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

Pulmonary Arterial Hypertension Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01795950
• Other study ID #: PLX-PH-101
• Status: Terminated
• Phase: Phase 1
• First received: February 12, 2013
• Last updated: February 15, 2016
• Start date: April 2013
• Est. completion date: January 2016

Study information

• Verified date: February 2016
• Source: United Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Health authority: Australia: Human Research Ethics CommitteeAustralia: Department of Health and Ageing Therapeutic Goods Administration
• Study type: Interventional

Clinical 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. 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 6
• Est. completion date: January 2016
• Est. primary completion date: December 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 75 Years

Eligibility

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

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hypertension
• Pulmonary Arterial Hypertension

Intervention

Drug:
• PLX-PAD
intravenous administration of a single dose of PLX-PAD cells

Locations

Country	Name	City	State
Australia	The Prince Charles Hospital	Brisbane	Queensland
Australia	The Alfred Hospital	Melbourne

Sponsors (1)

Lead Sponsor	Collaborator
United Therapeutics

Country where clinical trial is conducted

Australia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of treatment-emergent AEs (frequency and severity at each dose level)		12 weeks	Yes
Primary	Incidence of SAEs		1 year	Yes
Secondary	Change in Six Minute Walk distance		Baseline and 6 weeks	No
Secondary	Change in Dyspnea Score	Change in maximum level of dyspnea experienced during the six minute walk test using a 10 point scale.	Baseline and 6 weeks	No
Secondary	Change in WHO Functional Classification		Baseline and 6 weeks	No
Secondary	Change in Plasma NT-pro-BNP levels		Baseline and 6 weeks	No
Secondary	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)	Baseline and 6 weeks	No
Secondary	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)	Baseline and 6 weeks	No