Solid Tumor, AdultPhase 2NCT03274661
Pembrolizumab Activity in Patients With Homologous Recombination Competent and Deficient Tumors
A Phase 2 study testing Pembrolizumab 200 mg Q3W in people with Solid Tumor, Adult, run by Baptist Health South Florida at 1 site in United States.
Trial summary
Phase II trial in which patients with metastatic solid tumors experiencing progression after first line standard chemotherapy or for which there is no standard chemotherapy, and for which pembrolizumab does not have an FDA or compendia listing approved indication, will receive pembrolizumab intravenously at a dose of… Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- Solid Tumor, Adult
- What is being tested?
- Pembrolizumab 200 mg Q3W
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 52 participants
- Where
- 1 siteUnited States
- Study dates
- 3 Nov 2017to 19 Mar 2022
- Phase
- Phase 2
- 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 18 to 99 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Have measurable disease based on RECIST 1.1.
- Be willing to provide consent for retrieval of archival tumor material tissue from a previously obtained core or excisional biopsy of a tumor lesion.
- Have a tumor presentation at screening for which pembrolizumab does not have an FDA approved indication for commercial use.
- Have a performance status of 0, 1 or 2 on the ECOG Performance Scale.
- Demonstrate adequate organ function
- Female subject of childbearing potential should have a negative urine or serum pregnancy within 72 hours prior to receiving the first dose of study medication.
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- Expansion cohort - additional criteria
- Diagnosis of Endometrial Carcinoma or Sarcoma which is metastatic and has failed standard treatment or is recurrent, or for which standard chemotherapy is contraindicated or refused by patient.
- Sufficient tissue is available for correlative studies
- MSI studies have been performed, either by immunohistochemistry or next generation sequencing and results show that patient is MS low or stable.
Who cannot join
- Is currently participating and receiving study therapy or has participated in a study of an investigational agent and received study therapy, or used an investigational device within 4 weeks of the first dose of treatment.
- Has a diagnosis of immunodeficiency or is receiving systemic steroid therapy or any other form of immunosuppressive therapy within 7 days prior to the first dose of trial treatment.
- Has a known additional malignancy that is progressing or requires active treatment. Exceptions include basal cell carcinoma of the skin or squamous cell carcinoma of the skin that has undergone potentially curative therapy or in situ cervical cancer.
- Has a known history of active Bacillus Tuberculosis
- Hypersensitivity to pembrolizumab or any of its excipients.
- Has known active central nervous system (CNS) metastases and/or carcinomatous meningitis. Subjects with previously treated brain metastases may participate provided they are stable. This exception does not include carcinomatous meningitis which is excluded regardless of clinical stability.
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- Has active autoimmune disease that has required systemic treatment in the past 2 years (i.e. with use of disease modifying agents, corticosteroids or immunosuppressive drugs).
- Has immunohistochemically proven mismatch repair deficient cancer
- Has a history of (non-infectious) pneumonitis that required steroids or current pneumonitis
- Evidence of interstitial lung disease.
- Has an active infection requiring systemic therapy.
- Has known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial.
- Is pregnant or breastfeeding, or expecting to conceive or father children within the projected duration of the trial, starting with the pre-screening or screening visit through 120 days after the last dose of trial treatment.
- Has received prior therapy with an anti-PD-1, anti-PD-L1, or anti-PD-L2 agent.
- Has a known history of Human Immunodeficiency Virus (HIV) (HIV 1/2 antibodies).
- Has known active Hepatitis B (e.g., HBsAg reactive) or Hepatitis C (e.g., HCV RNA [qualitative] is detected).
- Has received a live vaccine within 30 days of planned start of study therapy. Note: Seasonal influenza vaccines for injection are generally inactivated flu vaccines and are allowed; however intranasal influenza vaccines (e.g., Flu-Mist®) are live attenuated vaccines, and are not allowed.
Trial locations and contacts
1 site in 1 country.
United States1 site
Miami Cancer Institute at Baptist Health, Inc.
Miami, Florida, 33176
About this study
Official title: Evaluating Immune Checkpoint Inhibition In Solid Tumor Patients With Homologous Recombination Repair Deficiency
Phase II trial in which patients with metastatic solid tumors experiencing progression after first line standard chemotherapy or for which there is no standard chemotherapy, and for which pembrolizumab does not have an FDA or compendia listing approved indication, will receive pembrolizumab intravenously at a dose of 200 mg every 3 weeks.
Read the detailed description
Patients with metastatic or recurrent solid malignancy who have progressed on first line standard of care treatment or for which defined standard of care does not exist or is not readily available are eligible to participate on this trial. Patients for which pembrolizumab has an FDA approved indication and for whom pembrolizumab is covered by their insurance should receive standard commercial pembrolizumab and will not be eligible for this trial.
The primary objective of this trial is to evaluate the Immune-Related Objective Response Rate (IR-ORR) achieved with pembrolizumab in patients with Fanconi Anemia Repair Pathway functionally competent and functionally deficient tumors.
