Phase 2
N=50
Pilot Study Using Avastin and Gleevec to Treat the Progression of Intraluminal Pulmonary Vein Stenosis
Pulmonary Veno Occlusive Disease
Bottom Line
View on ClinicalTrials.gov: NCT00891527 ↗Enrolled (actual)
50
Serious AEs
20.8%
Results posted
May 2017
Primary outcome: Primary: Number of Patients With Survival at 48 Weeks — 42 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Bevacizumab (Avastin) and Imatinib Mesylate (Gleevec) (Drug)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Boston Children's Hospital
- Primary completion
- Dec 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Patients With Survival at 48 Weeks |
42 | — |
| SECONDARY Number of Patients With Disease Progression at 48 Weeks |
5 | — |
| SECONDARY Number of Patients With Disease Stabilization at 48 Weeks |
15 | — |
Summary
The objective of this study is to conduct a pilot study using biologic agents Avastin and Gleevec to treat progression of multivessel intraluminal pulmonary vein stenosis in children.
Eligibility Criteria
Eligibility Criteria: (Both groups)
- Evidence of intraluminal pulmonary vein stenosis in > 1 vessel
- Evidence of myofibroblast neo-proliferation, if biopsies were obtained
- Acceptable organ function includes:
Creatinine or = 1,500/mm3, Hemoglobin > or = 10g/dl, Platelets > or = 100,000/mm3.
Group A Eligibility Criteria: (begin treatment with Gleevec® only)
- Significant concomitant congenital heart defect
- Disease severity for each vessel Category 5 or lower or Category 6 or 7 in no more than 1 vessel
Group B Eligibility Criteria: (begin treatment with Gleevec® and Avastin®)
- Primary PVS (i.e. without concomitant congenital heart defect or lung disease)
- Significant concomitant lung disease
- Patients with PVS and underlying CHD who have category 6 or 7 disease in at least 2 of their pulmonary veins even after surgical or cath-based interventions.
- Accepted organ function includes:
Urine protein < 1
Data sourced from ClinicalTrials.gov (NCT00891527). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.