Skip to main content
Eligibility
for people ages 7 years to 17 years
Location
at San Francisco, California and other locations
Dates
study started
estimated completion:
Principal Investigator

Description

Summary

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.

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

Keywords

PAH pediatric treprostinil transition Remodulin Epoprostenol Tezosentan

Eligibility

You can join if…

Open to people ages 7 years to 17 years

  • 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 splitting
  • 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)

You CAN'T join if...

  • 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

  • Lucile Packard Children's Hospital
    Palo Alto, California, 94304, USA

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
United Therapeutics
ID
NCT02276872
Phase
Phase 2
Lead Scientist
Jeffrey Fineman
Study Type
Interventional
Last Updated
February 2017