Evaluating the Safety and Efficacy of Decitabine in the Treatment of XMEN Patients

This is a single-arm, open-label, single-center, exploratory clinical trial evaluating the safety and efficacy of decitabine in male patients aged 1 month to 18 years with X-linked magnesium transporter 1 (MAGT1) deficiency. Eligible patients have a confirmed MAGT1 gene mutation leading to XMEN disease ( X-linked MAGT1 deficiency with increased susceptibility to Epstein-Barr virus (EBV) infection and N-linked glycosylation defect). The study will assess changes in liver function, immune function, and NKG2D expression, as well as adverse events, over four treatment cycles and the follow-up period.

NCT Number	NCT07555405
Lead Sponsor	Children's Hospital of Fudan University Collaborators: National Natural Science Foundation of China
Conditions	MAGT1 Deficiency
Enrollment	6 participants
Start Date	2026-04
Primary Completion	2028-12 (estimated)
Study Completion	2028-12 (estimated)
Updated on ClinicalTrials.gov	2026-04-29