CClinicalTrials.gg
CompletedNCT03289533Updated Sep 4, 2020Results posted

A Study Of Avelumab In Combination With Axitinib In Advanced HCC (VEGF Liver 100)

A Phase 1 interventional study of Avelumab (MSB0010718C) and Axitinib (AG-013736) in Carcinoma, Hepatocellular, sponsored by Pfizer. Completed at 7 sites in Japan. Open to participants aged 20 Years and older. Per ClinicalTrials.gov, last updated 2020-09-04.

Sponsored by Pfizer · Phase 1, Interventional, and Treatment

Phase
Phase 1
Study type
Interventional
Enrollment
22
Allocation
Non-randomized
Ages
20 Years and older
Sex
All
01

Study summary

To evaluate the safety, efficacy and PK of avelumab in combination with axitinib as first line treatment in patients with advanced HCC

02

Conditions studied

  • Carcinoma, Hepatocellular

Keywords

  • Cancer
  • Hepatocellular carcinoma
  • Liver disease
  • Avelumab
  • Axitinib
03

Who can participate

Ages eligible
20 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Diagnosis of locally advanced or metastatic HCC, obtained by histology/cytology (on a prior tumor biopsy) or by imaging with serum α-fetoprotein (AFP) ≥400 ng/mL.
  • All patients must provide at least 1 archival tumor specimen. If archival tumor specimen is no longer available, de novo tumor biopsy will be required during screening.
  • HCC not amenable to local therapy.
  • Measurable disease according to RECIST v. 1.1.
  • Child Pugh Class A disease.
  • BCLC stage B or C disease.
  • No evidence of uncontrolled hypertension as documented by 2 baseline blood pressure readings taken at least 1 hour apart.
  • ECOG performance status 0 or 1.
  • Adequate bone marrow function, renal and liver functions
  • Left ventricular ejection fraction (LVEF) ≥ lower limit of normal (LLN) as assessed by multigated acquisition (MUGA) scan or echocardiogram (ECHO).

Exclusion criteria

Exclusion Criteria:

  • Prior systemic treatment for advanced HCC, including prior treatment with approved or investigational drugs.
  • Any prior locoregional therapy within 4 weeks and radiotherapy or surgical procedure within 2 weeks (4 weeks for major surgery) of enrollment.
  • Patients with known symptomatic brain metastases requiring steroids.
  • Presence of hepatic encephalopathy (ie, Child Pugh score of 2 or 3) and/or clinically relevant ascites (ie, Child Pugh score of 3).
  • Presence of main portal vein invasion by HCC.
  • Any of the following within the 12 months prior to enrollment: myocardial infarction, severe/unstable angina, coronary/peripheral artery bypass graft, symptomatic congestive heart failure, LVEF less than LLN, clinically significant pericardial effusion, cerebrovascular accident, transient ischemic attack.
  • Active infection requiring systemic therapy except for hepatitis C virus (HCV) and hepatitis B virus (HBV).
04

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
22 participants (actual)

Study arms

  • Experimental
    Experimental 1

    Avelumab (MSB0010718C) in combination with axitinib (AG-013736)

    Drug: Avelumab (MSB0010718C) · Drug: Axitinib (AG-013736)

Interventions

  • DrugAvelumab (MSB0010718C)

    Patients will receive avelumab 10 mg/kg Q2W in combination with axitinib 5 mg BID.

  • DrugAxitinib (AG-013736)

    Patients will receive avelumab 10 mg/kg Q2W in combination with axitinib 5 mg BID.

05

What researchers measure

Primary outcomes

  1. Number of Participants With Treatment Emergent Adverse Events (TEAEs) by Severity as Graded by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version (v.) 4.03

    An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. AEs were graded by investigator according to NCI CTCAE v.4.03 as follows: Grade 1: mild AE, Grade 2: moderate AE, Grade 3: severe AE, Grade 4: life-threatening consequences and urgent intervention indicated, Grade 5: death related to AE.

    Time frame: From first dose of study drug up to 30 days after last dose of study drug or initiation of new anti-cancer drug therapy, whichever occurred first (maximum up to 21 months)

  2. Number of Participants With Abnormal Laboratory Parameter Values (Hematology) as Graded by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Event (CTCAE) Version 4.03

    As per NCI-CTCAE v 4.03, anemia Grade 1= Less than (\<) lower limit of normal (LLN) to 100 gram per liter (g/L),Grade 2= \<100 to 80 g/L; hemoglobin increased: Grade 1= increase of greater than (\>) 0 to 2 gram per deciliter(g/dL) above upper limit of normal \[ULN\]; lymphocyte count decreased: Grade 1= \<LLN to 0.8\*10\^9/L, Grade 2= \<0.8\*10\^9/L to 0.5\*10\^9/L, Grade 3= \<0.5\*10\^9/L to 0.2\*10\^9/L ; lymphocyte count increased: Grade 2= \>4\*10\^9/L to 20\*10\^9/L; neutrophil count decreased: Grade 1= \<LLN to 1.5\*10\^9/L ,Grade 2= \<1.5\*10\^9/L to 1.0\*10\^9/L; platelet count decreased: Grade 1= \<LLN to 75.0\*10\^9/L, Grade 2= \<75.0\*10\^9/L to 50.0\*10\^9/L; white blood cell decreased: Grade 1= \<LLN to 3\*10\^9/L, Grade 2= \<3\*10\^9/L to 2\*10\^9/L.

    Time frame: From first dose of study drug up to 30 days after last dose of study drug or initiation of new anti-cancer drug therapy, whichever occurred first (maximum up to 21 months)

  3. Number of Participants With Abnormal Laboratory Parameter Values (Chemistry) as Graded by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Event (CTCAE) Version (v) 4.03

    ALT,ALP,AST increased grades(g):g1\>ULN-3.0\*ULN,g2\>3.0-5.0\*ULN,g3\>5.0-20.0\*ULN; blood bilirubin increased:g1\>ULN-1.5\*ULN, g2\>1.5-3.0\*ULN, g3\>3.0-10.0\*ULN; \[cholesterol high:g1\>ULN-7.75, g2 \>7.75-10.34,g4 \>12.92\]millimoles per liter(mmol/L);creatine phosphokinase, gamma-glutamyl transferase(ggt) increased g1\>ULN-2.5\*ULN, g2\>2.5\*ULN-5\*ULN; Ggt increased g3 \>5.0-20.0\*ULN; Creatinine increased: g1\>ULN-1.5\*ULN; \[hypoalbuminemia:g1\<LLN-30,g2\<30-20\] grams per liter(g/L);\[hyperglycemia:g1\> ULN-8.9,g2\> 8.9-13.9,g3\> 13.9-27.8;hypermagnesemia:g1\>ULN-1.23;hypercalcemia:g1\>ULN -2.9;hyperkalemia:g1\>ULN-5.5,hypernatremia:g1\>ULN-150;hypertriglyceridemia g1:1.71-3.42,g2 \>3.42-5.7;hypocalcemia:g1\<LLN-2.0,hypoglycemia:g1\<LLN-3.0, g2\<3.0-2.2;hypokalemia:g2\<LLN-3.0,g4\<2.5,hypomagnesemia:g1\<LLN-0.5,hyponatremia:g1\<LLN-130, g3\<130-120,hypophosphatemia:g1\<LLN-0.8,g2\<0.8-0.6\]mmol/L;lipase increased:g1\>ULN-1.5\*ULN,g3 \>2.0-5.0\*ULN;serum amylase increased:g1\>ULN-1.5\*ULN, g2\>1.5-2.0\*ULN,g3\>2.0-5.0\*ULN.

    Time frame: From first dose of study drug up to 30 days after last dose of study drug or initiation of new anti-cancer drug therapy, whichever occurred first (maximum up to 21 months)

Secondary outcomes

  1. Time to Disease Progression (TTP)

    TTP as assessed by investigator according to Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1, was define as time (in months) from date of first dose of study drug to date of first documentation of progressive disease (PD) or data censoring date, whichever occurred first. PD was defined as greater than or equal to (\>=) 20 percent (%) increase in sum of diameters of target lesions, taking as a reference smallest sum on study treatment (this included baseline sum if that was smallest on study treatment), with a minimum absolute increase of at least 5 millimeter (mm), or appearance of \>=1 new lesions. TTP was analyzed by Kaplan-Meier method. TTP data was censored on the date the last adequate tumor assessment for participants without PD, for participants who start new anti-cancer treatment prior to PD, for participants who died without PD, or for participants with PD after \>=2 missing tumor assessments.

    Time frame: From first dose of study drug until first documentation of progressive disease or data censoring date, whichever occurred first (maximum up to 20 months)

  2. Progression Free Survival (PFS)

    PFS as assessed by investigator per RECIST v1.1, was defined as time (in months) from date of first dose of study drug to date of first documentation of PD or death due to any cause or data censoring date, whichever occurred first. PD: \>= 20% increase in sum of diameters of target lesions, taking as a reference smallest sum on study treatment (this included baseline sum if that was smallest on study treatment). The sum must also demonstrate absolute increase of \>=5 mm, or appearance of \>=1 new lesions. PFS was analyzed by Kaplan-Meier method. PFS data was censored on the date the last adequate tumor assessment for participants without an event (PD or death), for participants who started new anti-cancer treatment prior to PFS event, for participants with a PFS event after \>=2 missing tumor assessments.

    Time frame: From first dose of study drug until first documentation of PD or death due to any cause or data censoring date, whichever occurred first (maximum up to 20 months)

  3. Percentage of Participants With Objective Response (OR)

    OR as assessed by investigator per RECIST v.1.1, was defined as participants with confirmed best overall response of complete response (CR) or partial response (PR), were recorded from first dose of study drug until disease progression or death due to any cause. CR was defined as disappearance of all target and non-target lesions, and sustained for at least 4 weeks. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to less than (\<) 10 mm. PR was defined as \>=30% decrease in sum of diameters of target lesions, taking as reference the baseline sum diameters.

    Time frame: From first dose of study drug until disease progression or death due to any cause (maximum up to 20 months)

  4. Percentage of Participants With Disease Control (DC)

    Disease control as assessed by investigator according to RECIST v1.1, was defined as participants with CR, PR, stable disease (SD), or non-CR/non-PD. CR: disappearance of all target and non-target lesions and sustained for 4 weeks. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm. PR: \>= 30% decrease in sum of diameters of target lesions taking as reference baseline sum diameters. SD: neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study. PD: at least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study. The sum must also demonstrate an absolute increase of at least 5 mm or appearance of \>=1 new lesions. Non-CR/non-PD: Persistence of any non-target lesions and/or tumor marker level above the normal limit at \>=8 weeks after date of first dose of study treatment.

    Time frame: From first dose of study drug until first documentation of CR or PR or SD or till non-CR/non-PD (maximum up to 20 months)

  5. Time to Tumor Response (TTR)

    TTR as assessed by investigator according to RECIST v1.1 was defined as the time (in months) from the date of first dose of study drug to the first documentation of objective response (CR or PR) that was subsequently confirmed. CR was defined as disappearance of all target and non-target lesions, and sustained for at least 4 weeks. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm. PR: \>= 30% decrease in sum of diameter of target lesions taking as reference baseline sum diameters.

    Time frame: From first dose of study drug until first documentation of CR or PR (maximum up to 20 months)

  6. Duration of Response (DR)

    DR as assessed by investigator according to RECIST v1.1, was defined as the time from date of first documentation of objective response (CR or PR) to date of PD or death due to any cause, or data censoring date, whichever occurred first. CR: disappearance of all target and non-target lesions, and sustained for at least 4 weeks. Any pathological lymph nodes (target or non-target) reduced in short axis to \<10 mm. PR: \>= 30% decrease in sum of diameter of target lesions taking as reference baseline sum diameters. PD: at least a 20% increase in sum of diameters of target lesions, taking as reference the smallest sum on study treatment, with absolute increase of at least 5 mm or appearance of \>=1 new lesions. DR data was censored on the date of last adequate tumor assessment for participants without an event (CR, PR, PD or death), for participants who start new anti-cancer treatment prior to DR assessment, for participants with DR assessment after \>=2 missing tumor assessments.

    Time frame: From first documentation of CR or PR until first documentation of tumor progression or death due to any cause or data censoring date, whichever occurred first (maximum up to 20 months)

  7. Overall Survival (OS)

    OS was defined as the time (in months) from the date of first dose of study drug to the date of death due to any cause or data censoring date, whichever occurred first. Participants last known to be alive were censored at the date of last contact. OS was analyzed by Kaplan-Meier method.

    Time frame: From first dose of study drug to date of death from any cause or data censoring date, whichever occurred first (maximum up to 21 months)

  8. Maximum Observed Serum Concentration of Avelumab

    Time frame: Pre-dose, at the end of avelumab infusion on Day 1 of Cycle 2, 3, 4 (Duration of each cycle=14 days)

  9. Maximum Observed Plasma Concentration of Axitinib

    Time frame: Pre-dose, 2 hours post dose Axitinib administration on Day 1 of Cycle 2, 3 (Duration of each cycle=14 days)

  10. Pre-dose Serum Concentration of Avelumab

    Time frame: Pre-dose on Day 1 of Cycle 2, 3, 4, 6, 8, 12, 16, 20, 24, 28 (Duration of each cycle=14 days)

  11. Pre-dose Plasma Concentration of Axitinib

    Time frame: Pre-dose on Day 1 of Cycle 2 and 3 (Duration of each cycle=14 days)

  12. Number of Participants With Their Target Programmed Death-Ligand 1 (PD-L1) Status

    PD-L1 status was defined as positive when PD-L1 staining of any intensity was observed in tumor-associated immune cells covering \>= 1% of the tumor area. PD-L1 status was defined as negative when PD-L1 staining of any intensity was observed in tumor-associated immune cells covering \< 1% of the tumor area.

