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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT01795950




Registration number
NCT01795950
Ethics application status
Date submitted
12/02/2013
Date registered
21/02/2013
Date last updated
17/02/2016

Titles & IDs
Public title
Safety Study of PLX-PAD Cells to Treat Pulmonary Arterial Hypertension (PAH)
Scientific title
A Phase I Safety and Pharmacodynamic Study of Intravenous Infusion of PLX-PAD Cells in Patients With PAH
Secondary ID [1] 0 0
PLX-PH-101
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Pulmonary Arterial Hypertension 0 0
Condition category
Condition code
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Cardiovascular 0 0 0 0
Hypertension

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - PLX-PAD

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

Experimental: 1 M PLX-PAD - 1.0 million (M) PLX-PAD cells per kg body weight

Experimental: 2 M PLX-PAD - 2.0 million (M) PLX-PAD cells per kg body weight


Treatment: Drugs: PLX-PAD
intravenous administration of a single dose of PLX-PAD cells
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of treatment-emergent AEs (frequency and severity at each dose level)
Timepoint [1] 0 0
12 weeks
Primary outcome [2] 0 0
Incidence of SAEs
Timepoint [2] 0 0
1 year
Secondary outcome [1] 0 0
Change in Six Minute Walk distance
Timepoint [1] 0 0
Baseline and 6 weeks
Secondary outcome [2] 0 0
Change in Dyspnea Score
Timepoint [2] 0 0
Baseline and 6 weeks
Secondary outcome [3] 0 0
Change in WHO Functional Classification
Timepoint [3] 0 0
Baseline and 6 weeks
Secondary outcome [4] 0 0
Change in Plasma NT-pro-BNP levels
Timepoint [4] 0 0
Baseline and 6 weeks
Secondary outcome [5] 0 0
Change from Baseline in echocardiography parameters
Timepoint [5] 0 0
Baseline and 6 weeks
Secondary outcome [6] 0 0
Change in cardiopulmonary hemodynamics
Timepoint [6] 0 0
Baseline and 6 weeks

Eligibility
Key inclusion 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
Minimum age
18 Years
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/04/2013
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/01/2016
Sample size
Target
Accrual to date
Final
6
Recruitment in Australia
Recruitment state(s)
QLD
Recruitment hospital [1] 0 0
The Prince Charles Hospital - Brisbane
Recruitment hospital [2] 0 0
The Alfred Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
4032 - Brisbane
Recruitment postcode(s) [2] 0 0
- Melbourne

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
United Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
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 website
https://clinicaltrials.gov/ct2/show/NCT01795950
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Daniel Chambers, MRCP FRACP MD
Address 0 0
The Prince Charles Hospital
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT01795950