Ignite Creation Date:
2024-05-05 @ 11:24 AM
Last Modification Date:
2024-10-26 @ 9:06 AM
Study NCT ID:
NCT00023296
Status:
COMPLETED
Last Update Posted:
2019-12-17
First Post:
2001-09-03
Brief Title:
Nitric Oxide and Transfusion Therapy for Sickle Cell Patients With Pulmonary Hypertension
Sponsor:
National Heart Lung and Blood Institute NHLBI
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Inhaled Nitric Oxide and Transfusion Therapy for Patients With Sickle Cell Anemia and Secondary Pulmonary Hypertension
Status:
COMPLETED
Status Verified Date:
2015-11-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This study will test whether inhaling nitric oxide NO gas mixed with room air can improve pulmonary hypertension high blood pressure in the lungs in patients with sickle cell anemia
Patients with sickle cell disease 18 years of age or older may be eligible to participate in one or more parts of this three-stage study as follows
Stage 1
Patients undergo the following tests to determine the cause of their pulmonary hypertension blood tests echocardiogram heart ultrasound asthma test oxygen breathing study with measurement of arterial blood oxygen levels chest X-ray lung scans MRI of the heart 6-minute walk test night-time oxygen measurement while sleeping and exercise studies
Stage 2
Patients have a detailed MRI evaluation of the heart and are admitted to the NIH Clinical Center intensive care unit ICU for the following test A plastic tube is placed in a vein in the patients arm and another tube is placed in a deeper neck or leg vein A third tube is inserted through the vein into the heart and the lung artery to measure blood pressures in the heart and lungs directly Following baseline measurements three medications inhaled oxygen infused prostaglandin and inhaled NO are delivered for 2 hours each separated by a 30-minute washout period A small blood sample is drawn during the NO administration
Patients who cannot be treated with nitric oxide or for whom the treatment does not work may receive monthly exchange transfusions for 3 months For this procedure 3 to 5 five units of the patients blood is removed and replaced with 3 to 5 units that do not have sickle hemoglobin Some patients who do not respond to NO or exchange transfusions may receive an alternative therapy such as oxygen prostacyclin L-arginine bosentan or sidenafil
Stage 3
Patients remain in the ICU with catheters in place for another 24 hours During this time they breathe NO Lung pressures are measured every 4 hours and blood is drawn every 8 hours They then stay in the hospital 1 more day for observation Patients then breathe nitric oxide continuously for 2 months using a tank of gas that delivers the NO through tubes placed in the nose They may do this at home on an outpatient basis or may remain in the hospital for the 2 months
Patients have an echocardiogram and blood tests every week and do a 6-minute walk test every 2 weeks
Patients with sickle cell disease 18 years of age or older may be eligible to participate in one or more parts of this three-stage study as follows
Stage 1
Patients undergo the following tests to determine the cause of their pulmonary hypertension blood tests echocardiogram heart ultrasound asthma test oxygen breathing study with measurement of arterial blood oxygen levels chest X-ray lung scans MRI of the heart 6-minute walk test night-time oxygen measurement while sleeping and exercise studies
Stage 2
Patients have a detailed MRI evaluation of the heart and are admitted to the NIH Clinical Center intensive care unit ICU for the following test A plastic tube is placed in a vein in the patients arm and another tube is placed in a deeper neck or leg vein A third tube is inserted through the vein into the heart and the lung artery to measure blood pressures in the heart and lungs directly Following baseline measurements three medications inhaled oxygen infused prostaglandin and inhaled NO are delivered for 2 hours each separated by a 30-minute washout period A small blood sample is drawn during the NO administration
Patients who cannot be treated with nitric oxide or for whom the treatment does not work may receive monthly exchange transfusions for 3 months For this procedure 3 to 5 five units of the patients blood is removed and replaced with 3 to 5 units that do not have sickle hemoglobin Some patients who do not respond to NO or exchange transfusions may receive an alternative therapy such as oxygen prostacyclin L-arginine bosentan or sidenafil
Stage 3
Patients remain in the ICU with catheters in place for another 24 hours During this time they breathe NO Lung pressures are measured every 4 hours and blood is drawn every 8 hours They then stay in the hospital 1 more day for observation Patients then breathe nitric oxide continuously for 2 months using a tank of gas that delivers the NO through tubes placed in the nose They may do this at home on an outpatient basis or may remain in the hospital for the 2 months
Patients have an echocardiogram and blood tests every week and do a 6-minute walk test every 2 weeks
Detailed Description:
Sickle cell anemia is an autosomal recessive disorder and the most common genetic disease affecting African-Americans Approximately 015 of African-Americans are homozygous for sickle cell disease and 8 have sickle cell trait Acute pain crisis acute chest syndrome ACS and secondary pulmonary hypertension are common complications of sickle cell anemia Pulmonary hypertension has now been identified as a major cause of death in adults with sickle cell disease Inhaled nitric oxide NO has been proposed as a possible therapy for both primary and secondary pulmonary hypertension Furthermore a number of recent studies have suggested that NO may have a favorable impact on sickle red cells at the molecular level and could improve the abnormal microvascular perfusion that is characteristic of sickle cell anemia In addition chronic exchange transfusion therapy may reduce the progression andor severity of pulmonary hypertension in these patients
This clinical trial is designed 1 to determine the pathophysiologic processes that are associated with and potentially contribute to secondary pulmonary hypertension in adult patients with sickle cell anemia by comparing the cardio-pulmonary status of patients with sickle cell disease with and without pulmonary hypertension 2 to determine the relative acute vasodilatory effects of oxygen intravenous prostacyclin and inhaled nitric oxide on pulmonary artery pressures and other hemodynamic parameters in patients with secondary pulmonary hypertension and sickle cell anemia 3 to determine the effects of two months of inhaled nitric oxide on pulmonary artery pressures other hemodynamic parameters exercise tolerance and symptoms in this patient population and 4 to determine the effects of three months of exchange transfusion on pulmonary artery pressures other hemodynamic parameters exercise tolerance and symptoms in patients who do not receive or fail to respond to NO therapy
This clinical trial is designed 1 to determine the pathophysiologic processes that are associated with and potentially contribute to secondary pulmonary hypertension in adult patients with sickle cell anemia by comparing the cardio-pulmonary status of patients with sickle cell disease with and without pulmonary hypertension 2 to determine the relative acute vasodilatory effects of oxygen intravenous prostacyclin and inhaled nitric oxide on pulmonary artery pressures and other hemodynamic parameters in patients with secondary pulmonary hypertension and sickle cell anemia 3 to determine the effects of two months of inhaled nitric oxide on pulmonary artery pressures other hemodynamic parameters exercise tolerance and symptoms in this patient population and 4 to determine the effects of three months of exchange transfusion on pulmonary artery pressures other hemodynamic parameters exercise tolerance and symptoms in patients who do not receive or fail to respond to NO therapy
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 01-H-0223 | None | None | None |