Cholangiocarcinoma Non-resectablePhase 1NCT03267940
Study of PEGPH20 With Cisplatin (CIS) and Gemcitabine (GEM); PEGPH20 With Atezolizumab (ATEZO), CIS, and GEM; and CIS and GEM Alone in Participants With Previously Untreated, Unresectable, Locally Advanced, or Metastatic Intrahepatic and Extrahepatic Cholangiocarcinoma and Gallbladder Adenocarcinoma
A Phase 1 study testing PEGPH20, CIS and GEM in people with Cholangiocarcinoma Non-resectable and Cholangiocarcinoma, Intrahepatic, run by Halozyme Therapeutics at 36 sites in 3 countries.
Trial summary
The study is being conducted to assess the safety and tolerability of (1) PEGPH20 in combination with CIS and GEM (PEGCISGEM), and (2) PEGPH20 in combination with CIS, GEM, and atezolizumab (PEGCISGEMATEZO) compared with (3) cisplatin and gemcitabine (CISGEM). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cholangiocarcinoma Non-resectable, Cholangiocarcinoma, Intrahepatic, Cholangiocarcinoma, Extrahepatic
- What is being tested?
- PEGPH20, CIS and GEM
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 85 participants
- Where
- 36 sitesin 3 countries
- Study dates
- 2 Oct 2017to 11 Nov 2019
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- For both portions of the study, participants must satisfy all of the following inclusion criteria to be enrolled in the study:
- Written Institutional Review Board/Ethics Committee-approved informed consent form (ICF), signed by participant or legally authorized representative.
- Participants must be determined to have histologically confirmed unresectable, locally advanced or metastatic adenocarcinoma of the intra- and/or extra-hepatic bile ducts and/or gallbladder. Participants must have sufficient tissue with architectural integrity, including tumor and associated stroma, available for retrospective biomarker testing.
- One or more lesions measurable on computed tomography (CT) scan/magnetic resonance imaging (MRI) scan per Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 (v1.1).
- Participants having Eastern Cooperative Oncology Group (ECOG) Performance Status of 0 to 1.
- Life expectancy ≥3 months.
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- Males and females aged ≥18 years.
- Screening clinical laboratory values within pre-determined parameters
- Female participants of childbearing potential (WOCBP) must have a negative urine or serum pregnancy test within 7 days before Day 1 (first dose of study medication).
- For WOCBP and for men, agreement to use a highly effective contraceptive method from the time of screening throughout the study until 5 months (WOCBP) or 6 months (men) after administration of the last dose of any study medication. Highly effective contraceptive methods consist of prior sterilization, intrauterine device (IUD), intrauterine hormone releasing system (IUS), oral or injectable contraceptives, barrier methods, and/or true sexual abstinence.
Who cannot join
- Participants are ineligible for enrollment if they meet any of the following exclusion criteria:
- Clinical evidence of deep vein thrombosis or pulmonary embolism present during the screening period
- New York Heart Association Class III or IV cardiac disease, atrial fibrillation, unstable angina, or myocardial infarction within the past 12 months before screening.
- Participants with known brain metastases
- History of cerebrovascular accident or transient ischemic attack
- History of active bleeding within the last 3 months prior to screening requiring transfusion.
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- Participants must have received no previous radiotherapy, surgery, chemotherapy or investigational therapy for treatment of metastatic or locally advanced disease.
- Intolerance to non-steroidal anti-inflammatory drugs (NSAIDs).
- Active hepatitis B virus (HBV) infection, defined as having a positive hepatitis B surface antigen (HBsAg) test at screening
- Active hepatitis C virus (HCV) infection, defined as having a positive HCV antibody test at screening
- History of:
- Idiopathic pulmonary fibrosis, organizing pneumonia (for example, bronchiolitis obliterans), drug-induced pneumonitis, or idiopathic pneumonitis, or evidence of active pneumonitis on screening chest CT scan. History of radiation pneumonitis in the radiation field (fibrosis) is permitted.
- Or known cases of hepatobiliary diseases (for example, primary biliary cholangitis, primary sclerosing cholangitis, history of immune-mediated cholangitis);
- Participants with cholangitis attributed to infectious etiology (for example, ascending cholangitis, bacterial cholangitis) are eligible if the infection has been fully resolved prior to the screening visit.
- Or known cases of drug-induced hepatobiliary toxicities.
- Active or history of autoimmune diseases
- Uncontrolled hypercalcemia
Trial locations and contacts
36 sites in 3 countries.
