Ewing Sarcoma, Lymphoma, Non-rhabdomyosarcoma Soft Tissue Sarcoma (NRSTS), Osteosarcoma, Rhabdomyosarcoma, Solid Tumor
Conditions
Brief summary
The purpose of this study is to evaluate the safety and tolerability of surufatinib, thereby identifying the Maximum Tolerated Dose (MTD) and/or Recommended Phase 2 Dose (RP2D) of surufatinib administered in combination with gemcitabine in pediatric patients with recurrent or refractory solid tumors or lymphoma. The study will be conducted in 2 parts.
Detailed description
The purpose of this study is to evaluate the safety and tolerability of surufatinib, thereby identifying the Maximum Tolerated Dose (MTD) and/or Recommended Phase 2 Dose (RP2D) of surufatinib administered in combination with gemcitabine in pediatric patients with recurrent or refractory solid tumors or lymphoma. The study will be conducted in 2 parts: Part 1 - evaluation of tolerability and safety of surufatinib administered in combination with gemcitabine, and confirmation of the recommended clinical dose of surufatinib in pediatric patients with recurrent or refractory solid tumors or lymphoma. Part 2 - evaluation of anti-tumor activity and confirmation of tolerability of surufatinib administered in combination with gemcitabine in pediatric patients with recurrent or refractory osteosarcoma, Ewing sarcoma, RMS, and non-rhabdomyosarcoma soft tissue sarcoma (NRSTS). Part 1 will enroll 2 to 6 patients per dose level cohort (up to 4 cohorts) with recurrent or refractory solid tumors. During cycle 1, surufatinib will be administered, orally, once daily (QD), as a single agent for 14 days followed by surufatinib daily in combination with gemcitabine intravenously on days 15 and 22 (cycle 1 duration=35 days) and days 1 and 8 of all subsequent cycles (cycle duration=21 days). Assessment based on dose limiting toxicity (DLT) criteria will be performed in the first 35-day cycle (DLT Evaluation Period). This study will utilize a rolling 6 design for part 1, with 3 dose escalation levels and 1 de escalation level, if needed. Part 2 of the study will use a Simon 2-stage design with a maximum of 18 patients per cohort (osteosarcoma, Ewing sarcoma, RMS, and non-rhabdomyosarcoma soft tissue sarcoma (NRSTS)). Surufatinib will be administered orally at identified MTD/RP2D daily in combination with gemcitabine (1000 mg/m2 weekly × 2 doses) intravenously on days 1 and 8. In both parts 1 and 2, patients can remain on treatment until completing cycle 17, or until progressive disease, unacceptable toxicity, or death; whichever comes first.
Interventions
DRUGSurufatinib in combination with Gemcitabine
Surufatinib in combination with Gemcitabine
Sponsors
Hutchmed
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
The study will be conducted in 2 parts: Part 1 - evaluation of tolerability and safety of surufatinib administered in combination with gemcitabine, and confirmation of the recommended clinical dose of surufatinib in pediatric patients with recurrent or refractory solid tumors or lymphoma. Part 2 - evaluation of anti-tumor activity and confirmation of tolerability of surufatinib administered in combination with gemcitabine in pediatric patients with recurrent or refractory osteosarcoma, Ewing sarcoma, RMS, and NRSTS.
