Real-world Study of First Line Pembrolizumab- or Nivolumab-treated HPV-positive Recurrent/Metastatic Oropharyngeal Squamous Cell Carcinoma

Active, not recruiting

• Conditions: Oropharyngeal Squamous Cell Carcinoma
• Interventions: Drug: Pembrolizumab; Drug: Nivolumab

• Registration Number: NCT06771674
• Lead Sponsor: Hookipa Biotech GmbH

Brief Summary

Pembrolizumab and nivolumab (with or without chemotherapy) are used to treat head and neck cancer. The middle part of the throat (oropharynx) is a common location for head and neck cancer. This cancer is known as oropharyngeal squamous cell carcinoma (OPSCC) and is most often caused by human papillomavirus (HPV) infection. This real-world evidence study carried out in the United States (US) will assess patient demographic and clinical characteristics, treatment patterns, and effectiveness of pembrolizumab and nivolumab (with or without chemotherapy) in patients with HPV positive (HPV+) OPSCC after their cancer spread (metastatic) and/or returned (recurrent). The collected real-world data can be compared with data derived from matched study populations in clinical studies that test new therapies in patients with HPV+ OPSCC. This will allow a more reliable evaluation of the clinical benefits and better-informed design of future clinical studies in this patient population.

Detailed Description

This non-interventional study will be conducted as a single-phase, multicenter medical chart review of US patients with HPV+ recurrent and/or metastatic (R/M) OPSCC treated with first-line (1L) pembrolizumab or nivolumab (with or without chemotherapy). Participating physicians (Cardinal Health's Oncology Provider Extended Network) and clinical sites (Practice Research Network) will be asked to submit data on patient demographics, clinical characteristics, treatment patterns, and 1L treatment outcomes during a 10-week data collection period. The networks do not overlap, eliminating the chance of duplicate patient records. The completion of data collection defines the study completion date. The data collection period will be followed by a data validation period based on responses of the participating physicians and clinical sites to follow-up questions.

The data obtained in this study will create external control arms (ECAs) comprised of patients with HPV+ R/M OPSCC initiating 1L therapy with pembrolizumab monotherapy, or nivolumab with or without chemotherapy. The use of these ECAs will allow for a more accurate estimation of the effectiveness of therapies currently assessed in interventional clinical studies versus pembrolizumab or nivolumab (with or without chemotherapy) as assessed in this observational study in a non-randomized setting. This information will serve to increase the reliability of the study data from interventional clinical studies investigating therapies in matching indications and patient populations and improve the accuracy of the clinical data to inform future clinical trials.

Study Timeline

• Study Start: 2024-06-05
• Study First Submitted: 2025-01-08
• Study First Submitted QC: 2025-01-08
• Study First Posted: 2025-01-13
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-10
• Last Update Posted: 2025-03-11
• Primary Completion: 2025-04-30
• Study Completion: 2025-06-30

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 663

Inclusion Criteria

• Confirmed diagnosis of HPV+ R/M OPSCC (base and posterior one-third of tongue, the tonsils, soft palate, and lateral pharyngeal walls).
• Initiated and treated with pembrolizumab monotherapy, or nivolumab (monotherapy or in combination with chemotherapy) as 1L therapy for HPV+ R/M OPSCC on or after 05 June 2024 (may have had curative intent treatment including surgery, chemotherapy, and/or radiotherapy for non-metastatic/non-recurrent disease).
• Have access to baseline assessment scan within 30 days prior to initiating 1L therapy for HPV+ R/M OPSCC and response assessment confirmed by objective measurements within 26 weeks of initiation of 1L therapy.
• Must have been followed for 6 months or more since initiation of 1L therapy for R/M OPSCC.

Exclusion Criteria

• Participation in a controlled clinical trial for 1L R/M OPSCC.
• Central nervous system involvement or other concurrent malignancy at the time of 1L initiation.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
ECA 1	Pembrolizumab	A representative sample of real-world patients with HPV+ R/M OPSCC treated with 1L pembrolizumab monotherapy.
ECA 3	Pembrolizumab	A representative sample of real-world patients with HPV+ R/M OPSCC and high CPS (at least 20 or more) treated with 1L pembrolizumab monotherapy.
ECA 2	Nivolumab	A representative sample of real-world patients with HPV+ R/M OPSCC treated with 1L pembrolizumab or nivolumab with chemotherapy.
ECA 2	Pembrolizumab	A representative sample of real-world patients with HPV+ R/M OPSCC treated with 1L pembrolizumab or nivolumab with chemotherapy.
ECA 4	Nivolumab	A representative sample of real-world patients with HPV+ R/M OPSCC treated with 1L pembrolizumab or nivolumab with or without chemotherapy.
ECA 4	Pembrolizumab	A representative sample of real-world patients with HPV+ R/M OPSCC treated with 1L pembrolizumab or nivolumab with or without chemotherapy.

