Pulmonary Hypertension: Assessment of Cell Therapy

Phase 1

Completed

• Conditions: Hypertension, Pulmonary
• Interventions: Biological: eNOS transfected EPCs will be delivered via a PA line

• Registration Number: NCT00469027
• Lead Sponsor: Northern Therapeutics

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.

Study Timeline

• Study Start: 2006-05
• Study First Submitted: 2007-05-02
• Study First Submitted QC: 2007-05-03
• Study First Posted: 2007-05-04
• Primary Completion: 2012-07
• Study Completion: 2012-07
• Status Verified: 2016-10
• Last Update Submitted: 2016-10-13
• Last Update Posted: 2016-10-17

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 7

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
eNOS transfected EPCs	eNOS transfected EPCs will be delivered via a PA line	eNOS transfected EPCs will be delivered by injection via a PA line, incremental doses over three days

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (2)

Sir Mortimer B. Davis - Jewish General Hospital; 🇨🇦 Montreal, Quebec, Canada

St. Michael's Hospital; 🇨🇦 Toronto, Ontario, Canada