Clinical trial NCT00469027: Pulmonary Hypertension: Assessment of Cell Therapy
Official title: Phase I Trial to Establish Safety of Autologous Progenitor Cell-based Gene Therapy Delivery of heNOS in Patients With Severe Pulmonary Arterial Hypertension(PAH)Refractory to Conventional Treatment
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Hypertension, Pulmonary
Keywords: Pulmonary Arterial Hypertension (PAH)
Lead sponsor: Northern Therapeutics
Sponsor class: INDUSTRY
Collaborators: name: Unity Health Toronto
name: Sir Mortimer B. Davis - Jewish General Hospital
First posted: 2007-05-04
Enrollment: 7
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP

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.

Eligibility criteria: 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

Primary outcomes:
measure: The primary endpoints will be related to the tolerability and safety of injection of genetically engineered progenitor cells in patients with severe PAH.
timeFrame: 5 years
Secondary outcomes:
measure: Potential efficacy of this approach will be assessed by changes in hemodynamic pressures, patient perceived quality of life and exercise capacity
timeFrame: 3 month post cell delivery
Other outcomes:
Locations:
city: Toronto
country: Canada
facility: St. Michael's Hospital
state: Ontario
city: Montreal
country: Canada
facility: Sir Mortimer B. Davis - Jewish General Hospital
state: Quebec
Study officials:
affiliation: St. Michael's Hospital and University Health Network
name: John Granton, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Sir Mortimer B. Davis - Jewish General Hospital
name: David Langleben, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Granton J, Langleben D, Kutryk MB, Camack N, Galipeau J, Courtman DW, Stewart DJ. Endothelial NO-Synthase Gene-Enhanced Progenitor Cell Therapy for Pulmonary Arterial Hypertension: The PHACeT Trial. Circ Res. 2015 Sep 11;117(7):645-54. doi: 10.1161/CIRCRESAHA.114.305951. Epub 2015 Jul 20.
citation: O'Connell C, O'Callaghan DS, Humbert M. Novel medical therapies for pulmonary arterial hypertension. Clin Chest Med. 2013 Dec;34(4):867-80. doi: 10.1016/j.ccm.2013.08.002.