Pulmonary Artery Stenosis
Conditions
Brief summary
The primary objective of this study is to evaluate the safety and efficacy of transcatheter Cutting Balloon therapy for branch pulmonary artery stenosis resistant to low pressure dilation.
Detailed description
The proposed study will be a prospective, multi-center, randomized, blinded trial comparing 2 treatments for pulmonary artery stenosis. Specifically, vessels that fail to respond to balloon inflation at 8 ATM will be individually randomized to 1 of 2 treatment pathways. In the first treatment pathway, the vessel is dilated using a CB, followed by additional low pressure balloon dilation (\< 8ATM). In the second pathway,the vessel is dilated with high pressure balloon angioplasty, with balloon inflation pressures exceeding 15 ATM. Cross-over therapy will be permitted for failed high pressure treatment starting with enrollment of patient #15. The primary outcome assessment for efficacy will be a comparison of percent change in minimum lumen diameter before and after randomized therapy, as determined by a core laboratory blinded to pathway assignment. The primary outcome assessment for safety will be a comparison of the proportion of vessel dilations resulting in any serious adverse events, as determined by the Data and Safety Monitoring Board. Patient-level safety endpoints will also be tabulated. Patients can have multiple vessels enrolled; analytical techniques will adjust for potential intra-patient correlation.
Interventions
DEVICETranscatheter Cutting Balloon therapy
PROCEDUREHigh Pressure Balloon Angioplasty
Sponsors
Boston Children's Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
DIAGNOSTIC
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
0 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
Patient Inclusion Criteria (Determined Prior to Cardiac Catheterization) a. At least 1 branch pulmonary artery stenosis referred for planned catheterization for PA dilation b. At least one of the following: i.greater than one half systemic right ventricular pressure ii. regional decrease in pulmonary blood flow by lung scan iii. elevated pulmonary artery pressures (\> 20 mmHg MPAP) iv. cyanosis at least in part due to PA stenosis c. Informed consent of patient and/or parent/guardian; assent of mature minors d. Agreement to participate in protocol, including follow-up testing Vessel Inclusion Criteria (Determined During Cardiac Catheterization) 1. Native pulmonary artery or branch which fails balloon dilation up to 8 ATM; as defined by failure to eliminate a waist 2. Native pulmonary artery or branch with a balloon waist diameter less than 7.5 mm (i.e.,0.5 mm less than the largest available CB) with dilation at 8 ATM 3. All eligible vessels that are dilated during the catheterization must be enrolled as study vessels; "off-study" use of Cutting Balloons or high pressure dilations in eligible vessels is not allowed.
Exclusion criteria
3\. Patient
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Acute Change in Minimum Lumen Diameter Immediately Post-intervention | Pre-intervention to immediate post-intervention (approximately 10 minutes) | The primary efficacy outcome is the percent change in minimum lumen diameter from pre-intervention to immediately post-intervention as measured by angiography. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percent Change in Minimum Lumen Diameter at 3 Months Post-intervention | 3 months post-intervention | Late percent change in minimum lumen diameter from pre-intervention to follow-up angiography. |
Countries
United States
Contacts
PRINCIPAL_INVESTIGATORKathy Jenkins, MD, MPH
Boston Children's Hospital
Participant flow
Pre-assignment details
Among 73 patients, 331 stenotic vessels were identified. 86 vessels were not eligible and 245 vessels were potentially eligible. 72 vessels were considered successfully treated after low-pressure dilation. 173 met eligibility. 66 vessels were randomized to High Pressure Balloon (HPB) and 107 vessels were randomized to Cutting Balloon (CB).
Participants by arm
| Arm | Count |
|---|---|
| Patients With Resistant Pulmonary Artery Stenosis Vessels with resistant PA stenosis were identified during catheterization and eligible vessels were randomized to Cutting Balloon or High Pressure Balloon Dilation.
171 vessels in 73 patients were studied. Individual participants could have vessels randomized to one or both arms of the trial. | 73 |
| Patients With Resistant Pulmonary Artery Stenosis Vessels with resistant PA stenosis were identified during catheterization and eligible vessels were randomized to Cutting Balloon or High Pressure Balloon Dilation.
171 vessels in 73 patients were studied. Individual participants could have vessels randomized to one or both arms of the trial. | 173 |
| Total | 246 |
Baseline characteristics
| Characteristic | Patients With Resistant Pulmonary Artery Stenosis |
|---|---|
| Age, Categorical <=18 years | 72 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 1 Participants |
| Cyanosis in part due to PA stenosis | 19 Participants |
| Elevated PA pressure (mean >20) | 47 Participants |
| Greater than 1/2 systemic RV pressure | 60 Participants |
| Regional decrease in pulmonary flow | 58 Participants |
| Region of Enrollment United States | 73 participants |
| Sex: Female, Male Female | 33 Participants |
| Sex: Female, Male Male | 40 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| other Total, other adverse events | 16 / 73 |
| serious Total, serious adverse events | 0 / 73 |
Outcome results
Primary
Acute Change in Minimum Lumen Diameter Immediately Post-intervention
The primary efficacy outcome is the percent change in minimum lumen diameter from pre-intervention to immediately post-intervention as measured by angiography.
Time frame: Pre-intervention to immediate post-intervention (approximately 10 minutes)
Population: Vessels with resistant Pulmonary Artery Stenosis were identified during catheterization and eligible vessels were randomized to Cutting Balloon or High Pressure Balloon Dilation. Patients had variable number of eligible vessels. Each eligible vessel was randomized at the time of procedure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Patients With Resistant Pulmonary Artery Stenosis | Acute Change in Minimum Lumen Diameter Immediately Post-intervention | Cutting Balloon | 85.1 percentage change | Standard Deviation 77.1 |
| Patients With Resistant Pulmonary Artery Stenosis | Acute Change in Minimum Lumen Diameter Immediately Post-intervention | High Pressure Balloon Dilation | 52.4 percentage change | Standard Deviation 66 |
Secondary
Percent Change in Minimum Lumen Diameter at 3 Months Post-intervention
Late percent change in minimum lumen diameter from pre-intervention to follow-up angiography.
Time frame: 3 months post-intervention
Population: Vessels with resistant Pulmonary Artery Stenosis were identified during catheterization and eligible vessels were randomized to Cutting Balloon or High Pressure Balloon Dilation.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Patients With Resistant Pulmonary Artery Stenosis | Percent Change in Minimum Lumen Diameter at 3 Months Post-intervention | Cutting Balloon | 75.6 percent change | Standard Deviation 97.8 |
| Patients With Resistant Pulmonary Artery Stenosis | Percent Change in Minimum Lumen Diameter at 3 Months Post-intervention | High Pressure Balloon Dilation | 42.2 percent change | Standard Deviation 66.9 |