Trial Outcomes & Findings for Pazopanib Hydrochloride in Treating Patients With Advanced or Progressive Malignant Pheochromocytoma or Paraganglioma (NCT NCT01340794)
NCT ID: NCT01340794
Last Updated: 2017-09-21
Results Overview
Response and progression are evaluated in this study using the international criteria proposed by the Response Evaluation Criteria in Solid Tumors (RECIST) guidelines (version 1.1) Ninety-five percent confidence intervals for the true response proportion was calculated using the exact binomial test. Complete Response (CR): All of the following must be true: 1. Disappearance of all target and non-target lesions. 2. Each lymph node must have reduction in short axis to \<1.0 cm. Partial Response (PR): At least a 30% decrease in PBSD (sum of the longest diameter for all target lesions plus the sum of the short axis of all the target lymph nodes at current evaluation) taking baseline measures as reference. Overall Response (OR) was calculated by summing the number of patients with a CR or PR.
TERMINATED
PHASE2
7 participants
Up to 5 years
2017-09-21
Participant Flow
This study opened on 10/3/2011 and enrolled 2 patients prior to temporarily closing on 2/28/2012 due to safety concerns. After review of AEs, an addendum was implemented which instituted changes to treatment administration, namely, 2 - 14 day run-in periods where patients received pazopanib PO days 1-7 and then were assessed for safety.
A total of seven patients were recruited to this study. One of the 5 patients registered to the study post-addendum cancelled prior to initiating treatment.
Participant milestones
| Measure |
Treatment: Pazopanib With no Run-in
Patients receive 800 mg pazopanib hydrochloride PO QD on days 1-28 of a 28 day cycle.
|
Treatment: Pazopanib Run-in
Cycle 1:
Patients receive 400 mg pazopanib hydrochloride PO QD on days 1-7 of a 14 day cycle.
Cycle 2:
Patients receive 800 mg pazopanib hydrochloride PO QD on days 1-7 of a 14 day cycle.
Cycle 3 and beyond:
Patients receive 800 mg pazopanib hydrochloride PO QD on days 1-28 of a 28 day cycle.
|
|---|---|---|
|
Overall Study
STARTED
|
2
|
5
|
|
Overall Study
COMPLETED
|
2
|
4
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
Reasons for withdrawal
| Measure |
Treatment: Pazopanib With no Run-in
Patients receive 800 mg pazopanib hydrochloride PO QD on days 1-28 of a 28 day cycle.
|
Treatment: Pazopanib Run-in
Cycle 1:
Patients receive 400 mg pazopanib hydrochloride PO QD on days 1-7 of a 14 day cycle.
Cycle 2:
Patients receive 800 mg pazopanib hydrochloride PO QD on days 1-7 of a 14 day cycle.
Cycle 3 and beyond:
Patients receive 800 mg pazopanib hydrochloride PO QD on days 1-28 of a 28 day cycle.
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
0
|
1
|
Baseline Characteristics
Pazopanib Hydrochloride in Treating Patients With Advanced or Progressive Malignant Pheochromocytoma or Paraganglioma
Baseline characteristics by cohort
| Measure |
Treatment: Pazopanib Without Run-in
n=2 Participants
Patients receive 800 mg pazopanib hydrochloride PO QD on days 1-28 of a 28 day cycle.
|
Treatment: Pazopanib With Run-in
n=4 Participants
Cycle 1:
Patients receive 400 mg pazopanib hydrochloride PO QD on days 1-7 of a 14 day cycle.
Cycle 2:
Patients receive 800 mg pazopanib hydrochloride PO QD on days 1-7 of a 14 day cycle.
Cycle 3 and beyond:
Patients receive 800 mg pazopanib hydrochloride PO QD on days 1-28 of a 28 day cycle.
|
Total
n=6 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
42 years
n=5 Participants
|
64.5 years
n=7 Participants
|
57 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
2 participants
n=5 Participants
|
4 participants
n=7 Participants
|
6 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 5 yearsPopulation: One of the two patients that initiated treatment with no run-in was found to be ineligible for this endpoint, leaving one patient evaluable for this endpoint in this treatment group. For patient confidentiality, results are not entered for endpoints based on 1 patient measures. All 4 patients that initiated treatment with a run-in were evaluable
Response and progression are evaluated in this study using the international criteria proposed by the Response Evaluation Criteria in Solid Tumors (RECIST) guidelines (version 1.1) Ninety-five percent confidence intervals for the true response proportion was calculated using the exact binomial test. Complete Response (CR): All of the following must be true: 1. Disappearance of all target and non-target lesions. 2. Each lymph node must have reduction in short axis to \<1.0 cm. Partial Response (PR): At least a 30% decrease in PBSD (sum of the longest diameter for all target lesions plus the sum of the short axis of all the target lymph nodes at current evaluation) taking baseline measures as reference. Overall Response (OR) was calculated by summing the number of patients with a CR or PR.
