Locally Advanced Head and Neck Squamous Cell CarcinomaPhase 1NCT04947241
Toripalimab Combined With Gemcitabine and Cisplatin Treating Resectable Locally Advanced HNSCC
A Phase 1 study testing PD-1 inhibitor+ Gemcitabine + Cisplatin in people with Locally Advanced Head and Neck Squamous Cell Carcinoma and Neoadjuvant Therapy, run by Fifth Affiliated Hospital, Sun Yat-Sen University at 1 site in China.
Trial summary
Head and Neck Squamous Cell Carcinoma (HNSCC) is the most common malignant tumor of the head and neck, accounting for 90% of head and neck malignancies, and 16% to 40% of systemic malignancies. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Locally Advanced Head and Neck Squamous Cell Carcinoma, Neoadjuvant Therapy
- What is being tested?
- PD-1 inhibitor+ Gemcitabine + Cisplatin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 15 Dec 2020to 15 Jan 2027
- 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 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Untreated locally advanced head and neck squamous cell carcinoma confirmed by histology or cytology;
- Patients who are recommended to perform surgery;
- Patients between 18 and 70 years old;
- ECOG: 0～2 points;
- Estimated survival time ≥ 6 months;
- At least one measurable lesion should be detected according to the RECIST 1.1;
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- The major organs meet the following standards (no blood components and cell growth factors are injected within 14 days):
- Hemoglobin HB≥90 g/L; neutrophil ANC≥1.5×109/L; platelet count PLT≥100×109/L;
- Serum albumin ≥28g/L;
- Total bilirubin TBIL≤1.5×upper limit of normal, alanine aminotransferase ALT, aspartate aminotransferase AST≤2.5×upper limit of normal; if there is liver metastasis, ALT and AST≤5×upper limit of normal;
- Serum creatinine ≤1.5×upper limit of normal, and creatinine clearance ≥50 mL/min;
- Activated partial thromboplastin time (APTT) and international normalized ratio (INR) ≤ 1.5 × upper limit of normal (for the use of stable doses of anticoagulant therapy such as low molecular weight heparin or warfarin, and INR in the anticoagulant expected treatment can be filtered within the scope);
- TSH≤ upper limit of normal; if abnormal, the T3 and T4 levels should be examined, and the T3 and T4 levels are normal.
- Women of childbearing age should take contraceptive measures (such as intrauterine devices, contraceptives or condoms) during the medication period and within 3 months after the medication; the serum or urine pregnancy test is negative within 7 days before the study is enrolled, And must be a non-lactating patient, and the male should agree to take contraceptive measures during the study period and within 3 months after the end of the study period;
- The subjects voluntarily joined the study, signed an informed consent form, had good compliance, and cooperated with the follow-up.
Who cannot join
- Pregnant or lactating women;
- Allergic to anti-PD-1 monoclonal antibody, gemcitabine, or cisplatin;
- History of other malignant tumors in the past 5 years or at the same time, except for cured skin basal cell carcinoma, cervical carcinoma in situ, and thyroid papillary carcinoma;
- Uncontrollable clinical symptoms or diseases of the heart, such as: (1) Heart failure, NYHA Ⅱ, III or IV (2) Unstable angina (3) Myocardial infarction occurred within 1 year (4) Supraventricular in clinical significance or Patients with ventricular arrhythmia requiring clinical intervention;
- Have received any of the following treatments:
- Have received any research relevant drugs before enrolling in this research;
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- Enrolled in another clinical study at the same time, unless it is an observational (non-interventional) clinical study or intervention in a new clinical study follow-up;
- Patients who need to be given corticosteroids (more than 10 mg prednisone equivalent dose per day) or other immunosuppressive agents for systemic treatment within 2 weeks before giving medication for the first time, except for local inflammation and prevention of allergies, nausea and vomiting The case of corticosteroids. In the absence of active autoimmune diseases, inhaled or topical steroids and adrenal corticosteroids with a dose greater than 10 mg per day of prednisone curative dose are allowed to replace;
- Have been vaccinated with anti-tumor vaccine or have been vaccinated with live vaccine within 4 weeks before the first administration of study drug;
- Received major surgery or severe trauma within 4 weeks before using the study drug for the first time;
- The left ventricular ejection fraction of the heart is greater than or equal to 60%;
- Severe infection (CTC AE greater than grade 2) occurred within 4 weeks before the first use of the study drug, such as severe pneumonia, bacteremia, infection comorbidities that require hospitalization, etc.; baseline chest imaging examinations suggest active lung inflammation , There are symptoms and signs of infection 2 weeks before the first use of the study drug or the need for oral or intravenous antibiotic treatment (excluding prophylactic use of antibiotics);
- Have active autoimmune diseases, history of autoimmune diseases;
- A history of immunodeficiency, including a positive HIV test, or other acquired or congenital immunodeficiency diseases, or a history of organ transplantation and bone marrow transplantation;
- Patients with active tuberculosis infection found through medical history or CT examination, or patients with a history of active tuberculosis infection within 1 year before enrollment, or patients with a history of active tuberculosis infection but without formal treatment 1 year before being checked;
- The subject has active hepatitis (HBV DNA ≥ 2000IU/ml or 10,000 copies/ml), hepatitis C (hepatitis C antibody is positive, and HCV-RNA is higher than the lower limit of the analysis method);
- History of psychotropic drug abuse, alcohol and drug abuse;
- Symptomatic brain metastases (confirmed or suspected);
- The patients who are reckoned as not suitable for inclusion.
Trial locations and contacts
1 site in 1 country.
China1 site
Fifth Affilliated Hospital of Sun Yat-sen University
Zhuhai, Guangdong
About this study
Official title: Phase Ib Clinical Study of Toripalimab Combined With Gemcitabine and Cisplatin Neoadjuvant Chemotherapy in Patients With Resectable Locally Advanced Head and Neck Squamous Cell Carcinoma
Head and Neck Squamous Cell Carcinoma (HNSCC) is the most common malignant tumor of the head and neck, accounting for 90% of head and neck malignancies, and 16% to 40% of systemic malignancies. There are 60,000 new cases reported annually worldwide, and the incidence and mortality are increasing year by year, however,the 5-year survival rate under standard treatment is only 50%. 70%~80% of patients already developed into locally advanced status (stage II-IVa) when they are first diagnosed. The treatment principle is mainly determined by the clinical stage and location of the tumor, various factors affecting the prognosis and the patient's tolerance. Locally advanced head and neck squamous cell carcinoma has a higher probability of local/regional failure and distant metastasis after treatment. Therefore, in recent years, the use of neoadjuvant therapy (NAC) followed by surgery or radiotherapy has been advocated. Surgical treatment is still one of the preferred treatments for local head and neck squamous cell carcinoma. TPF (Docetaxel + Cisplatin + Fluorouracil) regimen is considered as the standard regimen of induced chemotherapy for head and neck squamous cell carcinoma (especially in laryngeal cancer), which can significantly reduce the patient's distant metastasis rate and prolong overall survival ( OS). Nevertheless, the therapeutic effect of neoadjuvant therapy on head and neck squamous cell carcinoma has reached a bottleneck. In recent years, PD-1 inhibitors have achieved significant effects in the field of tumor therapy and have been approved for the treatment of various tumors including head and neck tumors. And a number of clinical trials have shown that PD-1 inhibitors can significantly prolong the OS of patients.
Altogether, the investigators launch an open-label, single-arm, phase Ib clinical trial of PD-1 inhibitor plus chemotherapy in patients with resectable HNSCC to explore the safety and efficacy of the treatment. The study comprises two stages, run-in and case development.
Read the detailed description
In the first phase, we would observe the initial six patients lasting 90 days from the first day of treatment(or 30 days after surgery) to evaluate if the dose-limited- toxicity DLT would occur in two of them or more.
3、If the dose-limited- toxicity would be recorded in no more than one patient, the trail would continue until meeting the scheduled size of population, otherwise the trial would stop. The further investigation would be launch to review the data thoroughly and modify the protocol.
- Being tested
- PD-1 inhibitor+ Gemcitabine + Cisplatin
- Keywords
- Anti-PD-1 Monopoly Antibody, immunochemotherapy, head and neck squamous cell carcinoma
- Medical subject headings
- Squamous Cell Carcinoma of Head and Neck
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PD-1 inhibitor+ Gemcitabine + CisplatinDrug
Also called Toripalimab
Before surgery, patients will receive therapy as follows:
PD-1 inhibitor: 240mg (day1) , intravenous, Q3W, 2cycles; cisplatin: 80 mg/m2(day1), intravenous , intravenous, Q3W, 2cycles Gemcitabine: 1000mg/m2(day1and day8), intravenous, Q3W, 2cycles
| Study group | Role | What they receive |
|---|---|---|
| Neoadjuvant therapy | Experimental | Patients included are going to receive neoadjuvant therapy, surgery and adjuvamt therapy post surgery. Before surgery, patients will receive therapy as follows: PD-1 inhibitor: 240mg (day1) , intravenous, Q3W, 2cycles; cisplatin: 80 mg/m2(day1), intravenous , intravenous, Q3W, 2cycles Gemcitabine: 1000mg/m2(day1and day8), intravenous, Q3W, 2cycles Receives: Drug: PD-1 inhibitor+ Gemcitabine + Cisplatin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Major pathological response rate
The proportion of patients with less than 10% surviving cancer cells
Measured: 1 week after surgery
Objective Response Rate
The proportion of patients whose tumors have shrunk to a certain amount and maintained for a certain period of time, including complete response and partial response
Measured: 1 week after surgery
R0 resection rate
The proportion of no residue on the edge of the resection under the microscope after surgery
Measured: 1 week after surgery
the incidence of adverse event
the immune limited adverse event
Measured: 90 days from the first day of treatment
Secondary outcomes (9)
Progression Free Survival
The period of time between the start of treatment for patients with tumor disease and the observation of disease progression or death from any cause
Measured: 1 year after therapy
Progression Free Survival
The period of time between the start of treatment for patients with tumor disease and the observation of disease progression or death from any cause
Measured: 3 years after therapy
Progression Free Survival
The period of time between the start of treatment for patients with tumor disease and the observation of disease progression or death from any cause
Measured: 5 years after therapy
Overall Survival
The time from the start of treatment to death for any cause
Measured: 1 year after therapy
Overall Survival
The time from the start of treatment to death for any cause
Measured: 3 years after therapy
Overall Survival
The time from the start of treatment to death for any cause
Measured: 5 years after therapy
Disease Free Survival
The time from the start of treatment to the first tumor recurrence/metastasis or the death of the patient for any reason
Measured: 1 year after therapy
Disease Free Survival
The time from the start of treatment to the first tumor recurrence/metastasis or the death of the patient for any reason
Measured: 3 years after therapy
Disease Free Survival
The time from the start of treatment to the first tumor recurrence/metastasis or the death of the patient for any reason
Measured: 5 years after therapy
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jul 2021
- Study start15 Dec 2020
- Primary completion15 May 2022
- Study completion15 Jan 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Locally Advanced Head and Neck Squamous Cell CarcinomaStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fifth Affiliated Hospital, Sun Yat-Sen University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Zhigang Liu, Fifth Affiliated Hospital, Sun Yat-Sen University
- Investigators
- Zhigang Liu, M.D., Principal investigator, Fifth Affilliated Hospital of Sun Yat-sen University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04947241
- Sponsor's protocol ID
- No.ZDWY[2020]LunziNo.(K66-1)
- Status last verified
- Sep 2026
- First submitted
- 18 Jun 2021
- Last updated
- 9 Sep 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase Ib Clinical Study of Toripalimab Combined With Gemcitabine and Cisplatin Neoadjuvant Chemotherapy in Patients With Resectable Locally Advanced Head and Neck Squamous Cell Carcinoma
- ClinicalTrials.gov ID
- NCT04947241
- Acronym / sponsor ID
- No.ZDWY[2020]LunziNo.(K66-1)
- Keywords
- Anti-PD-1 Monopoly Antibody, immunochemotherapy, head and neck squamous cell carcinoma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Fifth Affiliated Hospital, Sun Yat-Sen UniversityUniversity, hospital or other
- Study start
- 15 Dec 2020 (actual)
- Primary completion
- 15 May 2022 (actual)
- Study completion
- 15 Jan 2027 (estimated)
- First posted
- 1 Jul 2021 (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.
Head and Neck Squamous Cell Carcinoma (HNSCC) is the most common malignant tumor of the head and neck, accounting for 90% of head and neck malignancies, and 16% to 40% of systemic malignancies. There are 60,000 new cases reported annually worldwide, and the incidence and mortality are increasing year by year, however,the 5-year survival rate under standard treatment is only 50%. 70%~80% of patients already developed into locally advanced status (stage II-IVa) when they are first diagnosed. The treatment principle is mainly determined by the clinical stage and location of the tumor, various factors affecting the prognosis and the patient's tolerance. Locally advanced head and neck squamous cell carcinoma has a higher probability of local/regional failure and distant metastasis after treatment. Therefore, in recent years, the use of neoadjuvant therapy (NAC) followed by surgery or radiotherapy has been advocated. Surgical treatment is still one of the preferred treatments for local head and neck squamous cell carcinoma. TPF (Docetaxel + Cisplatin + Fluorouracil) regimen is considered as the standard regimen of induced chemotherapy for head and neck squamous cell carcinoma (especially in laryngeal cancer), which can significantly reduce the patient's distant metastasis rate and prolong overall survival ( OS). Nevertheless, the therapeutic effect of neoadjuvant therapy on head and neck squamous cell carcinoma has reached a bottleneck. In recent years, PD-1 inhibitors have achieved significant effects in the field of tumor therapy and have been approved for the treatment of various tumors including head and neck tumors. And a number of clinical trials have shown that PD-1 inhibitors can significantly prolong the OS of patients.
