Clinical trial NCT00453414: Safety and Efficacy of Inhaled Iloprost in Pediatric Patients With Pulmonary Arterial Hypertension
Official title: Safety and Efficacy of Inhaled Iloprost in Pediatric Patients With Pulmonary Arterial Hypertension
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE2
Conditions: Pulmonary Arterial Hypertension
Keywords: PAH
Pediatric
Pulmonary Arterial Hypertension
Lead sponsor: Actelion
Sponsor class: INDUSTRY
First posted: 2007-03-29
Enrollment: 0
Sex: ALL
Minimum age (years): 3
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Change from baseline in 6-minute walk distance measured post-inhalation (for patients ≥ 8 years old) at Week 8 and Week 16
measure: Change from baseline in Borg Dyspnea Scale measured following 6-MWT (for patients ≥ 8 years old) at Week 8 and Week 16
measure: Change from baseline in NYHA/WHO functional class at Week 8 and Week 16
measure: 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
measure: 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
measure: Change from baseline in Child Health Questionnaire (CHQ-28) at Week 8 and Week 16
measure: 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
Secondary outcomes:
Other outcomes:
Locations:
city: Denver
country: United States
facility: The Children's Hospital
state: Colorado
city: New York
country: United States
facility: Columbia University Medical Center
state: New York
Study officials:
References: