AzTrials
AzTrials

Vulvar CancerPhase 2NCT04430699

Cisplatin+Pembrolizumab+RT in Vulvar Cancer

A Phase 2 study testing Cisplatin, Pembrolizumab and Radiation Therapy in people with Vulvar Cancer and Vulvar Squamous Cell Carcinoma, run by Massachusetts General Hospital at 2 sites in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial summary

This research is being done to see how well the combination of a standard of care drug, investigational drug, and radiation therapy work against unresectable vulvar squamous cell carcinoma. Read more about this study

Who is it for?
Ages 18 years and older, women only
Conditions the study is for
Vulvar Cancer, Vulvar Squamous Cell Carcinoma
What is being tested?
Cisplatin, Pembrolizumab and Radiation Therapy
Could I receive a placebo?
No, everyone receives an active treatment
How many people
24 participants
Where
2 sitesUnited States
Study dates
29 Jul 2020to 30 Jan 2028
Phase
Phase 2
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 18 years and older
  • Women only
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Participants must have histologically or cytologically confirmed unresectable, incompletely resected, recurrent, or metastatic squamous cell carcinoma of the vulva.Patients with unresectable disease are defined as T2 or T3 primary tumors (N0-3, M0) not amenable to surgical resection by standard radical vulvectomy.
  • Participants must have measurable disease based on RECIST 1.1. Lesions situated in a previously irradiated area are considered measurable if progression has been demonstrated in such lesions.
  • Have provided archival tumor tissue sample or newly obtained core or excisional biopsy of a tumor lesion not previously irradiated. Formalin-fixed, paraffin embedded (FFPE) tissue blocks are preferred to slides.
  • Prior therapy: Participants with no prior therapy are eligible and patients with recurrent disease must not have had more than two lines of cytotoxic therapy. Topical or hormonal therapy are not counted towards prior lines. Prior treatment with immunotherapy is allowed, provided treatment was not stopped for grade 2 or greater adverse events.
  • Time from prior therapy:
  • Systemic anti-neoplastic therapy: 5 half-lives or 4 weeks, whichever is shorter.
Show all 31Show fewer
  • Hormonal therapy is not considered anti-neoplastic therapy.
  • Radiotherapy: Any prior irradiation is acceptable provided the site being considered for study has not been previously irradiated.
  • Age ≥18 years. Because insufficient dosing or adverse event data are currently available on the use of pembrolizumab in combination with cisplatin-sensitized radiation therapy participants <18 years of age, children are excluded. Vulva cancer is rare in the pediatric population
  • ECOG performance status of 0 or 1.
  • Participants must have adequate organ and marrow function as defined below (Table 1):
  • Table 1: Adequate Organ Function Laboratory Values
  • Hematological
  • Absolute neutrophil count (ANC) ≥1500/μL
  • Platelets ≥100 000/μL
  • Hemoglobin ≥9.0 g/dL or ≥5.6 mmol/La
  • Renal
  • Creatinine OR Measured or calculated b creatinine clearance (GFR can also be used in place of creatinine or CrCl) ≤1.5 × ULN OR ≥50 mL/min for participant with creatinine
  • Hepatic
  • Total bilirubin ≤1.5 ×ULN OR direct bilirubin ≤ULN for participants with total bilirubin levels >1.5 × ULN
  • AST (SGOT) and ALT (SGPT) ≤2.5 × ULN (≤5 × ULN for participants with liver metastases)
  • Coagulation
  • International normalized ratio (INR) OR prothrombin time (PT) Activated partial thromboplastin time (aPTT) ≤1.5 × ULN unless participant is receiving anticoagulant therapy as long as PT or aPTT is within therapeutic range of intended use of anticoagulants
  • ALT (SGPT)=alanine aminotransferase (serum glutamic pyruvic transaminase); AST (SGOT)=aspartate aminotransferase (serum glutamic oxaloacetic transaminase); GFR=glomerular filtration rate; ULN=upper limit of normal.
  • Criteria must be met without erythropoietin dependency and without packed red blood cell (pRBC) transfusion within last 2 weeks.
  • Creatinine clearance (CrCl) should be calculated per institutional standard.
  • Note: This table includes eligibility-defining laboratory value requirements for treatment; laboratory value requirements should be adapted according to local regulations and guidelines for the administration of specific chemotherapies.
  • Participant must be female, and is eligible to participate if she is not pregnant (see Appendix B), not breastfeeding, and at least one of the following conditions applies:
  • Not a woman of childbearing potential (WOCBP) as defined in Appendix B OR
  • A WOCBP must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, during study treatment, and for at least twelve weeks after the last dose of study treatment. Should a woman become pregnant or suspect she is pregnant while she is pregnant while she is participating in this study, she should inform her treating physician immediately.
  • Ability to understand and the willingness to sign a written informed consent document.

