A Study of Patients Having Pulmonary Hypertension Associated With Sickle Cell Disease and Completing an ASSET Study
- Conditions
- Pulmonary HypertensionSickle Cell Anemia
- Registration Number
- NCT00360087
- Lead Sponsor
- Actelion
- Brief Summary
This study will assess the safety and efficacy of bosentan therapy (in a study known as ASSET) for patients who have high blood pressure in the lungs associated with sickle cell disease. That form of hypertension places people at risk for complications, including shortness of breath, pain, pneumonia, and death. Previous studies have shown that bosentan can be helpful in reducing pulmonary hypertension.
Patients ages 16 and older who have completed the 16-week treatment in the ASSET 1 or ASSET 2 study and who are not pregnant or breastfeeding may be eligible for this study. The research will be conducted in about 25 hospitals in the United States and Europe. Up to 30 participants will be enrolled. The screening visit will involve a physical examination, blood sample of about 3 teaspoons for laboratory tests, and a pregnancy test. Patients' doctors will give them bosentan tablets (62.5 mg each), to take one in the morning and one in the evening. After 1 month, patients will be told whether the dose should be increased to 125 mg tablets to take twice a day. Two weeks after the increase in dose, a blood test will be done to analyze the drug's effects on the liver. After the start of treatment, patients will return for visits every 6 months, when there will be a 6-minute walking test to measure exercise capacity and evaluate shortness of breath. There will be follow-up for patients up to the end of the study and for 28 days after the last dose of bosentan is taken, to collect information about side effects.
Some patients on bosentan have had changes in liver function and red blood cell count. Side effects commonly reported are headache, flushed appearance, inflammation of the throat and nasal passages, and gastrointestinal symptoms. If patients have sudden worsening in breathing in the first few weeks after taking bosentan, they should immediately tell their doctors, because it may be necessary to change the treatment.
- Detailed Description
The object of this study is to assess long-term safety, tolerability and efficacy of bosentan in patients with pulmonary hypertension (PH) associated with sickle cell disease (SCD). The study population will include male and female patients with sickle cell disease (SS,S-beta-Thalassemia) who have previously completed the 16-week treatment period of the double-blind study of bosentan (ASSET 1 or ASSET 2). Patients who meet all the inclusion criteria and none of the exclusion criteria will be started on 62.5 mg bid for 4 weeks and then start the maintenance dose of 125 mg bid (or stay on 62.5 mg if their weight is less than 40kg/90lbs). Patients will be divided into two groups. Group A will consist of patients who begin this study within 4 weeks of completing ASSET 1 or ASSET2. Group B will consist of patients who begin this study longer than 4 weeks after completing ASSET I or ASSET 2. Patients will remain on drug until the FDA approves the drug for use in patients with pulmonary hypertension or until the sponsor decides to stop the study.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 236
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Change from baseline to all assessed time points in 6MWT, in Borg dyspnea index, and in modified NYHA functional class.
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (24)
University of Illinois
🇺🇸Chicago, Illinois, United States
Ohio State University
🇺🇸Columbus, Ohio, United States
St. Louis University
🇺🇸St. Louis, Missouri, United States
Albert Einstein College of Medicine
🇺🇸Bronx, New York, United States
University of Alabama
🇺🇸Birmingham, Alabama, United States
University of California, Los Angeles
🇺🇸Los Angeles, California, United States
University of Kansas
🇺🇸Kansas City, Kansas, United States
University of Tennessee
🇺🇸Memphis, Tennessee, United States
Wayne State University Hutzel Hospital
🇺🇸Detroit, Michigan, United States
University of Colorado
🇺🇸Denver, Colorado, United States
Boston University School of medicine
🇺🇸Boston, Massachusetts, United States
Columbia University
🇺🇸New York, New York, United States
National Heart, Lung and Blood Institute (NHLBI), 9000 Rockville Pike
🇺🇸Bethesda, Maryland, United States
Henry Ford Health Systems
🇺🇸Detroit, Michigan, United States
University Hospitals of Ohio
🇺🇸Cleveland, Ohio, United States
Duke University
🇺🇸Durham, North Carolina, United States
University of Texas, Houston
🇺🇸Houston, Texas, United States
Temple University
🇺🇸Philadelphia, Pennsylvania, United States
Baylor College of Medicine
🇺🇸Houston, Texas, United States
Virginia Commonwealth University Medical Center
🇺🇸Richmond, Virginia, United States
Amsterdam Medical Center
🇳🇱Amsterdam, Netherlands
Royal Free Hospital
🇬🇧London, United Kingdom
University of North Carolina
🇺🇸Chapel Hill, North Carolina, United States
Thomas Jefferson University
🇺🇸Philadelphia, Pennsylvania, United States