Pembrolizumab in Advanced Hepatocellular Carcinoma as Second-line Treatment After Failure of Sorafenib

NCT03163992 | Completed Phase 2 Results FDA Drug

• 1 other identifier: 2016-12-127
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

Brief Summary

This is a single-arm, single-center, open-label trial of pembrolizumab (MK-3475) in subjects with advanced hepatocellular carcinoma as second-line treatment after failure of sorafenib. Approximately 60 subjects will be enrollment to evaluate the efficacy and safety of pembrolizumab. Enrollment will begin with all subjects without regard for PD-L1 expression status. An evaluable specimen for PD-L1 status must be available and confirmed prior to enrollment. All study subjects will be evaluated every 6 weeks (+/- 7 days) following the date of IP drug adminstration for the first 12 months and every 12 weeks (+/- 7 days) thereafter until progression of disease is documented with radiologic imaging (computed tomography or magnetic resonance imaging). The primary efficacy endpoint is ORR (objective response rate) per RECIST 1.1.

Conditions

Hepatocellular Carcinoma

Outcome Measures

Primary Outcomes (1)

• Overall Response Rate

  Overall response rate (ORR) is defined as the proportion of patients who have a partial or complete response to therapy; it does not include stable disease and is a direct measure of drug tumoricidal activity. (biomarker negative vs biomarker Advanced Hepatocellular Carcinoma) Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by CT or MRI Complete Response (CR); Disappearance of all target lesions Partial Response (PR); \>=30% decrease in the sum of the longest diameter of target lesions Overall Response (OR) = CR + PR

  through study completion, an average of 24 months

Secondary Outcomes (1)

• Progression-free Survival (PFS) and Overall Survival (OS)

  through study completion, an average of 24 months

Study Arms (1)

pembrolizumab

EXPERIMENTAL

Pembrolizumab (MK-3475) 200 mg every 3 weeks (Q3W)

Drug: Pembrolizumab

Interventions

Pembrolizumab DRUG

Pembrolizumab (MK-3475) 200 mg every 3 weeks (Q3W)

Also known as: MK3475

pembrolizumab

Eligibility Criteria

• Age: 20 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Be willing and able to provide written informed consent/assent for the trial. The subject may also provide consent for Biomedical Research. However, the subject may participate in the main trial without participating in Biomedical Research.
• Have histologically or cytologically confirmed diagnosis of HCC (fibrolamellar and mixed hepatocellular/cholangiocarcinoma subtypes are not eligible) based on pathology report.
• Have Barcelona Clinic Liver Cancer (BCLC) Stage C disease, or BCLC Stage B disease not amenable to locoregional therapy or refractory to locoregional therapy, and not amenable to a curative treatment approach.
• Have a Child-Pugh class A liver score
• Has experienced documented objective radiographic or clinical disease progression during first-line sorafenib therapy.
• Have measurable disease based on RECIST1.1 as determined by investigator.
• Be willing to provide fresh tissue for biomarker analysis, and, based on the adequacy of the tissue sample quality for assessment of biomarker status. Repeat samples may be required if adequate tissue is not provided. Newly obtained endoscopic biopsy specimens are preferred to archived samples and formalin-fixed, paraffin-embedded (FFPE) block specimens are preferred to slides.

You may not qualify if:

• Is currently participating and receiving study therapy or has participated in a study of an investigational agent and received study therapy or used an investigational device within 4 weeks of the first dose of treatment.
• Has received sorafenib within 14 days of first dose of study medication.
• Has had esophageal or gastric variceal bleeding within the last 6 months. All subjects will be screened for esophageal varices, unless such screening has been performed in the past 12 months before first dose of treatment. If varices are present, they should be treated according to institutional standards before starting study treatment.
• Had a solid organ transplant.
• Has active autoimmune disease that has required systemic treatment in past 2 years (i.e., with use of disease-modifying agents, corticosteroids, or immunosuppressive drugs). Replacement therapy (e.g., thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment.
• Has a diagnosis of immunodeficiency or is receiving systemic steroid therapy or any other form of immunosuppressive therapy within 7 days prior to the first dose of trial treatment.
• Has received locoregional therapy to liver (transcatheter chemoembolization \[TACE\], transcatheter embolization \[TAE\], radiation, radioembolization, or ablation) or major surgery to liver or other site within 4 weeks prior to the first dose of study drug. Minor surgery (e.g., simple excision, tooth extraction) must have occurred at least 7 days prior to the first dose of study treatment (Cycle 1, Day 1).

Sponsors & Collaborators

• Samsung Medical Center: lead

Study Sites (1)

Samsung Medical Center

Seoul, South Korea

Location

MeSH Terms

Conditions

Carcinoma, Hepatocellular

Interventions

pembrolizumab

Condition Hierarchy (Ancestors)

Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Liver Neoplasms; Digestive System Neoplasms; Neoplasms by Site; Digestive System Diseases; Liver Diseases

Results Point of Contact

• Title: Jeeyun Lee
• Organization: Samsung Medical Center

jyunlee@skku.edu

8223410-1779

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD, PhD

Model Details: Pembrolizumab (MK-3475) 200 mg every 3 weeks (Q3W)

Study Record Dates

• First Submitted: April 26, 2017
• First Posted: May 23, 2017
• Study Start: December 26, 2017
• Primary Completion: May 17, 2021
• Study Completion: May 17, 2021
• Last Updated: August 8, 2022
• Results First Posted: August 4, 2022

Record last verified: 2022-08

Locations

KR (1)