Non-small Cell Lung CancerNCT05908799
Emulation of the KEYNOTE-189 Trial Using Electronic Health Records
An observational study of Non-small Cell Lung Cancer and Metastatic Lung Cancer, run by Aetion, Inc. at 1 site in United States.
Trial summary
Investigators are building an empirical evidence base supporting the utility of real-world data through the emulation of randomized controlled trials in the oncology setting. The purpose of this work is to demonstrate whether real-world evidence studies can provide reliable conclusions on treatment effectiveness to… Read more about this study
- Who is it for?
- Any ageHealthy volunteers can take part
- Conditions the study is for
- Non-small Cell Lung Cancer, Metastatic Lung Cancer
- What is being tested?
- Pembrolizumab + Pemetrexed-Platinum and Pemetrexed-Platinum
- How many people
- 1,854 participants
- Where
- 1 siteUnited States
- Study dates
- 5 Jun 2023to 31 Aug 2023
- Phase
- Observational
- 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 Any age
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: Non-small cell lung cancer patients with evidence of metastatic disease and a subsequent record indicating a first-line treatment.
Who may be able to join
- Has evidence in electronic health record indicating a 'lung' tumor site, non-squamous morphology (e.g., adenocarcinoma, large cell, etc), and a stage IV or metastatic disease in the form of diagnosis codes or derived variables from physician notes or linked data from a tumor registry. There is no evidence of other lung cancer types (i.e., mesothelioma, small cell, etc.).
- Does not have evidence of HGVS codes in health record indicating EGFR or ALK mutations.
- Has no evidence of use of guideline-recommended systemic cancer therapy* for NSCLC on or after first date associated with stage IV (or metastatic) disease and prior to index treatment.
Who cannot join
- Has evidence of squamous cell lung cancer morphology in the form of diagnosis codes or derived variables from physician notes or linked information from a tumor registry.
- Has evidence of systemic cancer therapy utilization on or after first date associated with stage IV (or metastatic) disease and prior to index treatment. Patient has no evidence of biologic therapy at any time prior to the first record indicating an exposure of interest.
- Has evidence in medical record or tumor registry of primary malignancies beyond the lung tissue, except basal cell carcinoma of the skin, superficial bladder cancer, squamous cell carcinoma of the skin, in situ cervical cancer, or other in situ cancers.
- Has evidence of central nervous system (CNS) metastases and/or carcinomatous meningitis in the 2 weeks prior to initiation.
- Has a record of use of one of the following agents in the 2 years prior to the index date: methotrexate, sulfasalazine, hydroxychloroquine, leflunomide, azathioprine, etanercept, adalimumab, infliximab, certolizumab, golimumab, anakinra, tocilizumab, sarilumumab, abatacept, rituximab, tofacitinib, barivitinib, upadacitinib, mycophenolate.
- Has at least two records 60 days apart indicating use of one of the following agents in the 3 months prior to the index date: prednisone, prednisolone, methylprednisolone, dexamethasone, hydrocortisone.
- Has evidence of use of one of the following agents prior to the index date: pembrolizumab, nivolumab, cemiplimab, atezolizumab, durvalumab.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Emulation of a Comparative Effectiveness Study of Pembrolizumab and Chemotherapy vs. Chemotherapy for the First-line Treatment of Metastatic Non-small Cell Lung Cancer
Investigators are building an empirical evidence base supporting the utility of real-world data through the emulation of randomized controlled trials in the oncology setting. The purpose of this work is to demonstrate whether real-world evidence studies can provide reliable conclusions on treatment effectiveness to inform further applications of real-world data in pharmaceutical product label expansion, post-marketing safety, and other purposes that are complementary to RCTs.
Read the detailed description
This is a non-randomized observational study that is being conducted as a part of the Coalition to Advance Real-World Evidence through Randomized Controlled Trial Emulation (CARE) Initiative, a multi-stakeholder collaboration led by Aetion. The study is intended to emulate the trial listed below/above. Although many features of the trial cannot be directly replicated in real-world data sources, key elements of the study design, including outcomes, exposures, and inclusion/exclusion criteria, were selected to proxy those features from the trial as closely as possible. Investigators assume that the trial provides the reference standard treatment effect estimate and that failure to replicate trial findings is indicative of the inadequacy of the real-world data source for replication for a range of possible reasons and does not provide information on the validity of the original trial finding.
- Conditions
- Non-small Cell Lung Cancer, Metastatic Lung Cancer
- Being tested
- Pembrolizumab + Pemetrexed-Platinum, Pemetrexed-Platinum
- Keywords
- real-world evidence, real-world data, oncology, pharmacoepidemiology, comparative effectiveness research
- Medical subject headings
- Carcinoma, Non-Small-Cell Lung, Lung Neoplasms, Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Pembrolizumab + Pemetrexed-PlatinumDrug
Medication records in EHR database used to define exposure group
Pemetrexed-PlatinumDrug
Medication records in EHR database used to define comparator group
| Study group | Role | What they receive |
|---|---|---|
| Pembrolizumab + Chemotherapy | Exposure Group Receives: Drug: Pembrolizumab + Pemetrexed-Platinum | |
| Chemotherapy | Reference Group Receives: Drug: Pemetrexed-Platinum |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Survival - Mortality Hazard Ratio
Relative hazard of mortality due to any cause
Measured: Through study completion (earliest of 640 days or censoring)
Overall Survival - Mortality Risk
12-month survival probability
Measured: 1 year, year 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jun 2023
- Study start5 Jun 2023
- Primary completion31 Aug 2023
- Study completion31 Aug 2023
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 Non-small Cell Lung 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
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 1,854 participants (actual)
Sponsor and investigators
- Lead sponsor
- Aetion, Inc.
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, David Merola, Aetion, Inc.
- Investigators
- David Merola, PharmD, PhD, Principal investigator, Aetion, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05908799
- Sponsor's protocol ID
- 1
- Status last verified
- Sep 2023
- First submitted
- 7 Jun 2023
- Last updated
- 13 Sep 2023
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided: Data analysis code and de-identified patient-level data files may be made available, provided that it is determined that this will not violate the investigators' data usage agreements and patient privacy requirements.
Publications
Merola D, Campbell U, Gautam N, Rubens A, Schneeweiss S, Wang SV, Carrigan G, Chia V, Ovbiosa OE, Pinheiro S, Bruno A, Jiao X, Stewart M, Hendricks-Sturrup R, Rodriguez-Watson C, Khosla S, Zhang Y, Rimawi M, Huang J, Taylor A, Becnel L, McRoy L, Eckert J, Taylor B. The Aetion Coalition to Advance Real-World Evidence through Randomized Controlled Trial Emulation Initiative: Oncology. Clin Pharmacol Ther. 2023 Jun;113(6):1217-1222. doi: 10.1002/cpt.2800. Epub 2022 Dec 7.
PubMed 36408668
Study at a glance
From the registry record, last updated by the sponsor on 13 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Emulation of a Comparative Effectiveness Study of Pembrolizumab and Chemotherapy vs. Chemotherapy for the First-line Treatment of Metastatic Non-small Cell Lung Cancer
- ClinicalTrials.gov ID
- NCT05908799
- Acronym / sponsor ID
- 1
- Conditions
- Non-small Cell Lung Cancer, Metastatic Lung Cancer
- Keywords
- real-world evidence, real-world data, oncology, pharmacoepidemiology, comparative effectiveness research
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 1,854 participants (actual)
- Lead sponsor
- Aetion, Inc.University, hospital or other
- Study start
- 5 Jun 2023 (actual)
- Primary completion
- 31 Aug 2023 (actual)
- Study completion
- 31 Aug 2023 (actual)
- First posted
- 18 Jun 2023 (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.
Investigators are building an empirical evidence base supporting the utility of real-world data through the emulation of randomized controlled trials in the oncology setting. The purpose of this work is to demonstrate whether real-world evidence studies can provide reliable conclusions on treatment effectiveness to inform further applications of real-world data in pharmaceutical product label expansion, post-marketing safety, and other purposes that are complementary to RCTs.