Trial treatment should be administered on Day 1 of each cycle after all procedures/assessments have been completed. Trial treatment may be administered up to 3 days before or after the scheduled Day 1 of each cycle due to administrative reasons.
All trial treatments will be administered on an outpatient basis.
Pembrolizumab 200 mg will be administered as a 30 minute IV infusion every 3 weeks. Target infusion timing is 30 minutes.
Expansion Cohort:
Ten (10) additional subjects with the diagnosis of metastatic or recurrent endometrial carcinoma will be enrolled in the trial at the conclusion of regular enrollment.
- Conditions
- Solid Tumor, Adult
- Being tested
- Pembrolizumab 200 mg Q3W
- Keywords
- solid tumor, Pembrolizumab,
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Pembrolizumab 200 mg Q3WDrug
Also called Pembrolizumab 200 mg
Pembrolizumab 200 mg Intravenous Infusion every 3 weeks administered on Day 1 of each 3 week cycle
| Study group | Role | What they receive |
|---|---|---|
| Pembrolizumab 200 mg Q3W | Experimental | Pembrolizumab 200 mg Intravenous Infusion every 3 weeks administered on Day 1 of each 3 week cycle Receives: Drug: Pembrolizumab 200 mg Q3W |
What is being measured
The primary outcome is the main question the study is designed to answer.
Immune-related Objective Response Rate
Immune-related Response Criteria (irRC) will be utilized for assessment of response to therapy, defined as the percent of participants who achieved complete or partial response. Per irRC, for target lesions assessed by CT or MRI: Complete Response (CR), Disappearance of all lesions in two consecutive observations not less than 4 weeks apart; Partial Response (PR), ≥50% decrease in tumor burden compared with baseline in two observations at least 4 weeks apart.
Measured: 20 weeks
Secondary outcomes (2)
Progression-free Survival (PFS)
Immune-related Response Criteria (irRC) will be utilized for assessment of response to therapy. PFS will be defined as the time from treatment start to time of disease progression or death, whichever comes first. Per irRC, for target lesions assessed by CT or MRI: Progressive Disease (PD), at least 25% increase in tumor burden compared with nadir (at any single time point) in two consecutive observations at least 4 weeks apart.
Measured: 20 weeks
Overall Response
Immune-related Response Criteria (irRC) will be utilized for assessment of response to therapy, defined as the duration of time from start of treatment to end of study or death, whichever comes first. Per irRC, for target lesions assessed by CT or MRI:Complete Response (CR) is the disappearance of all lesions in two consecutive observations not less than 4 weeks apart; Partial Response (PR) is a ≥50% decrease in tumor burden compared with baseline in two observations at least 4 weeks apart; and Progressive Disease (PD) is at least 25% increase in tumor burden compared with nadir (at any single time point) in two consecutive observations at least 4 weeks apart. Everything else is considered Stable Disease (SD).
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Sep 2017
- Study start3 Nov 2017
- Primary completion20 Oct 2020
- Study completion19 Mar 2022
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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Solid Tumor, AdultStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Eligible participants will receive pembrolizumab intravenously at a dose of 200 mg every 3 weeks.
- Masking
- None (open label)
- Enrolment
- 52 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baptist Health South Florida
- Sponsor type
- University, hospital or other
- Collaborators
- Merck Sharp & Dohme LLC, Miami Cancer Institute
- Responsible party
- Sponsor
- Investigators
- John P Diaz, MD, Principal investigator, Miami Cancer Institute at Baptist Health, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03274661
- Sponsor's protocol ID
- MK-3475-663
- Status last verified
- Sep 2023
- First submitted
- 5 Sep 2017
- Last updated
- 19 Oct 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Villalona-Calero MA, Diaz JP, Duan W, Diaz Z, Schroeder ED, Aparo S, Gatcliffe T, Albrecht F, Venkatappa S, Guardiola V, Garrido S, Rubens M, DeZarraga F, Vuong H. Pembrolizumab activity in patients with Fanconi anemia repair pathway competent and deficient tumors. Biomark Res. 2022 Jun 3;10(1):39. doi: 10.1186/s40364-022-00386-0.
PubMed 35658948
Study at a glance
From the registry record, last updated by the sponsor on 19 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluating Immune Checkpoint Inhibition In Solid Tumor Patients With Homologous Recombination Repair Deficiency
- ClinicalTrials.gov ID
- NCT03274661
- Acronym / sponsor ID
- MK-3475-663
- Conditions
- Solid Tumor, Adult
- Keywords
- solid tumor, Pembrolizumab,
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 52 participants (actual)
- Lead sponsor
- Baptist Health South FloridaUniversity, hospital or other
- Collaborators
- Merck Sharp & Dohme LLC, Miami Cancer Institute
- Study start
- 3 Nov 2017 (actual)
- Primary completion
- 20 Oct 2020 (actual)
- Study completion
- 19 Mar 2022 (actual)
- First posted
- 7 Sep 2017 (actual)
- Results
- Posted 19 Oct 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Phase II trial in which patients with metastatic solid tumors experiencing progression after first line standard chemotherapy or for which there is no standard chemotherapy, and for which pembrolizumab does not have an FDA or compendia listing approved indication, will receive pembrolizumab intravenously at a dose of 200 mg every 3 weeks.
