Phase
Condition
Sickle Cell Disease
Red Blood Cell Disorders
Treatment
Red Blood Cell
Standard of care
Clinical Study ID
Ages > 18 All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
Age 18 years or older
Diagnosis of SCD: homozygous sickle cell disease, hemoglobin-SC, Sβ-thalassemia,hemoglobin-SO or hemoglobin-SD.
Patients not on a chronic exchange transfusion program for at least 60 days.
If patients are on a SCD drug (e.g. hydroxyurea, glutamine, or P-selectininhibitors), the doses must be stable for at least 60 days prior to randomization.At the time of randomization, participants must be off Oxbryta for at least 30 days.
Any one of the following vasculopathy biomarker clinical results (a, b, c, d or e)measured in the last 24 months before randomization that indicates a high-riskpatient:
Both a TRV 2.5- <3.0 m/sec and NT-proBNP plasma level ≥ 160 pg/mL,
TRV ≥ 3.0 m/sec,
Both a mean pulmonary artery pressure (PAP) by right heart catheterization 20-24 mmHg and NT-proBNP plasma level ≥ 160 pg/mL,
Mean PAP by right heart catheterization ≥ 25 mmHg,
Chronic kidney disease (CKD) due to SCD with abnormal measures on 2 separateoccasions as defined by: macroalbuminuria (albumin to creatinine ratio (ACR) >300 mg/g) or proteinuria (protein to creatinine ratio >30 mg/mmol), orestimated glomerular filtration rate (eGFR) < 60 mL/min/1.73 m2. (It isrecommended that local laboratories use Chronic Kidney Disease EpidemiologyCollaboration [CKD-EPI] equation without ethnic factors when estimating andreporting GFR).
Clinical results of these biomarkers measured locally at sites within 24 months prior to randomization are acceptable to determine eligibility. TRV, PAP, NT-proBNP, albumin to creatinine ratio, protein to creatinine ratio, or eGFR values must be measured in a steady state (defined as measured ≥ 14 days since an acute care pain event) on different days.
vi. Written informed consent obtained from patient to participate in the trial.
Exclusion
Exclusion Criteria:
RBC alloimmunization resulting in inability of blood bank to obtain compatiblecomponents for chronic exchange transfusions
Previous history of hyper-hemolysis syndrome
Previous history of severe transfusion reaction resulting in renal failure or due toserious complications such as hypotension or respiratory distress
More than 10 vaso-occlusive episodes in the past 12 months requiring admission to ahospital to receive treatment.
Religious objection to receiving blood transfusion
Diagnosis of ischemic stroke within the past 6 months
Clinical evidence of liver failure or advanced cirrhosis or any co-existing medicalcondition that in the Investigator's judgement will substantially increase the riskassociated with the patient's participation in the trial
Women of childbearing potential who have a positive pregnancy test at baseline
Study Design
Study Description
Connect with a study center
Hemorio
Rio De Janeiro,
BrazilSite Not Available
Kremlin-Bicêtre
Créteil,
FranceSite Not Available
Henri Mondor Hopital
Paris,
FranceSite Not Available
Imperial College Healthcare NSH Trust-Hammersmith Hospital
London, W12 0HS
United KingdomSite Not Available
University of Alabama
Tuscaloosa, Alabama 35401
United StatesSite Not Available
UCSF Benioff Children's Hospital Oakland
Oakland, California 94609
United StatesSite Not Available
Howard University Center for Sickle Cell Disease
Washington, District of Columbia 20060
United StatesSite Not Available
Emory University
Atlanta, Georgia 30322
United StatesSite Not Available
University of Illinois at Chicago
Chicago, Illinois 60607
United StatesSite Not Available
Johns Hopkins University
Baltimore, Maryland 21206
United StatesSite Not Available
University of Maryland
Baltimore, Maryland 21201
United StatesSite Not Available
Boston Medical Center
Boston, Massachusetts 02118
United StatesSite Not Available
Washington University-St. Louis
Saint Louis, Missouri 63110
United StatesSite Not Available
Washington University-St. Louis
St. Louis, Missouri 63110
United StatesSite Not Available
Icahn School of Medicine at Mount Sinai
New York, New York 10029
United StatesSite Not Available
Montefiore Medical Center
New York, New York 10461
United StatesSite Not Available
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina 27599
United StatesSite Not Available
Atrium Health
Charlotte, North Carolina 28204
United StatesSite Not Available
Duke University
Durham, North Carolina 27708
United StatesSite Not Available
East Carolina University
Greenville, North Carolina 27834
United StatesSite Not Available
University Hospitals Cleveland Medical Center
Cleveland, Ohio 44106
United StatesSite Not Available
Ohio State University
Columbus, Ohio 43210
United StatesSite Not Available
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania 15232
United StatesSite Not Available
University of Texas Health Science Center at Houston
Houston, Texas 77030
United StatesSite Not Available
Virginia Commonwealth University
Richmond, Virginia 23284
United StatesSite Not Available
Not the study for you?
Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.