Eligibility
Sex/Gender
ALL
Age
2 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
1. Age: At time of study enrollment, patients must be For US Sites: 1. Part 1 (including PK expansion cohort): ≥2 and \<18 years of age; 2. Part 2: ≥2 and ≤18 years of age; For EU/UK Sites: 1. Part 1 (including PK expansion cohort): from birth to \<18 years of age; 2. Part 2: from birth to \<18 years of age (except as noted below); * Note: Patients \<2 years of age will only be enrolled in Europe, however, enrollment of patients \<2 years of age will not begin until definitive juvenile animal toxicity data is available. 2. Diagnosis: 1. Part 1 - Patients with any recurrent or refractory solid tumors or lymphoma (not central nervous system \[CNS\]) that have a known or expected dysfunction of VEGFR 1, -2, and -3; FGFR-1, or CSF-1R pathways (based on literature) are eligible. Patients must have had histologic verification of malignancy at original diagnosis or relapse. 2. Part 2 - Recurrent or refractory osteosarcoma (US and EU), Ewing sarcoma (US and EU), RMS (US and EU), or NRSTS (EU only). Patients must have had histologic verification of malignancy at original diagnosis or relapse. 3. Disease status: Patients must have measureable or evaluable disease for part 1 dose escalation; for part 2, patients must have measurable disease by RECIST version 1.1. 4. Therapeutic options: Patient's current disease state must be one for which there is no known curative therapy. 5. Performance level: Karnofsky ≥50 for patients ≥16 and \<18 years of age and Lansky ≥50 for patients \<16 years of age, Eastern Cooperative Oncology Group (ECOG) ≤2 for patients ≥18 years of age. 6. Adequate organ and bone marrow function as defined in the current protocol. 7. Adequate cardiac function as indicated as defined in the current protocol. 8. Patients with known bone marrow metastatic disease will be eligible for the study provided they meet the blood counts in the inclusion criteria as defined in the current protocol. 9. Adequate BP control which is defined as a BP \<95th percentile (≤ grade 1) for age, height, and sex. 10. Informed consent: Provision of signed and dated written informed consent (parent/legal guardian if patient \<18 years of age) and assent (from patients aged \>7 years) prior to any study-specific procedures, sampling, and analyses. 11. Patient must meet all defined Inclusion criteria as defined in the current protocol.
Exclusion criteria
1. Patient must not meet any
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Patients With Clinically Significant Laboratory Abnormalities | From the first dose of study drug (Day 1) up to 30 + 7 days after the last dose of study drug, approximately 19 weeks | Blood and urine samples were collected to determine the clinical chemistry, hematology, and urinalysis laboratory abnormalities. |
| Number of Patients With Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | From the first dose of study drug (Day 1) up to 30 + 7 days after the last dose of study drug, approximately 19 weeks | Standard 12-lead ECGs were performed after the patient rested for 5 to 10 minutes. The ECG parameters included heart rate, PR interval, RR interval, QT interval, QTcF, and QRS interval from the triplicate 12-lead ECG. |
| Number of Patients With Dose-Limiting Toxicities (DLT) | From the first dose of study drug (Day 1) up to Day 35 of Cycle 1 | A DLT was defined as any of following events that were attributable to study treatment (at least possibly related). Adverse events (AE) were graded using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. * Any Grade 3 or greater nonhematological toxicity. * Grade 3 liver enzyme elevation. * Cases of Hy's law. * Any Grade 2 nonhematological toxicity that persisted for \>= 7 days. * Any Grade 4 hypertension. * Grade 3 corrected QT interval prolongation \> 500 millisecond. * Grade 4 thrombocytopenia for \> 7 days. * Grade 3 thrombocytopenia with clinically significant bleeding. * Grade 4 neutropenia that lasted for \> 7 days. * Myelosuppression that caused a delay of \> 7 days in the start of Cycle 2. |
| Number of Patients With Treatment Emergent Adverse Events (TEAEs) by Severity | From the first dose of study drug (Day 1) up to 30 + 7 days after the last dose of study drug, approximately 19 weeks | The AEs were graded using the NCI CTCAE version 5.0. The CTCAE displays Grades 1 through 5 where, Grade 1= mild, Grade 2= moderate, Grade 3= Severe, Grade 4= life-threatening consequences and Grade 5= death. |
