NCT00469027

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
7

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started May 2006

Longer than P75 for phase_1

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 1, 2006

Completed
1 year until next milestone

First Submitted

Initial submission to the registry

May 2, 2007

Completed
2 days until next milestone

First Posted

Study publicly available on registry

May 4, 2007

Completed
5.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2012

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2012

Completed
Last Updated

October 17, 2016

Status Verified

October 1, 2016

Enrollment Period

6.2 years

First QC Date

May 2, 2007

Last Update Submit

October 13, 2016

Conditions

Hypertension, Pulmonary

Keywords

Pulmonary Arterial Hypertension (PAH)

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (1)

  • 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

Study Arms (1)

eNOS transfected EPCs

EXPERIMENTAL

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

Interventions

eNOS transfected EPCs will be delivered via a PA lineBIOLOGICAL

incremental dosing over 3 days

Also known as: eNOS transfect EPCs
eNOS transfected EPCs

Eligibility Criteria

Age18 Years - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Clinical diagnosis of idiopathic Pulmonary Arterial Hypertension
  • Familial PAH or anorexigen induced PAH
  • Specified 6-minute walk distance

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

St. Michael's Hospital

Toronto, Ontario, M5B1W8, Canada

Location

Sir Mortimer B. Davis - Jewish General Hospital

Montreal, Quebec, H3T 1E2, Canada

Location

Related Publications (2)

  • Granton J, Langleben D, Kutryk MB, Camack N, Galipeau J, Courtman DW, Stewart DJ. Endothelial NO-Synthase Gene-Enhanced Progenitor Cell Therapy for Pulmonary Arterial Hypertension: The PHACeT Trial. Circ Res. 2015 Sep 11;117(7):645-54. doi: 10.1161/CIRCRESAHA.114.305951. Epub 2015 Jul 20.

    PMID: 26195220DERIVED

  • O'Connell C, O'Callaghan DS, Humbert M. Novel medical therapies for pulmonary arterial hypertension. Clin Chest Med. 2013 Dec;34(4):867-80. doi: 10.1016/j.ccm.2013.08.002.

    PMID: 24267310DERIVED

MeSH Terms

Conditions

Hypertension, PulmonaryPulmonary Arterial Hypertension

Condition Hierarchy (Ancestors)

Lung DiseasesRespiratory Tract DiseasesHypertensionVascular DiseasesCardiovascular Diseases

Study Officials

  • John Granton, MD

    St. Michael's Hospital and University Health Network

    PRINCIPAL INVESTIGATOR

  • David Langleben, MD

    Sir Mortimer B. Davis - Jewish General Hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 2, 2007

First Posted

May 4, 2007

Study Start

May 1, 2006

Primary Completion

July 1, 2012

Study Completion

July 1, 2012

Last Updated

October 17, 2016

Record last verified: 2016-10

Locations

CA(2)