Anemia, Sickle Cell Clinical Trial
Official title:
1503 BMT CTN STRIDE Biorepository
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)".
Status | Recruiting |
Enrollment | 200 |
Est. completion date | July 2023 |
Est. primary completion date | July 2023 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 15 Years to 40 Years |
Eligibility | 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 Sß), 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 |
Country | Name | City | State |
---|---|---|---|
United States | University of Michigan Medical Center | Ann Arbor | Michigan |
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 | Grady Hospital | Atlanta | Georgia |
United States | Augusta University Medical Center | Augusta | Georgia |
United States | Boston University | Boston | Massachusetts |
United States | Dana Farber Cancer Institute/Brigham and Women's Hospital | Boston | Massachusetts |
United States | Dana Farber Cancer Institute/Massachusetts General Hospital | Boston | Massachusetts |
United States | Montefiore Medical Center/Albert Einstein School of Medicine | Bronx | New York |
United States | New York Presbyterian Brooklyn Methodist Hospital | Brooklyn | New York |
United States | University of North Carolina Hospital at Chapel Hill | Chapel Hill | North Carolina |
United States | Medical University of South Carolina | Charleston | South Carolina |
United States | University of Virginia | Charlottesville | Virginia |
United States | University of Chicago | Chicago | Illinois |
United States | Ohio State University | Columbus | Ohio |
United States | Barbara Ann Karmanos Cancer Institute | Detroit | Michigan |
United States | Duke University Medical Center | Durham | North Carolina |
United States | University of Florida Gainsville | Gainesville | Florida |
United States | Hackensack University Medical Center | Hackensack | New Jersey |
United States | Foundation for Sickle Cell Research/Florida Sickle Inc. | Hollywood | Florida |
United States | Baylor College of Medicine/The Methodist Hospital | Houston | Texas |
United States | University of Texas Health Sciences Center | Houston | Texas |
United States | University of Texas/MD Anderson CRC | Houston | Texas |
United States | University of Iowa | Iowa City | Iowa |
United States | University of Miami | Miami | Florida |
United States | Cohen Children's Medical Center | New Hyde Park | New York |
United States | Children's Hospital of New Orleans | New Orleans | Louisiana |
United States | Oschner Medical Center | New Orleans | Louisiana |
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 | Newark Beth Israel Medical Center | Newark | New Jersey |
United States | Benioff Children's Hospital at Oakland | Oakland | California |
United States | University of Oklahoma | Oklahoma City | Oklahoma |
United States | Children's Hospital of Philadelphia | Philadelphia | Pennsylvania |
United States | Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania |
United States | Oregon Health Sciences University | Portland | Oregon |
United States | Virginia Commonwealth University | Richmond | Virginia |
United States | Washington University/St. Louis Children's Hospital | Saint Louis | Missouri |
United States | Children's National Medical Center | Washington | District of Columbia |
Lead Sponsor | Collaborator |
---|---|
Emory University | National Heart, Lung, and Blood Institute (NHLBI) |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Genetic variants in persons with sickle cell disease | A biorepository will be established for future genetic research of sickle cell disease. Blood samples will be drawn from participants at the Baseline Visit and will be stored until analyzed. Analysis will include learning more about the genetics behind complications of sickle cell disease. | Baseline Visit |
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