Trial Outcomes & Findings for A Study to Compare Vincristine to Sirolimus for Treatment of High Risk Vascular Tumors (NCT NCT02110069)
NCT ID: NCT02110069
Last Updated: 2023-09-21
Results Overview
Hematologic parameters are defined as a platelet count greater than 100,000/uL (or a 2 times increase in platelet count compared to baseline) and a fibrinogen level greater than 150mg/dl.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
4 participants
Primary outcome timeframe
2 months
Results posted on
2023-09-21
Participant Flow
Participant milestones
| Measure |
Vincristine
Induction phase: participants assigned to vincristine will receive vincristine weekly at a dose of 0.05mg/kg/dose IV (for participants less than 10kg) or a dose of 1.5 mg/m2/dose (for participants greater than 10kg) for 2 months.
Maintenance: if participants continue to receive vincristine weekly for 2 months, every 2 weeks for 5 months and every 3 weeks for 5 months.
Vincristine: Vincristine dose dependent upon weight. Weekly for 2 months (Induction); Weekly 2 months; every 2 weeks for next 5 months; every 3 weeks for 5 months (Maintenance)
|
Sirolimus
Sirolimus will be administered at a dose of 0.8mg/m2/dose twice a day on a continuous dosing schedule throughout the trial for participants randomized to Sirolimus or for participants who fail vincristine may cross-over to the sirolimus arm.
Sirolimus trough levels will be maintained between 10-15 ng/ml.
Sirolimus: Continuous dosing to maintain trough level of 10-15ng/ml.
|
|---|---|---|
|
Overall Study
STARTED
|
2
|
2
|
|
Overall Study
COMPLETED
|
0
|
2
|
|
Overall Study
NOT COMPLETED
|
2
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
There was no analysis as study was closed early
Baseline characteristics by cohort
| Measure |
Vincristine
n=2 Participants
Induction phase: participants assigned to vincristine will receive vincristine weekly at a dose of 0.05mg/kg/dose IV (for participants less than 10kg) or a dose of 1.5 mg/m2/dose (for participants greater than 10kg) for 2 months.
Maintenance: if participants continue to receive vincristine weekly for 2 months, every 2 weeks for 5 months and every 3 weeks for 5 months.
Vincristine: Vincristine dose dependent upon weight. Weekly for 2 months (Induction); Weekly 2 months; every 2 weeks for next 5 months; every 3 weeks for 5 months (Maintenance)
|
Sirolimus
n=2 Participants
Sirolimus will be administered at a dose of 0.8mg/m2/dose twice a day on a continuous dosing schedule throughout the trial for participants randomized to Sirolimus or for participants who fail vincristine may cross-over to the sirolimus arm.
Sirolimus trough levels will be maintained between 10-15 ng/ml.
Sirolimus: Continuous dosing to maintain trough level of 10-15ng/ml.
|
Total
n=4 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
2 Participants
n=2 Participants
|
2 Participants
n=2 Participants
|
4 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=2 Participants
|
0 Participants
n=2 Participants
|
0 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=2 Participants
|
0 Participants
n=2 Participants
|
0 Participants
n=4 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=2 Participants
|
0 Participants
n=2 Participants
|
0 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=2 Participants
|
2 Participants
n=2 Participants
|
4 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=2 Participants
|
0 Participants
n=2 Participants
|
0 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
2 Participants
n=2 Participants
|
2 Participants
n=2 Participants
|
4 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=2 Participants
|
0 Participants
n=2 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=2 Participants
|
0 Participants
n=2 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=2 Participants
|
1 Participants
n=2 Participants
|
2 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=2 Participants
|
0 Participants
n=2 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=2 Participants
|
0 Participants
n=2 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=2 Participants
|
1 Participants
n=2 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=2 Participants
|
0 Participants
n=2 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=2 Participants
|
0 Participants
n=2 Participants
|
0 Participants
n=4 Participants
|
|
Region of Enrollment
United States
|
2 participants
n=2 Participants
|
2 participants
n=2 Participants
|
4 participants
n=4 Participants
|
|
Number of participants with platelet count & fibrinogen levels
|
2 participants
n=2 Participants
|
2 participants
n=2 Participants
|
4 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: 2 monthsPopulation: no analysis was performed due to study being closed for low enrollment
Hematologic parameters are defined as a platelet count greater than 100,000/uL (or a 2 times increase in platelet count compared to baseline) and a fibrinogen level greater than 150mg/dl.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: 2 months; 12 monthsPopulation: study was closed early due to slow enrollment
Serious and non-serious adverse events will be recorded for all participants and graded using CTCAE v4.0 (Common Toxicity Criteria for Adverse Effects). Adverse event rates will be calculated for both sirolimus and vincristine.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 6 months; 12 monthsPopulation: no analysis was performed
Disease evaluation will be measured using a combination of quality of life assessments, clinical parameters, and radiologic images.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 6 months; 12 monthsPopulation: no data analyzed as study was closed early
Serious adverse events and adverse events will be graded according to CTCAE v4.0. Adverse event rates will be calculated for both sirolimus and vincristine.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, 2 months, 6 months, and 12 monthsPopulation: no analysis performed as study was closed early
The following KHE biomarkers will be evaluated vascular endothelial growth factor A, C, and D (VEGF-A, C, D\_, IL-8 (interleukin), Pleiotrophin, IGF-1 (insulin-like growth factor), endothelin-1, thrombospondin and angiopoietin 1 and 2.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: BaselinePopulation: study closed early due to slow enrollment
Single nucleotide polymorphism array analysis to obtain genetic information on variants in drug metabolism enzymes that affect sirolimus and vincristine metabolism.
Outcome measures
Outcome data not reported
Adverse Events
Vincristine
Serious events: 1 serious events
Other events: 1 other events
Deaths: 0 deaths
Sirolimus
Serious events: 1 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Vincristine
n=2 participants at risk
Induction phase: participants assigned to vincristine will receive vincristine weekly at a dose of 0.05mg/kg/dose IV (for participants less than 10kg) or a dose of 1.5 mg/m2/dose (for participants greater than 10kg) for 2 months.
Maintenance: if participants continue to receive vincristine weekly for 2 months, every 2 weeks for 5 months and every 3 weeks for 5 months.
Vincristine: Vincristine dose dependent upon weight. Weekly for 2 months (Induction); Weekly 2 months; every 2 weeks for next 5 months; every 3 weeks for 5 months (Maintenance)
|
Sirolimus
n=2 participants at risk
Sirolimus will be administered at a dose of 0.8mg/m2/dose twice a day on a continuous dosing schedule throughout the trial for participants randomized to Sirolimus or for participants who fail vincristine may cross-over to the sirolimus arm.
Sirolimus trough levels will be maintained between 10-15 ng/ml.
Sirolimus: Continuous dosing to maintain trough level of 10-15ng/ml.
|
|---|---|---|
|
Infections and infestations
Lung infection
|
50.0%
1/2 • Number of events 1 • 14 months for 2 subjects 12 months for 1 subject 6 months for 1 subject
|
0.00%
0/2 • 14 months for 2 subjects 12 months for 1 subject 6 months for 1 subject
|
|
Infections and infestations
Sepsis
|
0.00%
0/2 • 14 months for 2 subjects 12 months for 1 subject 6 months for 1 subject
|
50.0%
1/2 • Number of events 1 • 14 months for 2 subjects 12 months for 1 subject 6 months for 1 subject
|
Other adverse events
| Measure |
Vincristine
n=2 participants at risk
Induction phase: participants assigned to vincristine will receive vincristine weekly at a dose of 0.05mg/kg/dose IV (for participants less than 10kg) or a dose of 1.5 mg/m2/dose (for participants greater than 10kg) for 2 months.
Maintenance: if participants continue to receive vincristine weekly for 2 months, every 2 weeks for 5 months and every 3 weeks for 5 months.
Vincristine: Vincristine dose dependent upon weight. Weekly for 2 months (Induction); Weekly 2 months; every 2 weeks for next 5 months; every 3 weeks for 5 months (Maintenance)
|
Sirolimus
n=2 participants at risk
Sirolimus will be administered at a dose of 0.8mg/m2/dose twice a day on a continuous dosing schedule throughout the trial for participants randomized to Sirolimus or for participants who fail vincristine may cross-over to the sirolimus arm.
Sirolimus trough levels will be maintained between 10-15 ng/ml.
Sirolimus: Continuous dosing to maintain trough level of 10-15ng/ml.
|
|---|---|---|
|
Infections and infestations
Fever
|
50.0%
1/2 • Number of events 1 • 14 months for 2 subjects 12 months for 1 subject 6 months for 1 subject
|
50.0%
1/2 • Number of events 1 • 14 months for 2 subjects 12 months for 1 subject 6 months for 1 subject
|
|
Blood and lymphatic system disorders
anemia
|
0.00%
0/2 • 14 months for 2 subjects 12 months for 1 subject 6 months for 1 subject
|
100.0%
2/2 • Number of events 2 • 14 months for 2 subjects 12 months for 1 subject 6 months for 1 subject
|
|
Blood and lymphatic system disorders
Neutrophil count decreased
|
0.00%
0/2 • 14 months for 2 subjects 12 months for 1 subject 6 months for 1 subject
|
100.0%
2/2 • Number of events 2 • 14 months for 2 subjects 12 months for 1 subject 6 months for 1 subject
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place