36 sites in 3 countries
United States26 sites
Johns Hopkins
Baltimore, Maryland, 21287
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
Gabrail Cancer Center
Canton, Ohio, 44718
Medical University of South Carolina
Charleston, South Carolina, 29425
City of Hope
Duarte, California, 91010
Duke Cancer Institute
Durham, North Carolina, 27710
MD Anderson Cancer Center
Houston, Texas, 77030
Scripps
La Jolla, California, 92037
USC/Norris Comprehensive Cancer Center
Los Angeles, California, 90033
Froedtert Hospital And Medical College
Milwaukee, Wisconsin, 53226
Yale Cancer Center
New Haven, Connecticut, 06511
Mount Sinai
New York, New York, 10029
University of California Irvine Division of Hematology-Oncology, Department of Medicine UC Irvine Health
Orange, California, 92868
Mayo Clinic of Arizona
Phoenix, Arizona, 85054
University of Pittsburgh Cancer Institute
Pittsburgh, Pennsylvania, 15232
University of Rochester Medical Center
Rochester, New York, 14642
UC Davis
Sacramento, California, 95817
Huntsman Cancer Institute
Salt Lake City, Utah, 84112
UT Health Cancer Center
San Antonio, Texas, 78229
UCLA - David Geffen School of Medicine
Santa Monica, California, 90404
Virginia Mason
Seattle, Washington, 98101
Seattle Cancer Care Alliance
Seattle, Washington, 98109
Washington University School of Medicine
St Louis, Missouri, 63110
University of Arizona
Tucson, Arizona, 85724
Lombardi Cancer Center, Georgetown University
Washington D.C., District of Columbia, 20007
The Oncology Institute of Hope and Innovation
Whittier, California, 90603
South Korea7 sites
Seoul National University Bundang Hospital
Seongnam-si, Gyeonggi-do, 13620
Samsung Medical Center
Seoul, Gangnam-gu, 06351
Korea University Guro Hospital
Seoul, Guro-gu, 08308
Seoul National University Hospital
Seoul, Jongno-gu, 03080
The Catholic University of Korea Seoul St. Mary's Hospital
Seoul, Seocho-gu, 06591
Severance Hospital, Yonsei University Health System
Seoul, Seodaemun-Gu, 03722
Asan Medical Center
Seoul, Songpa-gu, 05505
Thailand3 sites
King Chulalongkorn Memorial Hospital
Bangkok, Patumwan, 10330
Maharaj Nakorn Chiang Mai Hospital
Chiang Mai, Muang, 50200
Prince of Songkla University
Hat Yai, Changwat Songkhla, 90110
About this study
Official title: A Phase 1B, Randomized, Open-Label Study of PEGylated Recombinant Human Hyaluronidase (PEGPH20) in Combination With Cisplatin Plus Gemcitabine and PEGPH20 in Combination With Atezolizumab and Cisplatin Plus Gemcitabine Compared With Cisplatin Plus Gemcitabine in Subjects With Previously Untreated, Unresectable, Locally Advanced, or Metastatic Intrahepatic and Extrahepatic Cholangiocarcinoma and Gallbladder Adenocarcinoma
The study is being conducted to assess the safety and tolerability of (1) PEGPH20 in combination with CIS and GEM (PEGCISGEM), and (2) PEGPH20 in combination with CIS, GEM, and atezolizumab (PEGCISGEMATEZO) compared with (3) cisplatin and gemcitabine (CISGEM).
Read the detailed description
The study will have a Run-in portion and an Expansion portion. The Run-in portion will be used to evaluate the safety profile of the PEGCISGEM and PEGCISGEMATEZO treatments prior to evaluating the efficacy and safety of PEGCISGEM and PEGCISGEMATEZO treatments compared with CISGEM treatment in the Expansion portion of the study. Treatment in both portions of the study will continue until death, withdrawal of consent from the study, disease progression, or unacceptable toxicity.
- Conditions
- Cholangiocarcinoma Non-resectable, Cholangiocarcinoma, Intrahepatic, Cholangiocarcinoma, Extrahepatic, Gallbladder Adenocarcinoma
- Being tested
- PEGPH20, CIS, GEM, Atezolizumab
- Keywords
- Extrahepatic Cholangiocarcinoma, Intrahepatic Cholangiocarcinoma, Gallbladder Adenocarcinoma, PEGylated Recombinant Human Hyaluronidase, PEGylated Recombinant Human Hyaluronidase with atezolizumab
- Medical subject headings
- Cholangiocarcinoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PEGPH20Drug
PEGPH20 will be administered as per the schedule specified in the respective arms.
CISDrug
CIS will be administered as per the schedule specified in the respective arms.
GEMDrug
GEM will be administered as per the schedule specified in the respective arms.
AtezolizumabDrug
Atezolizumab will be administered as per the schedule specified in the respective arms.