Who cannot join

  • Patients who in the opinion of the investigator cannot safely receive a minimum of 30 Gy in 10 fractions are not eligible for the trial.
  • Participants who have received prior systemic anti-cancer therapy including investigational agents within 4 weeks prior to first dose of study treatment. Note: If participant received major surgery, they must have recovered adequately from the toxicity and/or complications from the intervention prior to starting study treatment
  • Participants must have recovered from all AEs due to previous therapies to ≤Grade 1 or baseline.
  • Has received prior radiotherapy within 2 weeks of start of study treatment. Participants must have recovered from all radiation-related toxicities, not require corticosteroids, and not have had radiation pneumonitis. Re-irradiation to a previously treated site will not be permitted.
  • Participants who have received a live vaccine within 30 days prior to the first dose of study drug. Examples of live vaccines include, but are not limited to, the following: measles, mumps, rubella, varicella/zoster (chicken pox), yellow fever, rabies, Bacillus Calmette-Guérin (BCG), and typhoid vaccine. Seasonal influenza vaccines for injection are generally killed virus vaccines and are allowed; however, intranasal influenza vaccines (eg, FluMist®) are live attenuated vaccines and are not allowed.
  • Participants with vulvar melanomas, sarcomas, extramammary Paget's disease, or basal cell carcinoma
Show all 20Show fewer
  • Participants with a history of gastrointestinal or colovesicular fistulae
  • 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). Replacement therapy (eg., thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment.
  • Has known active CNS metastases and/or carcinomatous meningitis. Participants with previously treated brain metastases may participate provided they are radiologically stable, i.e. without evidence of progression for at least 4 weeks by repeat imaging (note that the repeat imaging should be performed during study screening), clinically stable and without requirement of steroid treatment for at least 14 days prior to first dose of study treatment.
  • Has a history of (non-infectious) pneumonitis that required steroids or has current pneumonitis.
  • Has an active infection requiring systemic therapy.
  • Patients with a history of other invasive malignancies, with the exception of nonmelanoma skin cancer, are excluded if there is any evidence of other malignancy being present within the last five years. Patients are also excluded if their previous cancer treatment contraindicates this protocol therapy.
  • Participants with uncontrolled intercurrent illness.
  • Participants with psychiatric illness/social situations that would limit compliance with study requirements.
  • Has a known history of Hepatitis B (defined as Hepatitis B surface antigen [HBsAg] reactive) or known active Hepatitis C virus (defined as HCV RNA [qualitative] is detected) infection. Note: no testing for Hepatitis B and Hepatitis C is required.
  • Has a known history of active TB (Bacillus Tuberculosis).
  • Pregnant or nursing women are excluded from this study because effects of agents used in this study on infants or the developing human fetus are unknown.
  • Presence of other malignancies unless they are considered cured by patient's oncologist.
  • Has a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the study, interfere with the subject's participation for the full duration of the study, or is not in the best interest of the subject to participate, in the opinion of the treating investigator.
  • Has a known history of Human Immunodeficiency Virus (HIV).

Trial locations and contacts

2 sites in 1 country.

United States2 sites

  • Massachusetts General Hospital Cancer Center

    Boston, Massachusetts, 02114

  • Beth Israel Deaconess Medical Center

    Boston, Massachusetts, 02215

Other trials in United States

About this study

Official title: A Phase 2 Study of Combined Chemo-immunotherapy With Cisplatin-pembrolizumab and Radiation for Unresectable Vulvar Squamous Cell Carcinoma

This research is being done to see how well the combination of a standard of care drug, investigational drug, and radiation therapy work against unresectable vulvar squamous cell carcinoma.

This research study involves the following:

  • Cisplatin (standard of care drug)
  • Pembrolizumab (investigational drug)
  • Radiation Therapy (standard of care intervention)
Read the detailed description

This is a single-arm phase 2 clinical trial involving women with unresectable, incompletely resected, recurrent, or metastatic vulva squamous cell carcinoma. This study combines cisplatin, pembrolizumab, and radiation therapy to see if this combination further increases participants' immune system's efficiency in killing their tumor, and if the combination decreases the chances of participants' cancer coming back.

The research study procedures include screening for eligibility and study treatment including evaluations and follow up visits.

This research study involves the following:

  • Cisplatin (standard of care drug)
  • Pembrolizumab (investigational drug)
  • Radiation Therapy (standard of care intervention)

Participants will receive study treatment for up to 36 weeks and will be followed for up to 3 years.

It is expected that about 24 people will take part in this research study.