Altogether, the investigators launch an open-label, single-arm, phase Ib clinical trial of PD-1 inhibitor plus chemotherapy in patients with resectable HNSCC to explore the safety and efficacy of the treatment. The study comprises two stages, run-in and case development.
Detailed description
In the first phase, we would observe the initial six patients lasting 90 days from the first day of treatment(or 30 days after surgery) to evaluate if the dose-limited- toxicity DLT would occur in two of them or more.
3、If the dose-limited- toxicity would be recorded in no more than one patient, the trail would continue until meeting the scheduled size of population, otherwise the trial would stop. The further investigation would be launch to review the data thoroughly and modify the protocol.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Neoadjuvant therapy | Experimental | Drug: PD-1 inhibitor+ Gemcitabine + CisplatinPatients included are going to receive neoadjuvant therapy, surgery and adjuvamt therapy post surgery. Before surgery, patients will receive therapy as follows: PD-1 inhibitor: 240mg (day1) , intravenous, Q3W, 2cycles; cisplatin: 80 mg/m2(day1), intravenous , intravenous, Q3W, 2cycles Gemcitabine: 1000mg/m2(day1and day8), intravenous, Q3W, 2cycles |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Untreated locally advanced head and neck squamous cell carcinoma confirmed by histology or cytology;
- Patients who are recommended to perform surgery;
- Patients between 18 and 70 years old;
- ECOG: 0～2 points;
- Estimated survival time ≥ 6 months;
Key exclusion criteria (19 listed)
- Pregnant or lactating women;
- Allergic to anti-PD-1 monoclonal antibody, gemcitabine, or cisplatin;
- History of other malignant tumors in the past 5 years or at the same time, except for cured skin basal cell carcinoma, cervical carcinoma in situ, and thyroid papillary carcinoma;
- Uncontrollable clinical symptoms or diseases of the heart, such as: (1) Heart failure, NYHA Ⅱ, III or IV (2) Unstable angina (3) Myocardial infarction occurred within 1 year (4) Supraventricular in clinical significance or Patients with ventricular arrhythmia requiring clinical intervention;
- Have received any of the following treatments:
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 |
|---|---|---|
| PD-1 inhibitor+ Gemcitabine + CisplatinAlso: ToripalimabBefore surgery, patients will receive therapy as follows: PD-1 inhibitor: 240mg (day1) , intravenous, Q3W, 2cycles; cisplatin: 80 mg/m2(day1), intravenous , intravenous, Q3W, 2cycles Gemcitabine: 1000mg/m2(day1and day8), intravenous, Q3W, 2cycles | Drug | Neoadjuvant therapy |
Primary outcome
Major pathological response rate
Time frame: 1 week after surgery
The proportion of patients with less than 10% surviving cancer cells
Objective Response Rate
Time frame: 1 week after surgery
The proportion of patients whose tumors have shrunk to a certain amount and maintained for a certain period of time, including complete response and partial response
R0 resection rate
Time frame: 1 week after surgery
The proportion of no residue on the edge of the resection under the microscope after surgery
the incidence of adverse event
Time frame: 90 days from the first day of treatment
the immune limited adverse event
9 secondary outcomes
Progression Free Survival
Time frame: 1 year after therapy
Progression Free Survival
Time frame: 3 years after therapy
Progression Free Survival
Time frame: 5 years after therapy
Overall Survival
Time frame: 1 year after therapy
Overall Survival
Time frame: 3 years after therapy
Overall Survival
Time frame: 5 years after therapy
Disease Free Survival
Time frame: 1 year after therapy
Disease Free Survival
Time frame: 3 years after therapy
Disease Free Survival
Time frame: 5 years after therapy
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 9 Sep 2026.
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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.
Zhigang Liu, M.D. principal investigator
Fifth Affilliated Hospital of Sun Yat-sen University
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 PD-1 inhibitor+ Gemcitabine + Cisplatin in people with Locally Advanced Head and Neck Squamous Cell Carcinoma and Neoadjuvant Therapy, run by Fifth Affiliated Hospital, Sun Yat-Sen University at 1 site in China.
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 15 inclusion and 19 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 9 Sep 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 China. 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?
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?
Major pathological response rate (time frame: 1 week after surgery). The study lists 4 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 9 Sep 2026.