NCT01795950

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.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
6

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Apr 2013

Typical duration for phase_1

Geographic Reach
1 country

2 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 12, 2013

Completed
9 days until next milestone

First Posted

Study publicly available on registry

February 21, 2013

Completed
1 month until next milestone

Study Start

First participant enrolled

April 1, 2013

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2015

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2016

Completed
Last Updated

February 17, 2016

Status Verified

February 1, 2016

Enrollment Period

2.7 years

First QC Date

February 12, 2013

Last Update Submit

February 15, 2016

Conditions

Pulmonary Arterial Hypertension

Keywords

cell therapyPulmonary arterial hypertension

Outcome Measures

Primary Outcomes (2)

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

    12 weeks

  • Incidence of SAEs

    1 year

Secondary Outcomes (6)

  • Change in Six Minute Walk distance

    Baseline and 6 weeks

  • Change in Dyspnea Score

    Baseline and 6 weeks

  • Change in WHO Functional Classification

    Baseline and 6 weeks

  • Change in Plasma NT-pro-BNP levels

    Baseline and 6 weeks

  • Change from Baseline in echocardiography parameters

    Baseline and 6 weeks

  • +1 more secondary outcomes

Study Arms (3)

0.5 M PLX-PAD

EXPERIMENTAL

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

Drug: PLX-PAD

1 M PLX-PAD

EXPERIMENTAL

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

Drug: PLX-PAD

2 M PLX-PAD

EXPERIMENTAL

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

Drug: PLX-PAD

Interventions

PLX-PADDRUG

intravenous administration of a single dose of PLX-PAD cells

Also known as: allogeneic Mesenchymal-like Adherent Stromal Cells (ASCs)
0.5 M PLX-PAD1 M PLX-PAD2 M PLX-PAD

Eligibility Criteria

Age18 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

The Prince Charles Hospital

Brisbane, Queensland, 4032, Australia

Location

The Alfred Hospital

Melbourne, Australia

Location

MeSH Terms

Conditions

Pulmonary Arterial Hypertension

Condition Hierarchy (Ancestors)

Hypertension, PulmonaryLung DiseasesRespiratory Tract Diseases

Study Officials

  • Daniel Chambers, MRCP FRACP MD

    The Prince Charles Hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 12, 2013

First Posted

February 21, 2013

Study Start

April 1, 2013

Primary Completion

December 1, 2015

Study Completion

January 1, 2016

Last Updated

February 17, 2016

Record last verified: 2016-02

Locations

AU(2)