Evaluating the Safety and Efficacy of Decitabine in the Treatment of XMEN Patients (NCT07555405) | Clinical Trial Compass
Not Yet RecruitingPhase 4
Evaluating the Safety and Efficacy of Decitabine in the Treatment of XMEN Patients
China6 participantsStarted 2026-04
Plain-language summary
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.
Who can participate
Age range1 Month – 18 Years
SexMALE
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. Male participants aged 1 month to 18 years old.
✓. Confirmed MAGT1 gene mutation by genetic testing.
✓. Clinical manifestations consistent with XMEN disease, including liver dysfunction and/or EBV infection.
✓. Reduced lymphocyte NKG2D expression.
✓. Vital signs within normal range at screening.
✓. Expected survival ≥ 6 months.
✓. Able to comply with study procedures.
✓. Guardian and participant provide written informed consent.
Exclusion criteria
✕. Hypersensitivity to decitabine or any excipient.
✕. Hematopoietic stem cell transplantation within 1 year before enrollment.
✕. Severe concurrent organ dysfunction or systemic disease.
What they're measuring
1
Improvement magnitude of serum liver enzyme levels
Timeframe: up to 6 months after the last dose
2
Changes in NKG2D expression levels
Timeframe: up to 6 months after the last dose
3
Cumulative incidence of grade ≥3 myelosuppression
Timeframe: up to 6-month follow-up period after the last dose