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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00453414
• Date of registration: 27/03/2007
• Prospective Registration: No
• Primary sponsor: Actelion
• Public title: Safety and Efficacy of Inhaled Iloprost in Pediatric Patients With Pulmonary Arterial Hypertension
• Scientific title: Safety and Efficacy of Inhaled Iloprost in Pediatric Patients With Pulmonary Arterial Hypertension
• Date of first enrolment: July 2006
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: http://clinicaltrials.gov/show/NCT00453414
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

United States

Key inclusion & exclusion 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



Age minimum: 3 Years
Age maximum: 18 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Pulmonary Arterial Hypertension

Intervention(s)

Drug: Iloprost Inhalation Solution (Ventavis)

Primary Outcome(s)

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 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 [Time Frame: No]

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

Secondary ID(s)

C200-007

Source(s) of Monetary Support

Please refer to primary and secondary sponsors