search
Back to results

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

Primary Purpose

Pulmonary Arterial Hypertension

Status
Terminated
Phase
Phase 1
Locations
Australia
Study Type
Interventional
Intervention
PLX-PAD
Sponsored by
United Therapeutics
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Pulmonary Arterial Hypertension focused on measuring cell therapy, Pulmonary arterial hypertension

Eligibility Criteria

18 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • The Prince Charles Hospital
  • The Alfred Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Experimental

Arm Label

0.5 M PLX-PAD

1 M PLX-PAD

2 M PLX-PAD

Arm Description

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

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

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

Outcomes

Primary Outcome Measures

Incidence of treatment-emergent AEs (frequency and severity at each dose level)
Incidence of SAEs

Secondary Outcome Measures

Change in Six Minute Walk distance
Change in Dyspnea Score
Change in maximum level of dyspnea experienced during the six minute walk test using a 10 point scale.
Change in WHO Functional Classification
Change in Plasma NT-pro-BNP levels
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)
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)

Full Information

First Posted
February 12, 2013
Last Updated
February 15, 2016
Sponsor
United Therapeutics
search

1. Study Identification

Unique Protocol Identification Number
NCT01795950
Brief Title
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

2. Study Status

Record Verification Date
February 2016
Overall Recruitment Status
Terminated
Study Start Date
April 2013 (undefined)
Primary Completion Date
December 2015 (Actual)
Study Completion Date
January 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
United Therapeutics

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pulmonary Arterial Hypertension
Keywords
cell therapy, Pulmonary arterial hypertension

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
6 (Actual)

8. Arms, Groups, and Interventions

Arm Title
0.5 M PLX-PAD
Arm Type
Experimental
Arm Description
0.5 million (M) PLX-PAD cells per kg body weight
Arm Title
1 M PLX-PAD
Arm Type
Experimental
Arm Description
1.0 million (M) PLX-PAD cells per kg body weight
Arm Title
2 M PLX-PAD
Arm Type
Experimental
Arm Description
2.0 million (M) PLX-PAD cells per kg body weight
Intervention Type
Drug
Intervention Name(s)
PLX-PAD
Other Intervention Name(s)
allogeneic Mesenchymal-like Adherent Stromal Cells (ASCs)
Intervention Description
intravenous administration of a single dose of PLX-PAD cells
Primary Outcome Measure Information:
Title
Incidence of treatment-emergent AEs (frequency and severity at each dose level)
Time Frame
12 weeks
Title
Incidence of SAEs
Time Frame
1 year
Secondary Outcome Measure Information:
Title
Change in Six Minute Walk distance
Time Frame
Baseline and 6 weeks
Title
Change in Dyspnea Score
Description
Change in maximum level of dyspnea experienced during the six minute walk test using a 10 point scale.
Time Frame
Baseline and 6 weeks
Title
Change in WHO Functional Classification
Time Frame
Baseline and 6 weeks
Title
Change in Plasma NT-pro-BNP levels
Time Frame
Baseline and 6 weeks
Title
Change from Baseline in echocardiography parameters
Description
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)
Time Frame
Baseline and 6 weeks
Title
Change in cardiopulmonary hemodynamics
Description
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)
Time Frame
Baseline and 6 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Daniel Chambers, MRCP FRACP MD
Organizational Affiliation
The Prince Charles Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
The Prince Charles Hospital
City
Brisbane
State/Province
Queensland
ZIP/Postal Code
4032
Country
Australia
Facility Name
The Alfred Hospital
City
Melbourne
Country
Australia

12. IPD Sharing Statement

Learn more about this trial

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

We'll reach out to this number within 24 hrs