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Safety, Tolerability, and Pharmacokinetics of Oral Treprostinil in Pediatric PAH Patients Aged 7 to 17 Years

Information source: United Therapeutics
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: PAH

Intervention: oral treprostinil (Drug)

Phase: Phase 2

Status: Recruiting

Sponsored by: United Therapeutics

Official(s) and/or principal investigator(s):
Dunbar Ivy, MD, Principal Investigator, Affiliation: Denver Children's Hospital


This is a multi-center, open-label, safety, tolerability and pharmacokinetic study of oral treprostinil in pediatric subjects with stable PAH aged 7 to 17 years who are, (1) transitioning from parenteral Remodulin therapy; (2) transitioning from inhaled prostacyclin therapy; or (3) not currently receiving prostacyclin therapy.

Clinical Details

Official title: A Multicenter, Open-Label, 24-Week, Uncontrolled Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Oral Treprostinil Extended Release Tablets Following Transition From Remodulin or Inhaled Prostacyclin Therapy or as Add-on to Current PAH Therapy in De Novo Prostacyclin Pediatric Subjects Aged 7 to 17 Years With Pulmonary Arterial Hypertension

Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome:

Successful transition from parenteral Remodulin to oral treprostinil

Successful initation of oral treprostinil

Frequency of adverse events

Secondary outcome:

Change in cardiopulmonary exercise testing (CPET) with progressive cycle ergometry

Change in symptoms of PAH

Change in Panama and WHO functional class

Change in six-minute walk distance (6MWD) with recovery monitoring

Change in dyspnea score

Change in quality of life assessed via the Pediatric Quality of Life Inventory (PedsQL) questionnaire

Change in plasma N-terminal pro-B-type natriuretic peptide (NT-Pro BNP)

Change in cardiac magnetic resonance imaging (cMRI)

Composite of treprostinil pharmacokinetics profiles


Minimum age: 7 Years. Maximum age: 17 Years. Gender(s): Both.


Inclusion Criteria:

- Between 7 and 17 years of age, inclusive, on the date informed consent is signed

- Cohort 3: The subject must weigh a minimum of 22 kg at Screening

- Current diagnosis of PAH (WHO Group I) associated with:

1. Idiopathic or heritable PAH 2. Persistent PAH for at least one year following surgical repair of a congenital systemic-to-pulmonary cardiac shunt, congenital heart disease, or other congenital heart lesions with no clinically significant residual defects and condition is stabilized hemodynamically 3. PAH in subjects with unrepaired restricted atrial septal defect, ventricular septal defect, or patent ductus arteriosus; subject must have a resting post- ductal oxygen saturation (off oxygen) of greater than 88%

- Cohort 1: The subject must have been receiving parenteral Remodulin for at least 90

days without dose change for at least 30 days prior to Baseline

- Cohort 2: The subject must have been receiving inhaled prostacyclin for at least 90

days and has been at the current stable dose without changes for at least 30 days prior to Baseline

- All Cohorts: The subject must have been receiving an approved oral endothelin

receptor antagonist, phosphodiesterase 5 inhibitor, and/or soluble guanylate cyclase stimulator for at least 90 days and has been at the current stable dose, other than weight-based adjustments for at least 30 days prior to first dose of study drug

- Willing and able to swallow intact tablets whole without chewing, breaking, or


- Willing and able to comply with the dietary requirements associated with the oral

treprostinil dosing regimen

- On stable doses of other medical therapy for 14 days prior to Baseline visit with no

dose adjustments, additions, or discontinuations (exception diuretics and anticoagulants) Exclusion Criteria:

- Diagnosis of large unrestrictive ventricular septal defect or patent ductus

arteriosus, Eisenmenger syndrome, congenital diaphragmatic hernia, or a chronic lung disease, such as bronchopulmonary dysplasia, or interstitial lung disease

- Current disease severity of Panama functional class IIIb or IV

- Previous exposure to oral treprostinil

- Current diagnosis of uncontrolled sleep apnea as defined by their physician

- Severe renal insufficiency as defined by an estimated creatinine clearance (CrCl) <30

mL/min (Schwartz Formula) or the requirement for dialysis at Screening

- Moderate to severe hepatic dysfunction; defined as elevated liver function tests (AST

or ALT) greater than or equal to three times the upper limit of normal at Screening, or Child Pugh class B or C hepatic disease

- Clinically significant anemia as defined by a hemoglobin and/or hematocrit level <75%

of the lower limit of normal ranges according to age and gender

- Down syndrome

- Uncontrolled systemic hypertension as evidenced by a systolic or diastolic blood

pressure greater than the 95th percentile for age, height, and gender at Screening or Baseline

- Subject and/or legal guardian has/have an unstable psychiatric condition or is/are

mentally incapable of understanding the objectives, nature, or consequences of the trial, or has any condition in which the Investigator's opinion would constitute an unacceptable risk to the subject's safety

- Active infection, or has any other cardiovascular, liver, renal, hematologic,

gastrointestinal, immunologic, endocrine, metabolic, or central nervous system disease or condition that, in the opinion of the Investigator, may adversely affect the safety of the subject or interfere with the interpretation of study assessments

- Actively listed for transplantation

- Receiving an investigational drug, has an investigational device in place or has

participated in an investigational drug or device study within 30 days prior to first dose of study drug

Locations and Contacts

Stanford Children's Hospital, Palo Alto, California 94304, United States; Recruiting
Keeley Phillips, Phone: 650-723-8922, Email: keeleyp@stanford.edu
Jeffrey Feinstein, MD, MPH, Principal Investigator

Children's Hospital Colorado, Aurora, Colorado 80045, United States; Recruiting
Tracy Urban, Email: Tracy.Urban@childrenscolorado.org
David Dunbar Ivy, MD, Principal Investigator

Boston Children's Hospital, Boston, Massachusetts 02115, United States; Recruiting
Hannah Stonebreaker, Phone: 617-355-5486, Email: hannah.stonebreaker@cardio.chboston.org
Mary Mullen, MD, PhD, Principal Investigator

Seattle Children's Hospital, Seattle, Washington 98105, United States; Recruiting
Amy Payne, RN, Phone: 206-987-5708, Email: amy.payne@seattlechildrens.org
Delphine Yung, MD, Principal Investigator

Additional Information

Starting date: December 2014
Last updated: June 16, 2015

Page last updated: August 23, 2015

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