Outcome measures
| Measure |
Treatment: Pazopanib With no Run-in
Patients receive 800 mg pazopanib hydrochloride PO QD on days 1-28 of a 28 day cycle.
|
Treatment: Pazopanib Run-in
n=4 Participants
Cycle 1:
Patients receive 400 mg pazopanib hydrochloride PO QD on days 1-7 of a 14 day cycle.
Cycle 2:
Patients receive 800 mg pazopanib hydrochloride PO QD on days 1-7 of a 14 day cycle.
Cycle 3 and beyond:
Patients receive 800 mg pazopanib hydrochloride PO QD on days 1-28 of a 28 day cycle.
|
|---|---|---|
|
Response Rate (RR) (Complete Response [CR] or Partial Response [PR]) Using RECIST Version 1.1
|
—
|
0 percentage of participants
Interval 0.0 to 60.0
|
SECONDARY outcome
Timeframe: Up to 5 yearsPopulation: One of the two patients that initiated treatment with no run-in was found to be ineligible for this endpoint, leaving one patient evaluable for this endpoint in this treatment group. For patient confidentiality, results are not entered for endpoints based on 1 patient measures. None of the 4 patients that initiated treatment with a run-in responded
Defined for all patients whose tumor met the criteria of CR or PR (using the RECIST criteria) as the date at which the patient's objective status is first noted to be either a CR or PR to the date progression is documented.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: The time from registration to death due to any cause, assessed up to 5 yearsPopulation: One of the two patients that initiated treatment with no run-in was found to be ineligible for this endpoint, leaving one patient evaluable for this endpoint in this treatment group. For patient confidentiality, results are not entered for endpoints based on 1 patient measures. All 4 patients that initiated treatment with a run-in were evaluable
Overall survival time is defined as the time from registration to death due to any cause and will be estimated using the Kaplan-Meier method.
Outcome measures
| Measure |
Treatment: Pazopanib With no Run-in
Patients receive 800 mg pazopanib hydrochloride PO QD on days 1-28 of a 28 day cycle.
|
Treatment: Pazopanib Run-in
n=4 Participants
Cycle 1:
Patients receive 400 mg pazopanib hydrochloride PO QD on days 1-7 of a 14 day cycle.
Cycle 2:
Patients receive 800 mg pazopanib hydrochloride PO QD on days 1-7 of a 14 day cycle.
Cycle 3 and beyond:
Patients receive 800 mg pazopanib hydrochloride PO QD on days 1-28 of a 28 day cycle.
|
|---|---|---|
|
Overall Survival Time
|
—
|
14.8 months
Interval 7.6 to 26.3
|
SECONDARY outcome
Timeframe: The time from registration to documentation of disease progression or death, whichever occurs first, assessed up to 5 yearsPopulation: One of the two patients that initiated treatment with no run-in was found to be ineligible for this endpoint, leaving one patient evaluable for this endpoint in this treatment group. For patient confidentiality, results are not entered for endpoints based on 1 patient measures. All 4 patients that initiated treatment with a run-in were evaluable
Progression-free survival is defined as the time from registration to documentation of disease progression. If a patient dies without a documentation of disease progression, the patient will be considered to have had tumor progression at the time of their death unless there is sufficient documented evidence to conclude no progression occurred prior to death. Progression-free survival time will be estimated using the Kaplan-Meier method.
Outcome measures
| Measure |
Treatment: Pazopanib With no Run-in
Patients receive 800 mg pazopanib hydrochloride PO QD on days 1-28 of a 28 day cycle.
|
Treatment: Pazopanib Run-in
n=4 Participants
Cycle 1:
Patients receive 400 mg pazopanib hydrochloride PO QD on days 1-7 of a 14 day cycle.
Cycle 2:
Patients receive 800 mg pazopanib hydrochloride PO QD on days 1-7 of a 14 day cycle.