| Study group | Role | What they receive |
|---|---|---|
| Run-in Portion: PEGCISGEM | Experimental | Participants will receive 3.0 micrograms per kilogram (mcg/kg) PEGPH20 on Days 1, 8, and 15 in combination with 25 milligrams per meter square (mg/m^2) of CIS plus 1000 mg/m^2 of GEM administered on Days 2 and 9 of each 21-day cycle by intravenous (IV) infusion. Treatment will continue until death, withdrawal of consent from the study, disease progression, or unacceptable toxicity. Receives: Drug: PEGPH20; Drug: CIS; Drug: GEM |
| Run-in Portion: PEGCISGEMATEZO | Experimental | After 6 participants from the PEGCISGEM arm are treated for at least 1 cycle without significant toxicities, new participants will be enrolled in this arm to receive 3.0 mcg/kg PEGPH20 on Days 1, 8, and 15 in combination with 1200 mg ATEZO (administered 1 to 3 hours after PEGPH20 on Day 1 of each 21-day cycle) plus 25 mg/m^2 of CIS and 1000 mg/m^2 GEM on Days 2 and 9 of each 21-day cycle by IV infusion. Treatment will continue until death, withdrawal of consent from the study, disease progression, or unacceptable toxicity. Receives: Drug: PEGPH20; Drug: CIS; Drug: GEM; Drug: Atezolizumab |
| Expansion Portion: PEGCISGEM | Experimental | After the Investigators and the Sponsor considers the study treatment with PEGCISGEM during the Run-in portion safe and tolerable, new participants will be enrolled to receive 3.0 mcg/kg PEGPH20 on Days 1, 8, and 15 in combination with 25 mg/m^2 CIS and 1000 mg/m^2 GEM on Days 2 and 9 of each 21-day cycle by IV infusion. Treatment will continue until death, withdrawal of consent from the study, disease progression, or unacceptable toxicity. Receives: Drug: PEGPH20; Drug: CIS; Drug: GEM |
| Expansion Portion: PEGCISGEMATEZO Twice Weekly | Experimental | After the Investigators and the Sponsor considers the study treatment with PEGCISGEM during the Run-in portion safe and tolerable, new participants will be enrolled to receive 3.0 mcg/kg PEGPH20 on Days 1, 8, and 15 in combination with 1200 mg ATEZO (administered 1 to 3 hours after PEGPH20 on Day 1 of each 21-day cycle) plus 25 mg/m^2 of CIS and 1000 mg/m^2 GEM on Days 2 and 9 of each 21-day cycle by IV infusion. Treatment will continue until death, withdrawal of consent from the study, disease progression, or unacceptable toxicity. Receives: Drug: PEGPH20; Drug: CIS; Drug: GEM; Drug: Atezolizumab |
| Expansion Portion: PEGCISGEMATEZO Once Weekly/Twice Weekly | Experimental | After the implementation of Protocol Amendment #3 and as communicated to the Investigators via a letter dated 22 March 2019, participants will receive 3.0 mcg/kg PEGPH20 on Days 1, 4, 8, 11, 15 and 18 in combination with 1200 mg ATEZO (administered 1 to 3 hours after PEGPH20 on Day 1 Cycle 1) plus 25 mg/m^2 of CIS and 1000 mg/m^2 GEM on Days 2 and 9 of Cycle 1 (cycle length = 21 days) by IV infusion. Participants will receive 3.0 mcg/kg PEGPH20 on Days 1, 8, and 15 in combination with 1200 mg ATEZO (administered 1 to 3 hours after PEGPH20 on Day 1 of each 21-day cycle) plus 25 mg/m^2 of CIS and 1000 mg/m^2 GEM on Days 2 and 9 of each 21-day cycle from Cycle 2 and beyond by IV infusion. Treatment will continue until death, withdrawal of consent from the study, disease progression, or unacceptable toxicity. Receives: Drug: PEGPH20; Drug: CIS; Drug: GEM; Drug: Atezolizumab |
| Expansion Portion: CISGEM | Active comparator | After the Investigators and the Sponsor considers the study treatment with PEGCISGEM during the run-in portion safe and tolerable, new participants will be enrolled to receive 25 mg/m^2 CIS and 1000 mg/m^2 GEM on Days 1 and 8 of each 21-day cycle by IV infusion. Treatment will continue until death, withdrawal of consent from the study, disease progression, or unacceptable toxicity. Receives: Drug: CIS; Drug: GEM |
What is being measured
The primary outcome is the main question the study is designed to answer.
Run-in Portion: Number of Participants With Adverse Events (AEs)
An AE is any unfavorable or unintended sign, symptom, or disease temporally associated with the use of a pharmaceutical product (for example, study drug), whether or not considered related to the pharmaceutical product. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
Measured: From first exposure to study drug through 30 days after the end of treatment visit (maximum exposure: 508 days)
Run-in Portion: Number of Participants With Clinically Significant Abnormalities in Laboratory Parameters (Hematology and Blood Chemistry)
Laboratory parameters evaluation included hematology (hemoglobin, hematocrit, red blood cell count, white blood cell [WBC] count, neutrophils [ANC], lymphocytes, monocytes, eosinophils, basophils, granulocytes, mean corpuscular hemoglobin, mean corpuscular volume, and platelet count) and blood chemistry (glucose, blood urea nitrogen [BUN], albumin, total bilirubin, alkaline phosphatase, aspartate aminotransferase [AST], alanine aminotransferase [ALT], electrolytes [including sodium, potassium, calcium, magnesium, chloride, and bicarbonate], and creatinine) evaluation. Clinical significance was defined as per Investigator's discretion. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
Measured: From first exposure to study drug through the end of treatment visit (maximum exposure: 508 days)
Run-in Portion: Number of Participants With Clinically Significant Abnormalities in Electrocardiogram (ECG) and Vital Signs
Clinical significance of ECGs was defined as per Investigator's discretion. Assessment of vital signs was to include the measurement of blood pressure (systolic and diastolic), pulse, respiratory rate, and body temperature. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
Measured: From first exposure to study drug through the end of treatment visit (maximum exposure: 508 days)
Run-in Portion: Number of Participants With AEs Leading to Dose Reduction or Interruption of Any Study Medication
Number of participants with AEs leading to dose reduction or interruption of any study medication (PEGPH20, CIS, GEM, or ATEZO) was reported. An AE is any unfavorable or unintended sign, symptom, or disease temporally associated with the use of a pharmaceutical product (for example, study drug), whether or not considered related to the pharmaceutical product. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
Measured: From first exposure to study drug through the end of treatment visit (maximum exposure: 508 days)
Expansion Portion: Objective Response Rate (ORR) Based on Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1: Percentage of Participants With Objective Response
ORR was defined as percentage of participants who achieved either a complete response (CR) or partial response (PR). CR was defined as disappearance of all target and non-target lesions; Any pathological or non-pathological lymph nodes (whether target or non-target) must have reduction in short axis to less than (<) 10 millimeters (mm). PR was defined as at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters.