Phase II clinical trials test the safety and effectiveness of an investigational drug to learn whether the drug works in treating a specific disease. "Investigational" means that the drug is being studied. The U.S. Food and Drug Administration (FDA) has approved cisplatin as a treatment option for vulva squamous cell carcinoma. The FDA has not approved pembrolizumab for vulva squamous cell carcinoma, but it has been approved for other uses. Cisplatin is a chemotherapy drug and will be given to participants per standard of care.

Radiation therapy will be given to you per standard of care. Pembrolizumab is a drug that may target participants immune systems to increase its efficiency in targeting and killing illnesses and diseases, such as unresectable vulvar squamous cell carcinoma.

Being tested
Cisplatin, Pembrolizumab, Radiation Therapy
Keywords
Vulvar Cancer, Vulvar Squamous Cell Carcinoma,, Immunotherapy, Radiation, Pembrolizumab
Medical subject headings
Vulvar Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CisplatinDrug

    Also called Platinol, Platinol-AQ, CDDP

    Participants will be given Cisplatin by intravenous infusion at a predetermined dose 1x weekly up to 8 weeks.

  • PembrolizumabDrug

    Also called Keytruda

    Participants will be given Pembrolizumab by intravenous infusion at a predetermined dose 1x every 3 weeks up to 36 weeks.

  • Radiation TherapyRadiation

    Standard of care radiation therapy 1x daily up to 8 weeks.

Study groups in NCT04430699
Study groupRoleWhat they receive
Pembrolizumab, Cisplatin and Radiation TherapyExperimentalTreatment period is 36 weeks with 21 day study cycles. Participants will receive cisplatin at a predetermined dose 1x weekly, pembrolizumab at a predetermined dose every 3 weeks, concurrently with daily radiation therapy from week 1 up to week 8. First 3 participants on the study, may skip 1 or 2 pembrolizumab dosages while receiving radiation therapy. Following completion of daily radiation therapy with 1x weekly cisplatin and 1x every 3 weeks pembrolizumab, participants will continue at a pre-determined maintenance dose of pembrolizumab 1x every 3 weeks for a total of 12 cycles or 36 weeks.
Receives: Drug: Cisplatin; Drug: Pembrolizumab; Radiation: Radiation Therapy

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Overall response rate (ORR)

    To determine the Overall response rate (ORR) in patients undergoing combination therapy with pembrolizumab and cisplatin-sensitized external beam radiation therapy by RECIST 1.1 criteria.

    This sample size calculation is based on an ORR of greater than or equal to 60%. For recurrent metastatic disease, there are is no standard of care.The ORR estimation is informed by data showing an ORR of 40% in patients with advanced or recurrent metastatic vulvar cancer treated with platinum-based combination therapy. Single agent chemotherapy has an ORR of about 12% (5). Patients with primary disease who are not candidates for upfront surgery have a reported response rates from 55% to 64%.

    Measured: Up to 36 weeks

Secondary outcomes (1)

  1. Recurrence Free Survival (RFS)-6 months

    To determine the 6-month Recurrence Free Survival (RFS) in patients with locally advanced, unresectable, or recurrent metastatic vulvar cancer treated with concurrent cisplatin-sensitized radiation therapy and pembrolizumab. A point estimate with exact 95% CI and Kaplan-Meier estimate will be used for the RFS distribution.

    Measured: 6 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 Jun 2020
  2. Study start29 Jul 2020
  3. Primary completion30 Apr 2026
  4. Study completion30 Jan 2028 (estimated)

Resources

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
Masking
None (open label)
Enrolment
24 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04430699
Sponsor's protocol ID
20-109
Status last verified
Apr 2026
First submitted
10 Jun 2020
Last updated
5 May 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: The Dana-Farber / Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to: \[contact information for Sponsor Investigator or designee\]. The protocol and statistical analysis plan will be made available on Clinical trials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.

Publications

  • Yeku O, Russo AL, Lee H, Spriggs D. A phase 2 study of combined chemo-immunotherapy with cisplatin-pembrolizumab and radiation for unresectable vulvar squamous cell carcinoma. J Transl Med. 2020 Sep 14;18(1):350. doi: 10.1186/s12967-020-02523-5.

    PubMed 32928237

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Cisplatin, Pembrolizumab and Radiation Therapy in people with Vulvar Cancer and Vulvar Squamous Cell Carcinoma, run by Massachusetts General Hospital at 2 sites 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 31 inclusion and 20 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: under way, not looking for participants, as of the sponsor's last update on 5 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites 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?

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?

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 response rate (ORR) (time frame: Up to 36 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 NCT04430699, 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 5 May 2026.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in