18 and older, any sex, with Medullary Thyroid Cancer (MTC). Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Clinical ResponsePrimary· 2 years
Participants with medullary thyroid cancer were administered a programmed cell death protein 1 (PD1) inhibitor to determine if any experienced a 50% or greater decline in calcitonin levels. A calcitonin response is defined as participants with a ≥50% decline from baseline that is then confirmed on a subsequent calcitonin assessment at least one week later.
Group
Value
95% CI
Cohort 1: Cancer Vaccine
0
Cohort 2: Participants That Have Had No Vaccine
0
Percentage of Participants With a Partial Response and Complete Response by Computed Tomography (CT) or Magnetic Resonance Imaging (MRI)Primary· 2 years
Participants were imaged by CT or MRI and followed for response using the Immune-Related Response Criteria (irRC). Partial Response is a ≥30% decrease in the sum of the largest diameter (SLD) compared with baseline confirmed by a consecutive assessment at least 4 weeks after the first documentation. Complete Response is a 100% disappearance of all lesions, whether measurable or not, and no new lesions, in two consecutive observations not less than 4 weeks from the date first documented.
Group
Value
95% CI
Cohort 1: Cancer Vaccine
0
Cohort 2: Participants That Have Had No Vaccine
0
Percentage Change in (Cluster of Differentiation 4 (CD4), CD8, Tregs, and Natural Killer (NK) Cells at Day 1 and 84 Days in All ParticipantsSecondary· Day 1 and 84 days
Regulatory T-Cells (CD4, CD8, Tregs, and NK cells) in peripheral blood mononuclear cell (PBMC)s were measured by 7-color flow cytometry.
CD4
Group
Value
95% CI
All Participants at Day 1
34.73
12.5 – 53.5
All Participants at Day 84
33.95
9.7 – 46.5
CD8
Group
Value
95% CI
All Participants at Day 1
13.70
6.17 – 26.3
All Participants at Day 84
12.50
6.5 – 24.22
Tregs
Group
Value
95% CI
All Participants at Day 1
0.70
0.0 – 1.2
All Participants at Day 84
0.77
0.46 – 1.8
NK
Group
Value
95% CI
All Participants at Day 1
10.47
6.6 – 17.7
All Participants at Day 84
10.26
4.6 – 19.5
Number of Participants With a Sustained Decline in Carcinoembryonic Antigen (CEA)Secondary· every 3 weeks while on treatment and post treatment, up to 2 years
A sustained 50% decline in CEA. A large magnitude decline in CEA may be associated with tumor responses.
Group
Value
95% CI
Cohort 1: Cancer Vaccine
0
Cohort 2: Participants That Have Had No Vaccine
0
Number of Participants With a Sustained Decline in CalcitoninSecondary· every 3 weeks while on treatment and post treatment, up to 2 years
A sustained 50% decline in calcitonin. A large magnitude decline in calcitonin may be associated with tumor responses.
Group
Value
95% CI
Cohort 1: Cancer Vaccine
0
Cohort 2: Participants That Have Had No Vaccine
0
Progression-free Survival (PFS)Secondary· 3 weeks for up to 2 years while on treatment than 2 weeks after last treatment
PFS is defined as the duration of time from start of treatment to time of progression or death, whichever occurs first. Progression, assessed by the Immune-Related Response Criteria (irRC), is defined as at least 20% increase in the sum of the largest diameter (SLD) compared with nadir (minimum recorded tumor burden) and an increase of at least 5mm over the nadir, confirmed by a repeat,consecutive observations at least 4 weeks from the date first documented.
Group
Value
95% CI
Cohort 1: Cancer Vaccine
210
Cohort 2: Participants That Have Had No Vaccine
55
Overall Survival at 2 YearsSecondary· 2 years
Percentage of participants who are alive at 2 years.
