Nasopharyngeal Cancinoma (NPC)Phase 2NCT06605131
GP Combined with Toripalimab Versus GP Induction Chemotherapy for Advanced Childhood Nasopharyngeal Carcinoma
A Phase 2 study testing GP+Toripalimab+CCRT and GP+CCRT in people with Nasopharyngeal Cancinoma (NPC), run by Sun Yat-sen University at 1 site in China.
Trial summary
Nasopharyngeal carcinoma (NPC) has a low incidence rate in children, accounting for only 1-2% of pediatric tumors. However, it is prone to metastasis, and most patients are already in advanced stages at the time of diagnosis. Chemoradiotherapy has been shown to effectively improve prognosis. Read more about this study
- Who is it for?
- Ages 6 to 18 years
- Conditions the study is for
- Nasopharyngeal Cancinoma (NPC)
- What is being tested?
- GP+Toripalimab+CCRT and GP+CCRT
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 72 participants
- Where
- 1 siteChina
- Study dates
- 17 Apr 2025to 25 Feb 2029
- 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 6 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients must be informed of the investigational nature of this study and give written informed consent.
- Age between 6 and 18 years, regardless of gender.
- Pathologically confirmed non-keratinizing nasopharyngeal carcinoma (differentiated or undifferentiated type, i.e., WHO Type II or III).
- Clinical stage II-III (according to AJCC 9th edition), excluding T3N0 and T3N1 (only with retropharyngeal lymph node metastasis); patients must be newly diagnosed with nasopharyngeal carcinoma.
- ECOG performance status of 0-1.
- Females of childbearing potential must agree to use contraception during the study period.
Show all 9Show fewer
- Hemoglobin (HGB) ≥ 90 g/L, white blood cell count (WBC) ≥ 4×10^9/L, platelet count (PLT) ≥ 100×10^9/L.
- Normal liver function test: ALT and AST < 2.5 x upper limit of normal (ULN), total bilirubin < 2.0×ULN.
- Adequate renal function: Serum creatinine < 1.5×ULN.
Who cannot join
- Older than 18 years.
- Presence of recurrence or distant metastasis.
- Pathologically diagnosed as keratinizing squamous cell carcinoma (WHO Type I).
- History of previous anti-tumor treatment.
- Pregnant or lactating women, and women of childbearing potential not using effective contraception.
- HIV positive.
Show all 17Show fewer
- History of malignancy within the past 5 years, except for patients with carcinoma in situ, adequately treated non-melanoma skin cancer, or papillary thyroid carcinoma.
- Patients with other immunodeficiency diseases or a history of organ transplantation.
- Patients with active autoimmune diseases, except for type I diabetes, hypothyroidism under replacement therapy, and skin conditions not requiring systemic treatment (e.g., vitiligo, psoriasis, or alopecia).
- Conditions requiring systemic corticosteroids (equivalent to prednisone greater than 10mg/d) or other immunosuppressive therapy within 28 days prior to signing informed consent. Patients on systemic corticosteroids equivalent to prednisone ≤10 mg/day or using inhaled or topical corticosteroids are permitted.
- Received a live vaccine within 30 days before signing informed consent or planning to receive a live vaccine in the near future.
- Patients with significant impairment in heart, liver, lung, kidney, or bone marrow function.
- Severe, uncontrolled medical diseases or infections.
- Concurrent use of other investigational drugs or participation in other clinical trials.
- Refusal or inability to sign the informed consent form to participate in the trial.
- Known allergies to large molecule protein products or any PD-1 antibody compounds, or those with other contraindications to the treatment.
