Could a chemo drug tame a rare immune disorder?
NCT ID NCT07555405
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests the drug decitabine in 6 boys with XMEN disease, a rare genetic condition that weakens the immune system and damages the liver. The goal is to see if decitabine can improve liver function and immune cell activity. It is a very early, small trial focused on safety and signs of benefit.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Decitabine (a chemotherapy drug given by IV)
- What this could lead to
- If it works, this could point toward a treatment for XMEN disease, a rare immune disorder that makes patients prone to severe infections and liver problems.
- What could go wrong
- This is a very small, early trial with only 6 participants, so results may not apply to everyone. Decitabine is a chemotherapy drug and can cause serious side effects like low blood cell counts.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 4
Runs after approval, following long-term safety and how well the treatment works in everyday use.
- Participants
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About 6 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Apr 2026
An estimate. Start dates often move.
- Expected to finish
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Dec 2028
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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1 month to 18 years
- Sex
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Male participants only
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Male participants aged 1 month to 18 years old. 2. Confirmed MAGT1 gene mutation by genetic testing. 3. Clinical manifestations consistent with XMEN disease, including liver dysfunction and/or EBV infection. 4. Reduced lymphocyte NKG2D expression. 5. Vital signs within normal range at screening. 6. Expected survival ≥ 6 months. 7. Able to comply with study procedures. 8. Guardian and participant provide written informed consent. Exclusion Criteria: 1. Hypersensitivity to decitabine or any excipient. 2. Hematopoietic stem cell transplantation within 1 year before enrollment. 3. Severe concurrent organ dysfunction or systemic disease. 4. Positive HBsAg, anti-HCV, syphilis, or HIV test. 5. Neurological or psychiatric disorders that impair compliance. 6. Participation in another clinical trial within 3 months. 7. Other conditions judged inappropriate by the investigator.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, 201102, China