ISRCTN ISRCTN14519481
DOI https://doi.org/10.1186/ISRCTN14519481
Secondary identifying numbers CT-PAH 001
Submission date
06/06/2006
Registration date
16/06/2006
Last edited
30/09/2008
Recruitment status
No longer recruiting
Overall study status
Completed
Condition category
Circulatory System
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data
Record updated in last year

Plain English summary of protocol

Not provided at time of registration

Contact information

Dr Michael Ward
Scientific

30 Bond Street
8th floor Queen wing
Toronto
M5B 1W8
Canada

Phone +1 416 864 5733
Email wardm@smh.toronto.on.ca

Study information

Study designPhase I, open-label, non-randomised, dose-escalation trial. Doses are assigned sequentially.
Primary study designInterventional
Secondary study designNon randomised controlled trial
Study setting(s)Not specified
Study typeTreatment
Scientific title
Study acronymThe PHACeT trial
Study objectivesThe primary objective of this phase I clinical trial is to establish the safety of autologous progenitor cell-based gene delivery of human nitric oxide synthase (heNOS) in patients with severe symptomatic pulmonary arterial hypertension (PAH) refractory to conventional treatment.

Please note that, as of 24/09/2008, the anticipated end date of this trial has been updated from 08/05/2008 to 31/10/2009.
Ethics approval(s)This study was approved by the Research Ethics Board (REB) of St. Michael's Hospital in May 2006 (ref: REB 04-253)
Health condition(s) or problem(s) studiedIdiopathic pulmonary arterial hypertension
InterventionA total of 18 patients will be studied using an open-label, dose-escalating 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.

Apheresis is performed to obtain mononuclear cells from the patientsÂ’ blood. These cells will then be engineered with human nitric oxide synthase (heNOS) and returned back to the patient (autologous) via the right ventricular port of a pulmonary arterial line in divided doses over a three-day elective hospitalisation. Follow-up hemodynamic measures are recorded at three months post-cell delivery.
Intervention typeDrug
Pharmaceutical study type(s)
PhasePhase I
Drug / device / biological / vaccine name(s)Nitric oxide
Primary outcome measure1. Tolerability and safety of the injection of genetically engineered progenitor cells in patients with severe PAH
2. Clinically significant changes in hemodynamic parameters
3. Time to clinical worsening
4. Contrast echo assessment of pulmonary arterial-venous shunting
5. Pulmonary function with diffusing capacity of the lung for carbon monoxide (DLCO)
6. Changes in ventilation perfusion scan
7. Dypnea by Borg index
8. Immune surveillance
9. Human nitric oxide synthase (heNOS) plasmid detection in systemic arterial blood pre- and post-cell delivery
Secondary outcome measuresPotential efficacy of this approach will be assessed by changes in hemodynamic pressures, patient perceived quality of life and exercise capacity.
Overall study start date08/05/2006
Completion date31/10/2009

Eligibility

Participant type(s)Patient
Age groupAdult
Lower age limit18 Years
SexBoth
Target number of participants18
Key inclusion criteria1. Age >=18 years and <=80 years
2. Clinical diagnosis of idiopathic PAH
3. Familial PAH or anorexigen-induced PAH
4. Specified 6-minute walk distance
Key exclusion criteria1. Intra or extra cardiac communication between the right- and left-sided circulations
2. Hemodynamic instability
3. Left ventricular ejection fraction <=40%
4. Thromboembolic event or recent hospitalisation for worsening right-sided heart failure in last three months
5. Central venous pressure (CVP) >20 mmHg at time of research heart catheterisation
6. Pregnancy
7. Concurrent hepatitis or HIV
Date of first enrolment08/05/2006
Date of final enrolment31/10/2009

Locations

Countries of recruitment

  • Canada

Study participating centre

30 Bond Street
Toronto
M5B 1W8
Canada

Sponsor information

Northern Therapeutics Inc (Canada)
Industry

c/o Dr Duncan Stewart
725 Parkdale Avenue
Ottawa
Ontario
K1Y 4E9
Canada

Phone +1 613 761 5341
Email djstewart@ohri.ca
Website http://www.northernther.com
ROR logo "ROR" https://ror.org/02pv1pj08

Funders

Funder type

Industry

Northern Therapeutics Inc (Canada)

No information available

Results and Publications

Intention to publish date
Individual participant data (IPD) Intention to shareNo
IPD sharing plan summaryNot provided at time of registration
Publication and dissemination planNot provided at time of registration
IPD sharing plan