- Individuals with personality or mental disorders, those without legal capacity or with limited legal capacity.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
Sun Yat-sen University Cancer Center
Guangzhou, Guangdong, 510060
Dong Hua Luo, 86-13560358839, luodh@sysucc.org.cn
About this study
Official title: Gemcitabine and Cisplatin in Combination with Toripalimab or Not As First-line Treatment in Advanced Childhood Nasopharyngeal Carcinoma: an Open, Randomised Phase II Trial
Nasopharyngeal carcinoma (NPC) has a low incidence rate in children, accounting for only 1-2% of pediatric tumors. However, it is prone to metastasis, and most patients are already in advanced stages at the time of diagnosis. Chemoradiotherapy has been shown to effectively improve prognosis. Induction chemotherapy combined with concurrent chemoradiotherapy with adjusted radiation doses has demonstrated good efficacy in children and adolescents with locally advanced NPC. Nevertheless, 10-15% of patients still experience treatment failure, and 15-20% of patients respond poorly to induction chemotherapy, necessitating higher doses of radiation, which in turn increases the incidence of treatment-related sequelae. Therefore, it is crucial to explore further treatment strategies that can enhance response rates, reduce acute and long-term treatment toxicities, and improve overall efficacy on the basis of induction chemotherapy followed by adjusted concurrent chemoradiotherapy.
The combination of gemcitabine and cisplatin (GP regimen) has been identified as the highest level of evidence-based induction chemotherapy regimen (Class 1A). However, the complete response rate of only 10% after three cycles of GP regimen induction chemotherapy in adults with locoregionally advanced NPC indicates the need for intensified induction treatment. PD-1 inhibitors combined with chemotherapy have demonstrated synergistic tumor-killing effects, providing additional curative opportunities for patients with locally advanced disease. Toripalimab, with its dual-blocking mechanism, is an ideal PD-1 monoclonal antibody model that can fully relieve T-cell-mediated antitumor immune suppression. Previous clinical trials have confirmed the efficacy and safety of toripalimab in treating nasopharyngeal carcinoma.
This study aims to conduct the first single-center, phase II randomized controlled clinical trial in children and adolescents with nasopharyngeal carcinoma, comparing the GP regimen combined with toripalimab induction chemotherapy versus the GP regimen alone. The goal is to optimize the treatment model for pediatric and adolescent NPC, enhance therapeutic efficacy, and provide high-level evidence-based medical support for the international treatment guidelines for pediatric NPC.
- Conditions
- Nasopharyngeal Cancinoma (NPC)
- Being tested
- GP+Toripalimab+CCRT, GP+CCRT
- Keywords
- nasopharyngeal carcinoma, adolescent and childhood, Toripalimab, GP
- Medical subject headings
- Nasopharyngeal Carcinoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GP+Toripalimab+CCRTDrug
- Induction Chemotherapy (GP Regimen combined with Toripalimab):
- GP Regimen Gemcitabine: 1000 mg/m² on Day 1 and Day 8; Cisplatin (DDP): 80 mg/m² on Day 1-3; every 3 weeks for 3 cycles.
- Toripalimab: For patients weighing ≥ 40 kg: 240 mg on Day 1; For patients weighing less than 40 kg: 3 mg/kg; every 3 weeks for 3 cycles.
- Concurrent Chemoradiotherapy (CCRT): Cisplatin (DDP): 100 mg/m², starting on the first day of radiotherapy; every 3 weeks for 3 cycles
GP+CCRTDrug
- Induction Chemotherapy (GP Regimen ): Gemcitabine: 1000 mg/m² on Day 1 and Day 8; Cisplatin (DDP): 80 mg/m² on Day 1-3; every 3 weeks for 3 cycles.
- Concurrent Chemoradiotherapy (CCRT): Cisplatin (DDP): 100 mg/m², starting on the first day of radiotherapy; every 3 weeks for 3 cycles
| Study group | Role | What they receive |
|---|---|---|
| GP combined with Toripalimab + CCRT | Experimental | 1. Induction Chemotherapy (GP Regimen combined with Toripalimab)
* GP Regimen: Gemcitabine: 1000 mg/m² on Day 1 and Day 8; Cisplatin (DDP): 80 mg/m² on Day 1-3; every 3 weeks for 3 cycles
* Toripalimab: For patients weighing ≥ 40 kg: 240 mg on Day 1; For patients weighing < 40 kg: 3 mg/kg; every 3 weeks for 3 cycles
2. Concurrent Chemoradiotherapy (CCRT):
Cisplatin (DDP): 100 mg/m², starting on the first day of radiotherapy; every 3 weeks for 3 cycles Receives: Drug: GP+Toripalimab+CCRT |
| GP regimen + CCRT | Active comparator | 1. Induction Chemotherapy (GP Regimen):
Gemcitabine: 1000 mg/m² on Day 1 and Day 8; Cisplatin (DDP): 80 mg/m² on Day 1-3, every 3 weeks for 3 cycles.
