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

United Therapeutics

Status and phase: Terminated; Phase 1

Conditions: Pulmonary Arterial Hypertension

Treatments: Drug: PLX-PAD

Study type: Interventional

Funder types: Industry

Identifiers

NCT01795950

PLX-PH-101

Details and patient eligibility

About

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.

Enrollment: 6 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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6 participants in 3 patient groups

0.5 M PLX-PAD

Experimental group

Description:

0.5 million (M) PLX-PAD cells per kg body weight

Treatment:

Drug: PLX-PAD

1 M PLX-PAD

Experimental group

Description:

1.0 million (M) PLX-PAD cells per kg body weight

Treatment:

Drug: PLX-PAD

2 M PLX-PAD

Experimental group

Description:

2.0 million (M) PLX-PAD cells per kg body weight

Treatment:

Drug: PLX-PAD