STRIDE Biorepository

Sponsor

Emory University (Other)

Overall Status

Recruiting

CT.gov ID

NCT02843347

Collaborator

National Heart, Lung, and Blood Institute (NHLBI) (NIH)

200

Enrollment

Locations

75.6

Anticipated Duration (Months)

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

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	Phase
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.

Study Design

Study Type:

Observational [Patient Registry]

Anticipated Enrollment :

200 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

1503 BMT CTN STRIDE Biorepository

Actual Study Start Date :

Mar 13, 2017

Anticipated Primary Completion Date :

Jul 1, 2023

Anticipated Study Completion Date :

Jul 1, 2023

Arms and Interventions

Arm	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.

Arm

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.

Outcome Measures

Primary Outcome Measures

Genetic variants in persons with sickle cell disease [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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

15 Years to 40 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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:

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.

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Benioff Children's Hospital at Oakland	Oakland	California	United States	94609
2	Children's National Medical Center	Washington	District of Columbia	United States	20010
3	University of Florida Gainsville	Gainesville	Florida	United States	32611
4	Foundation for Sickle Cell Research/Florida Sickle Inc.	Hollywood	Florida	United States	33021
5	University of Miami	Miami	Florida	United States	33136
6	Grady Hospital	Atlanta	Georgia	United States	30303
7	Children's Healthcare of Atlanta	Atlanta	Georgia	United States	30322
8	Emory Children's Center	Atlanta	Georgia	United States	30322
9	Emory University	Atlanta	Georgia	United States	30322
10	Augusta University Medical Center	Augusta	Georgia	United States	30912
11	University of Chicago	Chicago	Illinois	United States	60637
12	University of Iowa	Iowa City	Iowa	United States	52242
13	Children's Hospital of New Orleans	New Orleans	Louisiana	United States	70118
14	Oschner Medical Center	New Orleans	Louisiana	United States	70121
15	Dana Farber Cancer Institute/Brigham and Women's Hospital	Boston	Massachusetts	United States	02214
16	Boston University	Boston	Massachusetts	United States	02215
17	Dana Farber Cancer Institute/Massachusetts General Hospital	Boston	Massachusetts	United States	02215
18	University of Michigan Medical Center	Ann Arbor	Michigan	United States	48105
19	Barbara Ann Karmanos Cancer Institute	Detroit	Michigan	United States	48201
20	Washington University/St. Louis Children's Hospital	Saint Louis	Missouri	United States	63110
21	Hackensack University Medical Center	Hackensack	New Jersey	United States	07601
22	Newark Beth Israel Medical Center	Newark	New Jersey	United States	07112
23	Montefiore Medical Center/Albert Einstein School of Medicine	Bronx	New York	United States	10467
24	New York Presbyterian Brooklyn Methodist Hospital	Brooklyn	New York	United States	11215
25	Cohen Children's Medical Center	New Hyde Park	New York	United States	11040
26	Icahn School of Medicine at Mount Sinai	New York	New York	United States	10029
27	Weill Cornell Medical College	New York	New York	United States	10065
28	University of North Carolina Hospital at Chapel Hill	Chapel Hill	North Carolina	United States	27516
29	Duke University Medical Center	Durham	North Carolina	United States	27705
30	Ohio State University	Columbus	Ohio	United States	43210
31	University of Oklahoma	Oklahoma City	Oklahoma	United States	73104
32	Oregon Health Sciences University	Portland	Oregon	United States	97239
33	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania	United States	19104
34	Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania	United States	15224
35	Medical University of South Carolina	Charleston	South Carolina	United States	29435
36	University of Texas Health Sciences Center	Houston	Texas	United States	77004
37	Baylor College of Medicine/The Methodist Hospital	Houston	Texas	United States	77030
38	University of Texas/MD Anderson CRC	Houston	Texas	United States	77030
39	University of Virginia	Charlottesville	Virginia	United States	22908
40	Virginia Commonwealth University	Richmond	Virginia	United States	23298

Sponsors and Collaborators

Emory University
National Heart, Lung, and Blood Institute (NHLBI)

Investigators

Principal Investigator: Lakshmanan Krishnamurti, MD, Emory University

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Lakshmanan Krishnamurti, MD, Emory University

ClinicalTrials.gov Identifier:

NCT02843347

Other Study ID Numbers:

IRB00089102
1U01HL128566

First Posted:

Jul 25, 2016

Last Update Posted:

Feb 8, 2021

Last Verified:

Feb 1, 2021

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Keywords provided by Lakshmanan Krishnamurti, MD, Emory University

Biorepository

Genetics

Additional relevant MeSH terms:

Anemia, Sickle Cell

Study Results

No Results Posted as of Feb 8, 2021