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

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2014 by United Therapeutics
Sponsor:
Information provided by (Responsible Party):
United Therapeutics
ClinicalTrials.gov Identifier:
NCT01795950
First received: February 12, 2013
Last updated: August 6, 2014
Last verified: August 2014
  Purpose

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.


Condition Intervention Phase
Pulmonary Arterial Hypertension
Drug: PLX-PAD
Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase I Safety and Pharmacodynamic Study of Intravenous Infusion of PLX-PAD Cells in Patients With PAH

Resource links provided by NLM:


Further study details as provided by United Therapeutics:

Primary Outcome Measures:
  • Incidence of treatment-emergent AEs (frequency and severity at each dose level) [ Time Frame: 12 weeks ] [ Designated as safety issue: Yes ]
  • Incidence of SAEs [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Change in Six Minute Walk distance [ Time Frame: Baseline and 6 weeks ] [ Designated as safety issue: No ]
  • Change in Dyspnea Score [ Time Frame: Baseline and 6 weeks ] [ Designated as safety issue: No ]
    Change in maximum level of dyspnea experienced during the six minute walk test using a 10 point scale.

  • Change in WHO Functional Classification [ Time Frame: Baseline and 6 weeks ] [ Designated as safety issue: No ]
  • Change in Plasma NT-pro-BNP levels [ Time Frame: Baseline and 6 weeks ] [ Designated as safety issue: No ]
  • Change from Baseline in echocardiography parameters [ Time Frame: Baseline and 6 weeks ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline and 6 weeks ] [ Designated as safety issue: No ]
    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)


Estimated Enrollment: 9
Study Start Date: April 2013
Estimated Study Completion Date: January 2016
Estimated Primary Completion Date: March 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 0.5 M PLX-PAD
0.5 million (M) PLX-PAD cells per kg body weight
Drug: PLX-PAD
intravenous administration of a single dose of PLX-PAD cells
Other Name: allogeneic Mesenchymal-like Adherent Stromal Cells (ASCs)
Experimental: 1 M PLX-PAD
1.0 million (M) PLX-PAD cells per kg body weight
Drug: PLX-PAD
intravenous administration of a single dose of PLX-PAD cells
Other Name: allogeneic Mesenchymal-like Adherent Stromal Cells (ASCs)
Experimental: 2 M PLX-PAD
2.0 million (M) PLX-PAD cells per kg body weight
Drug: PLX-PAD
intravenous administration of a single dose of PLX-PAD cells
Other Name: allogeneic Mesenchymal-like Adherent Stromal Cells (ASCs)

  Eligibility

Ages Eligible for Study:   18 Years to 75 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01795950

Contacts
Contact: Kristan Rollins, PharmD 919-425-8167 krollins@unither.com

Locations
Australia, Queensland
The Prince Charles Hospital Recruiting
Brisbane, Queensland, Australia, 4032
Contact: Debra Enever       Debra_Enever@health.qld.gov.au   
Principal Investigator: Daniel Chambers, MRCP FRACP MD         
Australia
The Alfred Hospital Not yet recruiting
Melbourne, Australia
Contact: Christianne Manterfield       C.Manterfield@alfred.org.au   
Principal Investigator: Trevor Williams, MD         
Sponsors and Collaborators
United Therapeutics
Investigators
Principal Investigator: Daniel Chambers, MRCP FRACP MD The Prince Charles Hospital
  More Information

No publications provided

Responsible Party: United Therapeutics
ClinicalTrials.gov Identifier: NCT01795950     History of Changes
Other Study ID Numbers: PLX-PH-101
Study First Received: February 12, 2013
Last Updated: August 6, 2014
Health Authority: Australia: Human Research Ethics Committee
Australia: Department of Health and Ageing Therapeutic Goods Administration

Keywords provided by United Therapeutics:
cell therapy
Pulmonary arterial hypertension

Additional relevant MeSH terms:
Hypertension, Pulmonary
Hypertension
Lung Diseases
Respiratory Tract Diseases
Vascular Diseases
Cardiovascular Diseases

ClinicalTrials.gov processed this record on September 16, 2014