Group
Value
95% CI
Cohort 1: Cancer Vaccine
100
Cohort 2: Participants That Have Had No Vaccine
50
Number of Participants With Grade ≥1 Adverse Events Possibly, Probably, or Definitely Related to PembrolizumabSecondary· Date treatment consent signed to date off study, approximately 25 months and 28 days for cohort 1 and 18 months and 12 days for cohort 2.
Adverse events were assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0). Grade 1 is mild. Grade 2 is moderate. Grade 3 is severe or medically significant but not immediately life-threatening. Grade 4 is life-threatening; urgent intervention indicated. Grade 5 is death related to adverse event.
Acute kidney injury
Group
Value
95% CI
Cohort 1: Grade 1 Possibly Related
1
Cohort 1: Grade 1 Probably Related
0
Cohort 1: Grade 1 Definitely Related
0
Cohort 1: Grade 2 Possibly Related
1
Cohort 1: Grade 2 Probably Related
0
Cohort 1: Grade 2 Definitely Related
0
Cohort 1: Grade 3 Possibly Related
1
Cohort 1: Grade 3 Probably Related
0
Cohort 1: Grade 3 Definitely Related
0
Cohort 2: Grade 1 Possibly Related
0
Cohort 2: Grade 1 Probably Related
0
Cohort 2: Grade 1 Definitely Related
0
Alanine aminotransferase
Group
Value
95% CI
Cohort 1: Grade 1 Possibly Related
0
Cohort 1: Grade 1 Probably Related
0
Cohort 1: Grade 1 Definitely Related
0
Cohort 1: Grade 2 Possibly Related
1
Cohort 1: Grade 2 Probably Related
0
Cohort 1: Grade 2 Definitely Related
0
Cohort 1: Grade 3 Possibly Related
0
Cohort 1: Grade 3 Probably Related
0
Cohort 1: Grade 3 Definitely Related
0
Cohort 2: Grade 1 Possibly Related
0
Cohort 2: Grade 1 Probably Related
0
Cohort 2: Grade 1 Definitely Related
0
Allergic rhinitis
Group
Value
95% CI
Cohort 1: Grade 1 Possibly Related
1
Cohort 1: Grade 1 Probably Related
0
Cohort 1: Grade 1 Definitely Related
0
Cohort 1: Grade 2 Possibly Related
0
Cohort 1: Grade 2 Probably Related
0
Cohort 1: Grade 2 Definitely Related
0
Cohort 1: Grade 3 Possibly Related
0
Cohort 1: Grade 3 Probably Related
0
Cohort 1: Grade 3 Definitely Related
0
Cohort 2: Grade 1 Possibly Related
0
Cohort 2: Grade 1 Probably Related
0
Cohort 2: Grade 1 Definitely Related
0
Anemia
Group
Value
95% CI
Cohort 1: Grade 1 Possibly Related
0
Cohort 1: Grade 1 Probably Related
0
Cohort 1: Grade 1 Definitely Related
0
Cohort 1: Grade 2 Possibly Related
0
Cohort 1: Grade 2 Probably Related
0
Cohort 1: Grade 2 Definitely Related
0
Cohort 1: Grade 3 Possibly Related
0
Cohort 1: Grade 3 Probably Related
0
Cohort 1: Grade 3 Definitely Related
0
Cohort 2: Grade 1 Possibly Related
1
Cohort 2: Grade 1 Probably Related
0
Cohort 2: Grade 1 Definitely Related
0
Anorexia
Group
Value
95% CI
Cohort 1: Grade 1 Possibly Related
1
Cohort 1: Grade 1 Probably Related
0
Cohort 1: Grade 1 Definitely Related
1
Cohort 1: Grade 2 Possibly Related
0
Cohort 1: Grade 2 Probably Related
0
Cohort 1: Grade 2 Definitely Related
0
Cohort 1: Grade 3 Possibly Related
0
Cohort 1: Grade 3 Probably Related
0