Measured: From date of randomization until the date of first documented progression of disease or date of death from any cause, whichever came first (maximum exposure: 421 days)
Secondary outcomes (17)
Run-in Portion: ORR Based on RECIST v1.1: Percentage of Participants With Objective Response
ORR was defined as percentage of participants who achieved either a CR or PR. CR was defined as disappearance of all target and non-target lesions; Any pathological or non-pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm. PR was defined as at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters.
Measured: From date of randomization until the date of first documented progression of disease or date of death from any cause, whichever came first (maximum exposure: 508 days)
Expansion Portion: Duration of Response (DOR) Based on RECIST v1.1
DOR was considered the time from date of the first CR or PR until the date of first documentation of disease progression or date of death based on RECIST v1.1 for target lesions assessed by MRI/CT scans, as determined by independent radiologic review. CR was defined as the disappearance of all target lesions. PR was defined as at least a 30% decrease in the sum of diameters of all target lesions, taking as reference the baseline sum of diameters, in the absence of CR.
Measured: From date of the first CR or PR until the date of first documentation of disease progression or date of death, whichever came first (maximum exposure: 421 days)
Expansion Portion: Progression-Free Survival (PFS) Based on RECIST v1.1
PFS was based on RECIST v1.1 for target lesions assessed by MRI/CT scans, as determined by independent radiologic review and was defined as the time from randomization to radiological disease progression or death. Progressive disease (PD) was defined as at least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum of diameters on or prior to the current assessment (including baseline). In addition to the relative increase of 20%, the sum of diameters must also demonstrate an absolute increase of greater than or equal to (≥) 5 mm.
Measured: From date of randomization until date of progressive disease or death from any cause, whichever came first (maximum exposure: 421 days)
Expansion Portion: Disease Control Rate (DCR) Based on RECIST v1.1: Percentage of Participants With CR, PR or Stable Disease (SD)
DCR was defined as the percentage of participants with CR, PR, or SD based on RECIST v1.1 for target lesions assessed by MRI/CT scans, as determined by independent radiologic review. CR was defined as the disappearance of all target lesions. PR was defined as at least a 30% decrease in the sum of diameters of all target lesions, taking as reference the baseline sum of diameters, in the absence of CR. SD was defined as neither sufficient shrinkage to qualify for CR or PR nor sufficient increase to qualify for PD.
Measured: From date of randomization until date of progressive disease or death from any cause, whichever came first (maximum exposure: 421 days)
Expansion Portion: Overall Survival (OS)
Overall survival was defined as the time from randomization until death from any cause.
Measured: From randomization until death from any cause (maximum exposure: 421 days)
Expansion Portion: OS by PD-L1 Expression Levels
Overall survival was defined as the time from randomization until death from any cause.
Measured: From randomization until death from any cause (maximum exposure: 421 days)
Expansion Portion: Number of Participants With AEs
An AE is any unfavorable or unintended sign, symptom, or disease temporally associated with the use of a pharmaceutical product (for example, study drug), whether or not considered related to the pharmaceutical product. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
Measured: From first exposure to study drug through the end of treatment visit (maximum exposure: 421 days)
Expansion Portion: Number of Participants With Clinically Significant Abnormalities in Clinical Laboratory Parameters (Hematology and Blood Chemistry)
Laboratory parameters evaluation included hematology (hemoglobin, hematocrit, red blood cell count, WBC count, neutrophils [ANC], lymphocytes, monocytes, eosinophils, basophils, granulocytes, mean corpuscular hemoglobin, mean corpuscular volume, and platelet count) and blood chemistry (glucose, BUN, albumin, total bilirubin, alkaline phosphatase, AST, ALT, electrolytes [including sodium, potassium, calcium, magnesium, chloride, and bicarbonate], and creatinine) evaluation. Clinical significance was defined as per Investigator's discretion. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
Measured: From first exposure to study drug through the end of treatment visit (maximum exposure: 421 days)
Expansion Portion: Number of Participants With Clinically Significant Abnormalities in ECG and Vital Signs
Clinical significance of ECGs was defined as per Investigator's discretion. Assessment of vital signs was to include the measurement of blood pressure (systolic and diastolic), pulse, respiratory rate, and body temperature. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
Measured: From first exposure to study drug through the end of treatment visit (maximum exposure: 421 days)
Expansion Period: Number of Participants With AEs Leading to Dose Reduction or Interruption of Any Study Medication
Number of participants with AEs leading to dose reduction or interruption of any study medication (PEGPH20, CIS, GEM, or ATEZO) was reported. An AE is any unfavorable or unintended sign, symptom, or disease temporally associated with the use of a pharmaceutical product (for example, study drug), whether or not considered related to the pharmaceutical product. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
Measured: From first exposure to study drug through 30 days after the end of treatment visit (maximum exposure: 421 days)
Run-in and Expansion Portion : Maximum Observed Plasma Concentration (Cmax) of PEGPH20, ATEZO, GEM, and CIS
Plasma pharmacokinetic (PK) data were planned to be analyzed using a noncompartmental analysis approach.
