Quercetin for males with XIAP deficiency
Official title: A Pilot Study to Evaluate Safety and Tolerability, Pharmacokinetics, and Preliminary Efficacy of Quercetin in Patients With XIAP (X-linked Inhibitor of Apoptosis) Deficiency
Run by Children's Hospital Medical Center, Cincinnati · NCT07433621 · Data updated 2026-11-12
This trial will test whether taking quercetin by mouth safely lowers inflammation in males with XIAP deficiency.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Ages | 2 Years and up |
| Sex | Men only |
| Enrollment | 12 (estimated) |
| Sponsor | Children's Hospital Medical Center, Cincinnati Academic / other |
| Locations | 1 site (Cincinnati, Ohio) |
| Status | Recruiting |
| Started | February 26, 2026 |
| Main results expected | November 2028 |
| Study ends | June 2029 |
| Registry record updated | March 27, 2026 |
| Trial ID | NCT07433621 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Male patients aged 2 years and older with a confirmed diagnosis of XIAP deficiency, serum IL-18 ≥600 pg/mL, and the ability to take oral medication are the intended candidates for this study.
✕Probably not a fit
Patients with severe organ dysfunction (for example renal failure needing dialysis or very high liver tests), those on prohibited interacting medications, patients with unstable disease needing rapid escalation of care, or those unable to use required contraception are unlikely to be eligible or to benefit.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
XIAP deficiency is a rare genetic immune disorder that causes excessive inflammation and can lead to life-threatening complications such as HLH and severe inflammatory bowel disease. This Phase 1, interventional study gives oral quercetin to male patients with confirmed XIAP deficiency and elevated IL-18 to evaluate safety and tolerability. Participants must be able to take enteral medication and meet lab criteria, and they will be monitored for adverse events and changes in inflammatory markers including IL-18. The primary focus is safety, with secondary observations of inflammatory signal changes rather than definitive efficacy conclusions.
Why it matters
Potential benefit: If successful, quercetin could offer a low-risk, oral option to reduce inflammation and potentially delay or reduce the need for high-risk curative transplantation.
How similar studies have gone: Quercetin has shown anti-inflammatory effects in preclinical models and small clinical studies for other conditions, but its use in XIAP deficiency is novel and has not been validated in larger trials.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * Male patients * Diagnosis of XIAP deficiency * Patients ≥2 years of age * Serum IL-18 ≥600 pg/mL (approximately 25% above the lab-defined upper limit of normal of 477 pg/mL) * Able to take enteral medication Exclusion Criteria: * Renal failure requiring dialysis * Total bilirubin \>3 mg/dl and/or SGPT \>300 at time of enrollment * Patients receiving digoxin therapy, who are unable to discontinue treatment due to medical reasons * Patients receiving fluoroquinolone therapy, who are unable to discontinue treatment due to medical reasons * Patients who are at risk of pregnancy or fathering a child and are unable to use acceptable methods of birth control during the length of the study * Patients who have received quercetin or any over the counter anti-oxidant supplementation within last 1 month * Patients with unstable disease status or other medical issues requiring hospitalization or rapid escalation of medical care * Participating in another therapeutic study for XIAP deficiency
Where it's running
-
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States Recruiting
Study contacts
- Principal investigator: Rebecca Marsh, MD — Children's Hospital Medical Center, Cincinnati
- Study coordinator: Kelly McIntosh
- Email: Kelly.mcintosh@cchmc.org
- Phone: 513-803-0460
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.