Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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UNKNOWN
200 participants
OBSERVATIONAL
2017-03-13
2023-07-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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Biorepository substudy participants
Participants from the main study who give consent for the genetic testing substudy.
Blood draw
Three tubes of blood (28.5 mL in total) will be obtained at the Baseline Visit. The sample will be stored for future research.
Interventions
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Blood draw
Three tubes of blood (28.5 mL in total) will be obtained at the Baseline Visit. The sample will be stored for future research.
Eligibility Criteria
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Inclusion Criteria
* Severe sickle cell disease \[any clinically significant sickle genotype, for example, Hemoglobin SS (Hb SS), Hemoglobin SC (Hb SC) or Hemoglobin SBeta thalassemia (Hb Sβ), or Hemoglobin S-OArab genotype\] with at least 1 of the following manifestations:
1. Clinically significant neurologic event (stroke) or any neurological deficit lasting \> 24 hours;
2. History of two or more episodes of acute chest syndrome (ACS) in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. asthma therapy);
3. An average of three or more pain crises per year in the 2-year period preceding enrollment or referral (required intravenous pain management in the outpatient or inpatient hospital setting);
4. Administration of regular red blood cell (RBC) transfusion therapy, defined as receiving 8 or more transfusions per year(in the 12 months before enrollment to prevent vaso-occlusive clinical complications (i.e. pain, stroke, and acute chest syndrome);
5. An echocardiographic finding of tricuspid valve regurgitant jet (TRJ) velocity ≥ 2.7 m/sec;
6. Ongoing high impact chronic pain on a majority of days per month for at least 6 months.
* Adequate physical function as measured by all of the following:
1. Karnofsky/Lansky performance score \> or equal to 60
2. Cardiac function: Left ventricular ejection fraction (LVEF) \> 40%; or LV shortening fraction \> 26% by cardiac echocardiogram or by Multi Gated Acquisition (MUGA) Scan
3. Pulmonary function: Pulse oximetry with a baseline O2 saturation of ≥ 85% and diffusing capacity of the lung for carbon monoxide (DLCO) \> 40% (corrected for hemoglobin)
4. Renal function: Serum creatinine ≤ 1.5 x the upper limit of normal for age as per local laboratory and creatinine clearance \>70 mL/min; or GFR \> 70 mL/min/1.73 m2 by radionuclide Glomerular Filtration Rate (GFR)
5. Hepatic function: Serum conjugated (direct) bilirubin \< 2x upper limit of normal for age as per local laboratory; alanine aminotransferase (ALT) and aspartate aminotransferase (AST) \< 5 times upper limit of normal as per local laboratory.
Exclusion Criteria
* Uncontrolled bacterial, viral or fungal infection in the 6 weeks before enrollment.
* Seropositivity for HIV
* Previous HCT or solid organ transplant
* Participation in a clinical trial in which the patient received an investigational drug or device must be discontinued at enrollment.
* A history of substance abuse as defined by version IV of the Diagnostic \& Statistical Manual of Mental Disorders (DSM IV).
* Demonstrated lack of compliance with prior medical care (determined by referring physician).
* Pregnant or breast feeding females.
* Inability to receive HCT due to alloimmunization, defined as the inability to receive packed red blood cell (pRBC) transfusion therapy.
Additional Eligibility Criteria for Transplant after Biologic Assignment to the Donor Arm:
Participants assigned to the Donor Arm at the time of biologic assignment are subject to additional transplant eligibility criteria as specified below. Additional, repeat clinical assessments prior to transplant should be obtained in accordance with institutional policies and standards of care in the interest of good clinical practice.
* Participants must have liver magnetic resonance imaging (MRI) (at least 90 days prior to initiation of transplant conditioning) to document hepatic iron content is required for participants who are currently receiving ≥8 packed red blood cell transfusions for ≥1 year or have received ≥20 packed red blood cell transfusions (cumulative). Participants who have hepatic iron content ≥7 mg Fe/g liver dry weight by liver MRI must have a liver biopsy and histological examination/documentation of the absence of cirrhosis, bridging fibrosis, and active hepatitis (at least 90 days prior to initiation of transplant conditioning).
* Cerebral MRI/magnetic resonance angiogram (MRA) within 30 days prior to initiation of transplant conditioning. If there is clinical or radiologic evidence of a recent neurologic event (such as stroke or transient ischemic attack) subjects will be deferred for at least 6 months with repeat cerebral MRI/MRA to ensure stabilization of the neurologic event prior to proceeding to transplantation.
* Documentation of participant's willingness to use approved contraception method until discontinuation of all immunosuppressive medications. This is to be documented in the medical record corresponding with the consent conference.
* Have a suitably matched HLA donor
* Willing and able to donate bone marrow
* Absence of anti-donor HLA antibodies
15 Years
40 Years
ALL
No
Sponsors
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National Heart, Lung, and Blood Institute (NHLBI)
NIH
Emory University
OTHER
Responsible Party
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Lakshmanan Krishnamurti
MD
Principal Investigators
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Lakshmanan Krishnamurti, MD
Role: PRINCIPAL_INVESTIGATOR
Emory University
Locations
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Benioff Children's Hospital at Oakland
Oakland, California, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
University of Florida Gainsville
Gainesville, Florida, United States
Foundation for Sickle Cell Research/Florida Sickle Inc.
Hollywood, Florida, United States
University of Miami
Miami, Florida, United States
Grady Hospital
Atlanta, Georgia, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, United States
Emory Children's Center
Atlanta, Georgia, United States
Emory University
Atlanta, Georgia, United States
Augusta University Medical Center
Augusta, Georgia, United States
University of Chicago
Chicago, Illinois, United States
University of Iowa
Iowa City, Iowa, United States
Children's Hospital of New Orleans
New Orleans, Louisiana, United States
Oschner Medical Center
New Orleans, Louisiana, United States
Dana Farber Cancer Institute/Brigham and Women's Hospital
Boston, Massachusetts, United States
Boston University
Boston, Massachusetts, United States
Dana Farber Cancer Institute/Massachusetts General Hospital
Boston, Massachusetts, United States
University of Michigan Medical Center
Ann Arbor, Michigan, United States
Barbara Ann Karmanos Cancer Institute
Detroit, Michigan, United States
Washington University/St. Louis Children's Hospital
St Louis, Missouri, United States
Hackensack University Medical Center
Hackensack, New Jersey, United States
Newark Beth Israel Medical Center
Newark, New Jersey, United States
New York Presbyterian Brooklyn Methodist Hospital
Brooklyn, New York, United States
Cohen Children's Medical Center
New Hyde Park, New York, United States
Icahn School of Medicine at Mount Sinai
New York, New York, United States
Weill Cornell Medical College
New York, New York, United States
Montefiore Medical Center/Albert Einstein School of Medicine
The Bronx, New York, United States
University of North Carolina Hospital at Chapel Hill
Chapel Hill, North Carolina, United States
Duke University Medical Center
Durham, North Carolina, United States
Ohio State University
Columbus, Ohio, United States
University of Oklahoma
Oklahoma City, Oklahoma, United States
Oregon Health Sciences University
Portland, Oregon, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
University of Texas Health Sciences Center
Houston, Texas, United States
Baylor College of Medicine/The Methodist Hospital
Houston, Texas, United States
University of Texas/MD Anderson CRC
Houston, Texas, United States
University of Virginia
Charlottesville, Virginia, United States
Virginia Commonwealth University
Richmond, Virginia, United States
Countries
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Central Contacts
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Facility Contacts
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John Levine
Role: primary
Other Identifiers
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IRB00089102
Identifier Type: -
Identifier Source: org_study_id
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