Measured: PEGPH20 and ATEZO: Cycle 1: multiple timepoints on Days 1, 2, 8, 9, 15; ATEZO: Day 1 of subsequent cycles and end of treatment (EOT) (maximum exposure: 508 days for run-in and 421 days for expansion); CIS and GEM: Cycle 1: multiple timepoints on Days 2,9
Run-in and Expansion Portion : Minimum Observed Plasma Concentration (Cmin) of PEGPH20 and ATEZO
PK data were planned to be analyzed using a noncompartmental analysis approach.
Measured: PEGPH20 and ATEZO: Treatment Cycle 1: multiple timepoints on Days 1, 2, 8, 9, and 15; ATEZO: Day 1 of subsequent cycles and EOT visit (7 days after last 21-day cycle) (maximum exposure: 508 days for run-in portion and 421 days for expansion portion)
Run-in and Expansion Portion : Elimination Rate Constant (Kel) of PEGPH20
PK data were planned to be analyzed using a noncompartmental analysis approach.
Measured: Cycle 1: multiple timepoints on Days 1, 2, 8, 9, and 15
Run-in and Expansion Portion : Terminal Elimination Plasma Half-life (t1/2) of PEGPH20, ATEZO, GEM, and CIS
PK data were planned to be analyzed using a noncompartmental analysis approach.
Measured: PEGPH20 and ATEZO: Cycle 1: multiple timepoints on Days 1, 2, 8, 9, 15; ATEZO: Day 1 of subsequent cycles and EOT visit (maximum exposure: 508 days for run-in and 421 days for expansion portion); CIS and GEM: Cycle 1: multiple timepoints on Days 2, 9
Run-in and Expansion Portion : Clearance (CL) of PEGPH20, ATEZO, GEM, and CIS
PK data were planned to be analyzed using a noncompartmental analysis approach.
Measured: PEGPH20 and ATEZO: Cycle 1: multiple timepoints on Days 1, 2, 8, 9, 15; ATEZO: Day 1 of subsequent cycles and EOT visit (maximum exposure: 508 days for run-in and 421 days for expansion portion); CIS and GEM: Cycle 1: multiple timepoints on Days 2, 9
Run-in and Expansion Portion : Volume of Distribution (Vd) of PEGPH20, ATEZO, GEM, and CIS
PK data were planned to be analyzed using a noncompartmental analysis approach.
Measured: PEGPH20 and ATEZO: Cycle 1: multiple timepoints on Days 1, 2, 8, 9, 15; ATEZO: Day 1 of subsequent cycles and EOT visit (maximum exposure: 508 days for run-in and 421 days for expansion portion); CIS and GEM: Cycle 1: multiple timepoints on Days 2, 9
Run-in and Expansion Portion: Area Under the Concentration Time Curve (AUC) of PEGPH20, ATEZO, GEM, and CIS
PK data were planned to be analyzed using a noncompartmental analysis approach.
Measured: PEGPH20 and ATEZO: Cycle 1: multiple timepoints on Days 1, 2, 8, 9, 15; ATEZO: Day 1 of subsequent cycles and EOT visit (maximum exposure: 508 days for run-in and 421 days for expansion portion); CIS and GEM: Cycle 1: multiple timepoints on Days 2, 9
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Aug 2017
- Study start2 Oct 2017
- Primary completion11 Nov 2019
- Study completion11 Nov 2019
Why it stopped: Sponsor decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Cholangiocarcinoma Non-resectableStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 85 participants (actual)
Sponsor and investigators
- Lead sponsor
- Halozyme Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- VP, Medical, Regulatory and Drug Safety, Study director, Halozyme Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03267940
- Sponsor's protocol ID
- Halo-110-101
- Status last verified
- Jan 2020
- First submitted
- 25 Jul 2017
- Last updated
- 7 Feb 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Feb 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1B, Randomized, Open-Label Study of PEGylated Recombinant Human Hyaluronidase (PEGPH20) in Combination With Cisplatin Plus Gemcitabine and PEGPH20 in Combination With Atezolizumab and Cisplatin Plus Gemcitabine Compared With Cisplatin Plus Gemcitabine in Subjects With Previously Untreated, Unresectable, Locally Advanced, or Metastatic Intrahepatic and Extrahepatic Cholangiocarcinoma and Gallbladder Adenocarcinoma
- ClinicalTrials.gov ID
- NCT03267940
- Acronym / sponsor ID
- Halo-110-101
- Conditions
- Cholangiocarcinoma Non-resectable, Cholangiocarcinoma, Intrahepatic, Cholangiocarcinoma, Extrahepatic, Gallbladder Adenocarcinoma
- Keywords
- Extrahepatic Cholangiocarcinoma, Intrahepatic Cholangiocarcinoma, Gallbladder Adenocarcinoma, PEGylated Recombinant Human Hyaluronidase, PEGylated Recombinant Human Hyaluronidase with atezolizumab
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor decision
- Enrolment
- 85 participants (actual)
- Lead sponsor
- Halozyme TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 2 Oct 2017 (actual)
- Primary completion
- 11 Nov 2019 (actual)
- Study completion
- 11 Nov 2019 (actual)
- First posted
- 31 Aug 2017 (actual)
- Results
- Posted 7 Feb 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study is being conducted to assess the safety and tolerability of (1) PEGPH20 in combination with CIS and GEM (PEGCISGEM), and (2) PEGPH20 in combination with CIS, GEM, and atezolizumab (PEGCISGEMATEZO) compared with (3) cisplatin and gemcitabine (CISGEM).
Detailed description
The study will have a Run-in portion and an Expansion portion. The Run-in portion will be used to evaluate the safety profile of the PEGCISGEM and PEGCISGEMATEZO treatments prior to evaluating the efficacy and safety of PEGCISGEM and PEGCISGEMATEZO treatments compared with CISGEM treatment in the Expansion portion of the study. Treatment in both portions of the study will continue until death, withdrawal of consent from the study, disease progression, or unacceptable toxicity.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Run-in Portion: PEGCISGEM | Experimental | Drug: PEGPH20, Drug: CIS, Drug: GEMParticipants will receive 3.0 micrograms per kilogram (mcg/kg) PEGPH20 on Days 1, 8, and 15 in combination with 25 milligrams per meter square (mg/m^2) of CIS plus 1000 mg/m^2 of GEM administered on Days 2 and 9 of each 21-day cycle by intravenous (IV) infusion. Treatment will continue until death, withdrawal of consent from the study, disease progression, or unacceptable toxicity. |
| Run-in Portion: PEGCISGEMATEZO | Experimental | Drug: PEGPH20, Drug: CIS, Drug: GEM, Drug: AtezolizumabAfter 6 participants from the PEGCISGEM arm are treated for at least 1 cycle without significant toxicities, new participants will be enrolled in this arm to receive 3.0 mcg/kg PEGPH20 on Days 1, 8, and 15 in combination with 1200 mg ATEZO (administered 1 to 3 hours after PEGPH20 on Day 1 of each 21-day cycle) plus 25 mg/m^2 of CIS and 1000 mg/m^2 GEM on Days 2 and 9 of each 21-day cycle by IV infusion. Treatment will continue until death, withdrawal of consent from the study, disease progression, or unacceptable toxicity. |
| Expansion Portion: PEGCISGEM | Experimental | Drug: PEGPH20, Drug: CIS, Drug: GEMAfter the Investigators and the Sponsor considers the study treatment with PEGCISGEM during the Run-in portion safe and tolerable, new participants will be enrolled to receive 3.0 mcg/kg PEGPH20 on Days 1, 8, and 15 in combination with 25 mg/m^2 CIS and 1000 mg/m^2 GEM on Days 2 and 9 of each 21-day cycle by IV infusion. Treatment will continue until death, withdrawal of consent from the study, disease progression, or unacceptable toxicity. |
| Expansion Portion: PEGCISGEMATEZO Twice Weekly | Experimental | Drug: PEGPH20, Drug: CIS, Drug: GEM, Drug: AtezolizumabAfter the Investigators and the Sponsor considers the study treatment with PEGCISGEM during the Run-in portion safe and tolerable, new participants will be enrolled to receive 3.0 mcg/kg PEGPH20 on Days 1, 8, and 15 in combination with 1200 mg ATEZO (administered 1 to 3 hours after PEGPH20 on Day 1 of each 21-day cycle) plus 25 mg/m^2 of CIS and 1000 mg/m^2 GEM on Days 2 and 9 of each 21-day cycle by IV infusion. Treatment will continue until death, withdrawal of consent from the study, disease progression, or unacceptable toxicity. |
| Expansion Portion: PEGCISGEMATEZO Once Weekly/Twice Weekly | Experimental | Drug: PEGPH20, Drug: CIS, Drug: GEM, Drug: AtezolizumabAfter the implementation of Protocol Amendment #3 and as communicated to the Investigators via a letter dated 22 March 2019, participants will receive 3.0 mcg/kg PEGPH20 on Days 1, 4, 8, 11, 15 and 18 in combination with 1200 mg ATEZO (administered 1 to 3 hours after PEGPH20 on Day 1 Cycle 1) plus 25 mg/m^2 of CIS and 1000 mg/m^2 GEM on Days 2 and 9 of Cycle 1 (cycle length = 21 days) by IV infusion. Participants will receive 3.0 mcg/kg PEGPH20 on Days 1, 8, and 15 in combination with 1200 mg ATEZO (administered 1 to 3 hours after PEGPH20 on Day 1 of each 21-day cycle) plus 25 mg/m^2 of CIS and 1000 mg/m^2 GEM on Days 2 and 9 of each 21-day cycle from Cycle 2 and beyond by IV infusion. Treatment will continue until death, withdrawal of consent from the study, disease progression, or unacceptable toxicity. |
| Expansion Portion: CISGEM | Active comparator | Drug: CIS, Drug: GEMAfter the Investigators and the Sponsor considers the study treatment with PEGCISGEM during the run-in portion safe and tolerable, new participants will be enrolled to receive 25 mg/m^2 CIS and 1000 mg/m^2 GEM on Days 1 and 8 of each 21-day cycle by IV infusion. Treatment will continue until death, withdrawal of consent from the study, disease progression, or unacceptable toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- For both portions of the study, participants must satisfy all of the following inclusion criteria to be enrolled in the study:
- Written Institutional Review Board/Ethics Committee-approved informed consent form (ICF), signed by participant or legally authorized representative.
