Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors

Part of paid clinical trials in Bethesda, Maryland.

Sponsor
National Heart, Lung, and Blood Institute (NHLBI)
Study ID
NCT07599176
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
4 Years - 65 Years
Healthy Volunteers
Not accepted

Interventions

  • Total Body Irradiation — RADIATION
    Total Body Irradiation 400 cGy
  • Alemtuzumab — DRUG
    1 mg/kg
  • Abatacept — DRUG
    10 mg/kg x 6
  • Research blood draw — OTHER
    About 5 tablespoons of blood will be collected from donors for research purposes.

Study Details

This is a non-ablative (partial) stem cell transplant for patients with severe sickle cell disease or beta-thalassemia requiring red cell transfusions. The intensity of the transplant is slightly increased from our previous transplant regimens. The goal is to aim for higher percentage of donor cells to stably remain in the recipients long term.

Key Dates

Start date
Jun 16, 2026
Status verified
May 2026
Primary completion
Jun 30, 2032
Completion
Jun 30, 2035

Study Design

Enrollment
90 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Other: Matched related donors
    Donors for patients
  • Experimental: Transplant recipients
    Patients with symptomatic sickle cell disease or beta-thalassemia

Primary Outcome Measure

Donor myeloid chimerism [ Time Frame: 1 year ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
National Institutes of Health Clinical CenterBethesdaMaryland20892
NIH Clinical Center Office of Patient Recruitment (OPR)
[email protected]
800-411-1222

Find similar trials in Bethesda, MD

By condition
Sickle cell disease
By research site
National Institutes of Health Clinical Center· Bethesda, MD

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