| Number of Patients With Clinically Significant Physical Examination Abnormalities | From the first dose of study drug (Day 1) up to 30 + 7 days after the last dose of study drug, approximately 19 weeks | Physical examination included patient height, weight, and general condition, as well as an examination of the head, heart, chest (including the lungs), abdomen, extremities, skin, lymph nodes, nervous system, and additional areas/systems as clinically indicated. |
| Number of Patients With Clinically Significant Vital Signs Abnormalities | From the first dose of study drug (Day 1) up to 30 + 7 days after the last dose of study drug, approximately 19 weeks | Vital signs included systolic blood pressure (BP), diastolic BP, heart rate, height, weight, respiratory rate, and body temperature. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Objective Response Rate (ORR) | RECIST assessments performed at baseline (within 28 days before start of study treatment), Day 1 of Cycle 3 and until confirmed objective disease progression. Up to approximately 19 weeks. | The ORR was defined as the percentage of patients with a best overall response (BOR) of complete response (CR) or partial response (PR) as determined by the investigator using Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1. The BOR was defined as the best response recorded from the start of study treatment until documented RECIST version 1.1 progression, death, or withdrawal of consent. The CR was defined as disappearance of all target lesions. The PR was defined as at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum of diameters. |
| Disease Control Rate (DCR) | RECIST assessments performed at baseline (within 28 days before start of study treatment), Day 1 of Cycle 3 and until confirmed objective disease progression. Up to approximately 19 weeks. | The DCR was defined as the percentage of patients with a BOR of CR, PR, or stable disease as determined by the investigator using RECIST version 1.1. Stable disease was defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum on study. |
| Time to Response (TTR) | RECIST assessments performed at baseline (within 28 days before start of study treatment), Day 1 of Cycle 3 and until confirmed objective disease progression. Up to approximately 19 weeks. | The TTR was defined as the time from the start of study treatment until the date of the first occurrence of PR or CR for responders only. |
| Duration of Response (DoR) | RECIST assessments performed at baseline (within 28 days before start of study treatment), Day 1 of Cycle 3 and until confirmed objective disease progression. Up to approximately 19 weeks. | The DoR was defined as the time from the first occurrence of PR or CR whichever came first, until disease progression or death. |
| Progression-Free Survival (PFS) | RECIST assessments performed at baseline (within 28 days before start of study treatment), Day 1 of Cycle 3 and until confirmed objective disease progression. Up to approximately 19 weeks. | The PFS was defined as the time from the start of study treatment until the first radiographic documentation of objective progression as assessed by the investigator using RECIST version 1.1 or death from any cause. |
| Number of Patients Who Performed Taste and Palatability Survey of Surufatinib | Days 1 and 8 of Cycle 1 | The taste and palatability survey was assessed in patients who had taken surufatinib oral suspension. For pediatric patients, their parents completed the taste and palatability survey. Data for the evaluation of taste of surufatinib oral suspension was summarized on a scale of 1 (very bad) through 5 (very nice). |
Countries
United States
Participant flow
Recruitment details
This Phase 1/2 open-label study was conducted in pediatric, adolescent, and young adult patients with recurrent or refractory solid tumors at 8 study sites.
Pre-assignment details
This study consisted of a screening period (up to 28 days), followed by a treatment phase (Cycle 1: 35 days and subsequent cycles: 21 days) and safety follow-up period (up to 30 days). A total of 13 patients were enrolled in this study. The planned expansion phase for this study was not opened.
