Recruiting
Phase 1
Phase 2

Peripheral Blood Stem Cell Transplantation

Sponsor:

National Heart, Lung, and Blood Institute (NHLBI)

Code:

NCT04008368

Conditions

Myeloid Chimerism

Eligibility Criteria

Sex: All

Age: 2 - 70+

Healthy Volunteers: Accepted

Interventions

CliniMACS CD34 Reagent

Study Details

Brief summary:

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. Donors can be 2 years of age or older.

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....

Conditions

Myeloid Chimerism

Study ID

NCT04008368

Start date

Oct 24, 2019

Status verified date

Feb 2, 2026

Completion date

Dec 1, 2037

Anticipated

Primary completion date

Jan 30, 2030

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 2 - 70+

Healthy Volunteers: Accepted

  • INCLUSION CRITERIA:

Inclusion criteria- recipient

1. Patient with history of SCD or beta thalassemia who previously 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) or patients with recurrent beta thalassemia defined as clinical manifestations such as transfusion-dependence or evidence of extramedullary hematopoiesis. The HbS requirement may be waived by the PI or designee for reasons such as the patient is requiring chronic transfusion therapy or otherwise meets clinical criteria for return of SCD.
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% (Note: This criterion may be omitted in young children (e.g. near the age of 5 years old) or other individuals who may have difficulty understanding or complying with the instructions for accurate testing.)

Inclusion- donor

Related donor from original transplant, deemed suitable and eligible, and willing to donate, per clinical evaluations who are additionally willing to donate blood for research. Related donors will be evaluated in accordance with existing Standard NIH Policies and Procedures for determination of eligibility and suitability for clinical donation. Age >= 2 years <80 years old

EXCLUSION CRITERIA:

Exclusion criteria- recipient

1. ECOG performance status of 3 or more or if <16 years of age Lansky score of 50 or lower.
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. History of secondary malignancies (other than localized skin cancer)
7. Donor-specific HLA antibodies with mean fluorescent intensity >= 2,000

Exclusion- donor

None

Study Design

Enrollment

32 participants

Anticipated

Allocation

Non randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

other: 1

patients with HLA-matched sibling donors

other: 2

patients with haploidentical donors

Interventions

CliniMACS CD34 Reagent

Haploidentical recipients will receive CD34-selected cells using Miltenyi CliniMACS(R) CD34+ cell selection kits. The target CD34+ cell dose is at least 10 x 106/kg, and the minimum CD34+ cell dose is 5 x 106/kg. All of the cells collected during the apheresis procedure will be given. The cells will be cryopreserved and stored until the day of transplant.

Primary outcome measure

  • dichotomous positive/negative outcome where a positive response is defined by absence of graft rejection [ Time Frame: 5 years ]

Central Contacts and Locations

Central contacts

Julia M Varga, R.N.

(301) 402-3595julia.varga@nih.gov

Courtney F Joseph, M.D.

(301) 402-6496courtney.fitzhugh@nih.gov

Locations

National Institutes of Health Clinical Center

Recruiting

Bethesda, Maryland, United States, 20892

Contacts

For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)

800-411-1222prpl@cc.nih.gov

More Information

Sponsor

National Heart, Lung, and Blood Institute (NHLBI)

Last update posted

Feb 5, 2026

Last verified

Feb 2, 2026

Keywords

  • Stem Cell Transplant

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by National Heart, Lung, and Blood Institute (NHLBI) on 2026-02-05.