Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age4-65
SponsorNational Heart, Lung, and Blood Institute (NHLBI)
About this trial
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.
Eligibility criteria
Qualifiers
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
Non disease specific
Disqualifiers
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
Trial design
Treatments tested in this trial
- Total Body Irradiation
- Alemtuzumab
- Abatacept
- Research blood draw
Treatment groups
90 Participants
are divided into 2 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov