5 X upper limit of normal*. (*AST and ALT more than 5 X upper limit of normal if hepatic metastases are present).
- Subjects who have previously or are currently enrolled in another institutional pharmacogenomic genotyping study, or are known to have previously undergone pharmacogenomic genotyping for the gene(s) of interest via another commercial or other means.
- Inability to understand and give informed consent to participate.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Chicago Medical Center
Chicago, Illinois, 60637
About this study
Doctors leading this study hope to find out if giving study participants' genetic information to cancer care providers will help personalize chemotherapy dosing decisions and decrease common chemotherapy side effects. Doctors leading the study will collect genetic information from study participants using pharmacogenomics/genotyping. Pharmacogenomics is the study of how the differences in our genes can affect our unique response to medications.
This is a randomized study, which means that participants in this study will be randomly assigned (as if "by flip of a coin") to one of two different groups: a "pharmacogenomics group" or "control group".
- Conditions
- Gastrointestinal Cancer, Head and Neck Cancer, Dihydropyrimidine Dehydrogenase Deficiency, UGT1A1 Gene Mutation, Breast Cancer
- Being tested
- Availability of clinical decision support based on pharmacogenomic results.
- Keywords
- Pharmacogenomics
- Medical subject headings
- Gastrointestinal Neoplasms, Head and Neck Neoplasms, Dihydropyrimidine Dehydrogenase Deficiency, Breast Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Availability of clinical decision support based on pharmacogenomic results.Other
Availability of clinical decision support based on pharmacogenomic results. These results are designed to provide specific dosing information based on the participant's unique genetics/genomics.
| Study group | Role | What they receive |
|---|---|---|
| Control Group | No intervention | Participants assigned to the control group will receive standard chemotherapy without their doctors receiving any genetic information based on the participants' pharmacogenetic results. DNA (Deoxyribonucleic acid) samples for participants in this group will be stored and tested for genotyping six months later after treatment (or earlier if the participant experiences side effects). |
| Pharmacogenomics Group | Experimental | Participants enrolled in the pharmacogenomics group will give a DNA (deoxyribonucleic acid) sample for immediate pharmacogenomic genotyping. Once the genotyping results are in, cancer doctors caring for each participant will have immediate access to clinical decision support based on the participant's genetic results and can make dosing decisions/changes to the participant's chemotherapy prescription. Receives: Other: Availability of clinical decision support based on pharmacogenomic results. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dose Deviation Rate (Co-Primary Endpoint)
To assess the impact of prospective pharmacogenomic testing on dose intensity deviation rate of chemotherapy during the 1st treatment cycle, comparing control vs. pharmacogenomics-guided arms.
Measured: 15 months
Grade 3 or Higher Toxicity (Co-Primary Endpoint)
To determine the degree to which providing oncologists with comprehensive pharmacogenomic information impacts the incidence of Grade 3 or worse toxicities in subjects receiving chemotherapy. Toxicities will be assessed by Common Terminology Criteria for Adverse Events version 5.
Measured: 5 years
Secondary outcomes (4)
Cumulative Chemotherapy Dose Intensity
Cumulative drug dose intensity received (function of dose and frequency of drug administration).
Measured: 5 years.
Response Rate
Anti-cancer tumor response based on radiographic assessment (complete response, partial response, stable disease, progressive disease), by tumor type and disease setting.
Measured: 5 years.
Progression free survival (PFS)
Progression free survival (PFS) by tumor type and disease setting.
Measured: 5 years
Overall Survival
Overall survival (OS) by tumor type and disease setting.
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Sep 2020
- Study start7 Feb 2022
- Primary completion1 Oct 2027 (estimated)
- Study completion31 Mar 2028 (estimated)
Why it stopped: pending study amendment/re-design
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Gastrointestinal CancerStudies of the same condition, open and under way.
- More trials run by University of ChicagoThe sponsor's other registered studies.
- 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 N/A
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 860 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Chicago
- Sponsor type
- University, hospital or other
- Collaborators
- National Human Genome Research Institute (NHGRI)
- Responsible party
- Sponsor
- Investigators
- Peter H. O'Donnell, MD, Principal investigator, University of Chicago
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04541381
- Sponsor's protocol ID
- IRB20-0462
- Other IDs
- 5R01HG012273
- Status last verified
- May 2026
- First submitted
- 1 Sep 2020
- Last updated
- 7 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes
Publications
Cho Y, Elahi S, Jack MM, Campbell J, Smith E, Knoebel RW, George D, House L, Yeo KJ, Karrison T, Volchenboum SL, Meltzer DO, Szmulewitz RZ, Vokes EE, Ratain MJ, O'Donnell PH. Impact of First-of-Its-Kind Patient-Facing Pharmacogenetics Tool on Dosing Decisions and Treatment Outcomes. Clin Pharmacol Ther. 2025 Oct;118(4):906-916. doi: 10.1002/cpt.3747. Epub 2025 Jun 27.
