Fanconi AnemiaPhase 1NCT01720147
Quercetin in Children With Fanconi Anemia; a Pilot Study
A Phase 1 study testing Quercetin (dietary supplement) in people with Fanconi Anemia, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
Trial summary
Fanconi anemia (FA) is an autosomal recessive disease characterized by progressive bone marrow failure (BMF), congenital abnormalities and a predisposition to malignancy. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Fanconi Anemia
- What is being tested?
- Quercetin (dietary supplement)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2012to 26 Oct 2021
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of FA proven by DEB test or molecular testing
- Able to take enteral medication
- All age groups, including adults
Who cannot join
- Patients with morphological evidence of myelodysplasia or leukemia
- Renal failure requiring dialysis
- Total bilirubin > 3 mg/dl and/or SGPT >200 at time of enrollment
- 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 receiving cyclosporine or digoxin therapy or are unable to discontinue either treatment due to medical reasons
- Patients who have received quercetin supplementation or other antioxidants within the last 30 days
Trial locations and contacts
1 site in 1 country.
United States1 site
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
About this study
Fanconi anemia (FA) is an autosomal recessive disease characterized by progressive bone marrow failure (BMF), congenital abnormalities and a predisposition to malignancy.
Read the detailed description
Current therapies for children with Fanconi anemia (FA) and bone marrow failure, i.e. androgens or bone marrow transplantation, are associated with significant morbidity and mortality.
This is a pilot study aiming to assess feasibility, toxicity and pharmacokinetics of oral Quercetin therapy in patients with FA. This is a first step towards a clinical study of the efficacy of Quercetin therapy in delaying progression of BMF in FA.
Additional correlative studies will include assessment of impact of Quercetin on reduction of Reactive Oxygen Species (ROS), maintenance or improvement of hematopoietic stem cell (HSC) reserve, improvement of hematopoiesis (i.e. peripheral counts) and insulin sensitivity/glucose tolerance.
This study is an open-label single arm study.
Funding Source - FDA OOPD
Accrual closed for the main study. Expansion cohort added to observe additional patients at the desired dose.
Expansion Cohort:
The second and third cohort of participants completed the study treatment as expected, tolerating Quercetin well without any DLT. Based on the PK and ROS analyses, the dose was increased for the fourth cohort (subjects #10-12). To observe additional patients at the desired dose, an expansion cohort is being added. Up to 20 patients will be enrolled in the expansion cohort. The purpose of the expansion cohort is to assess the effects of quercetin supplement at the desired dose on clinical and biological endpoints.
Patients in the expansion cohort will receive quercetin treatment for the first 26 weeks. Patients will be able to continue quercetin supplement after the completion of the study period of 26 weeks if they wish. If patients decide to continue quercetin after the first 26 weeks, quercetin will be provided by the investigational pharmacy for up to 1 year. Patients who continue quercetin supplement after 26 weeks, but discontinue quercetin before 1 year will be followed through the 1 year visit. Patients who discontinue quercetin supplement any time after the 1 year visit will be followed through the 2 year visit.
Patients previously enrolled in the initial phase of the study (cohorts 1-4) may be considered for enrollment on the expansion cohort of the study provided they meet all inclusion and exclusion criteria.
- Conditions
- Fanconi Anemia
- Being tested
- Quercetin (dietary supplement)
- Keywords
- Fanconi Anemia, FA, Quercetin
- Medical subject headings
- Fanconi Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Quercetin (dietary supplement)Drug
Quercetin will be given orally on a twice a day schedule starting with weight adjusted dose for a maximum total daily dose of 1500 mg/day, for 4 months (16 weeks). Pharmacokinetics (PK) data will be analyzed after each cohort of 3 patients and will be used to optimize the dosing schedule (if required)for subsequent patients.
An expansion cohort has been added to the study protocol. Up to 20 patients may be enrolled. The dose utilized will be the same as the max weight adjusted dose that showed biological activity in our last cohort of patients (subjects #10-12 from above).
| Study group | Role | What they receive |
|---|---|---|
| Quercetin - Dietary Supplement | Experimental | Quercetin will be given orally on a twice a day schedule starting with weight adjusted dose for a maximum total daily dose of 1500 mg/day, for 4 months (16 weeks). Pharmacokinetics (PK) data will be analyzed after each cohort of 3 patients and will be used to optimize the dosing schedule (if required) for subsequent patients.
