Trial Search Results

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

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.

Stanford is currently accepting patients for this trial.

Lead Sponsor:

United Therapeutics

Stanford Investigator(s):


  • Drug: oral treprostinil


Phase 2


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

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

   - 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

Ages Eligible for Study

7 Years - 17 Years

Genders Eligible for Study


Now accepting new patients

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Henry Chen