2. Concurrent Chemoradiotherapy (CCRT):
Cisplatin (DDP): 100 mg/m², starting on the first day of radiotherapy, every 3 weeks for 3 cycles. Receives: Drug: GP+CCRT |
What is being measured
The primary outcome is the main question the study is designed to answer.
Complete Response (CR) Rate After Induction Chemotherapy
CR is assessed after induction chemotherapy by independent reviewers, according to the Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1. Complete response defined as the complete disappearance of the target and non-target lesion(s) identified at baseline after radiological evaluation by enhanced MRI (or enhanced CT if indicated).
Measured: when the induction chemotherapy complete
Secondary outcomes (10)
Overall survival
defined as the time from random assignment to death from any cause or censored at the date of last follow-up.
Measured: 3 years
Progression-free survival
defined as the time from random assignment to documented local or regional relapse, distant metastasis, or death from any cause, whichever occurred first
Measured: 3 years
Locoregional failure-free survival(LRRFS)
defined as the time from random assignment to local or regional relapse, or death from any cause.
Measured: 3 years
Distant metastasis-free survival(DMFS)
defined as the time from random assignment to distant metastasis, or death from any cause
Measured: 3 years
Incidence of acute and late toxicity
Incidence of acute toxicity is evaluated on basis of Common Terminology Criteria for Adverse Events (CTCAE) 5.0 criteria. Late radiation toxicities were assessed using the Radiation Therapy Oncology Group and European Organization for Research and Treatment of Cancer late radiation morbidity scoring scheme. Acute adverse events, occurring during study treatment, and radiation-related late adverse events, occurring from 3 months after completion of radiotherapy, and chemotherapy-induced late adverse events, occurring from 3 months after completion of chemotherapy until end of follow up.
Measured: 3 years
Hormone levels
Changes in levels of luteinizing hormone (LH), follicle stimulating hormone (FSH), thyroid stimulating hormone (TSH), and growth hormone (GH), will be monitored.
Measured: 3 years
Growth condition of Height
Patients will be monitored for height (in metres) before treatment, after treatment, and during follow-up.
Measured: 3 years
Growth condition of Weight
Patients will be monitored for Weight(in kilograms) before treatment, after treatment, and during follow-up.
Measured: 3 years
Growth condition of BMI
Patients will be monitored for BMI (in kg/m²) before treatment, after treatment, and during follow-up.
Measured: 3 years
Quality of life
The quality of life will be evaluated using the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire Core 30 (QLQ-C30, Version 3.0) and the EORTC Quality of Life Questionnaire Head and Neck Cancer Module (QLQ-H and N35, Version 1.0). For the EORTC QLQ-C30, scores range from 0 to 100. In the Functioning and General Health domains, higher scores reflect better functional status and quality of life, while in the Symptoms domain, higher scores indicate more symptoms or problems, signifying poorer quality of life. For the QLQ-H and N35, scores also range from 0 to 100, with higher scores consistently indicating poorer quality of life. We will calculate the scores for patients in each domain according to the scoring criteria of the respective questionnaires and explore the correlation of these scores with treatment plans, etc. Each domain and questionnaire will be treated as a separate outcome measure.
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Sep 2024
- Study start17 Apr 2025 (estimated)
- Primary completion25 Feb 2026 (estimated)
- Study completion25 Feb 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Nasopharyngeal Cancinoma (NPC)Studies of the same condition, open and under way.
- More trials run by Sun Yat-sen UniversityThe 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 72 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sun Yat-sen University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Hai-Qiang Mai,MD,PhD, Sun Yat-sen University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06605131
- Sponsor's protocol ID
- 2023-FXY-317
- Status last verified
- Mar 2025
- First submitted
- 11 Sep 2024
- Last updated
- 20 Mar 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Gemcitabine and Cisplatin in Combination with Toripalimab or Not As First-line Treatment in Advanced Childhood Nasopharyngeal Carcinoma: an Open, Randomised Phase II Trial
- ClinicalTrials.gov ID
- NCT06605131
- Acronym / sponsor ID
- 2023-FXY-317
- Conditions
- Nasopharyngeal Cancinoma (NPC)
- Keywords
- nasopharyngeal carcinoma, adolescent and childhood, Toripalimab, GP
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 72 participants (estimated)
- Lead sponsor
- Sun Yat-sen UniversityUniversity, hospital or other
- Study start
- 17 Apr 2025 (estimated)
- Primary completion
- 25 Feb 2026 (estimated)
- Study completion
- 25 Feb 2029 (estimated)
- First posted
- 20 Sep 2024 (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.
Nasopharyngeal carcinoma (NPC) has a low incidence rate in children, accounting for only 1-2% of pediatric tumors. However, it is prone to metastasis, and most patients are already in advanced stages at the time of diagnosis. Chemoradiotherapy has been shown to effectively improve prognosis. Induction chemotherapy combined with concurrent chemoradiotherapy with adjusted radiation doses has demonstrated good efficacy in children and adolescents with locally advanced NPC. Nevertheless, 10-15% of patients still experience treatment failure, and 15-20% of patients respond poorly to induction chemotherapy, necessitating higher doses of radiation, which in turn increases the incidence of treatment-related sequelae. Therefore, it is crucial to explore further treatment strategies that can enhance response rates, reduce acute and long-term treatment toxicities, and improve overall efficacy on the basis of induction chemotherapy followed by adjusted concurrent chemoradiotherapy.
The combination of gemcitabine and cisplatin (GP regimen) has been identified as the highest level of evidence-based induction chemotherapy regimen (Class 1A). However, the complete response rate of only 10% after three cycles of GP regimen induction chemotherapy in adults with locoregionally advanced NPC indicates the need for intensified induction treatment. PD-1 inhibitors combined with chemotherapy have demonstrated synergistic tumor-killing effects, providing additional curative opportunities for patients with locally advanced disease. Toripalimab, with its dual-blocking mechanism, is an ideal PD-1 monoclonal antibody model that can fully relieve T-cell-mediated antitumor immune suppression. Previous clinical trials have confirmed the efficacy and safety of toripalimab in treating nasopharyngeal carcinoma.
This study aims to conduct the first single-center, phase II randomized controlled clinical trial in children and adolescents with nasopharyngeal carcinoma, comparing the GP regimen combined with toripalimab induction chemotherapy versus the GP regimen alone. The goal is to optimize the treatment model for pediatric and adolescent NPC, enhance therapeutic efficacy, and provide high-level evidence-based medical support for the international treatment guidelines for pediatric NPC.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| GP combined with Toripalimab + CCRT | Experimental | Drug: GP+Toripalimab+CCRT1. Induction Chemotherapy (GP Regimen combined with Toripalimab) * GP Regimen: Gemcitabine: 1000 mg/m² on Day 1 and Day 8; Cisplatin (DDP): 80 mg/m² on Day 1-3; every 3 weeks for 3 cycles * Toripalimab: For patients weighing ≥ 40 kg: 240 mg on Day 1; For patients weighing < 40 kg: 3 mg/kg; every 3 weeks for 3 cycles 2. Concurrent Chemoradiotherapy (CCRT): Cisplatin (DDP): 100 mg/m², starting on the first day of radiotherapy; every 3 weeks for 3 cycles |
| GP regimen + CCRT | Active comparator | Drug: GP+CCRT1. Induction Chemotherapy (GP Regimen): Gemcitabine: 1000 mg/m² on Day 1 and Day 8; Cisplatin (DDP): 80 mg/m² on Day 1-3, every 3 weeks for 3 cycles. 2. Concurrent Chemoradiotherapy (CCRT): Cisplatin (DDP): 100 mg/m², starting on the first day of radiotherapy, every 3 weeks for 3 cycles. |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- Patients must be informed of the investigational nature of this study and give written informed consent.
- Age between 6 and 18 years, regardless of gender.
- Pathologically confirmed non-keratinizing nasopharyngeal carcinoma (differentiated or undifferentiated type, i.e., WHO Type II or III).
- Clinical stage II-III (according to AJCC 9th edition), excluding T3N0 and T3N1 (only with retropharyngeal lymph node metastasis); patients must be newly diagnosed with nasopharyngeal carcinoma.
- ECOG performance status of 0-1.
Key exclusion criteria (17 listed)
- Older than 18 years.
