Evaluating the Safety and Efficacy of Decitabine in the Treatment of XMEN Patients
Starting soon · Phase 4
Conditions studied: MAGT1 Deficiency
In brief
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.
Key facts
- Study ID
- NCT07555405
- Run by
- Children's Hospital of Fudan University
- People needed
- 6
- Starts
- 2026-04-01
- Expected to finish
- 2028-12-01
- Last updated by the study team
- 2026-04-29
Who can join
Age: 0 and older, up to 18. Sex: male. Healthy volunteers: not accepted.
You may qualify if…
- 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.
You may not qualify if…
- Hypersensitivity to decitabine or any excipient.
- Hematopoietic stem cell transplantation within 1 year before enrollment.
- Severe concurrent organ dysfunction or systemic disease.
- Positive HBsAg, anti-HCV, syphilis, or HIV test.
- Neurological or psychiatric disorders that impair compliance.
- Participation in another clinical trial within 3 months.
- Other conditions judged inappropriate by the investigator.
Where it is running
- Children's Hospital of Fudan University — Shanghai, Shanghai Municipality, China
Full record on ClinicalTrials.gov
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