Safety, Tolerability, and Pharmacokinetics of Oral Treprostinil in Pediatric PAH Patients Aged 7 … (NCT02276872) | Clinical Trial Compass
CompletedPhase 2
Safety, Tolerability, and Pharmacokinetics of Oral Treprostinil in Pediatric PAH Patients Aged 7 to 17 Years
United States32 participantsStarted 2014-12-18
Plain-language summary
This was 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 were (1) transitioning from parenteral Remodulin therapy; (2) transitioning from inhaled prostacyclin therapy; or (3) not currently receiving prostacyclin therapy.
Who can participate
Age range7 Years – 17 Years
SexALL
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Inclusion criteria
✓. Legal guardian informed consent and subject assent, if appropriate, to participate in the study was voluntarily given.
✓. The subject was between 7 and 17 years of age, inclusive, on the date informed consent was signed.
✓. Cohort 3: The subject weighed a minimum of 22 kg at Screening.
✓. The subject had a current diagnosis of PAH (WHO Group I) associated with:
✓. IPAH or HPAH
✓. Persistent PAH for at least 1 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 was stabilized hemodynamically
✓. PAH in subjects with unrepaired restricted atrial septal defect, ventricular septal defect, or patent ductus arteriosus; subject had a resting post-ductal oxygen saturation (off oxygen) of greater than 88%.
✓. The subject had a current diagnosis of PAH confirmed by RHC prior to the Screening Visit with the following parameters:
Exclusion criteria
✕. The subject had a 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.
✕. The subject had a current disease severity of Panama FC IIIb or IV.
What they're measuring
1
Number of Participants With Successful Transition From IV/SC Remodulin to Oral Treprostinil (Cohort 1), From Inhaled Prostacyclin to Oral Treprostinil (Cohort 2), or as an add-on to Current PAH Therapy in de Novo Prostacyclin Subjects (Cohort 3).
. The subject had previously been exposed to oral treprostinil.
✕. Cohort 1: The subject had previous intolerance to treprostinil or epoprostenol due to systemic adverse effects that resulted in discontinuation of therapy. This did not include site pain reactions or central venous catheter-related blood stream infections.
✕. Cohort 1 and 2: The subject was receiving IV/SC Remodulin or Tyvaso® (as the inhaled prostacyclin) for any other disease or condition other than the treatment of PAH in accordance with the IV/SC Remodulin or Tyvaso package inserts (ie, eligible subjects must have had a WHO Group I PAH classification as defined in inclusion criterion #4).
✕. Cohort 3: The subject had been previously exposed to a prostacyclin within 30 days of Screening, with the exception of vasoreactivity testing.
✕. The subject was pregnant or lactating.
✕. The subject had a current diagnosis of uncontrolled sleep apnea as defined by their physician.