STRIDE Biorepository
Purpose
The STRIDE Biorepository is an optional substudy available to participants in "Bone Marrow Transplantation vs Standard of Care in Patients with Severe Sickle Cell Disease (BMT CTN 1503) (STRIDE)".
Condition
- Anemia, Sickle Cell
Eligibility
- Eligible Ages
- Between 15 Years and 40 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Age at least 15 years old to less than 41 years old - 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
- Human Leukocyte Antigen (HLA) typing prior to referral (consultation with hematopoietic cell transplantation (HCT) physician). However, if a subject has had HLA typing with accompanying documentation that relatives were not HLA typed and that a search of the unrelated donor registry was not performed the subject will be considered eligible. Documentation will be reviewed and adjudicated by the Protocol Officer or his/her designee. - 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
Study Design
- Phase
- Study Type
- Observational [Patient Registry]
- Observational Model
- Cohort
- Time Perspective
- Prospective
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
| Biorepository substudy participants | Participants from the main study who give consent for the genetic testing substudy. |
|
Recruiting Locations
Benioff Children's Hospital at Oakland
Oakland, California 94609
Oakland, California 94609
Children's National Medical Center
Washington, District of Columbia 20010
Washington, District of Columbia 20010
University of Florida Gainsville
Gainesville, Florida 32611
Gainesville, Florida 32611
Children's Healthcare of Atlanta
Atlanta, Georgia 30322
Atlanta, Georgia 30322
Emory Children's Center
Atlanta, Georgia 30322
Atlanta, Georgia 30322
University of Chicago
Chicago, Illinois 60637
Chicago, Illinois 60637
Oschner Medical Center
New Orleans, Louisiana 70121
New Orleans, Louisiana 70121
University of Michigan Medical Center
Ann Arbor, Michigan 48105
Ann Arbor, Michigan 48105
Barbara Ann Karmanos Cancer Institute
Detroit, Michigan 48201
Detroit, Michigan 48201
Washington University/St. Louis Children's Hospital
Saint Louis, Missouri 63110
Saint Louis, Missouri 63110
Hackensack University Medical Center
Hackensack, New Jersey 07601
Hackensack, New Jersey 07601
Newark Beth Israel Medical Center
Newark, New Jersey 07112
Newark, New Jersey 07112
Montefiore Medical Center/Albert Einstein School of Medicine
Bronx, New York 10467
Bronx, New York 10467
New York Presbyterian Brooklyn Methodist Hospital
Brooklyn, New York 11215
Brooklyn, New York 11215
Icahn School of Medicine at Mount Sinai
New York, New York 10029
New York, New York 10029
Contact:
John Levine
John Levine
Weill Cornell Medical College
New York, New York 10065
New York, New York 10065
University of North Carolina Hospital at Chapel Hill
Chapel Hill, North Carolina 27516
Chapel Hill, North Carolina 27516
Duke University Medical Center
Durham, North Carolina 27705
Durham, North Carolina 27705
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
Philadelphia, Pennsylvania 19104
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania 15224
Pittsburgh, Pennsylvania 15224
Medical University of South Carolina
Charleston, South Carolina 29435
Charleston, South Carolina 29435
University of Texas Health Sciences Center
Houston, Texas 77004
Houston, Texas 77004
Baylor College of Medicine/The Methodist Hospital
Houston, Texas 77030
Houston, Texas 77030
University of Virginia
Charlottesville, Virginia 22908
Charlottesville, Virginia 22908
More Details
- NCT ID
- NCT02843347
- Status
- Unknown status
- Sponsor
- Emory University
Detailed Description
A subset of sites for the main study "Bone Marrow Transplantation vs Standard of Care in Patients with Severe Sickle Cell Disease (BMT CTN 1503) (STRIDE)" (NCT02766465) will also participate in the biorepository portion of the study. The purpose of the biorepository is to examine DNA to learn if certain genes predict who will have serious complications of sickle cell disease. The STRIDE Biorepository is an optional substudy available to individuals enrolled in the main study, who are at a participating site. Participants in the main study who consent to take part in the biorepository will have blood drawn at the Baseline Visit. This blood will be shipped to Emory University in Atlanta Georgia and stored for future research.