Combining Pembrolizumab with Chemotherapy for Advanced Head and Neck Cancer

EFFECT-neo: A Prospective, Open-label, Multicenter Phase III Study to Evaluate Efficacy and Safety of Pembrolizumab Combined With Standard Chemotherapy in the Neoadjuvant Treatment of Local Advanced (LA) HNSCC

This study is testing whether adding a drug called pembrolizumab to standard chemotherapy can help people with advanced head and neck cancer respond better to treatment.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 272 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Beijing Tongren Hospital (Academic / other)
• Drugs / interventions: pembrolizumab, chemotherapy, immunotherapy, radiation
• Locations: 1 site (Beijing, Beijing Municipality)
• Trial ID: NCT06102395 on ClinicalTrials.gov

What this trial studies

This phase III clinical trial evaluates the efficacy and safety of pembrolizumab combined with standard chemotherapy in patients with untreated stage IIIA to IVB head and neck squamous cell carcinoma. Participants are randomized to receive either pembrolizumab plus chemotherapy or chemotherapy alone for two cycles, followed by imaging evaluations to determine the next steps in treatment. The study aims to improve the rate of pathological complete response compared to traditional chemotherapy regimens. Close monitoring of adverse reactions and regular follow-ups will be conducted to assess patient outcomes.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18 and older with untreated stage IIIA-IVB head and neck squamous cell carcinoma.

Not a fit: Patients with distant metastasis or those who have previously received immunotherapy may not benefit from this study.

Why it matters

Potential benefit: If successful, this approach could significantly enhance treatment effectiveness and safety for patients with advanced head and neck cancer.

How similar studies have performed: Other studies have shown promising results with similar combinations of immunotherapy and chemotherapy, suggesting potential for success in this approach.

Eligibility criteria

Inclusion Criteria:

1. Patients with stage IIIA-IVB head and neck squamous cell carcinoma confirmed by histology and/or cytology;
2. Have not received immunotherapy in the past;
3. The researchers believe that he can safely receive pembrolizumab combined with chemotherapy or neoadjuvant chemotherapy;
4. Age ≥18 years;
5. ECOG 0-2;
6. Measurable disease as defined by RECIST v1.1;
7. Organs function normally;
8. Female and male participants of reproductive potential must agree to use appropriate contraception throughout the study period and for 180 days after the last study treatment;
9. Male participants must not donate sperm throughout the study and for 180 days after the last study treatment.

Exclusion Criteria:

1. Presence of distant metastasis;
2. Female subjects with a positive urine pregnancy test within 72 hours before the start of the study or within 24 hours after starting radiation therapy (with or without cisplatin);
3. received a live vaccine within 30 days before enrollment;
4. Diagnosed with immunodeficiency or receiving systemic steroid treatment or any other form of immunosuppressive treatment within 7 days before enrollment;
5. Have imaging detectable (even if asymptomatic and/or previously treated) central nervous system metastases and/or cancerous meningitis;
6. Have undergone surgery before commencing the study or have failed to recover adequately from toxicity or complications resulting from the intervention;
7. Previous allogeneic tissue/solid organ transplant;
8. Severe hypersensitivity reaction (≥Grade 3) to pembrolizumab or any of its excipients, radiotherapy, platinum, paclitaxel, 5-FU or their analogs;
9. Have an active autoimmune disease requiring systemic therapy in the past 2 years;
10. History of (non-infectious) pneumonia requiring steroid treatment;
11. Have a history of human immunodeficiency virus (HIV) infection;
12. Have a history of hepatitis B or be positive for hepatitis B virus (defined as a positive reaction to hepatitis B surface antigen \[HBsAg\]) or active hepatitis C (defined as detection of hepatitis C virus \[HCV\] ribonucleic acid).
13. Have any medical history, treatment, or laboratory abnormalities that could confound the study results, interfere with participant participation throughout the study, or be detrimental to the best interests of the participant (e.g., Hashimoto's thyroiditis, etc.);
14. Have a known history of mental illness or substance abuse disorder

Where this trial is running

Beijing, Beijing Municipality

• Beijing Tongren Hospital — Beijing, Beijing Municipality, China (Recruiting)

Study contacts

• Study coordinator: Zhigang Huang

Conditions Head and Neck Squamous Cell Carcinoma; Neoadjuvant Therapy; Pembrolizumab