Actively Recruiting

Phase 3
Age: 18Years +
All Genders
ID06102395

EFFECT-neo: A Phase III Study Evaluating Pembrolizumab Plus Standard Chemotherapy as Neoadjuvant Treatment for Locally Advanced Head and Neck Squamous Cell Carcinoma

Led by Beijing Tongren Hospital · Updated on 2023-10-30

272

Participants Needed

1

Research Sites

156 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

Researchers are evaluating the safety and effectiveness of pembrolizumab combined with standard chemotherapy as a neoadjuvant treatment for patients with untreated stage IIIA to IVB head and neck squamous cell carcinoma. This includes cancers of the oral cavity, oropharynx, hypopharynx, and larynx. The study is a phase III, prospective, open-label, multi-center trial aiming to improve the rate of complete pathological response before surgery or radiotherapy. Participants are randomly assigned to receive either pembrolizumab plus chemotherapy or chemotherapy alone for two cycles. The chemotherapy regimen includes drugs such as cisplatin, carboplatin, nedaplatin, nab-paclitaxel, docetaxel, liposomal paclitaxel, or fluorouracil. After neoadjuvant treatment, imaging assessments determine further treatment: complete responders receive radiotherapy with or without chemotherapy, while partial responders or those with stable disease proceed to surgery followed by standard care. Disease progression leads to standard treatment. Adverse reactions are closely monitored throughout. Participants undergo regular follow-ups every three months for the first year and every six months for the following three years. These visits include assessments of treatment response, adverse events, quality of life, and survival outcomes. Researchers measure the pathological complete response rate at 12 weeks as the primary outcome, alongside secondary outcomes such as response rates, event-free survival, overall survival, functional preservation, and treatment-related adverse events. The total study duration may extend up to several years to capture long-term results.

CONDITIONS

Brief Title

Evaluating the Efficacy and Safety of Pembrolizumab Plus Standard Chemotherapy in the Neoadjuvant Treatment of Local Advanced (LA) HNSCC

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Patients with stage IIIA to IVB head and neck squamous cell carcinoma confirmed by histology or cytology
  • No prior immunotherapy treatment
  • Able to safely receive pembrolizumab combined with chemotherapy or chemotherapy alone
  • Age 18 years or older
  • ECOG performance status 0 to 2
  • Measurable disease defined by RECIST v1.1
  • Normal organ function
  • Female and male participants of reproductive potential agree to use contraception during the study and for 180 days after last treatment
  • Male participants agree not to donate sperm during the study and for 180 days after last treatment
Not Eligible

You will not qualify if you...

  • Presence of distant metastasis
  • Positive urine pregnancy test within 72 hours before study start or within 24 hours after starting radiation therapy
  • Receipt of live vaccine within 30 days before enrollment
  • Immunodeficiency or use of systemic steroids or immunosuppressants within 7 days before enrollment
  • Imaging-detectable central nervous system metastases or cancerous meningitis
  • Previous surgery before study start or inadequate recovery from surgery complications
  • History of allogeneic tissue or organ transplant
  • Severe hypersensitivity (grade 3 or higher) to pembrolizumab or chemotherapy drugs
  • Active autoimmune disease requiring systemic therapy in past 2 years
  • History of pneumonia requiring steroid treatment
  • History of HIV infection
  • History or active hepatitis B or C infection
  • Medical conditions or treatments that could interfere with study participation or results
  • Known mental illness or substance abuse disorder

AI-Screening

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Treatment

Duration - Approximately 6 weeks (2 cycles of 3 weeks each)

Participants receive 2 cycles of neoadjuvant therapy with pembrolizumab plus standard chemotherapy or standard chemotherapy alone, given every 3 weeks.

2 treatment visits (in-person) every 3 weeks

Surgery and Immediate Post-operative Care

Duration - Up to 2 weeks

Participants with partial response or stable disease after treatment undergo surgery within 2 weeks followed by standard care.

1 surgery visit and immediate post-operative monitoring

Adjuvant Treatment

Duration - Duration varies based on radiotherapy schedule

Participants with complete response receive radiotherapy with or without chemotherapy as adjuvant treatment.

Visit frequency depends on adjuvant treatment plan

Follow-up

Duration - 4 years

All participants are followed for recurrence and survival, with assessments every 3 months for 1 year, then every 6 months for 3 years.

Regular follow-up visits every 3 months for 1 year, then every 6 months for 3 years

Trial Site Locations

Total: 1 location

1

Beijing Tongren Hospital

Beijing, Beijing Municipality, China, 100730

Actively Recruiting

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Research Team

Z

Zhigang Huang

Y

Yang Zhang

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

2

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