Cycle 3 and beyond:
Patients receive 800 mg pazopanib hydrochloride PO QD on days 1-28 of a 28 day cycle.
|
|---|---|---|
|
Progression-free Survival Time
|
—
|
6.5 months
Interval 2.1 to 26.3
|
SECONDARY outcome
Timeframe: Up to 5 years from registrationPopulation: One of the two patients that initiated treatment with no run-in was found to be ineligible for this endpoint, leaving one patient evaluable for this endpoint in this treatment group. For patient confidentiality, results are not entered for endpoints based on 1 patient measures. All 4 patients that initiated treatment with a run-in were evaluable.
The time from the date of registration to the date at which the patient is removed from treatment due to progression, toxicity, or refusal, assessed up to 5 years.
Outcome measures
| Measure |
Treatment: Pazopanib With no Run-in
Patients receive 800 mg pazopanib hydrochloride PO QD on days 1-28 of a 28 day cycle.
|
Treatment: Pazopanib Run-in
n=4 Participants
Cycle 1:
Patients receive 400 mg pazopanib hydrochloride PO QD on days 1-7 of a 14 day cycle.
Cycle 2:
Patients receive 800 mg pazopanib hydrochloride PO QD on days 1-7 of a 14 day cycle.
Cycle 3 and beyond:
Patients receive 800 mg pazopanib hydrochloride PO QD on days 1-28 of a 28 day cycle.
|
|---|---|---|
|
Time to Treatment Failure
|
—
|
2.4 months
Interval 0.8 to 5.7
|
Adverse Events
Treatment: Pazopanib Without Run-in
Treatment: Pazopanib With Run-in
Serious adverse events
| Measure |
Treatment: Pazopanib Without Run-in
n=2 participants at risk
Patients receive 800 mg pazopanib hydrochloride PO QD on days 1-28 of a 28 day cycle.
|
Treatment: Pazopanib With Run-in
n=4 participants at risk
Patients receive 800 mg pazopanib hydrochloride PO QD on days 1-28 of a 28 day cycle.
|
|---|---|---|
|
Blood and lymphatic system disorders
Anemia
|
50.0%
1/2 • Number of events 1
|
0.00%
0/4
|
|
Cardiac disorders
Myocardial infarction
|
50.0%
1/2 • Number of events 1
|
0.00%
0/4
|
|
Gastrointestinal disorders
Nausea
|
50.0%
1/2 • Number of events 1
|
0.00%
0/4
|
|
General disorders
Fatigue
|
50.0%
1/2 • Number of events 1
|
0.00%
0/4
|
|
Investigations
Aspartate aminotransferase increased
|
50.0%
1/2 • Number of events 1
|
0.00%
0/4
|
|
Investigations
Blood bilirubin increased
|
50.0%
1/2 • Number of events 1
|
0.00%
0/4
|
|
Investigations
CPK increased
|
50.0%
1/2 • Number of events 1
|
0.00%
0/4
|
|
Investigations
Cardiac troponin I increased
|
50.0%
1/2 • Number of events 1
|
0.00%
0/4
|
|
Investigations
GGT increased
|
50.0%
1/2 • Number of events 1
|
0.00%
0/4
|
|
Investigations
Platelet count decreased
|
50.0%
1/2 • Number of events 1
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
50.0%
1/2 • Number of events 1
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
Hyponatremia
|
50.0%
1/2 • Number of events 1
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
Metabolism and nutrition disorders - Other, specify
|
50.0%
1/2 • Number of events 1
|
0.00%
0/4
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
50.0%
1/2 • Number of events 1
|
0.00%
0/4
|
|
Nervous system disorders
Intracranial hemorrhage
|
50.0%
1/2 • Number of events 1
|
0.00%
0/4
|
|
Nervous system disorders
Syncope
|
50.0%
1/2 • Number of events 1
|
0.00%
0/4
|
|
Psychiatric disorders
Confusion
|
50.0%
1/2 • Number of events 1
|
0.00%
0/4
|
|
Renal and urinary disorders
Hematuria
|
50.0%
1/2 • Number of events 1
|
0.00%
0/4
|
|
Vascular disorders
Hypertension
|
50.0%
1/2 • Number of events 1
|
25.0%
1/4 • Number of events 1
|
|
Vascular disorders
Hypotension
|
50.0%
1/2 • Number of events 1
|
0.00%
0/4
|
Other adverse events
| Measure |
Treatment: Pazopanib Without Run-in
n=2 participants at risk
Patients receive 800 mg pazopanib hydrochloride PO QD on days 1-28 of a 28 day cycle.
