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STRIDE Biorepository

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ClinicalTrials.gov Identifier: NCT02843347
Recruitment Status : Recruiting
First Posted : July 25, 2016
Last Update Posted : August 7, 2019
Sponsor:
Collaborator:
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by (Responsible Party):
Lakshmanan Krishnamurti, Emory University

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)".

Condition or disease Intervention/treatment
Anemia, Sickle Cell Procedure: Blood draw

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.

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Study Type : Observational [Patient Registry]
Estimated Enrollment : 200 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 2 Years
Official Title: 1503 BMT CTN STRIDE Biorepository
Actual Study Start Date : March 13, 2017
Estimated Primary Completion Date : October 2021
Estimated Study Completion Date : October 2022

Group/Cohort Intervention/treatment
Biorepository substudy participants
Participants from the main study who give consent for the genetic testing substudy.
Procedure: 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.




Primary Outcome Measures :
  1. Genetic variants in persons with sickle cell disease [ Time Frame: Baseline Visit ]
    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.


Biospecimen Retention:   Samples With DNA
Blood will be collected, from participants who consent to the optional storage and future genetic testing of the sample provided.


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   15 Years to 40 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
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.
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02843347


Contacts
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Contact: Lakshmanan Krishnamurti, MD 404-785-1112 lakshmanan.krishnamurti@emory.edu

Locations
Show Show 40 study locations
Sponsors and Collaborators
Emory University
National Heart, Lung, and Blood Institute (NHLBI)
Investigators
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Principal Investigator: Lakshmanan Krishnamurti, MD Emory University
Additional Information:

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Responsible Party: Lakshmanan Krishnamurti, MD, Emory University
ClinicalTrials.gov Identifier: NCT02843347    
Other Study ID Numbers: IRB00089102
1U01HL128566 ( U.S. NIH Grant/Contract )
First Posted: July 25, 2016    Key Record Dates
Last Update Posted: August 7, 2019
Last Verified: August 2019

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Lakshmanan Krishnamurti, Emory University:
Biorepository
Genetics
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn