Try our beta test site
IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more...

Pulmonary Hypertension: Assessment of Cell Therapy (PHACeT)

This study has been completed.
St. Michael's Hospital, Toronto
Sir Mortimer B. Davis - Jewish General Hospital
Information provided by (Responsible Party):
Northern Therapeutics Identifier:
First received: May 2, 2007
Last updated: October 13, 2016
Last verified: October 2016
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.

Condition Intervention Phase
Hypertension, Pulmonary
Biological: eNOS transfected EPCs will be delivered via a PA line
Phase 1

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
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

Resource links provided by NLM:

Further study details as provided by Northern Therapeutics:

Primary Outcome Measures:
  • 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 ]

Secondary Outcome Measures:
  • 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 ]

Enrollment: 7
Study Start Date: May 2006
Study Completion Date: July 2012
Primary Completion Date: July 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: eNOS transfected EPCs
eNOS transfected EPCs will be delivered by injection via a PA line, incremental doses over three days
Biological: eNOS transfected EPCs will be delivered via a PA line
incremental dosing over 3 days
Other Name: eNOS transfect EPCs

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.

Ages Eligible for Study:   18 Years to 80 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00469027

Canada, Ontario
St. Michael's Hospital
Toronto, Ontario, Canada, M5B1W8
Canada, Quebec
Sir Mortimer B. Davis - Jewish General Hospital
Montreal, Quebec, Canada, H3T 1E2
Sponsors and Collaborators
Northern Therapeutics
St. Michael's Hospital, Toronto
Sir Mortimer B. Davis - Jewish General Hospital
Principal Investigator: John Granton, MD St. Michael's Hospital and University Health Network
Principal Investigator: David Langleben, MD Sir Mortimer B. Davis - Jewish General Hospital
  More Information

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Northern Therapeutics Identifier: NCT00469027     History of Changes
Other Study ID Numbers: CT-PAH 001
Study First Received: May 2, 2007
Last Updated: October 13, 2016

Keywords provided by Northern Therapeutics:
Pulmonary Arterial Hypertension (PAH)

Additional relevant MeSH terms:
Hypertension, Pulmonary
Vascular Diseases
Cardiovascular Diseases
Lung Diseases
Respiratory Tract Diseases processed this record on April 26, 2017