Primary Outcome Measures

Name	Time	Method
Patient demographics with ECA 1	Whole study period (05 June 2024 through 30 April 2025)	Patient demographics among real-world patients with HPV+ R/M OPSCC treated with 1L pembrolizumab monotherapy.
Clinical characteristics with ECA 1	Whole study period (05 June 2024 through 30 April 2025)	Clinical characteristics among real-world patients with HPV+ R/M OPSCC treated with 1L pembrolizumab monotherapy.
Treatment patterns with ECA 1	Whole study period (05 June 2024 through 30 April 2025)	Treatment patterns among real-world patients with HPV+ R/M OPSCC treated with 1L pembrolizumab monotherapy.
1L treatment outcomes with ECA 1	Whole study period (05 June 2024 through 30 April 2025)	1L treatment outcomes among real-world patients with HPV+ R/M OPSCC treated with 1L pembrolizumab monotherapy.
Patient demographics with ECA 2	Whole study period (05 June 2024 through 30 April 2025)	Patient demographics among real-world patients with HPV+ R/M OPSCC treated with 1L pembrolizumab or nivolumab with chemotherapy.
Clinical characteristics with ECA 2	Whole study period (05 June 2024 through 30 April 2025)	Describe the clinical characteristics among real-world patients with HPV+ R/M OPSCC treated with 1L pembrolizumab or nivolumab with chemotherapy.
1L treatment outcomes with ECA 2	Whole study period (05 June 2024 through 30 April 2025)	1L treatment outcomes among real-world patients with HPV+ R/M OPSCC treated with 1L pembrolizumab or nivolumab with chemotherapy.
Treatment patterns with ECA 2	Whole study period (05 June 2024 through 30 April 2025)	Treatment patterns among real-world patients with HPV+ R/M OPSCC treated with 1L pembrolizumab or nivolumab with chemotherapy.
Patient demographics with ECA3	Whole study period (05 June 2024 through 30 April 2025)	Patient demographics among real-world patients with HPV+ R/M OPSCC and with a CPS of at least 20 or more treated with 1L pembrolizumab monotherapy.
Clinical characteristics with ECA3	Whole study period (05 June 2024 through 30 April 2025)	Clinical characteristics among real-world patients with HPV+ R/M OPSCC and with a CPS of at least 20 or more treated with 1L pembrolizumab monotherapy.
Treatment patterns with ECA3	Whole study period (05 June 2024 through 30 April 2025)	Treatment patterns among real-world patients with HPV+ R/M OPSCC and with a CPS of at least 20 or more treated with 1L pembrolizumab monotherapy.
1L treatment outcomes with ECA3	Whole study period (05 June 2024 through 30 April 2025)	1L treatment outcomes among real-world patients with HPV+ R/M OPSCC and with a CPS of at least 20 or more treated with 1L pembrolizumab monotherapy.
Patient demographics with ECA 4	Whole study period (05 June 2024 through 30 April 2025)	Patient demographics among real-world patients with HPV+ R/M OPSCC treated with 1L pembrolizumab or nivolumab with or without chemotherapy.
Clinical characteristics with ECA 4	Whole study period (05 June 2024 through 30 April 2025)	Describe the clinical characteristics among real-world patients with HPV+ R/M OPSCC treated with 1L pembrolizumab or nivolumab with or without chemotherapy.
Treatment patterns with ECA 4	Whole study period (05 June 2024 through 30 April 2025)	Treatment patterns among real-world patients with HPV+ R/M OPSCC treated with 1L pembrolizumab or nivolumab with or without chemotherapy.
1L treatment outcomes with ECA 4	Whole study period (05 June 2024 through 30 April 2025)	1L treatment outcomes among real-world patients with HPV+ R/M OPSCC treated with 1L pembrolizumab or nivolumab with or without chemotherapy.

Secondary Outcome Measures

Name	Time	Method
Real-world ECA 1 population versus a population from an interventional clinical study	Whole study period (05 June 2024 through 30 April 2025)	Compare clinical outcomes of a matched patient population from an interventional clinical study with those of the representative ECA 1 real-world population with HPV+ R/M OPSCC treated with 1L pembrolizumab monotherapy from this study.
Real-world ECA 2 population versus a population from an interventional clinical study	Whole study period (05 June 2024 through 30 April 2025)	Compare clinical outcomes of a matched patient population from an interventional clinical study with those of the representative ECA 2 real-world population with HPV+ R/M OPSCC treated with pembrolizumab or nivolumab with chemotherapy from this study.
Real-world ECA 3 population versus a population from an interventional clinical study	Whole study period (05 June 2024 through 30 April 2025)	Compare clinical outcomes of a matched patient population from an interventional clinical study with those of the representative ECA 3 real-world population with HPV+ R/M OPSCC and with CPS of at least 20 or more treated with pembrolizumab monotherapy from this study.
Real-world ECA 4 population versus a population from an interventional clinical study	Whole study period (05 June 2024 through 30 April 2025)	Compare clinical outcomes of a matched patient population from an interventional clinical study with those of the representative ECA 4 real-world population with HPV+ R/M OPSCC treated with pembrolizumab or nivolumab with or without chemotherapy from this study.

Trial Locations

Locations (1)

Cardinal Health; 🇺🇸 Dublin, Ohio, United States