Cohort 1: Grade 3 Definitely Related
0
Cohort 2: Grade 1 Possibly Related
0
Cohort 2: Grade 1 Probably Related
0
Cohort 2: Grade 1 Definitely Related
0
Arthralgia
Group
Value
95% CI
Cohort 1: Grade 1 Possibly Related
1
Cohort 1: Grade 1 Probably Related
0
Cohort 1: Grade 1 Definitely Related
0
Cohort 1: Grade 2 Possibly Related
0
Cohort 1: Grade 2 Probably Related
0
Cohort 1: Grade 2 Definitely Related
0
Cohort 1: Grade 3 Possibly Related
0
Cohort 1: Grade 3 Probably Related
0
Cohort 1: Grade 3 Definitely Related
0
Cohort 2: Grade 1 Possibly Related
0
Cohort 2: Grade 1 Probably Related
0
Cohort 2: Grade 1 Definitely Related
0
Aspartate aminotransferase
Group
Value
95% CI
Cohort 1: Grade 1 Possibly Related
2
Cohort 1: Grade 1 Probably Related
0
Cohort 1: Grade 1 Definitely Related
0
Cohort 1: Grade 2 Possibly Related
0
Cohort 1: Grade 2 Probably Related
0
Cohort 1: Grade 2 Definitely Related
0
Cohort 1: Grade 3 Possibly Related
0
Cohort 1: Grade 3 Probably Related
0
Cohort 1: Grade 3 Definitely Related
0
Cohort 2: Grade 1 Possibly Related
0
Cohort 2: Grade 1 Probably Related
0
Cohort 2: Grade 1 Definitely Related
0
Diarrhea
Group
Value
95% CI
Cohort 1: Grade 1 Possibly Related
2
Cohort 1: Grade 1 Probably Related
1
Cohort 1: Grade 1 Definitely Related
0
Cohort 1: Grade 2 Possibly Related
0
Cohort 1: Grade 2 Probably Related
0
Cohort 1: Grade 2 Definitely Related
0
Cohort 1: Grade 3 Possibly Related
0
Cohort 1: Grade 3 Probably Related
0
Cohort 1: Grade 3 Definitely Related
0
Cohort 2: Grade 1 Possibly Related
1
Cohort 2: Grade 1 Probably Related
0
Cohort 2: Grade 1 Definitely Related
0
Number of Participants With Grade ≥1 Adverse Events Unlikely or Unrelated to PembrolizumabSecondary· Date treatment consent signed to date off study, approximately 25 months and 28 days for cohort 1 and 18 months and 12 days for cohort 2.
Adverse events were assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0). Grade 1 is mild. Grade 2 is moderate. Grade 3 is severe or medically significant but not immediately life-threatening. Grade 4 is life-threatening; urgent intervention indicated. Grade 5 is death related to adverse event.
Abdominal pain
Group
Value
95% CI
Cohort 1: Grade 1 Unlikely Related
0
Cohort 1: Grade 1 Unrelated
2
Cohort 1: Grade 2 Unlikely Related
0
Cohort 1: Grade 2 Unrelated
0
Cohort 1: Grade 3 Unlikely Related
0
Cohort 1: Grade 3 Unrelated
0
Cohort 2: Grade 1 Unlikely Related
1
Cohort 2: Grade 1 Unrelated
1
Cohort 2: Grade 2 Unlikely Related
0
Cohort 2: Grade 2 Unrelated
0
Cohort 2: Grade 3 Unlikely Related
0
Cohort 2: Grade 3 Unrelated
0
Alanine aminotransferase increased
Group
Value
95% CI
Cohort 1: Grade 1 Unlikely Related
0
Cohort 1: Grade 1 Unrelated
2
Cohort 1: Grade 2 Unlikely Related
0
Cohort 1: Grade 2 Unrelated
0
Cohort 1: Grade 3 Unlikely Related
0
Cohort 1: Grade 3 Unrelated
0
Cohort 2: Grade 1 Unlikely Related
0
Cohort 2: Grade 1 Unrelated
1
Cohort 2: Grade 2 Unlikely Related
0
Cohort 2: Grade 2 Unrelated
0
Cohort 2: Grade 3 Unlikely Related
0
Cohort 2: Grade 3 Unrelated
0
Allergic rhinitis
Group
Value
95% CI
Cohort 1: Grade 1 Unlikely Related
0
Cohort 1: Grade 1 Unrelated
1
Cohort 1: Grade 2 Unlikely Related
0
Cohort 1: Grade 2 Unrelated
1
Cohort 1: Grade 3 Unlikely Related
0
Cohort 1: Grade 3 Unrelated
0
Cohort 2: Grade 1 Unlikely Related
0
Cohort 2: Grade 1 Unrelated
0
Cohort 2: Grade 2 Unlikely Related
0
Cohort 2: Grade 2 Unrelated
0
Cohort 2: Grade 3 Unlikely Related
0
Cohort 2: Grade 3 Unrelated
0
Anemia
Group
Value
95% CI
Cohort 1: Grade 1 Unlikely Related
0
Cohort 1: Grade 1 Unrelated
2
Cohort 1: Grade 2 Unlikely Related
0
Cohort 1: Grade 2 Unrelated
0
Cohort 1: Grade 3 Unlikely Related
0
Cohort 1: Grade 3 Unrelated
0
Cohort 2: Grade 1 Unlikely Related
0
Cohort 2: Grade 1 Unrelated
0
Cohort 2: Grade 2 Unlikely Related
1
Cohort 2: Grade 2 Unrelated
0
Cohort 2: Grade 3 Unlikely Related
0
Cohort 2: Grade 3 Unrelated
0
Anorexia
Group
Value
95% CI
Cohort 1: Grade 1 Unlikely Related
0
Cohort 1: Grade 1 Unrelated
0
Cohort 1: Grade 2 Unlikely Related
0
Cohort 1: Grade 2 Unrelated
0
Cohort 1: Grade 3 Unlikely Related
0
Cohort 1: Grade 3 Unrelated
0
Cohort 2: Grade 1 Unlikely Related
0
Cohort 2: Grade 1 Unrelated
0
Cohort 2: Grade 2 Unlikely Related
0
Cohort 2: Grade 2 Unrelated
0
Cohort 2: Grade 3 Unlikely Related
0
Cohort 2: Grade 3 Unrelated
1
Aspartate aminotransferase increased
Group
Value
95% CI
Cohort 1: Grade 1 Unlikely Related
1
Cohort 1: Grade 1 Unrelated
2
Cohort 1: Grade 2 Unlikely Related
0
Cohort 1: Grade 2 Unrelated
0
Cohort 1: Grade 3 Unlikely Related
0
Cohort 1: Grade 3 Unrelated
0
Cohort 2: Grade 1 Unlikely Related
0
Cohort 2: Grade 1 Unrelated
0
Cohort 2: Grade 2 Unlikely Related
0
Cohort 2: Grade 2 Unrelated
0
Cohort 2: Grade 3 Unlikely Related
0
Cohort 2: Grade 3 Unrelated
0
Back pain
Group
Value
95% CI
Cohort 1: Grade 1 Unlikely Related
0
Cohort 1: Grade 1 Unrelated
2
Cohort 1: Grade 2 Unlikely Related
0
Cohort 1: Grade 2 Unrelated
0
Cohort 1: Grade 3 Unlikely Related
0
Cohort 1: Grade 3 Unrelated
0
Cohort 2: Grade 1 Unlikely Related
0
Cohort 2: Grade 1 Unrelated
0
Cohort 2: Grade 2 Unlikely Related
0
Cohort 2: Grade 2 Unrelated
0
Cohort 2: Grade 3 Unlikely Related
0
Cohort 2: Grade 3 Unrelated
0
Blood bilirubin increased
Group
Value
95% CI
Cohort 1: Grade 1 Unlikely Related
0
Cohort 1: Grade 1 Unrelated
1
Cohort 1: Grade 2 Unlikely Related
0
Cohort 1: Grade 2 Unrelated
1
Cohort 1: Grade 3 Unlikely Related
0
Cohort 1: Grade 3 Unrelated
0
Cohort 2: Grade 1 Unlikely Related
0
Cohort 2: Grade 1 Unrelated
1
Cohort 2: Grade 2 Unlikely Related
0
Cohort 2: Grade 2 Unrelated
0
Cohort 2: Grade 3 Unlikely Related
0
Cohort 2: Grade 3 Unrelated
0
Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0)Secondary· Date treatment consent signed to date off study, approximately 25 months and 28 days for cohort 1 and 18 months and 12 days for cohort 2.
