Sickle Cell Anemia Clinical Trial
Official title:
The Role of Endothelin-1 in Sickle Cell Disease
Verified date | April 2020 |
Source | Augusta University |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
The primary goal of the study is to determine the safety and tolerability of ambrisentan. It is also expected that ambrisentan will improve blood flow in the lungs, decrease inflammation, and reduce pain in sickle cell patients. An additional goal is to evaluate the use of select biomarkers in evaluating sickle nephropathy.
Status | Completed |
Enrollment | 26 |
Est. completion date | November 30, 2019 |
Est. primary completion date | May 30, 2019 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years to 65 Years |
Eligibility |
Inclusion Criteria: 1. SS or Sßo-thalassemia 2. Age 18-65 years 3. Microalbuminuria (24-hour albumin 150-300 mg) or macroalbuminuria (24-hour albumin >300 mq) OR random urine albumin-creatinine ratio (MA Random) = 30 µg/ mg creatinine 4. Subjects can have Stage 1, II, or III chronic kidney disease (CKD) 5. Subjects can be on hydroxyurea, ACE inhibitors (ACEi), or angiotensin receptor blockers (ARBs) for a period of 3 months or greater 6. Females of child bearing potential must agree to use two forms of birth control with one being a barrier method; abstinence is an acceptable form of birth control Exclusion Criteria: 1. Other genotypes of SCD 2. History of renal transplant 3. Chronic kidney disease (Stage IV and V including patients on hemo dialysis or peritoneal dialysis) 4. Patients on chronic transfusion therapy 5. Uncontrolled/poorly controlled hypertension or history of hypertension pre-dating proteinuria or 6. Known history of HIV, Hepatitis C, and/or diabetes 7. Peripheral edema 8. History of congestive heart failure or pulmonary edema 9. Recent history of coronary artery disease 10. Pregnant or breast feeding 11. Alanine Aminotransferase (ALT) or aspartate aminotransferase (AST) >3-fold upper limit of normal 12. Albumin <2.5 gm/dl 13. Hemoglobin < 6 gm/dL 14. History of non-compliance with medications and clinic visits; or Inability to give informed consent; or Patient deemed ineligible or unsuitable in the judgment of investigators |
Country | Name | City | State |
---|---|---|---|
United States | Augusta University | Augusta | Georgia |
Lead Sponsor | Collaborator |
---|---|
Augusta University | Gilead Sciences, National Heart, Lung, and Blood Institute (NHLBI) |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Safety and Tolerability of ambrisentan in patients with sickle cell disease measured by physical exam, vital signs, blood and urine testing, ECG (specified visits), concomitant medication review, adverse events review | Day 1 (Baseline) through Day 113 | ||
Secondary | Efficacy of ambrisentan in improving kidney function in patients with sickle cell disease measured by blood | measured by blood | Day 1 (Baseline), Day 15, Day 29, Day 57, Day 85, and Day 113 | |
Secondary | Efficacy of ambrisentan in decreasing TRJ velocity | measured by echocardiogram | Day 1 (Baseline) and at the end of the 12 week treatment period | |
Secondary | Efficacy of ambrisentan in decreasing inflammation | measurement of inflammatory markers in blood | Day 1 (Baseline) through Day 113 | |
Secondary | Efficacy of ambrisentan in improving micro-circulation | measured by forearm and skin blood flow measurements | Day 1 (Baseline) and at the end of the 12 week treatment period | |
Secondary | Efficacy of ambrisentan in improving macro-circulation | measured by Transcranial Doppler (TCD) | Day 1 (Baseline), and at the end of the 12 week treatment period | |
Secondary | Efficacy of ambrisentan in improving nociception/pain | performance of quantitative sensory testing | Day 1 (Baseline), and at the end of the 12 week treatment period | |
Secondary | Efficacy of ambrisentan in improving kidney function in patients with sickle cell disease measured by urine testing for microalbuminuria/proteinuria | measured by urine testing for microalbuminuria/proteinuria | Day 1 (Baseline), Day 15, Day 29, Day 43, Day 57, Day 71, Day 85, and Day 113 | |
Secondary | Efficacy of ambrisentan in improving nociception/pain | assessment of pain diaries/questionnaires | Day 1 (Baseline) through the 12 week treatment period | |
Secondary | Efficacy of ambrisentan in improving nociception/pain | assessment of adverse events | Day 1 (Baseline) through the 12 week treatment period |
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