    Time frame: Baseline (Day 1)

  13. Mean Percentage of CD8+ Cells in Per Unit Area of Invasive Margin, Center of Tumor Cells and Total Area of Tumor Cells

    CD8+ cells are the type of T-lymphocytes. Invasive margin is defined as the region on each side of the border between tumor cells. Expression of CD8+ cells in invasive margin, center of tumor cells, total area of tumor cells has been reported as mean percentage of CD8+cells per unit area. Area was measured in millimeter square (mm\^2).

    Time frame: From first dose of study drug up to end of treatment (maximum up to 20 months)

  14. Summary of Cluster of Differentiation 8 (CD8+) Cells Expression: Total Area Covered by CD8+ Cells in Center of Tumor Cells

    CD8+ cells are the type of T-lymphocytes.

    Time frame: From first dose of study drug up to end of treatment (up to 20 months)

  15. Number of Participants With Positive Anti-Drug Antibodies (ADAs) and Positive Neutralizing Antibodies (nAbs)

    ADA positive was defined as presence of at least one positive ADA sample. nAb positive was defined as presence of at least one positive nAb sample.

    Time frame: From first dose of study drug until 30 days after the last dose of study drug (maximum up to 21 months)

06

Results

Posted Sep 4, 2020

Participant flow

Participants with advanced hepatocellular carcinoma (HCC) who did not receive any prior systemic therapy were enrolled in this study.

Participant flow — Overall Study
MilestoneAvelumab + Axitinib
Started22
Completed0
Not completed22
Withdrew: Death12
Withdrew: Terminated from study by sponsor10

Outcome measures

PrimaryNumber of Participants With Treatment Emergent Adverse Events (TEAEs) by Severity as Graded by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version (v.) 4.03

An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. AEs were graded by investigator according to NCI CTCAE v.4.03 as follows: Grade 1: mild AE, Grade 2: moderate AE, Grade 3: severe AE, Grade 4: life-threatening consequences and urgent intervention indicated, Grade 5: death related to AE.

Time frame:
From first dose of study drug up to 30 days after last dose of study drug or initiation of new anti-cancer drug therapy, whichever occurred first (maximum up to 21 months)
Reported as:
Count of participants · Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) by Severity as Graded by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version (v.) 4.03
ParticipantsAvelumab + Axitinib
Grade 12
Grade 23
Grade 316
Grade 41
Grade 50
PrimaryNumber of Participants With Abnormal Laboratory Parameter Values (Hematology) as Graded by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Event (CTCAE) Version 4.03

As per NCI-CTCAE v 4.03, anemia Grade 1= Less than (\<) lower limit of normal (LLN) to 100 gram per liter (g/L),Grade 2= \<100 to 80 g/L; hemoglobin increased: Grade 1= increase of greater than (\>) 0 to 2 gram per deciliter(g/dL) above upper limit of normal \[ULN\]; lymphocyte count decreased: Grade 1= \<LLN to 0.8\*10\^9/L, Grade 2= \<0.8\*10\^9/L to 0.5\*10\^9/L, Grade 3= \<0.5\*10\^9/L to 0.2\*10\^9/L ; lymphocyte count increased: Grade 2= \>4\*10\^9/L to 20\*10\^9/L; neutrophil count decreased: Grade 1= \<LLN to 1.5\*10\^9/L ,Grade 2= \<1.5\*10\^9/L to 1.0\*10\^9/L; platelet count decreased: Grade 1= \<LLN to 75.0\*10\^9/L, Grade 2= \<75.0\*10\^9/L to 50.0\*10\^9/L; white blood cell decreased: Grade 1= \<LLN to 3\*10\^9/L, Grade 2= \<3\*10\^9/L to 2\*10\^9/L.

Time frame:
From first dose of study drug up to 30 days after last dose of study drug or initiation of new anti-cancer drug therapy, whichever occurred first (maximum up to 21 months)
Reported as:
Count of participants · Participants
Number of Participants With Abnormal Laboratory Parameter Values (Hematology) as Graded by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Event (CTCAE) Version 4.03
ParticipantsAvelumab + Axitinib
Anemia: Grade 113
Anemia: Grade 23
Hemoglobin increased: Grade 15
Lymphocyte count decreased: Grade 111
Lymphocyte count decreased: Grade 22
Lymphocyte count decreased: Grade 31
Lymphocyte count increased: Grade 21
Neutrophil count decreased: Grade 13
Neutrophil count decreased: Grade 25
Platelet count decreased: Grade 114
Platelet count decreased: Grade 22
White blood cell decreased: Grade 11
White blood cell decreased: Grade 24
PrimaryNumber of Participants With Abnormal Laboratory Parameter Values (Chemistry) as Graded by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Event (CTCAE) Version (v) 4.03

ALT,ALP,AST increased grades(g):g1\>ULN-3.0\*ULN,g2\>3.0-5.0\*ULN,g3\>5.0-20.0\*ULN; blood bilirubin increased:g1\>ULN-1.5\*ULN, g2\>1.5-3.0\*ULN, g3\>3.0-10.0\*ULN; \[cholesterol high:g1\>ULN-7.75, g2 \>7.75-10.34,g4 \>12.92\]millimoles per liter(mmol/L);creatine phosphokinase, gamma-glutamyl transferase(ggt) increased g1\>ULN-2.5\*ULN, g2\>2.5\*ULN-5\*ULN; Ggt increased g3 \>5.0-20.0\*ULN; Creatinine increased: g1\>ULN-1.5\*ULN; \[hypoalbuminemia:g1\<LLN-30,g2\<30-20\] grams per liter(g/L);\[hyperglycemia:g1\> ULN-8.9,g2\> 8.9-13.9,g3\> 13.9-27.8;hypermagnesemia:g1\>ULN-1.23;hypercalcemia:g1\>ULN -2.9;hyperkalemia:g1\>ULN-5.5,hypernatremia:g1\>ULN-150;hypertriglyceridemia g1:1.71-3.42,g2 \>3.42-5.7;hypocalcemia:g1\<LLN-2.0,hypoglycemia:g1\<LLN-3.0, g2\<3.0-2.2;hypokalemia:g2\<LLN-3.0,g4\<2.5,hypomagnesemia:g1\<LLN-0.5,hyponatremia:g1\<LLN-130, g3\<130-120,hypophosphatemia:g1\<LLN-0.8,g2\<0.8-0.6\]mmol/L;lipase increased:g1\>ULN-1.5\*ULN,g3 \>2.0-5.0\*ULN;serum amylase increased:g1\>ULN-1.5\*ULN, g2\>1.5-2.0\*ULN,g3\>2.0-5.0\*ULN.

Time frame:
From first dose of study drug up to 30 days after last dose of study drug or initiation of new anti-cancer drug therapy, whichever occurred first (maximum up to 21 months)
Reported as:
Count of participants · Participants
Number of Participants With Abnormal Laboratory Parameter Values (Chemistry) as Graded by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Event (CTCAE) Version (v) 4.03
ParticipantsAvelumab + Axitinib
Alanine aminotransferase (ALT) increased: Grade 115
ALT increased: Grade 22
ALT increased: Grade 31
Alkaline phosphatase (ALP) increased: Grade 111
ALP increased: Grade 22
ALP increased: Grade 33
Aspartate aminotransferase(AST) increased: Grade 115
AST increased: Grade 24
AST increased: Grade 32
Blood bilirubin increased: Grade 14
Blood bilirubin increased: Grade 22
Blood bilirubin increased: Grade 31
Cholesterol high: Grade 15
Cholesterol high: Grade 21
Cholesterol high: Grade 41
Creatine phosphokinase increased: Grade 12
Creatine phosphokinase increased: Grade 21
Creatinine increased: Grade 121
Gamma-glutamyl transferase(Ggt) increased: Grade 16
Ggt increased: Grade 25
Ggt increased: Grade 37
Hypercalcemia: Grade 110
Hyperglycemia: Grade 111
Hyperglycemia: Grade 22
Hyperglycemia: Grade 31
Hyperkalemia: Grade 14
Hypermagnesemia: Grade 14
Hypernatremia: Grade 13
Hypertriglyceridemia; Grade 19
Hypertriglyceridemia; Grade 23
Hypoalbuminemia: Grade 115
Hypoalbuminemia: Grade 23
Hypocalcemia: Grade 11
Hypoglycemia: Grade 12
Hypoglycemia: Grade 21
Hypokalemia: Grade 27
Hypokalemia: Grade 41
Hypomagnesemia: Grade 12
Hyponatremia: Grade 115
Hyponatremia: Grade 31
Hypophosphatemia: Grade 14
Hypophosphatemia: Grade 28
Lipase increased: Grade 18
Lipase increased: Grade 33
Serum amylase increased: Grade 15
Serum amylase increased: Grade 21
Serum amylase increased: Grade 31
SecondaryTime to Disease Progression (TTP)

TTP as assessed by investigator according to Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1, was define as time (in months) from date of first dose of study drug to date of first documentation of progressive disease (PD) or data censoring date, whichever occurred first. PD was defined as greater than or equal to (\>=) 20 percent (%) increase in sum of diameters of target lesions, taking as a reference smallest sum on study treatment (this included baseline sum if that was smallest on study treatment), with a minimum absolute increase of at least 5 millimeter (mm), or appearance of \>=1 new lesions. TTP was analyzed by Kaplan-Meier method. TTP data was censored on the date the last adequate tumor assessment for participants without PD, for participants who start new anti-cancer treatment prior to PD, for participants who died without PD, or for participants with PD after \>=2 missing tumor assessments.

