Clinical trial NCT06771674: Real-world Study of First Line Pembrolizumab- or Nivolumab-treated HPV-positive Recurrent/Metastatic Oropharyngeal Squamous Cell Carcinoma
Official title: Real-world Study of Patients with Human Papillomavirus-positive Recurrent/ Metastatic Oropharyngeal Squamous Cell Carcinoma Treated with First Line Pembrolizumab or Nivolumab with or Without Chemotherapy
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Oropharyngeal Squamous Cell Carcinoma
Keywords: oropharyngeal squamous cell carcinoma
throat
head and neck cancer
squamous cell cancer
OPSCC
HNSCC
human papillomavirus
HPV
immunotherapy
real-world study
PD-L1 therapy
checkpoint inhibitor
pembrolizumab
nivolumab
Lead sponsor: Hookipa Biotech GmbH
Sponsor class: INDUSTRY
Collaborators: name: Cardinal Health
First posted: 2025-01-13
Enrollment: 663
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Real-world patients with HPV+ R/M OPSCC who received 1L pembrolizumab monotherapy, or nivolumab with or without chemotherapy will be evaluated. The ECAs derived from this real-world study are matched to respective study populations from interventional clinical studies. Propensity score matching will be used to generate the ECAs. The baseline characteristics used for matching will encompass key demographic and clinical factors potentially associated with treatment outcomes and may include:

* Type of disease (only recurrent/metastatic including any recurrent)
* Smoking history
* Sex at birth
* Race/Ethnicity
* Hemoglobin

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Patient demographics with ECA 1
timeFrame: Whole study period (05 June 2024 through 30 April 2025)
measure: Clinical characteristics with ECA 1
timeFrame: Whole study period (05 June 2024 through 30 April 2025)
measure: Treatment patterns with ECA 1
timeFrame: Whole study period (05 June 2024 through 30 April 2025)
measure: 1L treatment outcomes with ECA 1
timeFrame: Whole study period (05 June 2024 through 30 April 2025)
measure: Patient demographics with ECA 2
timeFrame: Whole study period (05 June 2024 through 30 April 2025)
measure: Clinical characteristics with ECA 2
timeFrame: Whole study period (05 June 2024 through 30 April 2025)
measure: 1L treatment outcomes with ECA 2
timeFrame: Whole study period (05 June 2024 through 30 April 2025)
measure: Treatment patterns with ECA 2
timeFrame: Whole study period (05 June 2024 through 30 April 2025)
measure: Patient demographics with ECA3
timeFrame: Whole study period (05 June 2024 through 30 April 2025)
measure: Clinical characteristics with ECA3
timeFrame: Whole study period (05 June 2024 through 30 April 2025)
measure: Treatment patterns with ECA3
timeFrame: Whole study period (05 June 2024 through 30 April 2025)
measure: 1L treatment outcomes with ECA3
timeFrame: Whole study period (05 June 2024 through 30 April 2025)
measure: Patient demographics with ECA 4
timeFrame: Whole study period (05 June 2024 through 30 April 2025)
measure: Clinical characteristics with ECA 4
timeFrame: Whole study period (05 June 2024 through 30 April 2025)
measure: Treatment patterns with ECA 4
timeFrame: Whole study period (05 June 2024 through 30 April 2025)
measure: 1L treatment outcomes with ECA 4
timeFrame: Whole study period (05 June 2024 through 30 April 2025)
Secondary outcomes:
measure: Real-world ECA 1 population versus a population from an interventional clinical study
timeFrame: Whole study period (05 June 2024 through 30 April 2025)
measure: Real-world ECA 2 population versus a population from an interventional clinical study
timeFrame: Whole study period (05 June 2024 through 30 April 2025)
measure: Real-world ECA 3 population versus a population from an interventional clinical study
timeFrame: Whole study period (05 June 2024 through 30 April 2025)
measure: Real-world ECA 4 population versus a population from an interventional clinical study
timeFrame: Whole study period (05 June 2024 through 30 April 2025)
Other outcomes:
Locations:
city: Dublin
country: United States
facility: Cardinal Health
state: Ohio
Study officials:
References: