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Pazopanib

Phase 3

Carcinoma, Renal Cell | Small molecule | Oncology |Novartis AG|Last Updated: Mar 30, 2025

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindCONTROLLEDDMCBiomarker
Total Trials5
Total Enrollment1,876
FDA Designations
No designations recorded
Clinical Trials (5)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01064310Patient Preference Study of Pazopanib Versus Sunitinib in Advanced or Metastatic Kidney CancerPHASE3 COMPLETED 169May 17, 2010Nov 23, 2015Apr 17, 201751 Finland, France +3
NCT00720941Pazopanib Versus Sunitinib in the Treatment of Locally Advanced and/or Metastatic Renal Cell CarcinomaPHASE3 COMPLETED 1,110Aug 14, 2008Mar 24, 2021Mar 30, 2025227 United States, Australia +12
NCT01147822Pazopanib Versus Sunitinib in the Treatment of Asian Subjects With Locally Advanced and/or Metastatic Renal Cell CarcinomaPHASE2 COMPLETED 367May 19, 2010Sep 3, 2021Mar 28, 202521 China, South Korea +1
NCT02014636Safety and Efficacy Study of Pazopanib and MK 3475 in Advanced Renal Cell Carcinoma (RCC; KEYNOTE-018)PHASE1 COMPLETED 42Dec 27, 2013Feb 27, 2019Mar 3, 20206 United States, United Kingdom
NCT00387205Study To Assess Long Term Safety Of PazopanibPHASE1 COMPLETED 188Jun 20, 2006Mar 8, 2018Mar 6, 201919 United States, France +2
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Study Endpoints
Primary Endpoints
Number of Participants With Preference for Pazopanib Versus Sunitinib as Assessed by the Patient Preference Questionnaire (PPQ)
End of treatment of both study drugs (maximum of 22 weeks)

The PPQ is used to measure participants' preference for pazopanib or sunitinib for renal cell carcinoma management and is used to determine a participant's preference for 1 of the 2 drugs given in the 2 double-blind treatment periods. Participants were asked to select 1 of the following: 1. prefer the drug taken as the first treatment; 2. prefer the drug taken as the second treatment; or 3, no preference. Those participants who indicated a preference were asked to select the factors that had an influence on their treatment preference, as well as the most important reason for their preference.

Number of Participants Answering "Yes," "no," or Not Applicable (N/A) to the Question of Whether the Indicated Factors Influenced Their Preference for Sunitinib or Pazopanib Treatment as Assessed by the Patient Preference Questionnaire
End of treatment of both study drugs (maximum of 22 weeks)

The PPQ is used to measure participants' preference for pazopanib or sunitinib for renal cell carcinoma management and is used to determine a participant's preference for 1 of the 2 drugs given in the 2 double-blind treatment periods. Participants were asked to select 1 of the following: 1. prefer the drug taken as the first treatment; 2. prefer the drug taken as the second treatment; or 3, no preference. Those participants who indicated a preference were asked to select the factors that had an influence on their treatment preference, as well as the most important reason for their preference.

Progression-free Survival (PFS)
From randomization until the earliest date of disease progression or date of death from any cause, assessed up to approximately 39 months

PFS was defined as the interval between the date of randomization and the earliest date of progressive disease (PD), as defined by the Independent Review Committee (IRC), or death due to any cause. The IRC defined PD per Response Evaluation Criteria in Solid Tumors (RECIST), Version 1. Per RECIST, PD is defined as a \>=20% increase in the sum of the longest diameter of target lesions, taking as reference the smallest sum longest diameter recorded since the treatment started or the appearance of \>=1 new lesion.

Part 1: Incidence and severity of adverse events (AEs) and serious adverse events (SAEs )
From the start of study treatment (first dose) and, until the post-treatment follow-up visit (at least 30 days after the last dose of investigational product) for AEs, and until 90 days after last dose for SAEs
Part 1: To determine the dose limiting toxicity (DLT) and maximum tolerated regimen (MTR)
8 weeks

MTR is defined as the highest dose of pazopanib in combination with the highest dose of MK 3475 at which no more than 1 of 6 subjects experiences a DLT after a minimum of 8 weeks of treatment. DLT is defined as a drug-related AE starting in the first 8 weeks of treatment

Part 1: Number of subjects with permanent discontinuation of treatment, dose reductions, interruptions, or delays
24 months
Part 1: Change from baseline in laboratory parameters
Average of 4 years

Laboratory assessments include haematology, clinical chemistry, urine, coagulation and thyroid function test

Part 1: Change from baseline in vital signs
30 days after the last dose of study treatment

Vital sign measurements will include heart rate, temperature and blood pressure

Part 1: Change from baseline in cardiac parameters
24 months

Cardiac assessments will include Electrocardiogram (ECG) and Echocardiograms (ECHOs)

Part 1: Incidence and titer of anti MK 3475 antibodies
24 months

Subjects will be monitored for anti-MK 3475 antibodies throughout the study

Part 2: Progression-free survival (PFS)
Average of 4 years

PFS is defined as the interval between the date of randomization and the earlier date of disease progression (using RECIST v1.1) or death due to any cause.

To evaluate the long-term safety of repeat daily doses of pazopanib in cancer subjects with solid tumors
Subjects will stay on the study as long as they are benefiting from treatment, have not met one of the stopping criteria, or experienced a toxicity up to 72 months.

To evaluate the safety assessments; adverse events, vital signs, physical examinations, electrocardiograms, multi-gated acquisition scans or echocardiograms (only for patients on pazopanib and lapatinib combination therapy), and clinical laboratory assessments.

Secondary Endpoints
Change From Period Baseline (BL) in Fatigue as Assessed by the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Score
Day 1 (Period [P] 1 Pre-dose); Weeks 2, 4, 6, 8, and 10 of P 1; during 2-week Wash-out Period (Study Weeks 11 and 12); Weeks 2, 4, 6, and 8 of P 2 (Study Weeks 14, 16, 18, 20, and 22, respectively); End of Study (Week 10 of P 2 [Study Week 22])
Quality of Life as Assessed by the EuroQoL-5 Dimensions (EQ-5D) Thermometer and Utility Scores
Day 1 (Period 1 Pre-dose); during 2-week Wash-out Period (Study Weeks 11 and 12); and End of Study (Week 10 of Period 2 [Study Week 22])
Time to Dose Modification
End of second treatment period (maximum of 22 weeks)
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
pazopanib followed by sunitinibEXPERIMENTAL800mg pazopanib orally for 10 weeks followed by 50mg sunitinib orally for 10 weeks
sunitinib followed by pazopanibEXPERIMENTAL50mg sunitinib orally for 10 weeks followed by 800mg pazopanib orally for 10 weeks
SunitinibACTIVE_COMPARATORControl arm
PazopanibEXPERIMENTALExperimental arm
Part 1EXPERIMENTALPart 1 is a dose escalation phase in which subjects will receive pazopanib orally and the MK 3475 intravenously. Subjects will be evaluated for a minimum of 8 weeks before the next dose level cohort is enrolled.
Part 2EXPERIMENTALPart 2 is a randomized phase in which subjects will be enrolled in each treatment arm: Pazopanib monotherapy Pazopanib+MK-3475 MK-3475 monotherapy
Arm 1EXPERIMENTALPazopanib monotherapy or in combination with lapatinib or other approved anti-cancer medications
Interventions
NameTypeDescription
pazopanibDRUGoral anti-angiogenic treatment
sunitinibDRUGoral anti-angiogenic treatment
MK-3475DRUGMK 3475 is an intravenously administered 100 mg/ 4mL solution available in the potential dose range of 1 to 10 mg/kg.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites51

Inclusion Criteria: * Patients must provide written informed consent prior to performance of any study-specific procedures or assessments and must be willing to comply with treatment and follow up. Procedures conducted as part of the patient's routine clinical management (e.g. blood count, imaging ...

Countries:FinlandFranceGermanyItalyUnited KingdomUnited StatesAustraliaCanadaChinaIrelandJapanNetherlandsSouth KoreaSpainSwedenTaiwanSingapore
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