Comparing toripalimab with placebo in treating advanced nasopharyngeal carcinoma

Toripalimab Versus Placebo Combined With Induction Chemotherapy Followed by Concurrent Chemoradiotherapy for Locoregionally Advanced Nasopharyngeal Carcinoma: a Multicenter, Randomized, Placebo-controlled, Double-blind, Phase 3 Trial

This study is testing if adding a new treatment called toripalimab to standard chemotherapy can help people with advanced nasopharyngeal cancer live longer and feel better compared to those who only get chemotherapy.

Quick facts

• Phase: PHASE3
• Study type: Interventional
• Enrollment: 466 (estimated)
• Ages: 18 Years to 70 Years
• Sex: All
• Sponsor: Sun Yat-sen University (other)
• Drugs / interventions: toripalimab, chemotherapy, prednisone
• Locations: 1 site (Guangzhou, Guangdong)
• Trial ID: NCT06712888 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the efficacy and safety of toripalimab, an anti-PD-1 therapy, when combined with gemcitabine and cisplatin (GP) induction chemotherapy in patients with locoregionally advanced nasopharyngeal carcinoma. Participants will be randomly assigned to receive either the toripalimab combination or a placebo alongside GP chemotherapy. The study aims to determine if the addition of toripalimab improves progression-free survival, overall survival, and response rates compared to the placebo group. Additionally, it will assess safety profiles and explore potential predictive biomarkers related to treatment outcomes.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18-70 with pathologically confirmed locoregionally advanced nasopharyngeal carcinoma and no distant metastasis.

Not a fit: Patients with distant metastasis or those outside the specified age range may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve survival rates and reduce disease recurrence in patients with advanced nasopharyngeal carcinoma.

How similar studies have performed: Previous studies have shown promising results with similar combinations of anti-PD-1 therapy and chemotherapy in treating nasopharyngeal carcinoma, indicating potential for success.

Eligibility criteria

Inclusion Criteria:

1. Voluntarily participates and signs an informed consent form.
2. Aged 18-70 years, male or non-pregnant female.
3. Pathologically confirmed nasopharyngeal non-keratinizing carcinoma (differentiated or undifferentiated, i.e., WHO Type II or III).
4. Staging of any T, N2-3 or T4N1 (9th AJCC/UICC staging), with no distant metastasis.
5. ECOG performance status score of 0-1.
6. Hemoglobin (HGB) ≥90 g/L, neutrocyte count≥1.5×10⁹/L, platelets (PLT) ≥100×10⁹ /L.
7. Liver function: Alanine aminotransferase (ALT) and aspartate aminotransferase (AST)≤2.5×upper limit of normal (ULN), and bilirubin ≤ 1.5×ULN.
8. Adequate renal function: creatinine clearance rate ≥ 60 ml/min (Cockcroft-Gault formula).
9. Women of childbearing potential (WOCBP) who are sexually active must be willing to adhere to effective contraception during treatment and for 1 year after the last dose of the study drug. Men who are sexually active with WOCBP must be willing to adhere to effective contraception during treatment and for 1 year after the last dose of the study drug.

Exclusion Criteria:

1. Age \> 70 years or \< 18 years.
2. Patients with recurrence or distant metastases.
3. Pathologically confirmed nasopharyngeal keratinizing squamous cell carcinoma (WHO Type I).
4. Patients who have previously undergone radiotherapy or systemic chemotherapy.
5. Hepatitis B surface antigen (HBsAg) positive and hepatitis B virus DNA \>1000 copies/ml or 200IU/ml.
6. Hepatitis C virus (HCV) antibody positive.
7. Has active autoimmune disease, except type I diabetes, hypothyroidism treated with replacement therapy, and skin disease that doesn't require systemic treatment (e.g., vitiligo, psoriasis, or alopecia).
8. Has any condition that required systemic corticosteroid (equivalent to prednisone \>10mg/d) or other immunosuppressive therapy within 28 days before informed consent. Patients who received systemic corticosteroid equivalent to prednisone ≤10mg/d, inhale or topical corticosteroids will be allowed.
9. Has a known history of active bacillus tuberculosis within 1 year; patients with adequately treated active bacillus tuberculosis over 1 year ago will be allowed.
10. Has a known history of interstitial lung disease.
11. Has received a live vaccine within 30 days before informed consent or will receive a live vaccine in the near future.
12. Is pregnant or breastfeeding.
13. Prior malignancy within 5 years, except in situ cancer, adequately treated non-melanoma skin cancer and papillary thyroid carcinoma.
14. Has known allergy to large molecule protein products or any compound of toripalimab.
15. Has a known history of human immunodeficiency virus infection.
16. Any other condition, including symptomatic heart failure, unstable angina, myocardial infarction, active infection requiring systemic therapy, mental illness, or domestic/social factors, deemed by the investigator to be likely to interfere with a patient's ability to sign informed consent, cooperate and participate in the study or interferes with the interpretation of the results.

Where this trial is running

Guangzhou, Guangdong

• Sun Yat-sen University Cancer Center — Guangzhou, Guangdong, China (RECRUITING)

Study contacts

• Study coordinator: Hai-Qiang Mai, Ph. D.

Conditions: Nasopharyngeal Carcinoma, Anti-PD-1 therapy, induction chemotherapy