Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors

Recruiting now · Phase 1/Phase 2

Conditions studied: Sickle Cell Disease, Beta-thalassemia

In brief

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 facts

Study ID

NCT07599176

Run by

National Heart, Lung, and Blood Institute (NHLBI)

People needed

90

Starts

2026-07-08

Expected to finish

2035-06-30

Last updated by the study team

2026-07-29

Who can join

Age: 4 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• RECIPIENT:
• 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
• DONOR:
• Pregnant or breastfeeding
• Cognitively impaired subjects

Where it is running

• National Institutes of Health Clinical Center — Bethesda, Maryland, United States (enrolling)

Full record on ClinicalTrials.gov

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