Time frame:
From first dose of study drug until first documentation of progressive disease or data censoring date, whichever occurred first (maximum up to 20 months)
Reported as:
Median · Months
Time to Disease Progression (TTP)
MonthsAvelumab + Axitinib
Time to Disease Progression (TTP)5.52 (1.91 to 7.39)
SecondaryProgression Free Survival (PFS)

PFS as assessed by investigator per RECIST v1.1, was defined as time (in months) from date of first dose of study drug to date of first documentation of PD or death due to any cause or data censoring date, whichever occurred first. PD: \>= 20% increase in sum of diameters of target lesions, taking as a reference smallest sum on study treatment (this included baseline sum if that was smallest on study treatment). The sum must also demonstrate absolute increase of \>=5 mm, or appearance of \>=1 new lesions. PFS was analyzed by Kaplan-Meier method. PFS data was censored on the date the last adequate tumor assessment for participants without an event (PD or death), for participants who started new anti-cancer treatment prior to PFS event, for participants with a PFS event after \>=2 missing tumor assessments.

Time frame:
From first dose of study drug until first documentation of PD or death due to any cause or data censoring date, whichever occurred first (maximum up to 20 months)
Reported as:
Median · Months
Progression Free Survival (PFS)
MonthsAvelumab + Axitinib
Progression Free Survival (PFS)5.52 (1.91 to 7.39)
SecondaryPercentage of Participants With Objective Response (OR)

OR as assessed by investigator per RECIST v.1.1, was defined as participants with confirmed best overall response of complete response (CR) or partial response (PR), were recorded from first dose of study drug until disease progression or death due to any cause. CR was defined as disappearance of all target and non-target lesions, and sustained for at least 4 weeks. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to less than (\<) 10 mm. PR was defined as \>=30% decrease in sum of diameters of target lesions, taking as reference the baseline sum diameters.

Time frame:
From first dose of study drug until disease progression or death due to any cause (maximum up to 20 months)
Reported as:
Number · Percentage of participants
Percentage of Participants With Objective Response (OR)
Percentage of participantsAvelumab + Axitinib
Percentage of Participants With Objective Response (OR)13.6 (2.9 to 34.9)
SecondaryPercentage of Participants With Disease Control (DC)

Disease control as assessed by investigator according to RECIST v1.1, was defined as participants with CR, PR, stable disease (SD), or non-CR/non-PD. CR: disappearance of all target and non-target lesions and sustained for 4 weeks. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm. PR: \>= 30% decrease in sum of diameters of target lesions taking as reference baseline sum diameters. SD: neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study. PD: at least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study. The sum must also demonstrate an absolute increase of at least 5 mm or appearance of \>=1 new lesions. Non-CR/non-PD: Persistence of any non-target lesions and/or tumor marker level above the normal limit at \>=8 weeks after date of first dose of study treatment.

Time frame:
From first dose of study drug until first documentation of CR or PR or SD or till non-CR/non-PD (maximum up to 20 months)
Reported as:
Number · Percentage of participants
Percentage of Participants With Disease Control (DC)
Percentage of participantsAvelumab + Axitinib
Percentage of Participants With Disease Control (DC)68.2 (45.1 to 86.1)
SecondaryTime to Tumor Response (TTR)

TTR as assessed by investigator according to RECIST v1.1 was defined as the time (in months) from the date of first dose of study drug to the first documentation of objective response (CR or PR) that was subsequently confirmed. CR was defined as disappearance of all target and non-target lesions, and sustained for at least 4 weeks. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm. PR: \>= 30% decrease in sum of diameter of target lesions taking as reference baseline sum diameters.

Time frame:
From first dose of study drug until first documentation of CR or PR (maximum up to 20 months)
Reported as:
Median · Months
Time to Tumor Response (TTR)
MonthsAvelumab + Axitinib
Time to Tumor Response (TTR)1.91 (1.9 to 3.7)
SecondaryDuration of Response (DR)

DR as assessed by investigator according to RECIST v1.1, was defined as the time from date of first documentation of objective response (CR or PR) to date of PD or death due to any cause, or data censoring date, whichever occurred first. CR: disappearance of all target and non-target lesions, and sustained for at least 4 weeks. Any pathological lymph nodes (target or non-target) reduced in short axis to \<10 mm. PR: \>= 30% decrease in sum of diameter of target lesions taking as reference baseline sum diameters. PD: at least a 20% increase in sum of diameters of target lesions, taking as reference the smallest sum on study treatment, with absolute increase of at least 5 mm or appearance of \>=1 new lesions. DR data was censored on the date of last adequate tumor assessment for participants without an event (CR, PR, PD or death), for participants who start new anti-cancer treatment prior to DR assessment, for participants with DR assessment after \>=2 missing tumor assessments.

Time frame:
From first documentation of CR or PR until first documentation of tumor progression or death due to any cause or data censoring date, whichever occurred first (maximum up to 20 months)
Reported as:
Median · Months
Duration of Response (DR)
MonthsAvelumab + Axitinib
Duration of Response (DR)7.29 (3.71 to 12.94)
SecondaryOverall Survival (OS)

OS was defined as the time (in months) from the date of first dose of study drug to the date of death due to any cause or data censoring date, whichever occurred first. Participants last known to be alive were censored at the date of last contact. OS was analyzed by Kaplan-Meier method.

Time frame:
From first dose of study drug to date of death from any cause or data censoring date, whichever occurred first (maximum up to 21 months)
Reported as:
Median · Months
Overall Survival (OS)
MonthsAvelumab + Axitinib
Overall Survival (OS)14.05 (7.95 to NA)
SecondaryMaximum Observed Serum Concentration of Avelumab
Time frame:
Pre-dose, at the end of avelumab infusion on Day 1 of Cycle 2, 3, 4 (Duration of each cycle=14 days)
Reported as:
Geometric mean · Microgram per milliliter
Maximum Observed Serum Concentration of Avelumab
Microgram per milliliterAvelumab + Axitinib
Cycle 2 Day 1231.06 ± 24
Cycle 3 Day 1228.48 ± 25
Cycle 4 Day 1245.66 ± 26
SecondaryMaximum Observed Plasma Concentration of Axitinib
Time frame:
Pre-dose, 2 hours post dose Axitinib administration on Day 1 of Cycle 2, 3 (Duration of each cycle=14 days)
Reported as:
Geometric mean · Nanograms per milliliter
Maximum Observed Plasma Concentration of Axitinib
Nanograms per milliliterAvelumab + Axitinib
Cycle 2 Day 189.70 ± 23.015
Cycle 3 Day 1109.00 ± 17.275
SecondaryPre-dose Serum Concentration of Avelumab
Time frame:
Pre-dose on Day 1 of Cycle 2, 3, 4, 6, 8, 12, 16, 20, 24, 28 (Duration of each cycle=14 days)
Reported as:
Geometric mean · Microgram per milliliter
Pre-dose Serum Concentration of Avelumab
Microgram per milliliterAvelumab + Axitinib
Cycle 2 Day 133.80 ± 17.722
Cycle 3 Day 119.419 ± 64
Cycle 4 Day 119.220 ± 118
Cycle 6 Day 123.922 ± 68
Cycle 8 Day 124.814 ± 39
Cycle 12 Day 129.832 ± 37
Cycle 16 Day 135.388 ± 35
Cycle 20 Day 131.211 ± 36
Cycle 24 Day 130.070 ± 43
Cycle 28 Day 135.578 ± 19
SecondaryPre-dose Plasma Concentration of Axitinib
Time frame:
Pre-dose on Day 1 of Cycle 2 and 3 (Duration of each cycle=14 days)
Reported as:
Geometric mean · Nanograms per milliliter
Pre-dose Plasma Concentration of Axitinib
Nanograms per milliliterAvelumab + Axitinib
Cycle 2 Day 111.6439 ± 123
Cycle 3 Day 19.2226 ± 164
SecondaryNumber of Participants With Their Target Programmed Death-Ligand 1 (PD-L1) Status

PD-L1 status was defined as positive when PD-L1 staining of any intensity was observed in tumor-associated immune cells covering \>= 1% of the tumor area. PD-L1 status was defined as negative when PD-L1 staining of any intensity was observed in tumor-associated immune cells covering \< 1% of the tumor area.

Time frame:
Baseline (Day 1)
Reported as:
Count of participants · Participants
Number of Participants With Their Target Programmed Death-Ligand 1 (PD-L1) Status
ParticipantsAvelumab + Axitinib
PD-L1: Positive17
PD-L1: Negative3
SecondaryMean Percentage of CD8+ Cells in Per Unit Area of Invasive Margin, Center of Tumor Cells and Total Area of Tumor Cells

CD8+ cells are the type of T-lymphocytes. Invasive margin is defined as the region on each side of the border between tumor cells. Expression of CD8+ cells in invasive margin, center of tumor cells, total area of tumor cells has been reported as mean percentage of CD8+cells per unit area. Area was measured in millimeter square (mm\^2).

Time frame:
From first dose of study drug up to end of treatment (maximum up to 20 months)
Reported as:
Mean · Percentage of CD8+cells per mm^2
Mean Percentage of CD8+ Cells in Per Unit Area of Invasive Margin, Center of Tumor Cells and Total Area of Tumor Cells
Percentage of CD8+cells per mm^2Avelumab + Axitinib
CD8+ Cells in Invasive Margin2.12 ± 1.760
CD8+ Cells in Center of Tumor Cells0.91 ± 1.328
CD8+ Cells in Total Area of Tumor Cells1.02 ± 1.353
SecondarySummary of Cluster of Differentiation 8 (CD8+) Cells Expression: Total Area Covered by CD8+ Cells in Center of Tumor Cells

CD8+ cells are the type of T-lymphocytes.

