Phase 2
N=17
Pembrolizumab in Recurrent or Metastatic Medullary Thyroid Cancer
Medullary Thyroid Cancer (MTC)
Bottom Line
View on ClinicalTrials.gov: NCT03072160 ↗Enrolled (actual)
17
Serious AEs
23.5%
Results posted
Feb 2021
Primary outcome: Primary: Clinical Response — 0; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Pembrolizumab (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- National Cancer Institute (NCI)
- Primary completion
- Nov 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Clinical Response |
0; 0 | — |
| PRIMARY Percentage of Participants With a Partial Response and Complete Response by Computed Tomography (CT) or Magnetic Resonance Imaging (MRI) |
0; 0 | — |
| SECONDARY Percentage Change in (Cluster of Differentiation 4 (CD4), CD8, Tregs, and Natural Killer (NK) Cells at Day 1 and 84 Days in All Participants |
34.73; 33.95; 13.70; 12.50; 0.70; 0.77 | 0.926 |
| SECONDARY Number of Participants With a Sustained Decline in Carcinoembryonic Antigen (CEA) |
0; 0 | — |
| SECONDARY Number of Participants With a Sustained Decline in Calcitonin |
0; 0 | — |
| SECONDARY Progression-free Survival (PFS) |
210; 55 | — |
| SECONDARY Overall Survival at 2 Years |
100; 50 | — |
| SECONDARY Number of Participants With Grade ≥1 Adverse Events Possibly, Probably, or Definitely Related to Pembrolizumab |
1; 0; 0; 1; 0; 0 | — |
| SECONDARY Number of Participants With Grade ≥1 Adverse Events Unlikely or Unrelated to Pembrolizumab |
0; 2; 0; 0; 0; 0 | — |
| SECONDARY Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0) |
13; 4 | — |
Summary
Background:
Medullary thyroid cancer (MTC) is a tumor of the thyroid gland. Surgery is the only current treatment to cure it. The drug pembrolizumab (MK-3475) is a new type of cancer therapy. It works by allowing the immune system to detect and kill tumor cells.
Objective:
To test how pembrolizumab affects people with MTC and if it can offer them clinical benefit.
Eligibility:
People ages 18 and older with MTC
Patients who have recurrent or metastatic MTC, for whom surgery is not a curative option
Patients with some imaging evidence of MTC
Patients with minimal symptoms related to MTC
Design:
Participants will be screened with:
* Medical history
* Physical exam
* Blood, urine, and heart tests
* Computed tomography (CT) scan or magnetic resonance imaging (MRI): They lie in a machine that takes pictures of the body.
* Bone scan
Participants will be put in a group based on their treatment history:
* Group 1 if they have had an immune stimulating cancer vaccine
* Group 2 if they have had no vaccine
Participants will receive the study drug as a 30-minute intravenous (IV) infusion every 3 weeks. Treatment will continue for up to 2 years as long as they tolerate it and their disease does not get worse.
Participants will have physical exams and blood tests on the day of each infusion. They will have CT and bone scans every 3 months.
Participants may save biopsies before treatment and after starting treatment.
Participants will have a final visit 3-4 weeks after stopping treatment. This will include a physical exam and blood and heart tests.
After this study, participants can join a long-term follow-up study.
Eligibility Criteria
- INCLUSION CRITERIA:
- Diagnosis: Patients must have histologically confirmed medullary thyroid cancer by the Laboratory of Pathology or a pathology report and history consistent with medullary thyroid cancer. It is not uncommon for a secondary, minor pathologic focus of another form of thyroid cancer to be coincidentally found in 15-20% of patients with medullary thyroid cancer. In such cases, eligibility is based on the discretion of the investigator.
- Patients must have evidence of metastatic medullary thyroid cancer including disease that is evaluable on bone, computed tomography (CT) scan or magnetic resonance imaging (MRI). (Patients who are surgical candidates and potentially rendered disease free with surgical resection are not eligible.)
- Patients must have elevated calcitonin levels greater than 40 pg/mL
- Patients must have minimal or no disease related-symptoms (Minimal symptoms will include those that do not affect activities of daily living or pain that does not require regularly scheduled narcotics.)
- Patients must have evaluable disease on imaging
- No history of seizures, encephalitis, or multiple sclerosis.
- Age greater than or equal to 18 years
- Eastern Cooperative Oncology Group (ECOG) performance status of 0-1 at study entry (Karnofsky greater than or equal to 70).
- 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. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required.
- Female subjects of childbearing potential must be willing to use an adequate method of contraception, Contraception, for the course of the study through 120 days after the last dose of study medication. Note: Abstinence is acceptable if this is the usual lifestyle and preferred contraception for the subject
- Male subjects of childbearing potential must agree to use an adequate method of contraception. Contraception, starting with the first dose of study therapy through 120 days after the last dose of study therapy. Note: Abstinence is acceptable if this is the usual lifestyle and preferred contraception for the subject
- Willing to travel to the National Institutes of Health (NIH) for follow-up visits
- Able to understand and sign informed consent.
- Demonstrate adequate organ function, all screening labs should be performed within 10 days of treatment initiation.
- Adequate Organ Function Laboratory Values
- Hematological
---Absolute neutrophil count (ANC) greater than or equal to1,000 /mcL
- Platelets greater than or equal to 100,000 / mcL
- Hemoglobin greater than or equal to 9 g/dL or greater than or equal to 5.6 mmol/L without transfusion or erythropoietin (EPO) dependency (within 7 days of assessment)
- Renal
- Serum creatinine less than or equal to1.5 X upper limit of normal (ULN) OR
- Measured or calculated* creatinine clearance (Glomerular filtration rate (GFR) can also be used in place of creatinine or creatinine clearance (CrCl) greater than or equal to 60 mL/min for subject with creatinine levels > 1.5 X institutional ULN
- Hepatic
- Serum total bilirubin less than or equal to 1.5 X ULN OR Direct bilirubin less than or equal to ULN for subjects with total bilirubin levels > 1.5 ULN
- Aspartate aminotransferase (AST) Serum glutamic-oxaloacetic transaminase (SGOT) and Alanine aminotransferase (ALT) Serum glutamic pyruvic transaminase (SGPT) less than or equal to 2.5 X ULN OR less than or equal to 5 X ULN for subjects with liver metastases
- Albumin >2.5 mg/dL
- Coagulation
- International Normalized Ratio (INR) or Prothrombin Time (PT) less than or equal to1.5 X ULN unless subject is receiving anticoagulant therapy as long as PT or partial thromboplastin time (PTT) is within therapeutic range of intended use of anticoagulants
- Activated Partial Thromboplastin Time (aPTT) less than or equal to1.5 X ULN unless subject is receiving anticoagulant therapy as lon
Data sourced from ClinicalTrials.gov (NCT03072160). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.