Actively Recruiting
Pilot Study of Quercetin in Patients With Dyskeratosis Congenita and Telomere Biology Disorders
Led by Children's Hospital Medical Center, Cincinnati · Updated on 2026-06-05
12
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety of a vitamin-like substance called quercetin in people with a rare condition known as Dyskeratosis congenita (DC) or telomere biology disorders (TBD). This pilot study is open-label and single-arm, involving about 12 patients with DC or TBD. The study aims to understand how quercetin affects these patients over a 24-week period. Participants will receive quercetin orally twice daily, with doses adjusted based on their weight. The maximum total daily dose is 4000 mg, given in divided doses. For patients weighing 70 kg or more, the starting dose is set at the maximum level. The quercetin is supplied as a powder to be mixed with food like yogurt for home administration. During the study, participants will be monitored for any treatment-related side effects and for reasons that might cause them to stop taking quercetin. Assessments will include safety evaluations over the 24 weeks of treatment. The study is led by Children's Hospital Medical Center, Cincinnati, and aims to carefully track how participants respond to quercetin throughout the trial period.
CONDITIONS
Brief Title
Quercetin Dyskeratosis Congenita (DC)/Telomere Biology Disorders (TBD)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of Dyskeratosis Congenita or telomere biology disorder confirmed by specific genetic mutations or very short telomere length in blood cells
- Age 2 years or older (first three enrolled patients must be at least 10 years old)
- Able to take medication orally
You will not qualify if you...
- Renal failure requiring dialysis
- Total bilirubin over 3 mg/dl and/or SGPT over 300 unless related to DC/TBD
- Use of quercetin or any antioxidant supplements within the last month
- Current androgen therapy
- Receiving digoxin or fluoroquinolone therapy and unable to stop treatment
- Pregnant, breastfeeding, or at risk of pregnancy without acceptable birth control
- Evidence of myelodysplasia or leukemia
- Starting or currently receiving radiation, chemotherapy, or immunotherapy for cancer
- Unstable disease status needing hospitalization or rapid medical care escalation
- Participation in another therapeutic study for DC/TBD
- Within first 6 months after stem cell transplant
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 24 weeks
Participants receive oral quercetin twice daily for 24 weeks to evaluate safety and feasibility.
Visits at baseline and periodic follow-ups during treatment
Trial Site Locations
Total: 1 location
1
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
Research Team
S
Sara Loveless
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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