Trial Outcomes & Findings for Pilot Study Using Avastin and Gleevec to Treat the Progression of Intraluminal Pulmonary Vein Stenosis (NCT NCT00891527)

NCT ID: NCT00891527

Last Updated: 2019-06-19

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 50 participants
• Primary outcome timeframe: 48 weeks
• Results posted on: 2019-06-19

Participant Flow

Participant milestones

Measure	Avastin and/or Gleevec Patients were all treated with Gleevec (imatinib mesylate) and those without congenital heart disease and those who progressed were also treated with Avastin (bevacizumab).
Overall Study STARTED	50
Overall Study COMPLETED	48
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Measure	Avastin and/or Gleevec Patients were all treated with Gleevec (imatinib mesylate) and those without congenital heart disease and those who progressed were also treated with Avastin (bevacizumab).
Overall Study Withdrawal by Subject	2

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Measure	Avastin and/or Gleevec n=48 Participants Patients were all treated with Gleevec (imatinib mesylate) and those without congenital heart disease and those who progressed were also treated with Avastin (bevacizumab).
Age, Customized <3 months	3 Participants n=48 Participants
Age, Customized 3 to <6 months	15 Participants n=48 Participants
Age, Customized 6 to <12 months	17 Participants n=48 Participants
Age, Customized >/= 12 months	13 Participants n=48 Participants
Sex: Female, Male Female	21 Participants n=48 Participants
Sex: Female, Male Male	27 Participants n=48 Participants

PRIMARY outcome

Timeframe: 48 weeks

Outcome measures

Measure	Avastin and/or Gleevec n=48 Participants Patients were all treated with Gleevec (imatinib mesylate) and those without congenital heart disease, significant lung disease, and those who progressed were also treated with Avastin (bevacizumab).
Number of Patients With Survival at 48 Weeks	42 Participants

SECONDARY outcome

Timeframe: 48 weeks

Patients will be classified as having disease progression if at least 2 pulmonary veins have significantly worsened at 48 weeks. This determination is based on the study defined Pulmonary Vein Status Scale, which categorizes pulmonary veins on a scale from "1- None: No narrowing of the luminal contour," to "7- Distal atretic: Complete obliteration of the luminal contour extending >5mm within the vessel segment."

Outcome measures

Measure	Avastin and/or Gleevec n=48 Participants Patients were all treated with Gleevec (imatinib mesylate) and those without congenital heart disease, significant lung disease, and those who progressed were also treated with Avastin (bevacizumab).
Number of Patients With Disease Progression at 48 Weeks	5 Participants

SECONDARY outcome

Timeframe: 48 weeks

Outcome measures

Measure	Avastin and/or Gleevec n=48 Participants Patients were all treated with Gleevec (imatinib mesylate) and those without congenital heart disease, significant lung disease, and those who progressed were also treated with Avastin (bevacizumab).
Number of Patients With Disease Stabilization at 48 Weeks	15 Participants

Adverse Events

Avastin and/or Gleevec

Serious events: 10 serious events

Other events: 48 other events

Deaths: 11 deaths

Serious adverse events

Measure	Avastin and/or Gleevec n=48 participants at risk Patients were all treated with Gleevec (imatinib mesylate) and those without congenital heart disease and those who progressed were also treated with Avastin (bevacizumab).
Blood and lymphatic system disorders Bone Marrow Suppression	4.2% 2/48 • Number of events 4
Gastrointestinal disorders Gastrointestinal Toxicity	8.3% 4/48 • Number of events 4
Infections and infestations Infection	10.4% 5/48 • Number of events 7
General disorders Hospitalization	4.2% 2/48 • Number of events 6
General disorders Tremors	2.1% 1/48 • Number of events 1

Other adverse events

Measure	Avastin and/or Gleevec n=48 participants at risk Patients were all treated with Gleevec (imatinib mesylate) and those without congenital heart disease and those who progressed were also treated with Avastin (bevacizumab).
Blood and lymphatic system disorders Blood Bone Marrow Suppression	100.0% 48/48 • Number of events 602
Skin and subcutaneous tissue disorders Rash	27.1% 13/48 • Number of events 16
Gastrointestinal disorders Gastrointestinal Toxicity	56.2% 27/48 • Number of events 65
Infections and infestations Infection	45.8% 22/48 • Number of events 50
General disorders Liver Toxicity	66.7% 32/48 • Number of events 127
Renal and urinary disorders Renal Toxicity	64.6% 31/48 • Number of events 184
General disorders Transfusion	12.5% 6/48 • Number of events 22
General disorders Other	85.4% 41/48 • Number of events 447

Additional Information

Kathy Jenkins MD MPH

Boston Childrens Hospital

Phone: 6173557275

Email: kathy.jenkins@cardio.chboston.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place