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.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Masking
NONE
Enrollment
7
incremental dosing over 3 days
St. Michael's Hospital
Toronto, Ontario, Canada
Sir Mortimer B. Davis - Jewish General Hospital
Montreal, Quebec, Canada
The primary endpoints will be related to the tolerability and safety of injection of genetically engineered progenitor cells in patients with severe PAH.
Time frame: 5 years
Potential efficacy of this approach will be assessed by changes in hemodynamic pressures, patient perceived quality of life and exercise capacity
Time frame: 3 month post cell delivery
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