PubMed 40579854
Study at a glance
From the registry record, last updated by the sponsor on 7 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The PhOCus Trial: Implementation of Pharmacogenomic Testing in Oncology Care
- ClinicalTrials.gov ID
- NCT04541381
- Acronym / sponsor ID
- IRB20-0462
- Conditions
- Gastrointestinal Cancer, Head and Neck Cancer, Dihydropyrimidine Dehydrogenase Deficiency, UGT1A1 Gene Mutation, Breast Cancer
- Keywords
- Pharmacogenomics
- Study type
- Interventional
- Recruitment status
- SuspendedReason stopped: pending study amendment/re-design
- Enrolment
- 860 participants (estimated)
- Lead sponsor
- University of ChicagoUniversity, hospital or other
- Collaborators
- National Human Genome Research Institute (NHGRI)
- Study start
- 7 Feb 2022 (actual)
- Primary completion
- 1 Oct 2027 (estimated)
- Study completion
- 31 Mar 2028 (estimated)
- First posted
- 9 Sep 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Doctors leading this study hope to find out if giving study participants' genetic information to cancer care providers will help personalize chemotherapy dosing decisions and decrease common chemotherapy side effects. Doctors leading the study will collect genetic information from study participants using pharmacogenomics/genotyping. Pharmacogenomics is the study of how the differences in our genes can affect our unique response to medications.
This is a randomized study, which means that participants in this study will be randomly assigned (as if "by flip of a coin") to one of two different groups: a "pharmacogenomics group" or "control group".
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Control Group | No intervention | –Participants assigned to the control group will receive standard chemotherapy without their doctors receiving any genetic information based on the participants' pharmacogenetic results. DNA (Deoxyribonucleic acid) samples for participants in this group will be stored and tested for genotyping six months later after treatment (or earlier if the participant experiences side effects). |
| Pharmacogenomics Group | Experimental | Other: Availability of clinical decision support based on pharmacogenomic results.Participants enrolled in the pharmacogenomics group will give a DNA (deoxyribonucleic acid) sample for immediate pharmacogenomic genotyping. Once the genotyping results are in, cancer doctors caring for each participant will have immediate access to clinical decision support based on the participant's genetic results and can make dosing decisions/changes to the participant's chemotherapy prescription. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (0 listed)
Key exclusion criteria (8 listed)
- Subjects who have previously been exposed to the planned chemotherapy agent at any time (fluoropyrimidine and/or irinotecan).
- Subjects enrolled in an investigational trial which would preclude dose modifications of fluoropyrimidine and/or irinotecan chemotherapies.
- Subjects who have undergone, or are being actively considered for, bone marrow, liver or kidney transplantation.
- Subjects with a history of or active blood cancer (e.g., leukemia).
- Chronic kidney disease, as defined by glomerular filtration rate (GFR) < 30/mL/min/1.73m2, due to the risk of decreased drug excretion.
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 |
|---|---|---|
| Availability of clinical decision support based on pharmacogenomic results.Availability of clinical decision support based on pharmacogenomic results. These results are designed to provide specific dosing information based on the participant's unique genetics/genomics. | Other | Pharmacogenomics Group |
Primary outcome
Dose Deviation Rate (Co-Primary Endpoint)
Time frame: 15 months
To assess the impact of prospective pharmacogenomic testing on dose intensity deviation rate of chemotherapy during the 1st treatment cycle, comparing control vs. pharmacogenomics-guided arms.
Grade 3 or Higher Toxicity (Co-Primary Endpoint)
Time frame: 5 years
To determine the degree to which providing oncologists with comprehensive pharmacogenomic information impacts the incidence of Grade 3 or worse toxicities in subjects receiving chemotherapy. Toxicities will be assessed by Common Terminology Criteria for Adverse Events version 5.
4 secondary outcomes
Cumulative Chemotherapy Dose Intensity
Time frame: 5 years.
Response Rate
Time frame: 5 years.
Progression free survival (PFS)
Time frame: 5 years
Overall Survival
Time frame: 5 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 May 2026.
- Derived from this study: PubMed 40579854Cho Y, Elahi S, Jack MM, Campbell J, Smith E, Knoebel RW, George D, House L, Yeo KJ, Karrison T, Volchenboum SL, Meltzer DO, Szmulewitz RZ, Vokes EE, Ratain MJ, O'Donnell PH. Impact of First-of-Its-Kind Patient-Facing Pharmacogenetics Tool on Dosing Decisions and Treatment Outcomes. Clin Pharmacol Ther. 2025 Oct;118(4):906-916. doi: 10.1002/cpt.3747. Epub 2025 Jun 27.
- Individual participant dataSharing planned: Yes
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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.
Peter H. O'Donnell, MD principal investigator
University of Chicago
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Availability of clinical decision support based on pharmacogenomic results. in people with Gastrointestinal Cancer and Head and Neck Cancer, run by University of Chicago 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 0 inclusion and 8 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: paused, as of the sponsor's last update on 7 May 2026. 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.
Are results available for this study?
Results are not available in the source record.
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?
Dose Deviation Rate (Co-Primary Endpoint) (time frame: 15 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04541381, which also lists 1 linked publication.
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 7 May 2026.