Macitentan in Pulmonary Hypertension of Sickle Cell Disease (MENSCH)
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|ClinicalTrials.gov Identifier: NCT02651272|
Recruitment Status : Terminated (Unable to enroll IRB approved sample of participants.)
First Posted : January 8, 2016
Results First Posted : December 8, 2020
Last Update Posted : December 8, 2020
|Condition or disease||Intervention/treatment||Phase|
|Pulmonary Hypertension Sickle Cell Disease||Drug: macitentan||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||4 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Masking Description:||Study is Open Label|
|Official Title:||The Safety and Efficacy of Macitentan for Treatment of Pulmonary Hypertension in Sickle Cell Disease|
|Study Start Date :||July 2015|
|Actual Primary Completion Date :||December 18, 2019|
|Actual Study Completion Date :||December 18, 2019|
10mg macitentan tablets, taken once daily (QD), by mouth (PO), for the treatment period lasting 16 weeks.
10mg macitentan tablets
Other Name: opsumit
- Number of Participants With Treatment-emergent Adverse Events [ Time Frame: 20 weeks ]The occurrence of treatment emergent AEs includes having any of the following: vaso-occlusive crises requiring hospitalization; acute congestive heart failure; hypotension (defined as a mean arterial pressure less than 60mmHg); decrease in hemoglobin concentration by greater than 1 g/dL.
- Change in Right Arterial Pressure (RAP) [ Time Frame: Baseline, 16 weeks ]RAP will be assessed by right heart catheterization. Normal range is 2-6 mmHg.
- Change in Systolic Right Ventricular Pressure (RVSP) [ Time Frame: Baseline, 16 weeks ]RVSP will be assessed by right heart catheterization. Normal range is 15-25 mmHg.
- Change in Diastolic Pulmonary Artery Pressure (PADP) [ Time Frame: Baseline, 16 weeks ]PADP will be assessed by right heart catheterization. Normal range is 8-15 mmHg.
- Change in Systolic Pulmonary Artery Pressure (SPAP) [ Time Frame: Baseline, 16 weeks ]SPAP will be assessed by right heart catheterization. Normal range is 15-25 mm Hg.
- Change in Systemic Vascular Resistance Index (SVR) [ Time Frame: Baseline, Week 16 ]Systemic vascular resistance (SVR) will be assessed with this formula Systemic Vascular Resistance (SVR) = 80x(Mean Arterial Pressure - Mean Venous Pressure or CVP) / Cardiac Output. Normal range is 800 - 1200 dynes-sec/cm-5.
- Change in Cardiac Index (CI) [ Time Frame: Baseline to Week 16 ]Cardiac index (CI) will be measured in L/min/m^2. The normal range for CI is 2.5 to 4 L/min/m^2.
- Change in 6 Minute Walk Distance (6MWD) [ Time Frame: Baseline, 16 weeks ]The 6 minute walk test (6MWT) assesses distance walked over 6 minutes (6MWD) as a sub-maximal test of aerobic capacity/endurance. Participants will walk at their normal pace for 6 minutes.
- Assess Change of Borg Dyspnea Index [ Time Frame: Baseline, 16 weeks ]The Borg Dyspnea Index (BDI) is a 0 to 10 rated self reported numerical score used to measure dyspnea during submaximal exercise and will be administered immediately following the 6MWT. The higher the score, the more dyspnea.
- World Health Organization (WHO) Functional Classification [ Time Frame: 16 weeks ]
The WHO functional classification will be assessed and documented with the WHO Class.
Class I Patients with pulmonary hypertension (PH) but without resulting limitation of physical activity. Ordinary physical activity does not cause undue dyspnea or fatigue, chest pain or near syncope.
Class II Patients with PH resulting in a slight limitation of physical activity. They are comfortable at rest. Ordinary physical activity causes undue dyspnea or fatigue, chest pain or near syncope.
Class III Patients with PH resulting in marked limitation of physical activity. They are comfortable at rest. Less than ordinary activity causes undue dyspnea or fatigue, chest pain or near syncope.
Class IV Patients with PH with inability to carry out any physical activity without symptoms. These patients manifest signs of right heart failure. Dyspnea and/or fatigue may even be present at rest. Discomfort is increased by any physical activity.
The Class is inversely related to function.
- Change in NT-proB-type Natriuretic Peptide (NT-pro-BNP) [ Time Frame: Baseline, 16 weeks ]The normal range for NT-pro-BNP is less than 300 picograms of BNP per milliliter (pg/ml) of blood; higher levels are less favorable.
- Change in Cardiac Output (CO) [ Time Frame: Baseline, Week 16 ]Cardiac output (CO) will be measured in L/min/m^2. The normal range for CO is 4 to 8 L/min/m^2.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02651272
|United States, Massachusetts|
|Boston University School of Medicine|
|Boston, Massachusetts, United States, 02118|
|Principal Investigator:||Elizabeth Klings, MD||Boston University|