eligibility_summary
Adults/pediatrics with SCD: high-risk cerebrovascular (stroke, TCD≥200, silent infarct), renal, pulmonary (TRV≥2.5/PH), hepatic, or recurrent VOC/ACS despite therapy, plus ≥1 severe organ injury (CrCl<60/dialysis, EF 35–40, liver fibrosis/cirrhosis or ↑bili/ALT, DLCO 35–40) or no curative option. Age ≥18 (≥16 later), haploidentical donor, contraception. Donor haploidentical ≥4y/≥20kg. Exclude matched sibling, ECOG≥3, DLCO<35, O2 sat<85/PaO2<70, EF<35, infection, pregnant/breastfeeding, DSA≥2000, EBV− w/ EBV+ donor.
trial_source
clinical_trials.gov from Dec 2, 2025
annotation_status
ai
ai_summary
Phase I/II non-myeloablative haploidentical HCT for sickle cell disease with organ damage. Regimen adds briquilimab and abatacept to alemtuzumab–TBI–sirolimus, with filgrastim-mobilized donor PBSCs and post-transplant cyclophosphamide (PT-Cy, 1 vs 2 doses across cohorts). Drug types/mechanisms: briquilimab—anti-CD117 (c‑Kit) monoclonal antibody that depletes recipient HSCs to create marrow space, abatacept—CTLA4‑Ig fusion blocking CD28–CD80/86 costimulation to inhibit T‑cell activation/GVHD, alemtuzumab—anti‑CD52 mAb depleting T/B/NK cells, sirolimus—mTOR inhibitor suppressing T‑cell proliferation, PT‑Cy—alkylator eliminating proliferating alloreactive T cells, low-dose TBI—immunosuppression/space creation, filgrastim—G‑CSF to mobilize donor HSCs. Targets/pathways: CD117+ HSCs, CD52+ lymphocytes, T‑cell costimulation (CD28–CD80/86), mTOR signaling, proliferating allo‑T cells, marrow niche. Primary aim: engraftment without severe GVHD at 1 year.