Hypertension, PulmonaryPhase 1PHACeTNCT00469027
Pulmonary Hypertension: Assessment of Cell Therapy
A Phase 1 study testing eNOS transfected EPCs will be delivered via a PA line in people with Hypertension, Pulmonary, run by Northern Therapeutics at 2 sites in Canada.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Hypertension, Pulmonary
- What is being tested?
- eNOS transfected EPCs will be delivered via a PA line
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 2 sitesCanada
- Study dates
- May 2006to Jul 2012
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Clinical diagnosis of idiopathic Pulmonary Arterial Hypertension
- Familial PAH or anorexigen induced PAH
- Specified 6-minute walk distance
Who cannot join
- 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 locations and contacts
2 sites in 1 country.
Canada2 sites
Sir Mortimer B. Davis - Jewish General Hospital
Montreal, Quebec, H3T 1E2
St. Michael's Hospital
Toronto, Ontario, M5B1W8
About this study
Official title: 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
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.
Read the 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.
- Conditions
- Hypertension, Pulmonary
- Being tested
- eNOS transfected EPCs will be delivered via a PA line
- Keywords
- Pulmonary Arterial Hypertension (PAH)
- Medical subject headings
- Hypertension, Pulmonary, Pulmonary Arterial Hypertension
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
eNOS transfected EPCs will be delivered via a PA lineBiological
Also called eNOS transfect EPCs
incremental dosing over 3 days
| Study group | Role | What they receive |
|---|---|---|
| eNOS transfected EPCs | Experimental | eNOS transfected EPCs will be delivered by injection via a PA line, incremental doses over three days Receives: Biological: eNOS transfected EPCs will be delivered via a PA line |
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary endpoints will be related to the tolerability and safety of injection of genetically engineered progenitor cells in patients with severe PAH.
Measured: 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
Measured: 3 month post cell delivery
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 May 2007 (estimated)
- Study startMay 2006
- Primary completionJul 2012
- Study completionJul 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Hypertension, PulmonaryStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Northern Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Unity Health Toronto, Sir Mortimer B. Davis - Jewish General Hospital
- Responsible party
- Sponsor
- Investigators
- John Granton, MD, Principal investigator, St. Michael's Hospital and University Health NetworkDavid Langleben, MD, Principal investigator, Sir Mortimer B. Davis - Jewish General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00469027
- Sponsor's protocol ID
- CT-PAH 001
- Acronym
- PHACeT
- Status last verified
- Oct 2016
- First submitted
- 2 May 2007
- Last updated
- 17 Oct 2016
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 26195220O'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.
PubMed 24267310
Study at a glance
From the registry record, last updated by the sponsor on 17 Oct 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT00469027
- Acronym / sponsor ID
- PHACeT / CT-PAH 001
- Conditions
- Hypertension, Pulmonary
- Keywords
- Pulmonary Arterial Hypertension (PAH)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Northern TherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- Unity Health Toronto, Sir Mortimer B. Davis - Jewish General Hospital
- Study start
- May 2006
- Primary completion
- Jul 2012 (actual)
- Study completion
- Jul 2012 (actual)
- First posted
- 4 May 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| eNOS transfected EPCs | Experimental | Biological: eNOS transfected EPCs will be delivered via a PA lineeNOS transfected EPCs will be delivered by injection via a PA line, incremental doses over three days |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Clinical diagnosis of idiopathic Pulmonary Arterial Hypertension
- Familial PAH or anorexigen induced PAH
- Specified 6-minute walk distance
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| eNOS transfected EPCs will be delivered via a PA lineAlso: eNOS transfect EPCsincremental dosing over 3 days | Biological | eNOS transfected EPCs |
Primary outcome
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
1 secondary outcome
Potential efficacy of this approach will be assessed by changes in hemodynamic pressures, patient perceived quality of life and exercise capacity
Time frame: 3 month post cell delivery
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Oct 2016.
- Derived from this study: PubMed 26195220Granton 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.
- Derived from this study: PubMed 24267310O'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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
John Granton, MD principal investigator
St. Michael's Hospital and University Health Network
David Langleben, MD principal investigator
Sir Mortimer B. Davis - Jewish General Hospital
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing eNOS transfected EPCs will be delivered via a PA line in people with Hypertension, Pulmonary, run by Northern Therapeutics at 2 sites in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 17 Oct 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00469027, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Oct 2016.