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Pulmonary Arterial HypertensionPhase 1NCT01795950

Safety Study of PLX-PAD Cells to Treat Pulmonary Arterial Hypertension (PAH)

A Phase 1 study testing PLX-PAD in people with Pulmonary Arterial Hypertension, run by United Therapeutics at 2 sites in Australia.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

The purpose of this clinical study is to assess the safety of PLX-PAD to treat pulmonary arterial hypertension (PAH). PLX-PAD is a cell-based product made of allogeneic Mesenchymal-like Adherent Stromal Cells (ASCs), derived from human full-term placentas following an elective caesarean section. Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Pulmonary Arterial Hypertension
What is being tested?
PLX-PAD
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
6 participants
Where
2 sitesAustralia
Study dates
Apr 2013to Jan 2016
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 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Summary of inclusion and exclusion criteria.

Eligible subjects:

  • Are between 18 and 75 years of age
  • Have a minimum weight of 45 kg
  • Have a diagnosis of idiopathic or heritable PAH, PAH associated with connective tissue disease (CTD), PAH associated with repaired congenital systemic-to-pulmonary cardiac shunt (at least one year since repair), or PAH associated with appetite suppressant/drug or toxin use confirmed by RHC
  • Have a current WHO functional class II or III designation
  • Have been stabilized, without dose changes for at least 30 days prior to the Screening visit on at least two approved PAH medications (e.g., PDE-5 inhibitor, ERA, prostanoid [as inhalation or infusion]); or IV prostanoid monotherapy. Subjects on an IV prostanoid must have been receiving therapy for at least three months prior to the Screening visit.
  • Have a 6MWD equal to or greater than 200 meters (m) at the Screening and Baseline Visits.

Subjects must not:

  • Have any evidence of pulmonary thrombus, significant coronary artery disease (CAD), left ventricular dysfunction, or a restrictive or congestive cardiomyopathy
  • Have a history of malignancies within the past 5 years,with the exception of individuals with localized, non-metastatic basal cell carcinoma of the skin, in situ carcinoma of the cervix, or prostate cancer who are not currently or expected to undergo radiation therapy, chemotherapy and/or surgical intervention, or to initiate hormonal treatment during the study
  • Be listed for transplantation
  • Be pregnant or nursing

Trial locations and contacts

2 sites in 1 country.

Australia2 sites

  • The Prince Charles Hospital

    Brisbane, Queensland, 4032

  • The Alfred Hospital

    Melbourne

Other trials in Australia

About this study

Official title: A Phase I Safety and Pharmacodynamic Study of Intravenous Infusion of PLX-PAD Cells in Patients With PAH

The purpose of this clinical study is to assess the safety of PLX-PAD to treat pulmonary arterial hypertension (PAH). PLX-PAD is a cell-based product made of allogeneic Mesenchymal-like Adherent Stromal Cells (ASCs), derived from human full-term placentas following an elective caesarean section. This year-long study will evaluate the safety of three different dose levels of PLX-PAD, each given as a single intravenous infusion. This study will also evaluate effects that PLX-PAD may have on PAH, such as changes in the ability to exercise and on other tests used to measure the disease severity.

Being tested
PLX-PAD
Keywords
cell therapy, Pulmonary arterial hypertension
Medical subject headings
Pulmonary Arterial Hypertension

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • PLX-PADDrug

    Also called allogeneic Mesenchymal-like Adherent Stromal Cells (ASCs)

    intravenous administration of a single dose of PLX-PAD cells

Study groups in NCT01795950
Study groupRoleWhat they receive
0.5 M PLX-PADExperimental0.5 million (M) PLX-PAD cells per kg body weight
Receives: Drug: PLX-PAD
1 M PLX-PADExperimental1.0 million (M) PLX-PAD cells per kg body weight
Receives: Drug: PLX-PAD
2 M PLX-PADExperimental2.0 million (M) PLX-PAD cells per kg body weight
Receives: Drug: PLX-PAD

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of treatment-emergent AEs (frequency and severity at each dose level)

    Measured: 12 weeks

  2. Incidence of SAEs

    Measured: 1 year

Secondary outcomes (6)

  1. Change in Six Minute Walk distance

    Measured: Baseline and 6 weeks

  2. Change in Dyspnea Score

    Change in maximum level of dyspnea experienced during the six minute walk test using a 10 point scale.

    Measured: Baseline and 6 weeks

  3. Change in WHO Functional Classification

    Measured: Baseline and 6 weeks

  4. Change in Plasma NT-pro-BNP levels

    Measured: Baseline and 6 weeks

  5. Change from Baseline in echocardiography parameters

    Change in RV area at end systole and end diastole (for calculation of estimated RV ejection fraction, RV basal and mid diameter at end systole and end diastole, RV free wall thickness, tricuspid annular plane systolic excursion (TAPSE), maximal tricuspid regurgitant jet velocity TRJV) and pulmonary artery end diastolic pressure (PAEDP)

    Measured: Baseline and 6 weeks

  6. Change in cardiopulmonary hemodynamics

    mean pulmonary arterial pressure (PAPm), heart rate (HR), systolic systemic arterial pressure (SAPs), diastolic systemic arterial pressure (SAPd), mean systemic arterial pressure (SAPm), pulmonary artery systolic pressure (PAPs), pulmonary artery diastolic pressure (PAPd), mean right atrial pressure (RAPm), mean pulmonary capillary wedge pressure (PCWPm), and cardiac output (CO)

    Measured: Baseline and 6 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted21 Feb 2013 (estimated)
  2. Study startApr 2013
  3. Primary completionDec 2015
  4. Study completionJan 2016

Resources

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
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
6 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01795950
Sponsor's protocol ID
PLX-PH-101
Status last verified
Feb 2016
First submitted
12 Feb 2013
Last updated
17 Feb 2016
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing PLX-PAD in people with Pulmonary Arterial Hypertension, run by United Therapeutics at 2 sites in Australia.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

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: stopped early, as of the sponsor's last update on 17 Feb 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 Australia. 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 was stopped early. Results are not available in the source record.

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?

Incidence of treatment-emergent AEs (frequency and severity at each dose level) (time frame: 12 weeks). The study lists 2 primary outcomes in all. 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 NCT01795950.

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 Feb 2016.

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