An expansion cohort has been added to the study protocol. Up to 20 patients may be enrolled. The dose utilized will be the same as the max weight adjusted dose that showed biological activity in our last cohort of patients (subjects #10-12 from above). Receives: Drug: Quercetin (dietary supplement) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Measure the ability to administer twice daily oral quercetin therapy in patients with Fanconi Anemia (FA).
Measured: 4 months (16 weeks)
Measure safety of oral quercetin therapy in patients with FA
Measured: 4 months (16 weeks)
To measure pharmacokinetics (PK) of oral quercetin therapy in patients with FA
Measured: 4 months (16 weeks)
Secondary outcomes (4)
To measure the impact of quercetin therapy on reduction of Reactive Oxygen Species (ROS).
Measured: 4 months (16 weeks) and 1 year
Number of participants with improved hematopoiesis.
Measured: 4 months (16 weeks) and 1 year
Measure the preservation of hematopoietic stem cell reserve in patients with FA
Measured: 4 months (16 weeks) and 1 year
To measure the impact of quercetin therapy on changes in insulin sensitivity/glucose tolerance.
Measured: 4 month (16 weeks) and 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Nov 2012 (estimated)
- Study startJul 2012
- Primary completion26 Oct 2021
- Study completion26 Oct 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Fanconi AnemiaStudies of the same condition, open and under way.
- More trials run by Children's Hospital Medical Center, CincinnatiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Collaborators
- Food and Drug Administration (FDA)
- Responsible party
- Sponsor
- Investigators
- Parinda Mehta, MD, Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01720147
- Sponsor's protocol ID
- 2011-2049
- Other IDs
- 1R01FD004383-01A1
- Status last verified
- Jan 2024
- First submitted
- 25 Jul 2012
- Last updated
- 18 Jan 2024
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Mehta PA, Nelson A, Loveless S, Lane A, Fukuda T, Teusink-Cross A, Elder D, Lagory D, Miller E, Cancelas JA, Howell J, Zhao J, Mizuno K, Myers KC, Lake K, McIntosh K, Setchell KDR, Luebbering N, Edwards S, Chihanga T, Wells SI, Davies SM. Phase 1 study of quercetin, a natural antioxidant for children and young adults with Fanconi anemia. Blood Adv. 2025 Apr 22;9(8):1927-1939. doi: 10.1182/bloodadvances.2024015053.
PubMed 39820512
Study at a glance
From the registry record, last updated by the sponsor on 18 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Quercetin in Children With Fanconi Anemia; a Pilot Study
- ClinicalTrials.gov ID
- NCT01720147
- Acronym / sponsor ID
- 2011-2049
- Conditions
- Fanconi Anemia
- Keywords
- Fanconi Anemia, FA, Quercetin
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Collaborators
- Food and Drug Administration (FDA)
- Study start
- Jul 2012
- Primary completion
- 26 Oct 2021 (actual)
- Study completion
- 26 Oct 2021 (actual)
- First posted
- 2 Nov 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Fanconi anemia (FA) is an autosomal recessive disease characterized by progressive bone marrow failure (BMF), congenital abnormalities and a predisposition to malignancy.
Detailed description
Current therapies for children with Fanconi anemia (FA) and bone marrow failure, i.e. androgens or bone marrow transplantation, are associated with significant morbidity and mortality.
This is a pilot study aiming to assess feasibility, toxicity and pharmacokinetics of oral Quercetin therapy in patients with FA. This is a first step towards a clinical study of the efficacy of Quercetin therapy in delaying progression of BMF in FA.
Additional correlative studies will include assessment of impact of Quercetin on reduction of Reactive Oxygen Species (ROS), maintenance or improvement of hematopoietic stem cell (HSC) reserve, improvement of hematopoiesis (i.e. peripheral counts) and insulin sensitivity/glucose tolerance.
This study is an open-label single arm study.
Funding Source - FDA OOPD
Accrual closed for the main study. Expansion cohort added to observe additional patients at the desired dose.
Expansion Cohort:
The second and third cohort of participants completed the study treatment as expected, tolerating Quercetin well without any DLT. Based on the PK and ROS analyses, the dose was increased for the fourth cohort (subjects #10-12). To observe additional patients at the desired dose, an expansion cohort is being added. Up to 20 patients will be enrolled in the expansion cohort. The purpose of the expansion cohort is to assess the effects of quercetin supplement at the desired dose on clinical and biological endpoints.
Patients in the expansion cohort will receive quercetin treatment for the first 26 weeks. Patients will be able to continue quercetin supplement after the completion of the study period of 26 weeks if they wish. If patients decide to continue quercetin after the first 26 weeks, quercetin will be provided by the investigational pharmacy for up to 1 year. Patients who continue quercetin supplement after 26 weeks, but discontinue quercetin before 1 year will be followed through the 1 year visit. Patients who discontinue quercetin supplement any time after the 1 year visit will be followed through the 2 year visit.
Patients previously enrolled in the initial phase of the study (cohorts 1-4) may be considered for enrollment on the expansion cohort of the study provided they meet all inclusion and exclusion criteria.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Quercetin - Dietary Supplement | Experimental | Drug: Quercetin (dietary supplement)Quercetin will be given orally on a twice a day schedule starting with weight adjusted dose for a maximum total daily dose of 1500 mg/day, for 4 months (16 weeks). Pharmacokinetics (PK) data will be analyzed after each cohort of 3 patients and will be used to optimize the dosing schedule (if required) for subsequent patients. An expansion cohort has been added to the study protocol. Up to 20 patients may be enrolled. The dose utilized will be the same as the max weight adjusted dose that showed biological activity in our last cohort of patients (subjects #10-12 from above). |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Diagnosis of FA proven by DEB test or molecular testing
- Able to take enteral medication
- All age groups, including adults
Key exclusion criteria (6 listed)
- Patients with morphological evidence of myelodysplasia or leukemia
- Renal failure requiring dialysis
- Total bilirubin > 3 mg/dl and/or SGPT >200 at time of enrollment
- 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 receiving cyclosporine or digoxin therapy or are unable to discontinue either treatment due to medical reasons
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Quercetin (dietary supplement)Quercetin will be given orally on a twice a day schedule starting with weight adjusted dose for a maximum total daily dose of 1500 mg/day, for 4 months (16 weeks). Pharmacokinetics (PK) data will be analyzed after each cohort of 3 patients and will be used to optimize the dosing schedule (if required)for subsequent patients. An expansion cohort has been added to the study protocol. Up to 20 patients may be enrolled. The dose utilized will be the same as the max weight adjusted dose that showed biological activity in our last cohort of patients (subjects #10-12 from above). | Drug | Quercetin - Dietary Supplement |
Primary outcome
Measure the ability to administer twice daily oral quercetin therapy in patients with Fanconi Anemia (FA).
Time frame: 4 months (16 weeks)
Measure safety of oral quercetin therapy in patients with FA
Time frame: 4 months (16 weeks)
To measure pharmacokinetics (PK) of oral quercetin therapy in patients with FA
Time frame: 4 months (16 weeks)
4 secondary outcomes
To measure the impact of quercetin therapy on reduction of Reactive Oxygen Species (ROS).
Time frame: 4 months (16 weeks) and 1 year
Number of participants with improved hematopoiesis.
Time frame: 4 months (16 weeks) and 1 year
Measure the preservation of hematopoietic stem cell reserve in patients with FA
Time frame: 4 months (16 weeks) and 1 year
To measure the impact of quercetin therapy on changes in insulin sensitivity/glucose tolerance.
Time frame: 4 month (16 weeks) and 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jan 2024.
- Derived from this study: PubMed 39820512Mehta PA, Nelson A, Loveless S, Lane A, Fukuda T, Teusink-Cross A, Elder D, Lagory D, Miller E, Cancelas JA, Howell J, Zhao J, Mizuno K, Myers KC, Lake K, McIntosh K, Setchell KDR, Luebbering N, Edwards S, Chihanga T, Wells SI, Davies SM. Phase 1 study of quercetin, a natural antioxidant for children and young adults with Fanconi anemia. Blood Adv. 2025 Apr 22;9(8):1927-1939. doi: 10.1182/bloodadvances.2024015053.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Parinda Mehta, MD principal investigator
Children's Hospital Medical Center, Cincinnati
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Quercetin (dietary supplement) in people with Fanconi Anemia, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Measure the ability to administer twice daily oral quercetin therapy in patients with Fanconi Anemia (FA). (time frame: 4 months (16 weeks)). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01720147, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Jan 2024.