Detailed description
This is a non-randomized observational study that is being conducted as a part of the Coalition to Advance Real-World Evidence through Randomized Controlled Trial Emulation (CARE) Initiative, a multi-stakeholder collaboration led by Aetion. The study is intended to emulate the trial listed below/above. Although many features of the trial cannot be directly replicated in real-world data sources, key elements of the study design, including outcomes, exposures, and inclusion/exclusion criteria, were selected to proxy those features from the trial as closely as possible. Investigators assume that the trial provides the reference standard treatment effect estimate and that failure to replicate trial findings is indicative of the inadequacy of the real-world data source for replication for a range of possible reasons and does not provide information on the validity of the original trial finding.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Pembrolizumab + Chemotherapy | – | Drug: Pembrolizumab + Pemetrexed-PlatinumExposure Group |
| Chemotherapy | – | Drug: Pemetrexed-PlatinumReference Group |
Who may qualify
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Non-small cell lung cancer patients with evidence of metastatic disease and a subsequent record indicating a first-line treatment.
Key inclusion criteria (3 listed)
- Has evidence in electronic health record indicating a 'lung' tumor site, non-squamous morphology (e.g., adenocarcinoma, large cell, etc), and a stage IV or metastatic disease in the form of diagnosis codes or derived variables from physician notes or linked data from a tumor registry. There is no evidence of other lung cancer types (i.e., mesothelioma, small cell, etc.).
- Does not have evidence of HGVS codes in health record indicating EGFR or ALK mutations.
- Has no evidence of use of guideline-recommended systemic cancer therapy* for NSCLC on or after first date associated with stage IV (or metastatic) disease and prior to index treatment.
Key exclusion criteria (7 listed)
- Has evidence of squamous cell lung cancer morphology in the form of diagnosis codes or derived variables from physician notes or linked information from a tumor registry.
- Has evidence of systemic cancer therapy utilization on or after first date associated with stage IV (or metastatic) disease and prior to index treatment. Patient has no evidence of biologic therapy at any time prior to the first record indicating an exposure of interest.
- Has evidence in medical record or tumor registry of primary malignancies beyond the lung tissue, except basal cell carcinoma of the skin, superficial bladder cancer, squamous cell carcinoma of the skin, in situ cervical cancer, or other in situ cancers.
- Has evidence of central nervous system (CNS) metastases and/or carcinomatous meningitis in the 2 weeks prior to initiation.
- Has a record of use of one of the following agents in the 2 years prior to the index date: methotrexate, sulfasalazine, hydroxychloroquine, leflunomide, azathioprine, etanercept, adalimumab, infliximab, certolizumab, golimumab, anakinra, tocilizumab, sarilumumab, abatacept, rituximab, tofacitinib, barivitinib, upadacitinib, mycophenolate.
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 |
|---|---|---|
| Pembrolizumab + Pemetrexed-PlatinumMedication records in EHR database used to define exposure group | Drug | Pembrolizumab + Chemotherapy |
| Pemetrexed-PlatinumMedication records in EHR database used to define comparator group | Drug | Chemotherapy |
Primary outcome
Overall Survival - Mortality Hazard Ratio
Time frame: Through study completion (earliest of 640 days or censoring)
Relative hazard of mortality due to any cause
Overall Survival - Mortality Risk
Time frame: 1 year, year 1
12-month survival probability
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Sep 2023.
- Background: PubMed 36408668Merola D, Campbell U, Gautam N, Rubens A, Schneeweiss S, Wang SV, Carrigan G, Chia V, Ovbiosa OE, Pinheiro S, Bruno A, Jiao X, Stewart M, Hendricks-Sturrup R, Rodriguez-Watson C, Khosla S, Zhang Y, Rimawi M, Huang J, Taylor A, Becnel L, McRoy L, Eckert J, Taylor B. The Aetion Coalition to Advance Real-World Evidence through Randomized Controlled Trial Emulation Initiative: Oncology. Clin Pharmacol Ther. 2023 Jun;113(6):1217-1222. doi: 10.1002/cpt.2800. Epub 2022 Dec 7.
- Individual participant dataSharing planned: Undecided. Data analysis code and de-identified patient-level data files may be made available, provided that it is determined that this will not violate the investigators' data usage agreements and patient privacy requirements.
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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.
David Merola, PharmD, PhD principal investigator
Aetion, Inc.
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 Non-small Cell Lung Cancer and Metastatic Lung Cancer, run by Aetion, Inc. 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 13 Sep 2023. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Overall Survival - Mortality Hazard Ratio (time frame: Through study completion (earliest of 640 days or censoring)). 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 NCT05908799, 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 13 Sep 2023.