Phase Ib/II Study of INC280 + PDR001 or PDR001 Single Agent in Advanced HCC
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT02795429 |
Recruitment Status :
Active, not recruiting
First Posted : June 10, 2016
Last Update Posted : January 13, 2021
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Advanced Hepatocellular Carcinoma | Drug: PDR001 Drug: INC280 | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 90 participants |
Allocation: | Non-Randomized |
Intervention Model: | Parallel Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Phase Ib/II, Open-label, Multi-center Study of INC280 in Combination With PDR001 or PDR001 Single Agent in Advanced Hepatocellular Carcinoma. |
Actual Study Start Date : | June 15, 2016 |
Estimated Primary Completion Date : | March 25, 2021 |
Estimated Study Completion Date : | March 25, 2021 |

Arm | Intervention/treatment |
---|---|
Experimental: INC280+PDR001
PDR001 + INC280 treatment in Phase II
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Drug: PDR001
PDR001 will be administered intravenously Drug: INC280 INC280 will be administered orally |
Experimental: PDR001 single agent
PDR001 single agent treatment in Phase II
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Drug: PDR001
PDR001 will be administered intravenously |
- Number of participants with Dose Limiting Toxicities (DLTs) [ Time Frame: During the first 2 cycles (42 days) of treatment (Phase Ib) ]To identify the Maximum Tolerated Dose (MTD)/Recommended Phase 2 Dose (RP2D).
- Overall response rate (ORR) per Response Evaluation Criteria in Solid Tumors (RECIST v1.1) [ Time Frame: From cycle 1, every 6 weeks up to cycle 12, then every 3 cycles until progression of disease per irRC or patient withdrawal, for up to 3 years (Phase II) ]
- Best overall response (BOR) [ Time Frame: From cycle 1, every 6 weeks up to cycle 12, then every 3 cycles until progression of disease per irRC or patient withdrawal, for up to 3 years ]
- Duration of overall response (DOR) [ Time Frame: Baseline, every 6 weeks until progression, for up to 3 years (Phase II) ]
- Time to response (TTR) [ Time Frame: Baseline, every 6 weeks until response (Phase II), for up to 3 years ]
- Progression-free survival (PFS) [ Time Frame: Baseline, every 6 weeks until progression for up to 3 years ]
- Time to progression (TTP) [ Time Frame: Baseline, every 6 weeks until progression for up to 3 years ]
- Overall survival (OS) [ Time Frame: Every 12 weeks until the end of study for up to 3 years ]
- Overall response rate (ORR) [ Time Frame: Baseline, every 6 weeks until progression for up to 3 years (Phase Ib) ]
- Tiime to progression (TTP) [ Time Frame: Baseline, every 6 weeks until progression for up to 3 years ]
- Pharmacokinetics: Area under the plasma concentration versus time curve (AUC) (INC280) [ Time Frame: Day 1 of the first 6 cycles ]
- PK: Area under the serum concentration versus time curve (AUC) (PDR001) [ Time Frame: Days 1, 2, 3, 4, 8, 11 and 15 of Cycles 1 and 3, Day 1 of Cycles 2, 4, 5 and 6 ]
- PK: Plasma concentration vs. time profiles (INC280) [ Time Frame: Day 1 of the first 6 cycles ]
- PK:Serum concentration vs. time profiles (PDR001) [ Time Frame: Days 1, 2, 3, 4, 8, 11 and 15 of Cycles 1 and 3, Day 1 of Cycles 2, 4, 5 and 6 ]
- PK: Peak Plasma Concentration (Cmax) (INC280) [ Time Frame: Day 1 of the first 6 cycles ]
- PK:Time of Maximum concentration observed (Tmax) (INC280) [ Time Frame: Day 1 of the first 6 cycles ]
- PK: Peak Serum Concentration (Cmax) (PDR001) [ Time Frame: Days 1, 2, 3, 4, 8, 11 and 15 of Cycles 1 and 3, Day 1 of Cycles 2, 4, 5 and 6 ]
- PK: Time of Maximum concentration observed (Tmax) (PDR001) [ Time Frame: Days 1, 2, 3, 4, 8, 11 and 15 of Cycles 1 and 3, Day 1 of Cycles 2, 4, 5 and 6 ]

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Histologically or cytologically documented locally advanced recurrent or metastatic HCC or for patients with cirrhosis according to the American Association for the Study of Liver Diseases (AASLD) and Asian Pacific Association for the study of the liver (APASL) criteria. Current cirrhotic status of Child Pugh Class A (5-6 points), with no encephalopathy and/or clinically significant ascites (defined as requiring the use of diuretics or paracentesis treatment).
- Patients must have received prior systemic sorafenib treatment for HCC with documented progression during or after discontinuation of sorafenib treatment (for France only: patients must have received at least 8 weeks of prior sorafenib treatment), or are intolerant to sorafenib (defined as documented Grade 3 or 4 adverse events that led to sorafenib discontinuation),.
- ECOG Performance Status ≤ 1.
- Willing and able to swallow and retain oral medication. Other protocol defined Inclusion criteria may apply.
Exclusion Criteria:
- Use of any live vaccines within 4 weeks of initiation of study treatment.
- History of severe hypersensitivity reactions to other monoclonal antibodies (mAbs).
- Clinically significant pleural effusion that either required pleurocentesis or is associated with shortness of breath.
- Active autoimmune disease or a documented history of autoimmune disease.
- Clinically significant, uncontrolled heart diseases.
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Patient having out of range laboratory values defined as:
- Total bilirubin > 2 mg/dL, except for patients with Gilbert's syndrome who are excluded if total bilirubin > 3.0 x ULN or direct bilirubin > 1.5 x ULN
- Alanine aminotransferase (ALT) > 5 x ULN
- Aspartate aminotransferase (AST) > 5 x ULN
- Coagulation: Prothrombin Time (PT) > 4 seconds more than the ULN or International Normalized Ratio (INR) > 1.7
- Absolute neutrophil count (ANC) < 1.5 x 109/L
- Platelet count < 75 x 109/L
- Hemoglobin < 9 g/dL
- Creatinine clearance (calculated using Cockcroft-Gault formula, or measured) < 45 mL/min
- Asymptomatic serum amylase grade > 2 (1.5-2.0 x ULN). Patients with grade 1 or grade 2 serum amylase at the beginning of the study must be confirmed to have no signs or symptoms suggesting pancreatitis or pancreatic injury (e.g., elevated P-amylase, abnormal imaging findings of pancreas, etc.)
- Serum lipase > ULN
- Potassium, Magnesium, Phosphorus, total Calcium (corrected for serum albumin) outside of normal limits (patients may be enrolled if corrected to within normal limits with supplements during screening)
Other protocol-defined Exclusion criteria may apply.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02795429
Canada, Ontario | |
Novartis Investigative Site | |
Toronto, Ontario, Canada, M5G 2M9 | |
Canada, Quebec | |
Novartis Investigative Site | |
Montreal, Quebec, Canada, H3T 1E2 | |
China, Guangdong | |
Novartis Investigative Site | |
Guangzhou, Guangdong, China, 510515 | |
China, Shanghai | |
Novartis Investigative Site | |
Shanghai, Shanghai, China, 200032 | |
France | |
Novartis Investigative Site | |
Montpellier cedex 5, Herault, France, 34059 | |
Novartis Investigative Site | |
Lille Cedex, France, 59037 | |
Novartis Investigative Site | |
Toulouse Cedex 9, France, 31059 | |
Germany | |
Novartis Investigative Site | |
Heidelberg, Germany, 69120 | |
Novartis Investigative Site | |
Wuerzburg, Germany, 97080 | |
Hong Kong | |
Novartis Investigative Site | |
Hong Kong, Hong Kong | |
Italy | |
Novartis Investigative Site | |
Milano, MI, Italy, 20132 | |
Novartis Investigative Site | |
Rozzano, MI, Italy, 20089 | |
Novartis Investigative Site | |
Modena, MO, Italy, 41124 | |
Korea, Republic of | |
Novartis Investigative Site | |
Seoul, Korea, Republic of, 03080 | |
Novartis Investigative Site | |
Seoul, Korea, Republic of, 03722 | |
Taiwan | |
Novartis Investigative Site | |
Tainan, Taiwan, 70403 | |
Novartis Investigative Site | |
Taipei, Taiwan, 10002 |
Study Director: | Novartis Pharmaceuticals | Novartis Pharmaceuticals |
Responsible Party: | Novartis Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT02795429 |
Other Study ID Numbers: |
CINC280X2108 2015-005417-76 ( EudraCT Number ) |
First Posted: | June 10, 2016 Key Record Dates |
Last Update Posted: | January 13, 2021 |
Last Verified: | January 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Undecided |
Plan Description: | Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Phase Ib/II INC280 PDR001 |
checkpoint inhibitor PD-1 Liver cancer |
Carcinoma Carcinoma, Hepatocellular Neoplasms, Glandular and Epithelial Neoplasms by Histologic Type Neoplasms Adenocarcinoma |
Liver Neoplasms Digestive System Neoplasms Neoplasms by Site Digestive System Diseases Liver Diseases |