Safety and Efficacy of Inhaled Iloprost in Pediatric Patients With Pulmonary Arterial Hypertension

Safety and Efficacy of Inhaled Iloprost in Pediatric Patients With Pulmonary Arterial Hypertension

The purpose of this study is to evaluate the safety and effectiveness of iloprost for PAH in children who are between the ages of 3 and 18 years old.

Inclusion Criteria:

• Male or female, age 3-18 years
• Diagnosis of PAH due to IPAH, connective tissue disease, or congenital heart disease
• Pulmonary arterial hypertension confirmed by a cardiac catheterization, with mPAP > 25 mmHg at rest, PCWP or LVEDP ≤ 15 mmHg, and PVR ≥ 240 dynes/sec/cm-5 (ie, ≥ 3.0 units m2)
• NYHA/WHO functional Class II, III, or IV
• Have had a 6-minute walk test (6-MWT) performed within the 3 months preceding the screening visit (if ≥ 8 years of age)
• Have had cardiopulmonary exercise testing (CPET) performed within the 3 months preceding the screening visit (if ≥ 8 years of age and > 130 cms in height. CPET testing will be conducted on NYHA/WHO Class IV patients at the discretion of the Principal Investigator)
• If receiving therapy for PAH, on a stable dose and regimen for at least 3 months prior to the screening visit
• If receiving digoxin, diuretic, or oxygen therapy, on a stable dose and regimen for at least 30 days preceding the screening visit
• Must demonstrate the ability to appropriately use the nebulizer device as part of the screening process

Exclusion Criteria:

• Prior use of prostacyclins or prostacyclin analogues within 3 months prior to screening
• Portal hypertension or clinically relevant hepatic disease
• Eisenmenger syndrome with resting SpO2 <88% on room air
• Clinically relevant asthma or other chronic lung disease, such as bronchopulmonary dysplasia, cystic fibrosis, or repaired or unrepaired congenital diaphragmatic hernia
• Diagnosis or current evidence of active lung infection or clinically relevant hemoptysis
• Left-sided heart disease, including valvular disease or heart failure
• Initiation or dosage adjustment of PAH-specific therapy within 3 months prior to screening
• Initiation of treatment with digoxin, diuretics, anticoagulation, or oxygen therapy within 30 days prior to screening