STRIDE Biorepository
1503 BMT CTN STRIDE Biorepository
2 other identifiers
observational
200
1 country
40
Brief Summary
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)".
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Mar 2017
Longer than P75 for all trials
40 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 8, 2016
CompletedFirst Posted
Study publicly available on registry
July 25, 2016
CompletedStudy Start
First participant enrolled
March 13, 2017
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 1, 2023
CompletedStudy Completion
Last participant's last visit for all outcomes
July 1, 2023
CompletedFebruary 8, 2021
February 1, 2021
6.3 years
July 8, 2016
February 5, 2021
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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
Study Arms (1)
Biorepository substudy participants
Participants from the main study who give consent for the genetic testing substudy.
Interventions
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
The biorepository will consist of research participants from the main study who consent to having extra blood drawn and stored for the purpose of future genetic testing.
You may qualify if:
- 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:
- Clinically significant neurologic event (stroke) or any neurological deficit lasting \> 24 hours;
- 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);
- 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);
- 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);
- An echocardiographic finding of tricuspid valve regurgitant jet (TRJ) velocity ≥ 2.7 m/sec;
- 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:
- Karnofsky/Lansky performance score \> or equal to 60
- Cardiac function: Left ventricular ejection fraction (LVEF) \> 40%; or LV shortening fraction \> 26% by cardiac echocardiogram or by Multi Gated Acquisition (MUGA) Scan
- 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)
- 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)
- 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.
You may not qualify if:
- 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
- +2 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (40)
Benioff Children's Hospital at Oakland
Oakland, California, 94609, United States
Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
University of Florida Gainsville
Gainesville, Florida, 32611, United States
Foundation for Sickle Cell Research/Florida Sickle Inc.
Hollywood, Florida, 33021, United States
University of Miami
Miami, Florida, 33136, United States
Grady Hospital
Atlanta, Georgia, 30303, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322, United States
Emory Children's Center
Atlanta, Georgia, 30322, United States
Emory University
Atlanta, Georgia, 30322, United States
Augusta University Medical Center
Augusta, Georgia, 30912, United States
University of Chicago
Chicago, Illinois, 60637, United States
University of Iowa
Iowa City, Iowa, 52242, United States
Children's Hospital of New Orleans
New Orleans, Louisiana, 70118, United States
Oschner Medical Center
New Orleans, Louisiana, 70121, United States
Dana Farber Cancer Institute/Brigham and Women's Hospital
Boston, Massachusetts, 02214, United States
Boston University
Boston, Massachusetts, 02215, United States
Dana Farber Cancer Institute/Massachusetts General Hospital
Boston, Massachusetts, 02215, United States
University of Michigan Medical Center
Ann Arbor, Michigan, 48105, United States
Barbara Ann Karmanos Cancer Institute
Detroit, Michigan, 48201, United States
Washington University/St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
Hackensack University Medical Center
Hackensack, New Jersey, 07601, United States
Newark Beth Israel Medical Center
Newark, New Jersey, 07112, United States
New York Presbyterian Brooklyn Methodist Hospital
Brooklyn, New York, 11215, United States
Cohen Children's Medical Center
New Hyde Park, New York, 11040, United States
Icahn School of Medicine at Mount Sinai
New York, New York, 10029, United States
Weill Cornell Medical College
New York, New York, 10065, United States
Montefiore Medical Center/Albert Einstein School of Medicine
The Bronx, New York, 10467, United States
University of North Carolina Hospital at Chapel Hill
Chapel Hill, North Carolina, 27516, United States
Duke University Medical Center
Durham, North Carolina, 27705, United States
Ohio State University
Columbus, Ohio, 43210, United States
University of Oklahoma
Oklahoma City, Oklahoma, 73104, United States
Oregon Health Sciences University
Portland, Oregon, 97239, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
Medical University of South Carolina
Charleston, South Carolina, 29435, United States
University of Texas Health Sciences Center
Houston, Texas, 77004, United States
Baylor College of Medicine/The Methodist Hospital
Houston, Texas, 77030, United States
University of Texas/MD Anderson CRC
Houston, Texas, 77030, United States
University of Virginia
Charlottesville, Virginia, 22908, United States
Virginia Commonwealth University
Richmond, Virginia, 23298, United States
Biospecimen
Blood will be collected, from participants who consent to the optional storage and future genetic testing of the sample provided.
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Lakshmanan Krishnamurti, MD
Emory University
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Target Duration
- 2 Years
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- MD
Study Record Dates
First Submitted
July 8, 2016
First Posted
July 25, 2016
Study Start
March 13, 2017
Primary Completion
July 1, 2023
Study Completion
July 1, 2023
Last Updated
February 8, 2021
Record last verified: 2021-02