Efficacy and Safety of Bosentan in Sickle Cell Disease (SCD) Patients With Pulmonary Arterial Hyp… (NCT00310830) | Clinical Trial Compass
TerminatedPhase 3
Efficacy and Safety of Bosentan in Sickle Cell Disease (SCD) Patients With Pulmonary Arterial Hypertension (PAH)
Stopped: Slow enrollment
14 participantsStarted 2006-03
Plain-language summary
The study will assess the effect of bosentan on pulmonary vascular resistance and exercise capacity in sickle cell disease (SCD) patients diagnosed with pulmonary arterial hypertension. It consists of 3 phases: Screening, Treatment and Follow-up. During the Screening visit, the study doctor will decide if patients meet the study requirements. All potential patients will have a diagnosis of increased pulmonary artery pressures that is shown by right heart catheterization conducted shortly prior to start of study treatment. Patients will be asked to perform exercise capacity test (walking as far as possible for 6 minutes). Following the Baseline visit, the treatment phase consists of 4 additional clinic visits during which the good and bad effects of the drug are reviewed and exercise capacity test will be repeated. Patients will be treated for 16 weeks. Blood samples will be collected every month, or more often, if needed. At the end of the study, patients will be asked to repeat the right heart catheterization and exercise capacity test. After completion of the study, patients will have the option of enrolling in a long-term follow-up study where all patients will receive active drug. Patients electing not to participate in the extension study will be followed up for safety assessments for about 28 days after the end of the study treatment.
Who can participate
Age range12 Years
SexALL
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Inclusion criteria
✓. Males or females ≥ 12 years of age with a documented history of SCD
✓. Patients with symptomatic PAH associated with shortness of breath
✓. Patients with tricuspid regurgitation jet (TRJ) velocity of \> 2.9 m/sec based on echo/Doppler conducted within 6 months prior to randomization and not during SCD crisis
✓. Signed written informed consent is obtained from the patient or patient's parent/legal representative prior to initiation of any study related procedure
✓. Patients with hemoglobin (Hb) SS or Hb S/β0 genotype and with Hb A ≤ 10%
✓. Six-minute walk test (6MWT) distance ≥ 150 m and ≤ 450 m
✓. PAH confirmed by right heart catheterization (RHC) performed at the study site within 3 months of the randomization visit and defined as:
✓. Women of childbearing potential must have a negative result on their serum pregnancy test and use reliable methods of contraception during study treatment and for 3 months after study treatment termination
Exclusion criteria
What they're measuring
1
Change from Baseline to End of Study in 6MWT distance. A mean difference from placebo of at least 35 m is considered clinically relevant.