- Participants must be determined to have histologically confirmed unresectable, locally advanced or metastatic adenocarcinoma of the intra- and/or extra-hepatic bile ducts and/or gallbladder. Participants must have sufficient tissue with architectural integrity, including tumor and associated stroma, available for retrospective biomarker testing.
- One or more lesions measurable on computed tomography (CT) scan/magnetic resonance imaging (MRI) scan per Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 (v1.1).
- Participants having Eastern Cooperative Oncology Group (ECOG) Performance Status of 0 to 1.
Key exclusion criteria (17 listed)
- Participants are ineligible for enrollment if they meet any of the following exclusion criteria:
- Clinical evidence of deep vein thrombosis or pulmonary embolism present during the screening period
- New York Heart Association Class III or IV cardiac disease, atrial fibrillation, unstable angina, or myocardial infarction within the past 12 months before screening.
- Participants with known brain metastases
- History of cerebrovascular accident or transient ischemic attack
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PEGPH20PEGPH20 will be administered as per the schedule specified in the respective arms. | Drug | Expansion Portion: PEGCISGEM, Expansion Portion: PEGCISGEMATEZO Once Weekly/Twice Weekly, Expansion Portion: PEGCISGEMATEZO Twice Weekly, Run-in Portion: PEGCISGEM, Run-in Portion: PEGCISGEMATEZO |
| CISCIS will be administered as per the schedule specified in the respective arms. | Drug | Expansion Portion: CISGEM, Expansion Portion: PEGCISGEM, Expansion Portion: PEGCISGEMATEZO Once Weekly/Twice Weekly, Expansion Portion: PEGCISGEMATEZO Twice Weekly, Run-in Portion: PEGCISGEM, Run-in Portion: PEGCISGEMATEZO |
| GEMGEM will be administered as per the schedule specified in the respective arms. | Drug | Expansion Portion: CISGEM, Expansion Portion: PEGCISGEM, Expansion Portion: PEGCISGEMATEZO Once Weekly/Twice Weekly, Expansion Portion: PEGCISGEMATEZO Twice Weekly, Run-in Portion: PEGCISGEM, Run-in Portion: PEGCISGEMATEZO |
| AtezolizumabAtezolizumab will be administered as per the schedule specified in the respective arms. | Drug | Expansion Portion: PEGCISGEMATEZO Once Weekly/Twice Weekly, Expansion Portion: PEGCISGEMATEZO Twice Weekly, Run-in Portion: PEGCISGEMATEZO |
Primary outcome
Run-in Portion: Number of Participants With Adverse Events (AEs)
Time frame: From first exposure to study drug through 30 days after the end of treatment visit (maximum exposure: 508 days)
An AE is any unfavorable or unintended sign, symptom, or disease temporally associated with the use of a pharmaceutical product (for example, study drug), whether or not considered related to the pharmaceutical product. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
Run-in Portion: Number of Participants With Clinically Significant Abnormalities in Laboratory Parameters (Hematology and Blood Chemistry)
Time frame: From first exposure to study drug through the end of treatment visit (maximum exposure: 508 days)
Laboratory parameters evaluation included hematology (hemoglobin, hematocrit, red blood cell count, white blood cell [WBC] count, neutrophils [ANC], lymphocytes, monocytes, eosinophils, basophils, granulocytes, mean corpuscular hemoglobin, mean corpuscular volume, and platelet count) and blood chemistry (glucose, blood urea nitrogen [BUN], albumin, total bilirubin, alkaline phosphatase, aspartate aminotransferase [AST], alanine aminotransferase [ALT], electrolytes [including sodium, potassium, calcium, magnesium, chloride, and bicarbonate], and creatinine) evaluation. Clinical significance was defined as per Investigator's discretion. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
Run-in Portion: Number of Participants With Clinically Significant Abnormalities in Electrocardiogram (ECG) and Vital Signs
Time frame: From first exposure to study drug through the end of treatment visit (maximum exposure: 508 days)
Clinical significance of ECGs was defined as per Investigator's discretion. Assessment of vital signs was to include the measurement of blood pressure (systolic and diastolic), pulse, respiratory rate, and body temperature. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
Run-in Portion: Number of Participants With AEs Leading to Dose Reduction or Interruption of Any Study Medication
Time frame: From first exposure to study drug through the end of treatment visit (maximum exposure: 508 days)
Number of participants with AEs leading to dose reduction or interruption of any study medication (PEGPH20, CIS, GEM, or ATEZO) was reported. An AE is any unfavorable or unintended sign, symptom, or disease temporally associated with the use of a pharmaceutical product (for example, study drug), whether or not considered related to the pharmaceutical product. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
Expansion Portion: Objective Response Rate (ORR) Based on Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1: Percentage of Participants With Objective Response
Time frame: From date of randomization until the date of first documented progression of disease or date of death from any cause, whichever came first (maximum exposure: 421 days)
ORR was defined as percentage of participants who achieved either a complete response (CR) or partial response (PR). CR was defined as disappearance of all target and non-target lesions; Any pathological or non-pathological lymph nodes (whether target or non-target) must have reduction in short axis to less than (<) 10 millimeters (mm). PR was defined as at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters.
