Clinical trial NCT01795950: Safety Study of PLX-PAD Cells to Treat Pulmonary Arterial Hypertension (PAH)
Official title: A Phase I Safety and Pharmacodynamic Study of Intravenous Infusion of PLX-PAD Cells in Patients With PAH
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
Conditions: Pulmonary Arterial Hypertension
Keywords: cell therapy
Pulmonary arterial hypertension
Lead sponsor: United Therapeutics
Sponsor class: INDUSTRY
First posted: 2013-02-21
Enrollment: 6
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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

Primary outcomes:
measure: Incidence of treatment-emergent AEs (frequency and severity at each dose level)
timeFrame: 12 weeks
measure: Incidence of SAEs
timeFrame: 1 year
Secondary outcomes:
measure: Change in Six Minute Walk distance
timeFrame: Baseline and 6 weeks
measure: Change in Dyspnea Score
timeFrame: Baseline and 6 weeks
measure: Change in WHO Functional Classification
timeFrame: Baseline and 6 weeks
measure: Change in Plasma NT-pro-BNP levels
timeFrame: Baseline and 6 weeks
measure: Change from Baseline in echocardiography parameters
timeFrame: Baseline and 6 weeks
measure: Change in cardiopulmonary hemodynamics
timeFrame: Baseline and 6 weeks
Other outcomes:
Locations:
city: Brisbane
country: Australia
facility: The Prince Charles Hospital
state: Queensland
city: Melbourne
country: Australia
facility: The Alfred Hospital
Study officials:
affiliation: The Prince Charles Hospital
name: Daniel Chambers, MRCP FRACP MD
role: PRINCIPAL_INVESTIGATOR
References: