Pulmonary Arterial Hypertension
Conditions
Keywords
cell therapy, Pulmonary arterial hypertension
Brief 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. 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.
Interventions
DRUGPLX-PAD
intravenous administration of a single dose of PLX-PAD cells
Sponsors
United Therapeutics
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of treatment-emergent AEs (frequency and severity at each dose level) | 12 weeks |
| Incidence of SAEs | 1 year |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in WHO Functional Classification | Baseline and 6 weeks | — |
| Change in Plasma NT-pro-BNP levels | Baseline and 6 weeks | — |
| Change in Six Minute Walk distance | Baseline and 6 weeks | — |
| Change in cardiopulmonary hemodynamics | Baseline and 6 weeks | 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) |
| Change from Baseline in echocardiography parameters | Baseline and 6 weeks | 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) |
| Change in Dyspnea Score | Baseline and 6 weeks | Change in maximum level of dyspnea experienced during the six minute walk test using a 10 point scale. |
Countries
Australia
Outcome results
None listed