Evaluation of the Length of Treatment With PD-1/PD-L1 Inhibitors in Patients With Advanced Solid Tumors


Based on the overwhelming positive response to this survey and the large number of patients being treated with PD-1/PD-L1 therapy in the UPMC system, the investigators are proposing a trial that will randomize patients who have disease stability to stop treatment at 1 year or continue treatment until disease progression. The investigators anticipate that the results of this study will answer questions regarding the optimal duration of treatment. therapy.

Full Title of Study: “A Randomized Non-inferiority Trial Evaluating the Length of Treatment With PD-1/PD-L1 Inhibitors in Patients With Advanced Solid Tumors”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: October 2025

Detailed Description

Within the UPMC system, approximately 2,300 patients received PD-1/PD-L1 therapy for a variety of advanced solid tumors within the past year. It is anticipated that this number will increase as the clinical indications for treatment with these agents also increase. The investigators conducted a survey of 60 Medical Oncologists within the UPMC system regarding their interest in a trial that will attempt to address the question of optimal length of PD-1/PD-L1 treatment. Fifty-two (86.7%) physicians indicated that they would participate in a clinical trial that had a primary goal of determining whether it was feasible to stop immunotherapy after 1 year of treatment.


  • Drug: Continue PD-1/PD-L1 Inhibitors treatment
    • Continued treatment with PD-1/PD-L1-1 inhibitor
  • Other: Discontinue PD-1/PD-L1-1 inhibitor
    • Discontinued treatment with PD-1/PD-L1-1 inhibitor

Arms, Groups and Cohorts

  • Active Comparator: Continue Treatment with PD-1/PD-L1 inhibitor
    • Continued standard of care treatment with PD-1/PD-L1 -1 checkpoint inhibitor after 12 months of checkpoint inhibitor treatment.
  • Experimental: Discontinue Treatment with PD-1/PD-L1-1 inhibitor
    • Discontinued standard of care treatment with PD-1/PD-L1 -1 checkpoint inhibitor after 12 months of checkpoint inhibitor treatment.

Clinical Trial Outcome Measures

Primary Measures

  • Time to next treatment
    • Time Frame: Up to 36 months
    • In patients who have already been treated with a PD-1 or PD-L1 inhibitor for one year, the difference in progression-free survival (time to next treatment, progression or death, whichever occurs first) between patients who stop treatment and patients who continue treatment.

Secondary Measures

  • Incidence of irAEs (Immune-Related Adverse Events)
    • Time Frame: Up to 36 months
    • Proportion of participants in a disease stratum and treatment arm who experience at least one AE of any grade (per Common Terminology Criteria for Adverse Events (CTCAE v5.0)), at least possibly related to treatment in the categories of colitis, hepatitis, pnemonitis, hypophysitis or hypopituitarism, hypothyroidism, fatigue, diarrhea, rash, arthritis, arthralgia, back pain, musculoskeletal pain or myalgia, or any other category that is felt to be related to treatment.
  • Overall Survival (OS)
    • Time Frame: Up to 36 months
    • The length of time from the start of treatment that patients are still alive.
  • Best Objective Response (BOR)
    • Time Frame: Up to 36 months
    • Proportions of participants who restart for disease progression in each disease stratum, who experience a best objective response (progressive disease, stable disease, partial response, complete response) per RECIST v1.1 (Response Evaluation Criteria in Solid Tumors);Complete Response (CR): Disappearance of all target lesions. Any pathological lymph nodes (target or non-target) with reduction in short axis to <10 mm;Partial Response (PR): ≥ 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum of diameters;Stable Disease (SD): Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, (reference smallest sum diameters);Progressive Disease (PD): ≥ 20% increase in the sum of diameters of target lesions (reference smallest sum diameters); the sum must also demonstrate an absolute increase of at least 5 mm; (appearance ≥ 1 new lesions is considered progression).

Participating in This Clinical Trial

Inclusion Criteria

  • All patients must have an advanced solid tumor malignancy (specifically NSCLC, bladder, HNSCC, renal, melanoma, cervical, Merkel cell, MMR/MSI [colon, rectal, cholangio, esophageal, ovarian, uterine], anal, gastric and GE junction, hepatocellular, triple negative breast cancer) that is being treated with a PD-1/PD-L1 inhibitor including pembrolizumab, nivolumab, atezolizumab, durvalumab, or avelumab according to standard of care treatment. – Patients who initially started treatment with another agent in combination with the PD-1/PD-L1 inhibitor, i.e. chemotherapy, ipilumumab, are eligible. – Patients must have at least stable disease as evidenced by scans performed within 6 weeks of randomization. – Signed Informed consent allowing randomization to stopping immunotherapy at 1 year ± 4 weeks versus continued treatment beyond 1 year. – Patients can have measurable or non-measurable disease per iRECIST. – Patients cannot be enrolled in a clinical trial. Exclusion Criteria:

  • Patients with documented progressive disease prior to randomization. – Patients with an immune-related toxicity preventing the continuation of treatment beyond 1 year at the treating physician's discretion.

Gender Eligibility: All

Minimum Age: 18 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Antoinette J Wozniak
  • Provider of Information About this Clinical Study
    • Sponsor-Investigator: Antoinette J Wozniak, Professor, Department of Otolaryngology, of Immunology, and of Radiation Oncology – University of Pittsburgh
  • Overall Official(s)
    • Antoinette J Wozniak, MD, FACP, Principal Investigator, UPMC Hillman Cancer Center
  • Overall Contact(s)
    • Ruth Jen, BSN, ruthj2@upmc.edu, ruthj2@upmc.edu

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