Advanced CancerCC N-of-1NCT07343024
Methods of Identifying Effective Off-Guideline Treatments for Advanced Cancer Patients
An observational study of Advanced Cancer, run by Cancer Commons at 1 site in United States.
Trial summary
Many FDA-approved drugs are not available to patients with a particular cancer because there has been no successful clinical trial conducted for that drug against that cancer. In the absence of such a successful clinical trial, the drug is not included in the National Comprehensive Cancer Network (NCCN) guidelines… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Advanced Cancer
- How many people
- 300 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Mar 2026to Dec 2028
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: The study will include advanced cancer patients who have test result that can be used by the Scientific Team to identify Promising Off-guideline Drugs. The study is open to patients with any cancer.
Who may be able to join
- Participant must have a valid genomic or functional test or willing to undergo a new test
- Analysis of the participant's test must produce a set of Promising Off-guideline Drug(s)
- Oncologist must be willing to administer one or more Promising Off-guideline Drug(s)
- The study team must have an approved method of paying for the administered Off-Guideline Drug(s). Health insurance and self-pay are the two most common sources of payment
- Participant must sign the Informed Consent
Who cannot join
- No set of Promising Off-guideline Drug(s)
- Oncologist not willing to administer one or more Promising Off-guideline Drug(s)
- No approved method of paying for the administered off-guideline Drug(s)
- No signed Informed Consent
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Cancer Commons
Palo Alto, California, 94306
Clifford Reid, PhD, 877-971-1200, StudyTeam_CCCS001@cancercommons.org
Clifford Reid, PhD
About this study
Official title: Methods of Identifying Effective Off-Guideline Treatments for Advanced Cancer Patients (CC N-of-1)
Many FDA-approved drugs are not available to patients with a particular cancer because there has been no successful clinical trial conducted for that drug against that cancer. In the absence of such a successful clinical trial, the drug is not included in the National Comprehensive Cancer Network (NCCN) guidelines list of approved drugs for that cancer. The absence of a drug in the NCCN Guidelines for a particular cancer is usually not an indication that the drug has been shown to be ineffective for patients with that cancer. Rather, it is an indication that there is insufficient clinical trial evidence to include it. A drug that is FDA-approved for one or more cancers but is not in the NCCN Guidelines for a particular cancer is called an "off-guideline" drug for that cancer.
This study is being done to measure and compare the reliability of multiple different treatment selection tests to predict a participant's response to an off- guideline cancer therapy. The results can guide oncologists to effective off-guideline drugs that would otherwise not be available to their advanced cancer patients. As this is an observational study, all the data gathered and analyzed will be generated in the normal practice of medicine, not by the study.
Read the detailed description
- Significance: This is the first precision medicine study that aims to generate predictive accuracy of functional assays in selecting effective off-guideline treatments. Since the clinical evidence of these assays is severely limited, and as a result, oncologists are reluctant to use them. This study will add to that body of clinical evidence, and if successful, accelerate the acceptance of these functional assays for treatment selection in clinical practice. B) Specific Aims: This study will calculate the predictive accuracy of multiple functional assays (genomic and/or DST) to identify the tests that can be most helpful to patients, and to quantify the potential benefits of these tests. C) Methodology: The Participant's genomic and/ or DST results will be collected and reviewed by the Cancer Commons Scientific team. Furthermore, a literature review relevant to the Participant's cancer will be performed to identify the use of off-guideline drugs. Based on this analysis, the Scientific Team will write a Scientific Report that identifies any Promising Off-guideline Drugs and share with the Participant's oncologist to determine the merit of administering any off-guideline drug or drugs. If a Participant's oncologist is willing to administer one or more off-guideline drugs, the Participant is enrolled in the study. The Participant's oncologist administers one or more off-guideline drugs, and the subsequent Participant results are recorded in the study database. Note that the Participant's oncologist makes all treatment decisions unilaterally. Arms of the Study: The study will consist of multiple assay arms that belong to the categories shown below. Each arm will belong to a specific test. 1. Genomic Mutation Match: Select one or more off-guideline drugs that are indicated for a specific mutation which a genomic test shows to be present in the participant's cancer DNA, 2. Genomic Pathway Match: Select one or more off-guideline drugs that are not indicated for a specific mutation which a genomic test shows to be present in the participant's cancer DNA, but are indicated for a cancer pathway that genomics experts believe are strongly related to a specific mutation which a genomic test shows to be present in the participant's cancer DNA, 3. Drug Sensitivity Assays: Select an off-guideline drug or drugs that are designed as "response" by the test. D) Patient Population: The study will include advanced cancer patients who have test result that can be used by the Scientific Team to identify Promising Off-guideline Drugs. The study is open to patients with any cancer.