Participants by arm
| Arm | Count |
|---|---|
| Dose Level 1 Patients received surufatinib 120 mg/m\^2 oral capsule QD as a single agent for 14 days followed by surufatinib daily in combination with gemcitabine 1000 mg/m\^2/dose IV infusion on Days 15 and 22 of a 35-day Cycle 1 and on Days 1 and 8 of 21-day subsequent cycles until PD, unacceptable toxicity, or death; whichever came first. | 10 |
| Dose Level 2 Patients received surufatinib 160 mg/m\^2 oral capsule QD as a single agent for 14 days followed by surufatinib daily in combination with gemcitabine 1000 mg/m\^2/dose IV infusion on Days 15 and 22 of a 35-day Cycle 1 and on Days 1 and 8 of 21-day subsequent cycles until PD, unacceptable toxicity, or death; whichever came first. | 3 |
| Total | 13 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Death | 6 | 2 |
| Overall Study | Other | 4 | 1 |
Baseline characteristics
| Characteristic | Dose Level 1 | Dose Level 2 | Total |
|---|---|---|---|
| Age, Continuous | 11.8 years STANDARD_DEVIATION 5.55 | 13.7 years STANDARD_DEVIATION 2.89 | 12.2 years STANDARD_DEVIATION 5.02 |
| Race/Ethnicity, Customized Black or African American | 2 Participants | 0 Participants | 2 Participants |
| Race/Ethnicity, Customized Hispanic or Latino | 1 Participants | 2 Participants | 3 Participants |
| Race/Ethnicity, Customized Not Hispanic or Latino | 9 Participants | 1 Participants | 10 Participants |
| Race/Ethnicity, Customized Not Reported | 1 Participants | 1 Participants | 2 Participants |
| Race/Ethnicity, Customized White | 7 Participants | 2 Participants | 9 Participants |
| Sex: Female, Male Female | 4 Participants | 1 Participants | 5 Participants |
| Sex: Female, Male Male | 6 Participants | 2 Participants | 8 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 6 / 10 | 2 / 3 |
| other Total, other adverse events | 10 / 10 | 3 / 3 |
| serious Total, serious adverse events | 7 / 10 | 1 / 3 |
Outcome results
Primary
Number of Patients With Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities
Standard 12-lead ECGs were performed after the patient rested for 5 to 10 minutes. The ECG parameters included heart rate, PR interval, RR interval, QT interval, QTcF, and QRS interval from the triplicate 12-lead ECG.
Time frame: From the first dose of study drug (Day 1) up to 30 + 7 days after the last dose of study drug, approximately 19 weeks
Population: The Safety analysis set included all patients who received at least 1 dose of surufatinib or gemcitabine.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Dose Level 1 | Number of Patients With Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | 1 Participants |
| Dose Level 2 | Number of Patients With Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | 0 Participants |
Primary
Number of Patients With Clinically Significant Laboratory Abnormalities
Blood and urine samples were collected to determine the clinical chemistry, hematology, and urinalysis laboratory abnormalities.
Time frame: From the first dose of study drug (Day 1) up to 30 + 7 days after the last dose of study drug, approximately 19 weeks
Population: The Safety analysis set included all patients who received at least 1 dose of surufatinib or gemcitabine.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Dose Level 1 | Number of Patients With Clinically Significant Laboratory Abnormalities | 0 Participants |
| Dose Level 2 | Number of Patients With Clinically Significant Laboratory Abnormalities | 1 Participants |
Primary
Number of Patients With Clinically Significant Physical Examination Abnormalities
Physical examination included patient height, weight, and general condition, as well as an examination of the head, heart, chest (including the lungs), abdomen, extremities, skin, lymph nodes, nervous system, and additional areas/systems as clinically indicated.
Time frame: From the first dose of study drug (Day 1) up to 30 + 7 days after the last dose of study drug, approximately 19 weeks
Population: The Safety analysis set included all patients who received at least 1 dose of surufatinib or gemcitabine.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Dose Level 1 | Number of Patients With Clinically Significant Physical Examination Abnormalities | 6 Participants |
| Dose Level 2 | Number of Patients With Clinically Significant Physical Examination Abnormalities | 1 Participants |
Primary
Number of Patients With Clinically Significant Vital Signs Abnormalities
Vital signs included systolic blood pressure (BP), diastolic BP, heart rate, height, weight, respiratory rate, and body temperature.
Time frame: From the first dose of study drug (Day 1) up to 30 + 7 days after the last dose of study drug, approximately 19 weeks
Population: The Safety analysis set included all patients who received at least 1 dose of surufatinib or gemcitabine.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Dose Level 1 | Number of Patients With Clinically Significant Vital Signs Abnormalities | 0 Participants |
| Dose Level 2 | Number of Patients With Clinically Significant Vital Signs Abnormalities | 0 Participants |
Primary
Number of Patients With Dose-Limiting Toxicities (DLT)
A DLT was defined as any of following events that were attributable to study treatment (at least possibly related). Adverse events (AE) were graded using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. * Any Grade 3 or greater nonhematological toxicity. * Grade 3 liver enzyme elevation. * Cases of Hy's law. * Any Grade 2 nonhematological toxicity that persisted for \>= 7 days. * Any Grade 4 hypertension. * Grade 3 corrected QT interval prolongation \> 500 millisecond. * Grade 4 thrombocytopenia for \> 7 days. * Grade 3 thrombocytopenia with clinically significant bleeding. * Grade 4 neutropenia that lasted for \> 7 days. * Myelosuppression that caused a delay of \> 7 days in the start of Cycle 2.
