Back to search results

Partial Stem Cell Transplant for Sickle Cell Disease or Beta-thalassemia

This study is testing a partial stem cell transplant for people with severe sickle cell disease or beta-thalassemia who need regular blood transfusions. The transplant uses a combination of treatments: Total Body Irradiation (a type of radiation), Alemtuzumab, and Abatacept. The goal is to help more donor cells stay in your body long-term, which could improve your condition. Researchers will collect about 5 tablespoons of blood from donors for research. Success will be measured by how many donor cells are present in your body one year after the transplant. You may be able to join if you are between 4 and 65 years old and have severe sickle cell disease with organ damage or complications, or beta-thalassemia requiring transfusions.

Study design
This is an interventional study with a planned enrollment of 90 participants. It is not specified if it is randomized or blinded.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Donor myeloid chimerism will be measured at 1 year after the transplant.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07599176

Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors

Recruiting
PHASE1Ages 4–65InterventionalTreatment
National Heart, Lung, and Blood Institute (NHLBI)
~90 participants
Updated 2026-07-29 on ClinicalTrials.gov
What's tested:Total Body IrradiationAlemtuzumabAbataceptResearch blood draw

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Donor myeloid chimerism
Measured over 1 year

Conditions

Sickle Cell Disease
Beta-thalassemia

Explore related trials

Beta ThalassemiaSickle Cell Disease

Where it's being run

1 sites across 1 states
Maryland1

Study leadership

  • John F Tisdale, M.D. · PRINCIPAL_INVESTIGATOR · National Heart, Lung, and Blood Institute (NHLBI)

Who to contact

Kelly S Norris, R.N.
[email protected]
(301) 529-7104
Email the study team

Opens a ready-to-send draft in your own email app — review before sending.

Do you actually qualify for this trial?

Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.

Check eligibility for this trial ~2 min · HIPAA-protected · delete anytime
Eligibility criteria

Inclusion

Portal fibrosis by liver biopsy
Inadequate chelation history (defined as failure to maintain adequate compliance with chelation with deferoxamine initiated within 18 months of the first transfusion and administered at least 5 days each week)
Hepatomegaly of greater than 2 cm below the costochondral margin or by other imaging scans 3. Non disease specific
Ages \>=4 years and less than 65 years old
Fully matched human leukocyte antigen (HLA) donors at A, B, C, and DR loci (8 of 8 or 10 of 10)
Ability to comprehend and willing to sign an informed consent, assent obtained from minors when applicable. Negative serum or urine beta-HCG, when applicable
Agree to use birth control throughout the study and 3 months after abatacept or sirolimus administration.
Female subjects must agree to use a medically acceptable method of birth control such as oral contraceptive, intrauterine device, barrier and spermicide, or implant/injection from start of screening until immunosuppression is stopped.
Male subjects must agree to use effective contraception (including condoms) from start of screening until immunosuppression is stopped.
Fully matched human leukocyte antigen (HLA) donors at A, B, C, and DR loci (8 of 8 or 10 of 10) are intended for this study.
Donors age 4 or older and \>=15 kg (or weight deemed acceptable by IR for line placement, DTM for apheresis, and pediatric consult service) eligible to donate hematopoietic stem cells, are eligible for this study.
Donors will be evaluated in accordance with existing Standard NIH Policies and Procedures for determination of eligibility and suitability for clinical donation. Donors will sign on a separate protocol, 20-H-0099 NHLBI standard of care protocol for the mobilization and collection of HSCs. Note that participation in this study is offered to all eligible donors, but is not required for a donor to make a stem cell donation, so it is possible that not all donors will enroll onto this study.

Exclusion

Karnofsky or Lanksy performance status of \<40
Diffusing capacity of carbon monoxide \<35% predicted: DLCO corrected for hemoglobin or KCO (corrected for lung volume). This criterion may be omitted in young children (e.g. near age 5) or other individuals who may have difficulty understanding or complying with instructions of testing.
Baseline oxygen saturation of \<85% or PaO2 \<70
Left ventricular ejection fraction: \<35% estimated by ECHO
Transaminases \>5x upper limit of normal for age
Evidence of uncontrolled bacterial, viral, or fungal infections (currently taking medication and progression of clinical symptoms) within one month prior to starting the conditioning regimen
Major anticipated illness or organ failure incompatible with survival from HCT
Pregnant or breastfeeding
Pregnant or breastfeeding
Cognitively impaired subjects

What this trial measures

  • Donor myeloid chimerism1 year

    percentage of donor myeloid chimerism