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

Phase 1

Terminated

• Conditions: Pulmonary Arterial Hypertension
• Interventions: Drug: PLX-PAD

• Registration Number: NCT01795950
• Lead Sponsor: United Therapeutics

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2013-02-12
• Study First Submitted QC: 2013-02-18
• Study First Posted: 2013-02-21
• Study Start: 2013-04
• Primary Completion: 2015-12
• Study Completion: 2016-01
• Status Verified: 2016-02
• Last Update Submitted: 2016-02-15
• Last Update Posted: 2016-02-17

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 6

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
1 M PLX-PAD	PLX-PAD	1.0 million (M) PLX-PAD cells per kg body weight
0.5 M PLX-PAD	PLX-PAD	0.5 million (M) PLX-PAD cells per kg body weight
2 M PLX-PAD	PLX-PAD	2.0 million (M) PLX-PAD cells per kg body weight

Primary Outcome Measures

Name	Time	Method
Incidence of treatment-emergent AEs (frequency and severity at each dose level)	12 weeks
Incidence of SAEs	1 year

Secondary Outcome Measures

Name	Time	Method
Change in Six Minute Walk distance	Baseline and 6 weeks
Change in WHO Functional Classification	Baseline and 6 weeks
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.
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 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 in Plasma NT-pro-BNP levels	Baseline and 6 weeks

Trial Locations

Locations (2)

The Prince Charles Hospital; 🇦🇺 Brisbane, Queensland, Australia

The Alfred Hospital; 🇦🇺 Melbourne, Australia