Detailed description
Patients with metastatic or recurrent solid malignancy who have progressed on first line standard of care treatment or for which defined standard of care does not exist or is not readily available are eligible to participate on this trial. Patients for which pembrolizumab has an FDA approved indication and for whom pembrolizumab is covered by their insurance should receive standard commercial pembrolizumab and will not be eligible for this trial.
The primary objective of this trial is to evaluate the Immune-Related Objective Response Rate (IR-ORR) achieved with pembrolizumab in patients with Fanconi Anemia Repair Pathway functionally competent and functionally deficient tumors.
Trial treatment should be administered on Day 1 of each cycle after all procedures/assessments have been completed. Trial treatment may be administered up to 3 days before or after the scheduled Day 1 of each cycle due to administrative reasons.
All trial treatments will be administered on an outpatient basis.
Pembrolizumab 200 mg will be administered as a 30 minute IV infusion every 3 weeks. Target infusion timing is 30 minutes.
Expansion Cohort:
Ten (10) additional subjects with the diagnosis of metastatic or recurrent endometrial carcinoma will be enrolled in the trial at the conclusion of regular enrollment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Eligible participants will receive pembrolizumab intravenously at a dose of 200 mg every 3 weeks.
| Arm | Type | Receives |
|---|---|---|
| Pembrolizumab 200 mg Q3W | Experimental | Drug: Pembrolizumab 200 mg Q3WPembrolizumab 200 mg Intravenous Infusion every 3 weeks administered on Day 1 of each 3 week cycle |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Have measurable disease based on RECIST 1.1.
- Be willing to provide consent for retrieval of archival tumor material tissue from a previously obtained core or excisional biopsy of a tumor lesion.
- Have a tumor presentation at screening for which pembrolizumab does not have an FDA approved indication for commercial use.
- Have a performance status of 0, 1 or 2 on the ECOG Performance Scale.
- Demonstrate adequate organ function
Key exclusion criteria (17 listed)
- Is currently participating and receiving study therapy or has participated in a study of an investigational agent and received study therapy, or used an investigational device within 4 weeks of the first dose of treatment.
- Has a diagnosis of immunodeficiency or is receiving systemic steroid therapy or any other form of immunosuppressive therapy within 7 days prior to the first dose of trial treatment.
- Has a known additional malignancy that is progressing or requires active treatment. Exceptions include basal cell carcinoma of the skin or squamous cell carcinoma of the skin that has undergone potentially curative therapy or in situ cervical cancer.
- Has a known history of active Bacillus Tuberculosis
- Hypersensitivity to pembrolizumab or any of its excipients.
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 200 mg Q3WAlso: Pembrolizumab 200 mgPembrolizumab 200 mg Intravenous Infusion every 3 weeks administered on Day 1 of each 3 week cycle | Drug | Pembrolizumab 200 mg Q3W |
Primary outcome
Immune-related Objective Response Rate
Time frame: 20 weeks
Immune-related Response Criteria (irRC) will be utilized for assessment of response to therapy, defined as the percent of participants who achieved complete or partial response. Per irRC, for target lesions assessed by CT or MRI: Complete Response (CR), Disappearance of all lesions in two consecutive observations not less than 4 weeks apart; Partial Response (PR), ≥50% decrease in tumor burden compared with baseline in two observations at least 4 weeks apart.
2 secondary outcomes
Progression-free Survival (PFS)
Time frame: 20 weeks
Overall Response
Time frame: 3 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Oct 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 35658948Villalona-Calero MA, Diaz JP, Duan W, Diaz Z, Schroeder ED, Aparo S, Gatcliffe T, Albrecht F, Venkatappa S, Guardiola V, Garrido S, Rubens M, DeZarraga F, Vuong H. Pembrolizumab activity in patients with Fanconi anemia repair pathway competent and deficient tumors. Biomark Res. 2022 Jun 3;10(1):39. doi: 10.1186/s40364-022-00386-0.
- MCI - WebsiteLink provided by the sponsor.
- 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. This study is not recruiting at the moment.
John P Diaz, MD principal investigator
Miami Cancer Institute at Baptist Health, Inc.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Pembrolizumab 200 mg Q3W in people with Solid Tumor, Adult, run by Baptist Health South Florida 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 10 inclusion and 17 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 19 Oct 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 19 Oct 2023). 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?
Immune-related Objective Response Rate (time frame: 20 weeks). 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 NCT03274661, 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 19 Oct 2023; AzTrials last refreshed its copy on 8 Nov 2026.