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

Northern Therapeutics2 sites in 1 country7 target enrollmentMay 2006

ConditionsHypertension, Pulmonary

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Hypertension, Pulmonary
Sponsor: Northern Therapeutics
Enrollment: 7
Locations: 2
Primary Endpoint: The primary endpoints will be related to the tolerability and safety of injection of genetically engineered progenitor cells in patients with severe PAH.
Status: Completed
Last Updated: 9 years ago

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

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.

Registry

clinicaltrials.gov

Start Date

May 2006

End Date

July 2012

Last Updated

9 years ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Northern Therapeutics

Responsible Party

Sponsor

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

Outcomes

Primary Outcomes

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 Outcomes

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)