Safety Study of PLX-PAD Cells to Treat Pulmonary Arterial... | NCT01795950

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

NCT01795950

Sponsor

United Therapeutics

Status

Terminated

Last Update Posted

Feb 17, 2016Estimated

Enrollment

6Actual

Phase

Phase 1

Conditions

Pulmonary Arterial Hypertension

Interventions

PLX-PAD

Countries

Australia

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Feb 2016

Overall Recruitment Status or Expanded Access Status

Terminated

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Apr 2013

Primary Completion Date

Dec 2015Actual

Completion Date

Jan 2016Actual

First Submitted Date

Feb 12, 2013

First Submission Date that Met QC Criteria

Feb 18, 2013

First Posted Date

Feb 21, 2013Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Feb 15, 2016

Last Update Posted Date

Feb 17, 2016Estimated

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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 provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

6Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
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

Name	Type	Description	Arm Group Labels	Other Names
PLX-PAD	Drug	intravenous administration of a single dose of PLX-PAD cells	0.5 M PLX-PAD 1 M PLX-PAD 2 M PLX-PAD

Outcomes Module

Primary Outcomes

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

Secondary Outcomes

Measure	Description	Time Frame
Change in Six Minute Walk distance		Baseline and 6 weeks
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

Other Outcomes

Not provided

Eligibility Module

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

75 Years

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Facility	Status	City	State	ZIP	Country	Contacts
The Prince Charles Hospital		Brisbane	Queensland	4032	Australia
The Alfred Hospital

ID	Term
D006976	Hypertension, Pulmonary
D008171	Lung Diseases
D012140	Respiratory Tract Diseases