|
Treatment: Pazopanib With Run-in
n=4 participants at risk
Patients receive 800 mg pazopanib hydrochloride PO QD on days 1-28 of a 28 day cycle.
|
|---|---|---|
|
Blood and lymphatic system disorders
Anemia
|
50.0%
1/2 • Number of events 3
|
0.00%
0/4
|
|
Endocrine disorders
Hypothyroidism
|
50.0%
1/2 • Number of events 27
|
25.0%
1/4 • Number of events 1
|
|
Gastrointestinal disorders
Abdominal pain
|
50.0%
1/2 • Number of events 1
|
0.00%
0/4
|
|
Gastrointestinal disorders
Anal pain
|
50.0%
1/2 • Number of events 1
|
0.00%
0/4
|
|
Gastrointestinal disorders
Diarrhea
|
50.0%
1/2 • Number of events 28
|
50.0%
2/4 • Number of events 2
|
|
Gastrointestinal disorders
Nausea
|
50.0%
1/2 • Number of events 14
|
50.0%
2/4 • Number of events 7
|
|
Gastrointestinal disorders
Vomiting
|
50.0%
1/2 • Number of events 12
|
25.0%
1/4 • Number of events 1
|
|
General disorders
Fatigue
|
100.0%
2/2 • Number of events 32
|
100.0%
4/4 • Number of events 15
|
|
Investigations
Alanine aminotransferase increased
|
50.0%
1/2 • Number of events 1
|
25.0%
1/4 • Number of events 1
|
|
Investigations
Alkaline phosphatase increased
|
50.0%
1/2 • Number of events 19
|
25.0%
1/4 • Number of events 1
|
|
Investigations
Aspartate aminotransferase increased
|
100.0%
2/2 • Number of events 31
|
50.0%
2/4 • Number of events 3
|
|
Investigations
Blood bilirubin increased
|
50.0%
1/2 • Number of events 1
|
0.00%
0/4
|
|
Investigations
Electrocardiogram QT corrected interval prolonged
|
0.00%
0/2
|
25.0%
1/4 • Number of events 1
|
|
Investigations
Hemoglobin increased
|
50.0%
1/2 • Number of events 1
|
0.00%
0/4
|
|
Investigations
Investigations - Other, specify
|
50.0%
1/2 • Number of events 1
|
0.00%
0/4
|
|
Investigations
Lymphocyte count decreased
|
50.0%
1/2 • Number of events 2
|
0.00%
0/4
|
|
Investigations
Neutrophil count decreased
|
50.0%
1/2 • Number of events 17
|
0.00%
0/4
|
|
Investigations
Platelet count decreased
|
100.0%
2/2 • Number of events 28
|
0.00%
0/4
|
|
Investigations
White blood cell decreased
|
100.0%
2/2 • Number of events 25
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
Anorexia
|
0.00%
0/2
|
50.0%
2/4 • Number of events 4
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/2
|
25.0%
1/4 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Flank pain
|
0.00%
0/2
|
25.0%
1/4 • Number of events 1
|
|
Nervous system disorders
Dysgeusia
|
0.00%
0/2
|
25.0%
1/4 • Number of events 7
|
|
Nervous system disorders
Headache
|
0.00%
0/2
|
25.0%
1/4 • Number of events 1
|
|
Psychiatric disorders
Anxiety
|
50.0%
1/2 • Number of events 1
|
0.00%
0/4
|
|
Psychiatric disorders
Depression
|
50.0%
1/2 • Number of events 1
|
25.0%
1/4 • Number of events 2
|
|
Renal and urinary disorders
Hematuria
|
50.0%
1/2 • Number of events 1
|
0.00%
0/4
|
|
Renal and urinary disorders
Proteinuria
|
50.0%
1/2 • Number of events 1
|
25.0%
1/4 • Number of events 6
|
|
Skin and subcutaneous tissue disorders
Skin hypopigmentation
|
100.0%
2/2 • Number of events 33
|
25.0%
1/4 • Number of events 4
|
|
Vascular disorders
Hypertension
|
100.0%
2/2 • Number of events 32
|
100.0%
4/4 • Number of events 15
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60