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New treatment option for Dyskeratosis Congenita
Official title Quercetin Dyskeratosis Congenita (DC)/Telomere Biology Disorders (TBD)
ClinicalTrials.gov ID: NCT07628972
What this study is testing
What is Quercetin?
Quercetin is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for dyskeratosis congenita.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The purpose of this study is to see if a vitamin-like substance called quercetin is safe for people who have a rare condition called Dyskeratosis congenita (DC) or telomere biology disorders (TBD).
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 and older
You may be able to join if
- Diagnosis of DC/TBD deficiency as defined by at least one of the following:
- Age adjusted mean-telomere length of \<1 percentile in all tested peripheral blood cells such as granulocytes, lymphocytes, B-cells, naïve T-cells...
- A pathogenic or likely pathogenic mutation in DKC1, TERC, TERT, NOP10, NHP2, TINF2, CTC1, PARN, RTEL1, ACD, NAF1, ZCCHC8, or WRAP53
- Patients ≥ 2.0 years of age\
- The first three enrolled patients must be ≥ 10.0 years of age
You likely can't join if
- Renal failure requiring dialysis
- Total bilirubin \>3 mg/dl and/or SGPT \>300 at time of enrollment, unless elevation thought to be related to DC/TBD
- Patients who have received quercetin or any over-the-counter antioxidant supplementation within last 1 month
- Patients currently taking androgen therapy
- 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
See the full eligibility criteria
- Diagnosis of DC/TBD deficiency as defined by at least one of the following:
- Age adjusted mean-telomere length of \<1 percentile in all tested peripheral blood cells such as granulocytes, lymphocytes, B-cells, naïve T-cells, memory T-cells, and NK cells
- A pathogenic or likely pathogenic mutation in DKC1, TERC, TERT, NOP10, NHP2, TINF2, CTC1, PARN, RTEL1, ACD, NAF1, ZCCHC8, or WRAP53
- Patients ≥ 2.0 years of age\
- The first three enrolled patients must be ≥ 10.0 years of age
- Able to take medication orally
- Renal failure requiring dialysis
- Total bilirubin \>3 mg/dl and/or SGPT \>300 at time of enrollment, unless elevation thought to be related to DC/TBD
- Patients who have received quercetin or any over-the-counter antioxidant supplementation within last 1 month
- Patients currently taking androgen therapy
- 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 pregnant or breastfeeding or are at risk of pregnancy and are unable to use acceptable methods of birth control during the length of the study
- Patients with morphologic or cytogenetic evidence of myelodysplasia or leukemia.
- Patients needing to start or actively receiving radiation therapy, chemotherapy or immunotherapy for treatment of SCC or other cancers.
- Patients with unstable disease status or other medical issues requiring hospitalization or rapid escalation of medical care
- Participating in another therapeutic study for DC/TBD
- Patients who are in the early post-stem cell transplant period (i.e. first 6 months post-transplant)
The study team makes the final eligibility decision.
Where it's taking place
- Cincinnati, Ohio, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Cincinnati, Ohio, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.