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Protocol Details

Repeat Peripheral Blood Stem Cell Transplantation for Patients with Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels

This study is currently recruiting participants.

Summary | Eligibility | Citations | Contacts

Summary

Number

19-H-0118

Sponsoring Institute

National Heart, Lung and Blood Institute (NHLBI)

Recruitment Detail

Type: Participants currently recruited/enrolled
Gender: Male & Female
Min Age: 4
Max Age: 80

Referral Letter Required

Yes

Population Exclusion(s)

Pregnant Women and Fetuses;
Neonates

Special Instructions

Currently Not Provided

Keywords

Graft-Versus-Host Disease;
Donor Apheresis;
Host-Donor Chimerism

Recruitment Keyword(s)

None

Condition(s)

Sickle Cell Disease

Investigational Drug(s)

None

Investigational Device(s)

Miltenyi CliniMACS CD34+ cell selection kit

Intervention(s)

Device: CliniMACS CD34 Reagent

Supporting Site

National Heart, Lung, and Blood Institute

Background:

Sickle cell disease can often be treated with blood stem cell transplants. But for some people the disease returns. This study will give a second transplant to people whose disease has returned but still have some donor cells in their body.

Objective:

To cure people s sickle cell disease by giving a second treatment that makes more room in their bone marrow for donor cells.

Eligibility:

People ages 4 and older with sickle cell disease who had a transplant but the disease returned, and their donor relatives

Design:

Participants will be screened with medical history, physical exam, and blood tests.

Recipients will also be screened with heart and breathing tests, x-rays, a bone marrow sample, and teeth and eye exams. They must have a caregiver.

Donors will have 7-8 visits. They will take a drug for 5-6 days to prepare them for the donation. For the donation, blood is taken from a vein in the arm or groin. The stem cells are collected. The rest of the blood is returned. This may be repeated.

Recipients will get a long IV line in their arm or chest for about 1-2 months. They will take drugs to help their body accept the donor cells. They will get the donor cells and red blood cell transfusions through the line. They will stay in the hospital about 30 days after the transfusion of donor cells.

In first 3 months after the infusion, recipients will have many visits. Then they will have visits every 6 months to 1 year for 5 years. During those visits they will repeat some of the screening tests.

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Eligibility

INCLUSION CRITERIA:

Inclusion criteria- recipient

1. Patient with history of SCD who underwent allogeneic hematopoietic stem cell transplantation (HSCT)

2. Patient with recurrent SCD defined as HbS greater than or equal to 50% for donors with sickle cell trait and greater than or equal to 10% for donors with HbAA with recurrent clinical manifestations (for example but not limited to recurrent painful crises, acute chest syndrome, priapism, or severe anemia)

3. Persistent donor chimerism levels

4. Age greater than or equal to 4 years

5. Negative beta-HCG

6. Ejection fraction greater than or equal to 35%

7. DLCO greater than or equal to 35%

Inclusion- donor

1. Original donor from the patient s prior transplant

2. Age greater than or equal to 18 years

3. Fit to receive filgrastim (G-CSF) and to give peripheral blood stem cells (blood counts and blood pressure within DTM standards)

4. Ability to comprehend and willing to sign an informed consent

EXCLUSION CRITERIA:

Exclusion criteria- recipient

1. ECOG performance status of 3 or more (see Appendix B)

2. Evidence of uncontrolled bacterial, viral, or fungal infections (currently taking medication and progression of clinical symptoms) within one month prior to signing the consent

3. Patients with fever or suspected minor infection should await resolution of symptoms before signing the consent

4. Major anticipated illness or organ failure incompatible with survival from PBSC transplant

5. Pregnant or breastfeeding

6. Baseline alanine aminotransferase or direct bilirubin >3x upper limit of normal

7. History of liver cirrhosis

8. History of secondary malignancies (other than localized skin cancer)

Exclusion- donor

1. Pregnant or breastfeeding

2. HIV positive

3. Hemoglobin S >50%

4. History of congestive heart failure, unstable angina, or stroke


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Citations:

Not Provided

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Contacts:

Principal Investigator

Referral Contact

For more information:

Courtney D. Fitzhugh, M.D.
National Heart, Lung and Blood Institute (NHLBI)



Julia M. Varga
National Heart, Lung and Blood Institute (NHLBI)
National Institutes of Health
Building 10
Room 6-5140
10 Center Drive
Bethesda, Maryland 20892
(301) 402-3595
julia.varga@nih.gov

Office of Patient Recruitment
National Institutes of Health Clinical Center (CC)
Building 61, 10 Cloister Court
Bethesda, Maryland 20892
Toll Free: 1-800-411-1222
Local Phone: 301-451-4383
TTY: 1-866-411-1010
PRPL@cc.nih.gov

Clinical Trials Number:

NCT04008368

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