eligibility_summary
Include: confirmed CGD, significant complications or low oxidase (Q1–2), age 4–65, unrelated matched donor (no matched related), able to stay within 1 hr of NIH for 3 mo with companion, provide health-care DPOA, contraception required (start ≥1 mo pre-baseline to 1 yr if can conceive, to 3 mo if can impregnate). Exclude: ECOG ≥3, LVEF <40%, AST/ALT >5×ULN, CRP >100 mg/dL, major illness/organ failure, severe psych issues, pregnant/breastfeeding, HIV+, seizures, noncompliance, not eligible/consenting for alemtuzumab program.
trial_source
clinical_trials.gov from Dec 2, 2025
annotation_status
ai
ai_summary
NCT05600907: Early Phase 1, single-arm pilot in CGD assessing JSP191 in conditioning for matched-unrelated donor HSCT. Interventions/mechanisms: - JSP191 (biologic, anti-CD117/c-Kit monoclonal antibody): blocks SCF–c-Kit signaling to deplete/disable recipient hematopoietic stem/progenitor cells, facilitating donor engraftment (busulfan-sparing). - Alemtuzumab (biologic, anti-CD52 mAb): lymphodepletes T and B cells (and some dendritic/monocytes) to reduce rejection/GvHD. - Total body irradiation: cytotoxic to marrow and immune cells, aiding space/immune suppression. - Post-transplant cyclophosphamide (alkylating agent): preferentially eliminates proliferating alloreactive T cells to prevent GvHD. Targets/pathways: CD117+ hematopoietic stem/progenitor cells (c-Kit/SCF pathway), CD52+ lymphocytes, alloreactive T cells, and broader hematopoietic/immune compartments via TBI. Primary aim: improve donor engraftment.