A Study of Patients Having Pulmonary Hypertension Associated With Sickle Cell Disease and Completing an ASSET Study
Status:
Terminated
Trial end date:
2007-12-01
Target enrollment:
Participant gender:
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.