Hypertension, Pulmonary
Conditions
Keywords
Pulmonary Arterial Hypertension (PAH)
Brief summary
The primary objective is to establish the safety of autologous progenitor cell-based gene therapy of heNOS in patients with severe Pulmonary Arterial Hypertension(PAH) refractory to conventional treatment.
Detailed description
This is a two centre, phase I clinical trial. A total of 18 patients will be studied using an open-label, dose escaling protocol; three patients will be entered into each of the five dosing panels. An additional three patients will be entered into the final dose panel to establish safety at the maximum tolerated dose.
Interventions
BIOLOGICALeNOS transfected EPCs will be delivered via a PA line
incremental dosing over 3 days
Sponsors
Unity Health Toronto
Sir Mortimer B. Davis - Jewish General Hospital
Northern Therapeutics
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
* Clinical diagnosis of idiopathic Pulmonary Arterial Hypertension * Familial PAH or anorexigen induced PAH * Specified 6-minute walk distance
Exclusion criteria
* Intra or extra cardiac communication between the right and left sided circulations * Hemodynamic instability * Left ventricular ejection fraction \< 40% * Thromboembolic event or recent hospitalization for worsening right sided heart failure in past 3 months * CVP\>20mmHg at time of research heart catheterization * Pregnancy * Concurrent hepatitis or HIV
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary endpoints will be related to the tolerability and safety of injection of genetically engineered progenitor cells in patients with severe PAH. | 5 years |
Secondary
| Measure | Time frame |
|---|---|
| Potential efficacy of this approach will be assessed by changes in hemodynamic pressures, patient perceived quality of life and exercise capacity | 3 month post cell delivery |
Countries
Canada
Outcome results
None listed