- Presence of recurrence or distant metastasis.
- Pathologically diagnosed as keratinizing squamous cell carcinoma (WHO Type I).
- History of previous anti-tumor treatment.
- Pregnant or lactating women, and women of childbearing potential not using effective contraception.
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 |
|---|---|---|
| GP+Toripalimab+CCRT1. Induction Chemotherapy (GP Regimen combined with Toripalimab): 1. GP Regimen Gemcitabine: 1000 mg/m² on Day 1 and Day 8; Cisplatin (DDP): 80 mg/m² on Day 1-3; every 3 weeks for 3 cycles. 2. Toripalimab: For patients weighing ≥ 40 kg: 240 mg on Day 1; For patients weighing less than 40 kg: 3 mg/kg; every 3 weeks for 3 cycles. 2. Concurrent Chemoradiotherapy (CCRT): Cisplatin (DDP): 100 mg/m², starting on the first day of radiotherapy; every 3 weeks for 3 cycles | Drug | GP combined with Toripalimab + CCRT |
| GP+CCRT1. Induction Chemotherapy (GP Regimen ): Gemcitabine: 1000 mg/m² on Day 1 and Day 8; Cisplatin (DDP): 80 mg/m² on Day 1-3; every 3 weeks for 3 cycles. 2. Concurrent Chemoradiotherapy (CCRT): Cisplatin (DDP): 100 mg/m², starting on the first day of radiotherapy; every 3 weeks for 3 cycles | Drug | GP regimen + CCRT |
Primary outcome
Complete Response (CR) Rate After Induction Chemotherapy
Time frame: when the induction chemotherapy complete
CR is assessed after induction chemotherapy by independent reviewers, according to the Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1. Complete response defined as the complete disappearance of the target and non-target lesion(s) identified at baseline after radiological evaluation by enhanced MRI (or enhanced CT if indicated).
10 secondary outcomes
Overall survival
Time frame: 3 years
Progression-free survival
Time frame: 3 years
Locoregional failure-free survival(LRRFS)
Time frame: 3 years
Distant metastasis-free survival(DMFS)
Time frame: 3 years
Incidence of acute and late toxicity
Time frame: 3 years
Hormone levels
Time frame: 3 years
Growth condition of Height
Time frame: 3 years
Growth condition of Weight
Time frame: 3 years
Growth condition of BMI
Time frame: 3 years
Quality of life
Time frame: 3 years
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 Mar 2025.
- Individual participant dataSharing planned: No
Related trials for Nasopharyngeal Cancinoma (NPC)
Not yet recruitingPhase 2
Risk-Adapted Anlotinib and PD-L1 for Locoregionally Advanced Nasopharyngeal Carcinoma
Nasopharyngeal Cancinoma (NPC)
Not yet recruitingPhase 2
Dynamic Plasma EBV DNA-Guided Risk-Adapted Treatment With Becotatug Vedotin (MRG003) Plus PD-1 Blockade in Locoregionally Advanced Nasopharyngeal Carcinoma
Nasopharyngeal Cancinoma (NPC), Nasopharyngeal Cancer
RecruitingPhase 3
Prophylactic Nanocrystalline Megestrol Acetate Versus Placebo for Chemoradiotherapy-Related Anorexia in Nasopharyngeal Carcinoma
Nasopharyngeal Cancinoma (NPC)
RecruitingPhase 1
Intratumoral Injection of Recombinant Human Endostatin Adenovirus (EDS01) for the Treatment of Recurrent or Metastatic Head and Neck Tumors
Head & Neck Cancer, Nasopharyngeal Cancinoma (NPC)
Not yet recruitingPhase 2
Platinum-Free Regimen in Nasopharyngeal Carcinoma
Nasopharyngeal Cancinoma (NPC)
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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing GP+Toripalimab+CCRT and GP+CCRT in people with Nasopharyngeal Cancinoma (NPC), run by 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 9 inclusion and 17 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: opening soon, as of the sponsor's last update on 20 Mar 2025. 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 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?
Contact Dong Hua Luo (86-020-87343643, luodh@sysucc.org.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Complete Response (CR) Rate After Induction Chemotherapy (time frame: when the induction chemotherapy complete). 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 Mar 2025.