Time frame:
From first dose of study drug up to end of treatment (up to 20 months)
Reported as:
Mean · mm^2
Summary of Cluster of Differentiation 8 (CD8+) Cells Expression: Total Area Covered by CD8+ Cells in Center of Tumor Cells
mm^2Avelumab + Axitinib
Summary of Cluster of Differentiation 8 (CD8+) Cells Expression: Total Area Covered by CD8+ Cells in Center of Tumor Cells53.88 ± 48.832
SecondaryNumber of Participants With Positive Anti-Drug Antibodies (ADAs) and Positive Neutralizing Antibodies (nAbs)

ADA positive was defined as presence of at least one positive ADA sample. nAb positive was defined as presence of at least one positive nAb sample.

Time frame:
From first dose of study drug until 30 days after the last dose of study drug (maximum up to 21 months)
Reported as:
Count of participants · Participants
Number of Participants With Positive Anti-Drug Antibodies (ADAs) and Positive Neutralizing Antibodies (nAbs)
ParticipantsAvelumab + Axitinib
ADA positive3
nAb positive3

Adverse events

Collected over From first dose of study drug up to 30 days after last dose of study drug or initiation of new anti-cancer drug therapy, whichever occurred first (maximum up to 21 months). Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Avelumab + Axitinib12/22 (54.5%)8/22 (36.4%)22/22 (100%)
Most frequent serious events
Most frequent serious events
EventAvelumab + Axitinib
FatigueGeneral disorders2/22
Acute myocardial infarctionCardiac disorders1/22
DiarrhoeaGastrointestinal disorders1/22
Diverticulum intestinal haemorrhagicGastrointestinal disorders1/22
Gastric ulcerGastrointestinal disorders1/22
VomitingGastrointestinal disorders1/22
MalaiseGeneral disorders1/22
Hepatic function abnormalHepatobiliary disorders1/22
HeadacheNervous system disorders1/22
Most frequent other events
Showing 10 of 48
Most frequent other events
EventAvelumab + Axitinib
HypertensionVascular disorders17/22
Decreased appetiteMetabolism and nutrition disorders12/22
DysphoniaRespiratory, thoracic and mediastinal disorders11/22
Palmar-plantar erythrodysaesthesia syndromeSkin and subcutaneous tissue disorders11/22
StomatitisGastrointestinal disorders9/22
Weight decreasedInvestigations9/22
HypothyroidismEndocrine disorders7/22
ConstipationGastrointestinal disorders7/22
MalaiseGeneral disorders7/22
DiarrhoeaGastrointestinal disorders6/22

Baseline characteristics

Full analysis set included participants who received at least one dose of study drug.

Age, Continuous
Age, Continuous(years)Avelumab + Axitinib
Mean65.4 ± 14.98
Sex: Female, Male
Sex: Female, Male(Participants)Avelumab + Axitinib
Female2
Male20
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Avelumab + Axitinib
Hispanic or Latino0
Not Hispanic or Latino22
Unknown or Not Reported0
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Avelumab + Axitinib
American Indian or Alaska Native0
Asian22
Native Hawaiian or Other Pacific Islander0
Black or African American0
White0
More than one race0
Unknown or Not Reported0
07

Study locations

7 sites
  • Aichi Cancer Center Hospital
    Nagoya, Aichi 464-8681, Japan
  • Iizuka Hospital
    Iizuka, Fukuoka 820-8505, Japan
  • Kindai University Hospital, Department of Gastroenterology and Hepatology
    Osaka-Sayama, Osaka 589-8511, Japan
  • National Cancer Center Hospital
    Chuo-ku, Tokyo 104-0045, Japan
  • Kyorin University Hospital, Department of Medical Oncology
    Mitaka-shi, Tokyo 181-8611, Japan
  • Japanese Red Cross Musashino Hospital
    Musashino, Tokyo 180-8610, Japan
  • National Hospital Organization Kyushu Medical Center
    Fukuoka, 810-8563, Japan
08

References and documents

Study documents

  • Study protocol · May 18, 2017
  • Statistical analysis plan · Feb 20, 2019

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: No — Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

09

Registry details

Key details

Study ID
NCT03289533
Lead sponsor
Pfizer
Responsible party
Sponsor
First posted
Sep 21, 2017
Start date
Sep 8, 2017
Primary completion
Aug 27, 2019
Completion
Oct 25, 2019
Results posted
Sep 4, 2020
Last update
Sep 4, 2020

Study contacts

Pfizer CT.gov Call Center
study director · Pfizer

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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