Iloprost for the Treatment of Pulmonary Hypertension in Adults With Congenital Heart Disease

Not Applicable

Terminated

Conditions: Eisenmenger's Syndrome
Pulmonary Arterial Hypertension
Congenital Heart Disease

Interventions: Drug: Iloprost

Registration Number: NCT01319045

Lead Sponsor: University of California, Los Angeles

Brief Summary: Pulmonary arterial hypertension (PAH), or high blood pressure in the lungs, is common in patients with congenital heart disease. Historically these patients suffered significant morbidity and mortality due to a lack of effective therapies. More recently, advanced therapies which target the mechanisms underlying the development and progression of PAH have been introduced into clinical care. Oral, intravenous, subcutaneous, and inhaled therapies are all available for the treatment of PAH. Patients with PAH are first treated with oral agents (including sildenafil and bosentan). However, if these agents fail to achieve the desired effect for the patient, intravenous or inhaled therapies may be initiated. Combination therapy with multiple agents is common in routine clinical care. However, the most efficacious therapeutic regimen has yet to be delineated. The present study seeks to evaluate the efficacy of one specific regimen: iloprost, an inhaled prostacyclin derivative, used in combination with oral therapy (sildenafil and/or bosentan). Iloprost has been approved by the FDA for use in this patient population. Adults with PAH already receiving oral therapy will be invited to participate in this study. Iloprost will be added to their current therapeutic regimen for a period of three months, with pre- and post-treatment assessments. These will include a cardiopulmonary exercise test, BNP (a blood test), six minute walking distance, and a quality of life questionnaire.

Detailed Description: Not available

Recruitment & Eligibility

Status: TERMINATED

Sex: All

Target Recruitment: 5

Inclusion Criteria

Age greater than or equal to 18 years old
Congenital heart disease with pulmonary arterial hypertension and cyanosis (resting oxygen saturation < 90% on room air)
Stable on oral therapy (PDE5 inhibitor and/or endothelin blockade) for at least three months

Exclusion Criteria

Age < 18 years old
Current intravenous or subcutaneous prostacyclin therapy
Resting Systemic Hypotension (Systolic blood pressure < 85 mmHg)
Women who are pregnant or may become pregnant (unwilling to utilize effective contraception), as well as nursing mothers
Inability to ambulate
Planned surgical procedure during the study period

Study & Design

Study Type: INTERVENTIONAL

Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Iloprost	Iloprost	Participants will be administered iloprost at 5 mcg/dose x 6 doses daily for 3 months.

Primary Outcome Measures

Name	Time	Method
Safety and Tolerability	3 months	Number of Participants with adverse events, specifically mortality and heart failure.

Secondary Outcome Measures

Name	Time	Method
Serum Brain Natriuretic Peptide (BNP)	3 months	Change in serum BNP level
Exercise Capacity	3 months	Change in exercise duration (modified Bruce protocol), maximal oxygen consumption (VO2 max), and/or VE/VCO2 ratio.
Quality of Life	3 months	Change in quality of life as assessed by SF-36 QOL