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

• Status: Terminated
• Trial end date: 2016-01-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: United Therapeutics

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