Here is the number of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one
Group
Value
95% CI
Cohort 1: Cancer Vaccine
13
Cohort 2: Participants That Have Had No Vaccine
4
Adverse events — posted to ClinicalTrials.gov
Time frame: Date treatment consent signed to date off study, approximately 25 months and 28 days for cohort 1 and 18 months and 12 days for cohort 2..
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Cohort 1: Cancer Vaccine
Serious: 2/13 (15%)
Deaths: 0/13
Cohort 2: Participants That Have Had No Vaccine
Serious: 2/4 (50%)
Deaths: 2/4
Serious adverse events (6 terms)
Reaction
System
Cohort 1: Cancer Vaccine
Cohort 2: Participants Tha…
Acute kidney injury
Renal and urinary disorders
—
—
Headache
Nervous system disorders
—
—
Renal and urinary disorders - Other, Acute interstitial nephritis
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.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT07572123 — Evaluating the Addition of Maintenance Immunotherapy Compared to the Usual Treatment of Chemotherapy and Autologous Stem
· Phase 2, PHASE3
· not yet recruiting
NCT07275216 — Pembrolizumab in Combination With Chemotherapy for the Treatment of Frail Hodgkin Lymphoma Patients Ineligible for Stand
· Phase 2
· not yet recruiting
NCT07302347 — A Study of Pembrolizumab in Japanese Pediatric Participants With Solid Tumors or Lymphomas and Japanese Adult Participan
· Phase 1, PHASE2
· recruiting
NCT06724042 — Study of ISM5939 in Patients With Advanced and/or Metastatic Solid Tumors
· Phase 1
· not yet recruiting
NCT07383441 — Adding Biotherapy or Placebo to Standard Treatment for Advanced Kidney Cancer
· Phase 3
· not yet recruiting
Other recruiting trials for Medullary Thyroid Cancer (MTC)
Currently open trials in the same condition.
NCT07142005 — Cultural Adaptation of an Educational Tool in Medullary Thyroid Cancer
· recruiting
Other National Cancer Institute (NCI) trials
Trials by the same sponsor.
NCT07147231 — Testing the Effectiveness of the Anti-cancer Drug Pidnarulex (CX-5461), in Combination With Another Anti-cancer Drug Cem
· Phase 1, PHASE2
· recruiting
NCT07572123 — Evaluating the Addition of Maintenance Immunotherapy Compared to the Usual Treatment of Chemotherapy and Autologous Stem
· Phase 2, PHASE3
· not yet recruiting
NCT07281417 — Testing the Addition of Cemiplimab (REGN2810) to Chemotherapy Treatment Given Prior to Surgery in Patients With Sinonasa
· Phase 2
· recruiting
NCT07012044 — A Study to Find the Highest Dose of Cedazuridine and Decitabine Combination With Filgrastim as a Treatment Option After
· Phase 1
· not yet recruiting
NCT07437950 — Comparing Different Treatment Lengths for Venetoclax in Older People With Newly Diagnosed Acute Myeloid Leukemia (A Myel
· Phase 2
· not yet recruiting
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by National Cancer Institute (NCI)
Last refreshed: 11 February 2021
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03072160.