Pulmonary Hypertension: Assessment of Cell Therapy
| ISRCTN | ISRCTN14519481 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN14519481 |
| Protocol serial number | CT-PAH 001 |
| Sponsor | Northern Therapeutics Inc (Canada) |
| Funder | Northern Therapeutics Inc (Canada) |
- 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
Plain English summary of protocol
Not provided at time of registration
Contact information
Scientific
30 Bond Street
8th floor Queen wing
Toronto
M5B 1W8
Canada
| Phone | +1 416 864 5733 |
|---|---|
| wardm@smh.toronto.on.ca |
Study information
| Primary study design | Interventional |
|---|---|
| Study design | Phase I, open-label, non-randomised, dose-escalation trial. Doses are assigned sequentially. |
| Secondary study design | Non randomised controlled trial |
| Scientific title | |
| Study acronym | The PHACeT trial |
| Study objectives | The 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) studied | Idiopathic pulmonary arterial hypertension |
| Intervention | A 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 type | Drug |
| Phase | Phase I |
| Drug / device / biological / vaccine name(s) | Nitric oxide |
| Primary outcome measure(s) |
1. Tolerability and safety of the injection of genetically engineered progenitor cells in patients with severe PAH |
| Key secondary outcome measure(s) |
Potential efficacy of this approach will be assessed by changes in hemodynamic pressures, patient perceived quality of life and exercise capacity. |
| Completion date | 31/10/2009 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Adult |
| Lower age limit | 18 Years |
| Sex | All |
| Target sample size at registration | 18 |
| Key inclusion criteria | 1. 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 criteria | 1. 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 enrolment | 08/05/2006 |
| Date of final enrolment | 31/10/2009 |
Locations
Countries of recruitment
- Canada
Study participating centre
M5B 1W8
Canada
Results and Publications
| Individual participant data (IPD) Intention to share | No |
|---|---|
| IPD sharing plan summary | Not provided at time of registration |
| IPD sharing plan |
