eligibility_summary
Eligibility: Male, <6 years, ELAPRASE-naive, confirmed MPS II (I2S <=10% LLN with normal other sulfatase) plus documented severe IDS mutation predictive of persistent anti-idursulfase antibodies, negative anti-idursulfase Ab. Exclude: recent investigational drug/study (<30 d), idursulfase-IT, prior growth hormone/cord blood/BMT, transfusion <90 d, noncompliance, hypersensitivity (incl prophylactic ITR), interfering illness, or meds affecting disease/drug (<=30 d).
trial_source
clinical_trials.gov from Dec 2, 2025
annotation_status
ai
ai_summary
Trial: NCT05494593 (Phase 4, open-label, single-arm) in ELAPRASE-naïve boys with MPS II at high risk for persistent anti-drug antibodies. Interventions and mechanisms: • ELAPRASE (idursulfase) – recombinant enzyme replacement therapy, restores iduronate-2-sulfatase activity, enabling lysosomal degradation of dermatan/heparan sulfate via mannose-6-phosphate receptor–mediated cellular uptake. • Rituximab – anti-CD20 monoclonal antibody, depletes CD20+ B cells to prevent neutralizing antibody formation. • Methotrexate – antimetabolite immunosuppressant (DHFR inhibitor), suppresses B- and T-lymphocyte proliferation and reduces T-cell help for antibody responses. • IVIG – pooled immunoglobulin, immunomodulates via FcγR blockade, complement scavenging, and anti-idiotype effects to dampen humoral responses. Targets/pathways: CD20+ B cells (humoral immunity), germinal center/antibody production pathways, monitoring includes lymphocyte counts and CD19% recovery, lysosomal GAG catabolism restored by idursulfase.