Pulmonary Hypertension: Assessment of Cell Therapy (PHACeT)

Northern Therapeutics

Status and phase: Completed; Phase 1

Conditions: Hypertension, Pulmonary

Treatments: Biological: eNOS transfected EPCs will be delivered via a PA line

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT00469027

CT-PAH 001

Details and patient eligibility

About

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.

Full 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.

Enrollment: 7 patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

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

Trial design

7 participants in 1 patient group

eNOS transfected EPCs

Experimental group

Description:

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

Treatment:

Biological: eNOS transfected EPCs will be delivered via a PA line