Pulmonary Hypertension: Assessment of Cell Therapy (PHACeT)

October 13, 2016 updated by: Northern Therapeutics

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

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.

Study Overview

Status

Completed

Conditions

Intervention / 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.

Study Type

Interventional

Enrollment (Actual)

7

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M5B1W8
        • St. Michael's Hospital
    • Quebec
      • Montreal, Quebec, Canada, H3T 1E2
        • Sir Mortimer B. Davis - Jewish General Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 80 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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 Names:
  • eNOS transfect EPCs

What is the study measuring?

Primary Outcome Measures

Outcome 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.
Time Frame: 5 years
5 years

Secondary Outcome Measures

Outcome Measure
Time Frame
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
3 month post cell delivery

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Northern Therapeutics

Collaborators

Unity Health Toronto
Sir Mortimer B. Davis - Jewish General Hospital

Investigators

  • 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

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

May 1, 2006

Primary Completion (Actual)

July 1, 2012

Study Completion (Actual)

July 1, 2012

Study Registration Dates

First Submitted

May 2, 2007

First Submitted That Met QC Criteria

May 3, 2007

First Posted (Estimate)

May 4, 2007

Study Record Updates

Last Update Posted (Estimate)

October 17, 2016

Last Update Submitted That Met QC Criteria

October 13, 2016

Last Verified

October 1, 2016

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CT-PAH 001

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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