Stage II Vulvar Cancer AJCC v8Phase 1NCT04169763
Nelfinavir, Cisplatin, and External Beam Radiation Therapy for the Treatment of Locally Advanced Vulvar Cancer That Cannot Be Removed by Surgery
A Phase 1 study testing Cisplatin, External Beam Radiation Therapy and Nelfinavir in people with Stage II Vulvar Cancer AJCC v8 and Stage III Vulvar Cancer AJCC v8, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
This phase I trial studies the side effects and best dose of nelfinavir when given together with cisplatin and external beam radiation therapy in treating patients with vulvar cancer that has spread to nearby tissue or lymph nodes (locally advanced) and cannot be removed by surgery. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Stage II Vulvar Cancer AJCC v8, Stage III Vulvar Cancer AJCC v8, Stage IIIA Vulvar Cancer AJCC v8
- What is being tested?
- Cisplatin, External Beam Radiation Therapy and Nelfinavir
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 1 siteUnited States
- Study dates
- 7 Aug 2020to 31 Dec 2026
- Phase
- Phase 1
- 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
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- All patients with primary, previously untreated, histologically confirmed invasive carcinoma of the vulva (any cell type) not amenable to surgical excision. Clinical stages T2-T4, N0-3, M0. Hematoxylin \& eosin (H \& E) stained slide showing documentation of the primary invasive cancer is required. All specimens of primary tumor require documentation of type
- Absolute neutrophil count (ANC) >= 1,500/microliter (performed within 28 days from signing consent form)
- Platelet count >= 100,000/microliter (performed within 28 days from signing consent form)
- Creatinine < 2.0 mg/dL (performed within 28 days from signing consent form)
- Total bilirubin =< 1.5 times normal (performed within 28 days from signing consent form)
- Glutamic-oxaloacetic transaminase (SGOT) =< 3 times normal (performed within 28 days from signing consent form)
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- Patients with an Eastern Cooperative Oncology Group/Gynecologic Oncology Group (ECOG/GOG) performance status of 0, 1, or 2
- Patients with ureteral obstruction must be treated with stent or nephrostomy tube
- Patients must be consented within twelve (12) weeks of diagnosis or must be restaged
- Patients of childbearing potential must use an effective form of birth control. "Patients receiving oral contraceptives should be instructed that alternate or additional contraceptive measures should be used during therapy with VIRACEPT."
- Confirmed seronegative HIV status within 3 months of signing consent
- Patients must have signed an approved informed consent and authorization permitting release of personal health information
Who cannot join
- Patients with stage T1N0 disease
- Patients who have known metastases to other organs outside the radiation field at the time of the original clinical and surgical staging
- Patients who have received previous pelvic or abdominal radiation, cytotoxic chemotherapy, or previous therapy of any kind for this malignancy
- Patients with septicemia or severe infection
- Patients who have circumstances that will not permit completion of this study or the required follow-up
- Patients who are pregnant at the time of diagnosis and do not wish pregnancy termination prior to initiation of treatment
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- Patients with renal abnormalities, such as pelvic kidney, horseshoe kidney, or renal transplantation, that would require modification of radiation fields
- Patients with other concomitant malignancies (with the exception of non-melanoma skin cancer), who had (or have) any evidence of other cancer present within the last 5 years
- Patients with gastrointestinal (GI) tract disease resulting in an inability to take oral medication, malabsorption syndrome, a requirement for intravenous (IV) alimentation, prior surgical procedures affecting absorption, uncontrolled inflammatory GI disease (e.g., Crohn's, ulcerative colitis)
- Patients with poorly controlled diabetes mellitus despite medication
- Patients taking anti-arrhythmic agents such as amiodarone, quinidine, rifampin, ergot derivatives such as ergotamine, St John's wort, human menopausal gonadotropin (HMG)-CoA reductase inhibitors such as lovastatin, neuroleptic such as pimozide, sedatives such as midazolam and triazolam among other CYP3A4 and CYP2C19 substrates
- Patients with phenylketonuria
- Patients with estimated glomerular filtration rate (eGFR) < 30
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
M D Anderson Cancer Center
Houston, Texas, 77030
Lilie L. Lin, 713-563-2300
Lilie L. Lin
About this study
Official title: A Phase I Study of Nelfinavir and Cisplatin Chemotherapy Concurrent With Pelvic Radiation for Locally Advanced Vulvar Cancer Not Amenable to Surgical Resection
This phase I trial studies the side effects and best dose of nelfinavir when given together with cisplatin and external beam radiation therapy in treating patients with vulvar cancer that has spread to nearby tissue or lymph nodes (locally advanced) and cannot be removed by surgery. Nelfinavir is an antiviral drug normally used to treat human immunodeficiency virus (HIV). Drugs used in chemotherapy, such as cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Radiation therapy uses high energy beams to kill tumor cells and shrink tumors. Giving nelfinavir, cisplatin, and external beam radiation therapy may work better than giving only cisplatin and external beam radiation therapy in treating patients with vulvar cancer.
