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

Pulmonary Arterial Hypertension Clinical Trial

Official title:

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

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT00453414
• Other study ID #: C200-007
• Status: Withdrawn
• Phase: Phase 2
• First received: March 27, 2007
• Last updated: February 12, 2010
• Start date: July 2006
• Est. completion date: October 2006

Study information

• Verified date: February 2010
• Source: Actelion
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: October 2006
• Est. primary completion date: October 2006
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 3 Years to 18 Years

Eligibility

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

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hypertension
• Pulmonary Arterial Hypertension

Intervention

Drug:
• Iloprost Inhalation Solution (Ventavis)

Locations

Country	Name	City	State
United States	The Children's Hospital	Denver	Colorado
United States	Columbia University Medical Center	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
Actelion

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from baseline in 6-minute walk distance measured post-inhalation (for patients = 8 years old) at Week 8 and Week 16
Primary	Change from baseline in Borg Dyspnea Scale measured following 6-MWT (for patients = 8 years old) at Week 8 and Week 16
Primary	Change from baseline in NYHA/WHO functional class at Week 8 and Week 16
Primary	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
Primary	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
Primary	Change from baseline in Child Health Questionnaire (CHQ-28) at Week 8 and Week 16
Primary	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			No