NCT02843347Unknown不适用
1503 BMT CTN STRIDE Biorepository
适应症
试验速览
- 阶段
- 不适用
- 入组人数
- 200
- 试验地点
- 40
- 主要终点
- Genetic variants in persons with sickle cell disease
研究概览
简要总结
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)".
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 15 Years 至 40 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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
结局指标
主要结局
Genetic variants in persons with sickle cell disease
时间窗: 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.
次要结局
未报告次要终点
研究者
Lakshmanan Krishnamurti
MD
Emory University
研究点 (40)
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