Time frame: From the first dose of study drug (Day 1) up to Day 35 of Cycle 1
Population: The DLT evaluable set included all patients enrolled in dose escalation phase of study who received at least 80% of surufatinib dose and both doses of gemcitabine during DLT evaluation period or who discontinued treatment due to a DLT.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Dose Level 1 | Number of Patients With Dose-Limiting Toxicities (DLT) | 0 Participants |
| Dose Level 2 | Number of Patients With Dose-Limiting Toxicities (DLT) | 2 Participants |
Primary
Number of Patients With Treatment Emergent Adverse Events (TEAEs) by Severity
The AEs were graded using the NCI CTCAE version 5.0. The CTCAE displays Grades 1 through 5 where, Grade 1= mild, Grade 2= moderate, Grade 3= Severe, Grade 4= life-threatening consequences and Grade 5= death.
Time frame: From the first dose of study drug (Day 1) up to 30 + 7 days after the last dose of study drug, approximately 19 weeks
Population: The Safety analysis set included all patients who received at least 1 dose of surufatinib or gemcitabine.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Dose Level 1 | Number of Patients With Treatment Emergent Adverse Events (TEAEs) by Severity | Grade 2 | 1 Participants |
| Dose Level 1 | Number of Patients With Treatment Emergent Adverse Events (TEAEs) by Severity | Grade 4 | 3 Participants |
| Dose Level 1 | Number of Patients With Treatment Emergent Adverse Events (TEAEs) by Severity | Grade 1 | 0 Participants |
| Dose Level 1 | Number of Patients With Treatment Emergent Adverse Events (TEAEs) by Severity | Grade 5 | 2 Participants |
| Dose Level 1 | Number of Patients With Treatment Emergent Adverse Events (TEAEs) by Severity | Grade 3 | 4 Participants |
| Dose Level 2 | Number of Patients With Treatment Emergent Adverse Events (TEAEs) by Severity | Grade 5 | 0 Participants |
| Dose Level 2 | Number of Patients With Treatment Emergent Adverse Events (TEAEs) by Severity | Grade 1 | 0 Participants |
| Dose Level 2 | Number of Patients With Treatment Emergent Adverse Events (TEAEs) by Severity | Grade 2 | 1 Participants |
| Dose Level 2 | Number of Patients With Treatment Emergent Adverse Events (TEAEs) by Severity | Grade 3 | 2 Participants |
| Dose Level 2 | Number of Patients With Treatment Emergent Adverse Events (TEAEs) by Severity | Grade 4 | 0 Participants |
Secondary
Disease Control Rate (DCR)
The DCR was defined as the percentage of patients with a BOR of CR, PR, or stable disease as determined by the investigator using RECIST version 1.1. Stable disease was defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum on study.
Time frame: RECIST assessments performed at baseline (within 28 days before start of study treatment), Day 1 of Cycle 3 and until confirmed objective disease progression. Up to approximately 19 weeks.
Population: The Safety analysis set included all patients who received at least 1 dose of surufatinib or gemcitabine.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Dose Level 1 | Disease Control Rate (DCR) | 20.0 percentage of patients |
| Dose Level 2 | Disease Control Rate (DCR) | 0 percentage of patients |
Secondary
Duration of Response (DoR)
The DoR was defined as the time from the first occurrence of PR or CR whichever came first, until disease progression or death.
Time frame: RECIST assessments performed at baseline (within 28 days before start of study treatment), Day 1 of Cycle 3 and until confirmed objective disease progression. Up to approximately 19 weeks.
Population: The Safety analysis set included all patients who received at least 1 dose of surufatinib or gemcitabine. No responders in this study.