Read the detailed description
PRIMARY OBJECTIVES:
- To determine the safety and dose limiting toxicities of nelfinavir in combination with cisplatin plus inguinal +/-pelvic radiation therapy for treatment of patients with unresectable T2-4, N0-3 vulvar carcinoma.
II. To determine the recommended phase II dose of nelfinavir combined with chemoradiotherapy.
SECONDARY OBJECTIVES:
- To determine recurrence site (local/distant), progression-free survival and overall survival.
II. To determine the levels of Akt activity (and downstream effectors such as pGSK3, pEBP1) and p16INK4A in addition to the presence of human papilloma virus (HPV) 16 and 18, and E6/E7 ribonucleic acid (RNA) in vulvar biopsy specimens of patients at up to two(2) different time points (1. pre nelfinavir, pre-radiation, 2. while on nelfinavir, pre-radiation).
OUTLINE:
Patients receive nelfinavir orally (PO) twice daily (BID) for up to 8 weeks. Starting week 2, patients also receive cisplatin intravenously (IV) over 60-90 minutes once weekly during weeks 2-8. Patients undergo external beam radiation therapy (EBRT) for 5 consecutive days between weeks 2-8. Treatment continues in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up every 3-6 months for 1 year.
- Conditions
- Stage II Vulvar Cancer AJCC v8, Stage III Vulvar Cancer AJCC v8, Stage IIIA Vulvar Cancer AJCC v8, Stage IIIB Vulvar Cancer AJCC v8, Stage IIIC Vulvar Cancer AJCC v8, Stage IVA Vulvar Cancer AJCC v8
- Being tested
- Cisplatin, External Beam Radiation Therapy, Nelfinavir
- 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 Abiplatin, Blastolem, Briplatin, CDDP
Given IV
External Beam Radiation TherapyRadiation
Also called Definitive Radiation Therapy, EBRT, External Beam Radiation, External Beam Radiotherapy
Undergo EBRT
NelfinavirDrug
Given PO
| Study group | Role | What they receive |
|---|---|---|
| Treatment (nelfinavir, cisplatin, EBRT) | Experimental | Patients receive nelfinavir PO BID for up to 8 weeks. Starting week 2, patients also receive cisplatin IV over 60-90 minutes once weekly during weeks 2-8. Patients undergo EBRT for 5 consecutive days between weeks 2-8. Treatment continues in the absence of disease progression or unacceptable toxicity. Receives: Drug: Cisplatin; Radiation: External Beam Radiation Therapy; Drug: Nelfinavir |
What is being measured
The primary outcome is the main question the study is designed to answer.
Recommended phase II dose (RP2D) of nelfinavir
Defined as the dose for which the isotonic estimate of the dose limiting toxicity (DLT) rate is closest to the target DLT rate of 30%. If there are ties, the higher dose will be chosen as the RP2D when the isotonic estimate is lower than 30%, and the lower dose will be chosen when the isotonic estimate is greater than or equal to 30%.