- Conditions
- Advanced Cancer
- Keywords
- Off-Guideline, Advanced Cancer Treatments, Functional Precision Medicine, Drug Sensitivity Tests, Treatment Selection Tests, Genomic Tests, Off-label drugs, Predictive Accuracy of tests
What is being measured
The primary outcome is the main question the study is designed to answer.
The predictive accuracy for each drug sensitivity testing (DST) used in the study
For each participant in the study who is treated with an off-guideline drug, the oncologist will provide the study with a binary assessment of "response" or "no response" from the participant, using the RECIST criteria or other criteria proposed by the treating oncologist. For each participant, we will compare the test prediction of "response" or "no response" to the oncologist assessment of "response" and "no response" to determine if the test prediction was correct or incorrect. For each test, we will compute the predictive accuracy of the test by dividing the number of correct predictions by dividing the number of correct predictions by the total number of predictions made by the test in the course of the study. For example, if a test made fifty (50) predictions and thirty five (35) of those predictions were correct, then the predictive accuracy of the test would be 35/50 = 70%.
Measured: Through study completion, an average of 1 year
Secondary outcomes (1)
The overall patient response rate for each DST-guided therapy and for each cancer.
To compare individual outcomes (response and disease-free survival) in patients treated with DST-guided therapy as compared to non-DST guided (conventional) therapy.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jan 2026
- Study start30 Mar 2026
- Primary completionDec 2028 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Advanced CancerStudies 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
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 300 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Cancer Commons
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07343024
- Sponsor's protocol ID
- CCCS001
- Acronym
- CC N-of-1
- Status last verified
- Sep 2026
- First submitted
- 11 Dec 2025
- Last updated
- 8 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Sadik H, Pritchard D, Keeling DM, Policht F, Riccelli P, Stone G, Finkel K, Schreier J, Munksted S. Impact of Clinical Practice Gaps on the Implementation of Personalized Medicine in Advanced Non-Small-Cell Lung Cancer. JCO Precis Oncol. 2022 Oct;6:e2200246. doi: 10.1200/PO.22.00246.
PubMed 36315914Liu R, Wang L, Rizzo S, Garmhausen MR, Pal N, Waliany S, McGough S, Lin YG, Huang Z, Neal J, Copping R, Zou J. Systematic analysis of off-label and off-guideline cancer therapy usage in a real-world cohort of 165,912 US patients. Cell Rep Med. 2024 Mar 19;5(3):101444. doi: 10.1016/j.xcrm.2024.101444. Epub 2024 Feb 29.
PubMed 38428426Acanda de la Rocha AM, Berlow NE, Azzam DJ. Functional precision medicine: the future of cancer care. Trends Mol Med. 2025 May;31(5):404-408. doi: 10.1016/j.molmed.2024.10.015. Epub 2024 Nov 19.
PubMed 39567286Kornauth C, Pemovska T, Vladimer GI, Bayer G, Bergmann M, Eder S, Eichner R, Erl M, Esterbauer H, Exner R, Felsleitner-Hauer V, Forte M, Gaiger A, Geissler K, Greinix HT, Gstottner W, Hacker M, Hartmann BL, Hauswirth AW, Heinemann T, Heintel D, Hoda MA, Hopfinger G, Jaeger U, Kazianka L, Kenner L, Kiesewetter B, Krall N, Krajnik G, Kubicek S, Le T, Lubowitzki S, Mayerhoefer ME, Menschel E, Merkel O, Miura K, Mullauer L, Neumeister P, Noesslinger T, Ocko K, Ohler L, Panny M, Pichler A, Porpaczy E, Prager GW, Raderer M, Ristl R, Ruckser R, Salamon J, Schiefer AI, Schmolke AS, Schwarzinger I, Selzer E, Sillaber C, Skrabs C, Sperr WR, Srndic I, Thalhammer R, Valent P, van der Kouwe E, Vanura K, Vogt S, Waldstein C, Wolf D, Zielinski CC, Zojer N, Simonitsch-Klupp I, Superti-Furga G, Snijder B, Staber PB. Functional Precision Medicine Provides Clinical Benefit in Advanced Aggressive Hematologic Cancers and Identifies Exceptional Responders. Cancer Discov. 2022 Feb;12(2):372-387. doi: 10.1158/2159-8290.CD-21-0538. Epub 2021 Oct 11.
PubMed 34635570
Study at a glance
From the registry record, last updated by the sponsor on 8 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Methods of Identifying Effective Off-Guideline Treatments for Advanced Cancer Patients (CC N-of-1)
- ClinicalTrials.gov ID
- NCT07343024
- Acronym / sponsor ID
- CC N-of-1 / CCCS001
- Conditions
- Advanced Cancer
- Keywords
- Off-Guideline, Advanced Cancer Treatments, Functional Precision Medicine, Drug Sensitivity Tests, Treatment Selection Tests, Genomic Tests, Off-label drugs, Predictive Accuracy of tests
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- Cancer CommonsUniversity, hospital or other
- Study start
- 30 Mar 2026 (actual)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 15 Jan 2026 (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.
Many FDA-approved drugs are not available to patients with a particular cancer because there has been no successful clinical trial conducted for that drug against that cancer. In the absence of such a successful clinical trial, the drug is not included in the National Comprehensive Cancer Network (NCCN) guidelines list of approved drugs for that cancer. The absence of a drug in the NCCN Guidelines for a particular cancer is usually not an indication that the drug has been shown to be ineffective for patients with that cancer. Rather, it is an indication that there is insufficient clinical trial evidence to include it. A drug that is FDA-approved for one or more cancers but is not in the NCCN Guidelines for a particular cancer is called an "off-guideline" drug for that cancer.
This study is being done to measure and compare the reliability of multiple different treatment selection tests to predict a participant's response to an off- guideline cancer therapy. The results can guide oncologists to effective off-guideline drugs that would otherwise not be available to their advanced cancer patients. As this is an observational study, all the data gathered and analyzed will be generated in the normal practice of medicine, not by the study.
Detailed description
- Significance: This is the first precision medicine study that aims to generate predictive accuracy of functional assays in selecting effective off-guideline treatments. Since the clinical evidence of these assays is severely limited, and as a result, oncologists are reluctant to use them. This study will add to that body of clinical evidence, and if successful, accelerate the acceptance of these functional assays for treatment selection in clinical practice. B) Specific Aims: This study will calculate the predictive accuracy of multiple functional assays (genomic and/or DST) to identify the tests that can be most helpful to patients, and to quantify the potential benefits of these tests. C) Methodology: The Participant's genomic and/ or DST results will be collected and reviewed by the Cancer Commons Scientific team. Furthermore, a literature review relevant to the Participant's cancer will be performed to identify the use of off-guideline drugs. Based on this analysis, the Scientific Team will write a Scientific Report that identifies any Promising Off-guideline Drugs and share with the Participant's oncologist to determine the merit of administering any off-guideline drug or drugs. If a Participant's oncologist is willing to administer one or more off-guideline drugs, the Participant is enrolled in the study. The Participant's oncologist administers one or more off-guideline drugs, and the subsequent Participant results are recorded in the study database. Note that the Participant's oncologist makes all treatment decisions unilaterally. Arms of the Study: The study will consist of multiple assay arms that belong to the categories shown below. Each arm will belong to a specific test. 1. Genomic Mutation Match: Select one or more off-guideline drugs that are indicated for a specific mutation which a genomic test shows to be present in the participant's cancer DNA, 2. Genomic Pathway Match: Select one or more off-guideline drugs that are not indicated for a specific mutation which a genomic test shows to be present in the participant's cancer DNA, but are indicated for a cancer pathway that genomics experts believe are strongly related to a specific mutation which a genomic test shows to be present in the participant's cancer DNA, 3. Drug Sensitivity Assays: Select an off-guideline drug or drugs that are designed as "response" by the test. D) Patient Population: The study will include advanced cancer patients who have test result that can be used by the Scientific Team to identify Promising Off-guideline Drugs. The study is open to patients with any cancer.
Study design
- Observational model
- Other
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The study will include advanced cancer patients who have test result that can be used by the Scientific Team to identify Promising Off-guideline Drugs. The study is open to patients with any cancer.
Key inclusion criteria (5 listed)
- Participant must have a valid genomic or functional test or willing to undergo a new test
- Analysis of the participant's test must produce a set of Promising Off-guideline Drug(s)
- Oncologist must be willing to administer one or more Promising Off-guideline Drug(s)
- The study team must have an approved method of paying for the administered Off-Guideline Drug(s). Health insurance and self-pay are the two most common sources of payment
- Participant must sign the Informed Consent
Key exclusion criteria (4 listed)
- No set of Promising Off-guideline Drug(s)
- Oncologist not willing to administer one or more Promising Off-guideline Drug(s)
- No approved method of paying for the administered off-guideline Drug(s)
- No signed Informed Consent
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
The predictive accuracy for each drug sensitivity testing (DST) used in the study
Time frame: Through study completion, an average of 1 year
For each participant in the study who is treated with an off-guideline drug, the oncologist will provide the study with a binary assessment of "response" or "no response" from the participant, using the RECIST criteria or other criteria proposed by the treating oncologist. For each participant, we will compare the test prediction of "response" or "no response" to the oncologist assessment of "response" and "no response" to determine if the test prediction was correct or incorrect. For each test, we will compute the predictive accuracy of the test by dividing the number of correct predictions by dividing the number of correct predictions by the total number of predictions made by the test in the course of the study. For example, if a test made fifty (50) predictions and thirty five (35) of those predictions were correct, then the predictive accuracy of the test would be 35/50 = 70%.
1 secondary outcome
The overall patient response rate for each DST-guided therapy and for each cancer.
Time frame: 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Oct 2026.
- Background: PubMed 36315914Sadik H, Pritchard D, Keeling DM, Policht F, Riccelli P, Stone G, Finkel K, Schreier J, Munksted S. Impact of Clinical Practice Gaps on the Implementation of Personalized Medicine in Advanced Non-Small-Cell Lung Cancer. JCO Precis Oncol. 2022 Oct;6:e2200246. doi: 10.1200/PO.22.00246.
- Background: PubMed 38428426Liu R, Wang L, Rizzo S, Garmhausen MR, Pal N, Waliany S, McGough S, Lin YG, Huang Z, Neal J, Copping R, Zou J. Systematic analysis of off-label and off-guideline cancer therapy usage in a real-world cohort of 165,912 US patients. Cell Rep Med. 2024 Mar 19;5(3):101444. doi: 10.1016/j.xcrm.2024.101444. Epub 2024 Feb 29.
- Background: PubMed 39567286Acanda de la Rocha AM, Berlow NE, Azzam DJ. Functional precision medicine: the future of cancer care. Trends Mol Med. 2025 May;31(5):404-408. doi: 10.1016/j.molmed.2024.10.015. Epub 2024 Nov 19.
- Background: PubMed 34635570Kornauth C, Pemovska T, Vladimer GI, Bayer G, Bergmann M, Eder S, Eichner R, Erl M, Esterbauer H, Exner R, Felsleitner-Hauer V, Forte M, Gaiger A, Geissler K, Greinix HT, Gstottner W, Hacker M, Hartmann BL, Hauswirth AW, Heinemann T, Heintel D, Hoda MA, Hopfinger G, Jaeger U, Kazianka L, Kenner L, Kiesewetter B, Krall N, Krajnik G, Kubicek S, Le T, Lubowitzki S, Mayerhoefer ME, Menschel E, Merkel O, Miura K, Mullauer L, Neumeister P, Noesslinger T, Ocko K, Ohler L, Panny M, Pichler A, Porpaczy E, Prager GW, Raderer M, Ristl R, Ruckser R, Salamon J, Schiefer AI, Schmolke AS, Schwarzinger I, Selzer E, Sillaber C, Skrabs C, Sperr WR, Srndic I, Thalhammer R, Valent P, van der Kouwe E, Vanura K, Vogt S, Waldstein C, Wolf D, Zielinski CC, Zojer N, Simonitsch-Klupp I, Superti-Furga G, Snijder B, Staber PB. Functional Precision Medicine Provides Clinical Benefit in Advanced Aggressive Hematologic Cancers and Identifies Exceptional Responders. Cancer Discov. 2022 Feb;12(2):372-387. doi: 10.1158/2159-8290.CD-21-0538. Epub 2021 Oct 11.
- Individual participant dataSharing planned: No
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Advanced Cancer, run by Cancer Commons 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 5 inclusion and 4 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: looking for participants now, as of the sponsor's last update on 8 Oct 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The predictive accuracy for each drug sensitivity testing (DST) used in the study (time frame: Through study completion, an average of 1 year). 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 NCT07343024, which also lists 4 linked publications.
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 8 Oct 2026.