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

• Register: ClinicalTrials.gov
• Last refreshed on: 23 February 2016
• Main ID: NCT01795950
• Date of registration: 12/02/2013
• Prospective Registration: Yes
• Primary sponsor: United Therapeutics
• 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
• Date of first enrolment: April 2013
• Target sample size: 6
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT01795950
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1

Countries of recruitment

Australia

Contacts

• Name: Daniel Chambers, MRCP FRACP MD
• Affiliation: The Prince Charles Hospital

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: 75 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Pulmonary Arterial Hypertension

Intervention(s)

Drug: PLX-PAD

Primary Outcome(s)

Incidence of SAEs [Time Frame: 1 year]

Incidence of treatment-emergent AEs (frequency and severity at each dose level) [Time Frame: 12 weeks]

Secondary Outcome(s)

Change from Baseline in echocardiography parameters [Time Frame: Baseline and 6 weeks]

Change in Six Minute Walk distance [Time Frame: Baseline and 6 weeks]

Change in WHO Functional Classification [Time Frame: Baseline and 6 weeks]

Change in Dyspnea Score [Time Frame: Baseline and 6 weeks]

Change in Plasma NT-pro-BNP levels [Time Frame: Baseline and 6 weeks]

Change in cardiopulmonary hemodynamics [Time Frame: Baseline and 6 weeks]

Secondary ID(s)

PLX-PH-101

Source(s) of Monetary Support

Please refer to primary and secondary sponsors