Measured: 1 year
Incidence of adverse events
Results from the dose escalation phase of study will be summarized by a tabulation of the number of patients treated and the number who experience a DLT at each dose level tested. Comprehensive safety data on all toxicities will be tabulated by type, grade, duration, attribution to treatment, and administered dose level. A patient level summary by worst grade toxicity will be included. Laboratory data and concomitant medications associated with episodes of toxicity will be summarized. Will also report the number of patients who discontinue therapy and the reasons for discontinuation.
Measured: 1 year
Secondary outcomes (2)
Progression-free survival
Will be estimated using the product-limit method developed by Kaplan and Meier. Will report rates at specific times (e.g., at 6 and 12 months) and medians, if attained, with corresponding 95% confidence interval.
Measured: 1 year
Overall survival
Will be estimated using the product-limit method developed by Kaplan and Meier. Will report rates at specific times (e.g., at 6 and 12 months) and medians, if attained, with corresponding 95% confidence interval.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Nov 2019
- Study start7 Aug 2020
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Stage II Vulvar Cancer AJCC v8Studies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (estimated)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Lilie L Lin, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04169763
- Sponsor's protocol ID
- 2019-0629
- Other IDs
- NCI-2019-07570, 2019-0629
- Status last verified
- May 2026
- First submitted
- 18 Nov 2019
- Last updated
- 20 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Study of Nelfinavir and Cisplatin Chemotherapy Concurrent With Pelvic Radiation for Locally Advanced Vulvar Cancer Not Amenable to Surgical Resection
- ClinicalTrials.gov ID
- NCT04169763
- Acronym / sponsor ID
- 2019-0629
- Conditions
- Stage II Vulvar Cancer AJCC v8, Stage III Vulvar Cancer AJCC v8, Stage IIIA Vulvar Cancer AJCC v8, Stage IIIB Vulvar Cancer AJCC v8, Stage IIIC Vulvar Cancer AJCC v8, Stage IVA Vulvar Cancer AJCC v8
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 7 Aug 2020 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 20 Nov 2019 (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.
This phase I trial studies the side effects and best dose of nelfinavir when given together with cisplatin and external beam radiation therapy in treating patients with vulvar cancer that has spread to nearby tissue or lymph nodes (locally advanced) and cannot be removed by surgery. Nelfinavir is an antiviral drug normally used to treat human immunodeficiency virus (HIV). Drugs used in chemotherapy, such as cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Radiation therapy uses high energy beams to kill tumor cells and shrink tumors. Giving nelfinavir, cisplatin, and external beam radiation therapy may work better than giving only cisplatin and external beam radiation therapy in treating patients with vulvar cancer.
Detailed description
PRIMARY OBJECTIVES:
- To determine the safety and dose limiting toxicities of nelfinavir in combination with cisplatin plus inguinal +/-pelvic radiation therapy for treatment of patients with unresectable T2-4, N0-3 vulvar carcinoma.
II. To determine the recommended phase II dose of nelfinavir combined with chemoradiotherapy.
SECONDARY OBJECTIVES:
- To determine recurrence site (local/distant), progression-free survival and overall survival.
II. To determine the levels of Akt activity (and downstream effectors such as pGSK3, pEBP1) and p16INK4A in addition to the presence of human papilloma virus (HPV) 16 and 18, and E6/E7 ribonucleic acid (RNA) in vulvar biopsy specimens of patients at up to two(2) different time points (1. pre nelfinavir, pre-radiation, 2. while on nelfinavir, pre-radiation).
OUTLINE:
Patients receive nelfinavir orally (PO) twice daily (BID) for up to 8 weeks. Starting week 2, patients also receive cisplatin intravenously (IV) over 60-90 minutes once weekly during weeks 2-8. Patients undergo external beam radiation therapy (EBRT) for 5 consecutive days between weeks 2-8. Treatment continues in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up every 3-6 months for 1 year.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (nelfinavir, cisplatin, EBRT) | Experimental | Drug: Cisplatin, Radiation: External Beam Radiation Therapy, Drug: NelfinavirPatients receive nelfinavir PO BID for up to 8 weeks. Starting week 2, patients also receive cisplatin IV over 60-90 minutes once weekly during weeks 2-8. Patients undergo EBRT for 5 consecutive days between weeks 2-8. Treatment continues in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- All patients with primary, previously untreated, histologically confirmed invasive carcinoma of the vulva (any cell type) not amenable to surgical excision. Clinical stages T2-T4, N0-3, M0. Hematoxylin \& eosin (H \& E) stained slide showing documentation of the primary invasive cancer is required. All specimens of primary tumor require documentation of type
- Absolute neutrophil count (ANC) >= 1,500/microliter (performed within 28 days from signing consent form)
- Platelet count >= 100,000/microliter (performed within 28 days from signing consent form)
- Creatinine < 2.0 mg/dL (performed within 28 days from signing consent form)
- Total bilirubin =< 1.5 times normal (performed within 28 days from signing consent form)
Key exclusion criteria (13 listed)
- Patients with stage T1N0 disease
- Patients who have known metastases to other organs outside the radiation field at the time of the original clinical and surgical staging
- Patients who have received previous pelvic or abdominal radiation, cytotoxic chemotherapy, or previous therapy of any kind for this malignancy
- Patients with septicemia or severe infection
- Patients who have circumstances that will not permit completion of this study or the required follow-up
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 |
|---|---|---|
| CisplatinAlso: Abiplatin, Blastolem, Briplatin, CDDPGiven IV | Drug | Treatment (nelfinavir, cisplatin, EBRT) |
| External Beam Radiation TherapyAlso: Definitive Radiation Therapy, EBRT, External Beam Radiation, External Beam RadiotherapyUndergo EBRT | Radiation | Treatment (nelfinavir, cisplatin, EBRT) |
| NelfinavirGiven PO | Drug | Treatment (nelfinavir, cisplatin, EBRT) |
Primary outcome
Recommended phase II dose (RP2D) of nelfinavir
Time frame: 1 year
Defined as the dose for which the isotonic estimate of the dose limiting toxicity (DLT) rate is closest to the target DLT rate of 30%. If there are ties, the higher dose will be chosen as the RP2D when the isotonic estimate is lower than 30%, and the lower dose will be chosen when the isotonic estimate is greater than or equal to 30%.
Incidence of adverse events
Time frame: 1 year
Results from the dose escalation phase of study will be summarized by a tabulation of the number of patients treated and the number who experience a DLT at each dose level tested. Comprehensive safety data on all toxicities will be tabulated by type, grade, duration, attribution to treatment, and administered dose level. A patient level summary by worst grade toxicity will be included. Laboratory data and concomitant medications associated with episodes of toxicity will be summarized. Will also report the number of patients who discontinue therapy and the reasons for discontinuation.
2 secondary outcomes
Progression-free survival
Time frame: 1 year
Overall survival
Time frame: 1 year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 May 2026.
- Related InfoLink provided by the sponsor.
Related trials for Stage II Vulvar Cancer AJCC v8
Recruiting
A Blended e-Health Intervention to Improve Fear of Progression in Women With Gynecologic or Breast Cancer
Anatomic Stage I Breast Cancer AJCC v8, Anatomic Stage II Breast Cancer AJCC v8
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.
Lilie L Lin principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Cisplatin, External Beam Radiation Therapy and Nelfinavir in people with Stage II Vulvar Cancer AJCC v8 and Stage III Vulvar Cancer AJCC v8, run by M.D. Anderson Cancer Center 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 12 inclusion and 13 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: looking for participants now, as of the sponsor's last update on 20 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 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Contact Lilie L Lin (713-563-2300, lllin@mdanderson.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Recommended phase II dose (RP2D) of nelfinavir (time frame: 1 year). 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?
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 20 May 2026; AzTrials last refreshed its copy on 8 Nov 2026.