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

Study Overview
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Brief Summary
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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.

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

Conditions
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Pulmonary Arterial Hypertension

Intervention / Treatment
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* Drug: Iloprost Inhalation Solution (Ventavis)


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 3 Years
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: Iloprost Inhalation Solution (Ventavis)|nan|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in 6-minute walk distance measured post-inhalation (for patients ≥ 8 years old) at Week 8 and Week 16 |  |  | 
| Change from baseline in Borg Dyspnea Scale measured following 6-MWT (for patients ≥ 8 years old) at Week 8 and Week 16 |  |  | 
| Change from baseline in NYHA/WHO functional class at Week 8 and Week 16 |  |  | 
| Change from baseline in hemodynamic parameters measured at Week 16, and in patients who are able to undergo iloprost inhalation while consciously sedated, hemodynamic parameters measured prior to dosing and 15-30 minutes after dosing |  |  | 
| Time to clinical worsening, defined as any one of the following: death due to underlying PAH, transplantation, atrial septostomy, hospitalization for PAH or right heart failure, >15% worsening in 6-minute walk distance in concert with a ≥ 1 class deteri |  |  | 
| Change from baseline in Child Health Questionnaire (CHQ-28) at Week 8 and Week 16 |  |  | 
| Change from baseline in exercise capacity, as measured by post-inhalation CPET, at Week 8 and Week 16 (if ≥ 8 years old and > 130 cms in height. CPET testing will be conducted on NYHA/WHO Class IV patients at the discretion of the Principal Investigat |  |  | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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PAH, Pediatric, Pulmonary Arterial Hypertension