Secondary
Number of Patients Who Performed Taste and Palatability Survey of Surufatinib
The taste and palatability survey was assessed in patients who had taken surufatinib oral suspension. For pediatric patients, their parents completed the taste and palatability survey. Data for the evaluation of taste of surufatinib oral suspension was summarized on a scale of 1 (very bad) through 5 (very nice).
Time frame: Days 1 and 8 of Cycle 1
Population: The Safety analysis set included all patients who received at least 1 dose of surufatinib or gemcitabine. Only patients who performed the survey are reported.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Dose Level 1 | Number of Patients Who Performed Taste and Palatability Survey of Surufatinib | Cycle 1 Day 1: Very Nice | 0 Participants |
| Dose Level 1 | Number of Patients Who Performed Taste and Palatability Survey of Surufatinib | Cycle 1 Day 1: Nice | 1 Participants |
| Dose Level 1 | Number of Patients Who Performed Taste and Palatability Survey of Surufatinib | Cycle 1 Day 1: Not Nice, not Bad | 0 Participants |
| Dose Level 1 | Number of Patients Who Performed Taste and Palatability Survey of Surufatinib | Cycle 1 Day 1: Bad | 1 Participants |
| Dose Level 1 | Number of Patients Who Performed Taste and Palatability Survey of Surufatinib | Cycle 1 Day 1: Very Bad | 0 Participants |
| Dose Level 1 | Number of Patients Who Performed Taste and Palatability Survey of Surufatinib | Cycle 1 Day 8: Very Nice | 0 Participants |
| Dose Level 1 | Number of Patients Who Performed Taste and Palatability Survey of Surufatinib | Cycle 1 Day 8: Nice | 1 Participants |
| Dose Level 1 | Number of Patients Who Performed Taste and Palatability Survey of Surufatinib | Cycle 1 Day 8: Not Nice, not Bad | 0 Participants |
| Dose Level 1 | Number of Patients Who Performed Taste and Palatability Survey of Surufatinib | Cycle 1 Day 8: Bad | 1 Participants |
| Dose Level 1 | Number of Patients Who Performed Taste and Palatability Survey of Surufatinib | Cycle 1 Day 8: Very Bad | 0 Participants |
Secondary
Objective Response Rate (ORR)
The ORR was defined as the percentage of patients with a best overall response (BOR) of complete response (CR) or partial response (PR) as determined by the investigator using Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1. The BOR was defined as the best response recorded from the start of study treatment until documented RECIST version 1.1 progression, death, or withdrawal of consent. The CR was defined as disappearance of all target lesions. The PR was defined as at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum of diameters.
Time frame: RECIST assessments performed at baseline (within 28 days before start of study treatment), Day 1 of Cycle 3 and until confirmed objective disease progression. Up to approximately 19 weeks.
Population: The Safety analysis set included all patients who received at least 1 dose of surufatinib or gemcitabine.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Dose Level 1 | Objective Response Rate (ORR) | 0 percentage of patients |
| Dose Level 2 | Objective Response Rate (ORR) | 0 percentage of patients |
Secondary
Progression-Free Survival (PFS)
The PFS was defined as the time from the start of study treatment until the first radiographic documentation of objective progression as assessed by the investigator using RECIST version 1.1 or death from any cause.
Time frame: RECIST assessments performed at baseline (within 28 days before start of study treatment), Day 1 of Cycle 3 and until confirmed objective disease progression. Up to approximately 19 weeks.
Population: The Safety analysis set included all patients who received at least 1 dose of surufatinib or gemcitabine.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Dose Level 1 | Progression-Free Survival (PFS) | 1.8 months |
| Dose Level 2 | Progression-Free Survival (PFS) | 1.5 months |
Secondary
Time to Response (TTR)
The TTR was defined as the time from the start of study treatment until the date of the first occurrence of PR or CR for responders only.
Time frame: RECIST assessments performed at baseline (within 28 days before start of study treatment), Day 1 of Cycle 3 and until confirmed objective disease progression. Up to approximately 19 weeks.
Population: The Safety analysis set included all patients who received at least 1 dose of surufatinib or gemcitabine. No responders in this study.