17 secondary outcomes
Run-in Portion: ORR Based on RECIST v1.1: Percentage of Participants With Objective Response
Time frame: From date of randomization until the date of first documented progression of disease or date of death from any cause, whichever came first (maximum exposure: 508 days)
Expansion Portion: Duration of Response (DOR) Based on RECIST v1.1
Time frame: From date of the first CR or PR until the date of first documentation of disease progression or date of death, whichever came first (maximum exposure: 421 days)
Expansion Portion: Progression-Free Survival (PFS) Based on RECIST v1.1
Time frame: From date of randomization until date of progressive disease or death from any cause, whichever came first (maximum exposure: 421 days)
Expansion Portion: Disease Control Rate (DCR) Based on RECIST v1.1: Percentage of Participants With CR, PR or Stable Disease (SD)
Time frame: From date of randomization until date of progressive disease or death from any cause, whichever came first (maximum exposure: 421 days)
Expansion Portion: Overall Survival (OS)
Time frame: From randomization until death from any cause (maximum exposure: 421 days)
Expansion Portion: OS by PD-L1 Expression Levels
Time frame: From randomization until death from any cause (maximum exposure: 421 days)
Expansion Portion: Number of Participants With AEs
Time frame: From first exposure to study drug through the end of treatment visit (maximum exposure: 421 days)
Expansion Portion: Number of Participants With Clinically Significant Abnormalities in Clinical Laboratory Parameters (Hematology and Blood Chemistry)
Time frame: From first exposure to study drug through the end of treatment visit (maximum exposure: 421 days)
Expansion Portion: Number of Participants With Clinically Significant Abnormalities in ECG and Vital Signs
Time frame: From first exposure to study drug through the end of treatment visit (maximum exposure: 421 days)
Expansion Period: Number of Participants With AEs Leading to Dose Reduction or Interruption of Any Study Medication
Time frame: From first exposure to study drug through 30 days after the end of treatment visit (maximum exposure: 421 days)
Run-in and Expansion Portion : Maximum Observed Plasma Concentration (Cmax) of PEGPH20, ATEZO, GEM, and CIS
Time frame: PEGPH20 and ATEZO: Cycle 1: multiple timepoints on Days 1, 2, 8, 9, 15; ATEZO: Day 1 of subsequent cycles and end of treatment (EOT) (maximum exposure: 508 days for run-in and 421 days for expansion); CIS and GEM: Cycle 1: multiple timepoints on Days 2,9
Run-in and Expansion Portion : Minimum Observed Plasma Concentration (Cmin) of PEGPH20 and ATEZO
Time frame: PEGPH20 and ATEZO: Treatment Cycle 1: multiple timepoints on Days 1, 2, 8, 9, and 15; ATEZO: Day 1 of subsequent cycles and EOT visit (7 days after last 21-day cycle) (maximum exposure: 508 days for run-in portion and 421 days for expansion portion)
Run-in and Expansion Portion : Elimination Rate Constant (Kel) of PEGPH20
Time frame: Cycle 1: multiple timepoints on Days 1, 2, 8, 9, and 15
Run-in and Expansion Portion : Terminal Elimination Plasma Half-life (t1/2) of PEGPH20, ATEZO, GEM, and CIS
Time frame: PEGPH20 and ATEZO: Cycle 1: multiple timepoints on Days 1, 2, 8, 9, 15; ATEZO: Day 1 of subsequent cycles and EOT visit (maximum exposure: 508 days for run-in and 421 days for expansion portion); CIS and GEM: Cycle 1: multiple timepoints on Days 2, 9
Run-in and Expansion Portion : Clearance (CL) of PEGPH20, ATEZO, GEM, and CIS
Time frame: PEGPH20 and ATEZO: Cycle 1: multiple timepoints on Days 1, 2, 8, 9, 15; ATEZO: Day 1 of subsequent cycles and EOT visit (maximum exposure: 508 days for run-in and 421 days for expansion portion); CIS and GEM: Cycle 1: multiple timepoints on Days 2, 9
Run-in and Expansion Portion : Volume of Distribution (Vd) of PEGPH20, ATEZO, GEM, and CIS
Time frame: PEGPH20 and ATEZO: Cycle 1: multiple timepoints on Days 1, 2, 8, 9, 15; ATEZO: Day 1 of subsequent cycles and EOT visit (maximum exposure: 508 days for run-in and 421 days for expansion portion); CIS and GEM: Cycle 1: multiple timepoints on Days 2, 9
Run-in and Expansion Portion: Area Under the Concentration Time Curve (AUC) of PEGPH20, ATEZO, GEM, and CIS
Time frame: PEGPH20 and ATEZO: Cycle 1: multiple timepoints on Days 1, 2, 8, 9, 15; ATEZO: Day 1 of subsequent cycles and EOT visit (maximum exposure: 508 days for run-in and 421 days for expansion portion); CIS and GEM: Cycle 1: multiple timepoints on Days 2, 9
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Feb 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
VP, Medical, Regulatory and Drug Safety study director
Halozyme Therapeutics
36 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing PEGPH20, CIS and GEM in people with Cholangiocarcinoma Non-resectable and Cholangiocarcinoma, Intrahepatic, run by Halozyme Therapeutics at 36 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 7 Feb 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 36 sites in United States, South Korea, Thailand. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 Feb 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Run-in Portion: Number of Participants With Adverse Events (AEs) (time frame: From first exposure to study drug through 30 days after the end of treatment visit (maximum exposure: 508 days)). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Feb 2020.