Now Available: Final Rule for FDAAA 801 and NIH Policy on Clinical Trial Reporting

Quercetin in Children With Fanconi Anemia; a Pilot Study

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2016 by Children's Hospital Medical Center, Cincinnati
Sponsor:
Information provided by (Responsible Party):
Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier:
NCT01720147
First received: July 25, 2012
Last updated: August 19, 2016
Last verified: August 2016
  Purpose
Fanconi anemia (FA) is an autosomal recessive disease characterized by progressive bone marrow failure (BMF), congenital abnormalities and a predisposition to malignancy.

Condition Intervention Phase
Fanconi Anemia
Drug: Quercetin (dietary supplement)
Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Prevention
Official Title: Quercetin in Children With Fanconi Anemia; a Pilot Study

Resource links provided by NLM:


Further study details as provided by Children's Hospital Medical Center, Cincinnati:

Primary Outcome Measures:
  • Measure the ability to administer twice daily oral quercetin therapy in patients with Fanconi Anemia (FA). [ Time Frame: 4 months (16 weeks) ] [ Designated as safety issue: Yes ]
  • Measure safety of oral quercetin therapy in patients with FA [ Time Frame: 4 months (16 weeks) ] [ Designated as safety issue: Yes ]
  • To measure pharmacokinetics (PK) of oral quercetin therapy in patients with FA [ Time Frame: 4 months (16 weeks) ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • To measure the impact of quercetin therapy on reduction of Reactive Oxygen Species (ROS). [ Time Frame: 4 months (16 weeks) and 1 year ] [ Designated as safety issue: Yes ]
  • Number of participants with improved hematopoiesis. [ Time Frame: 4 months (16 weeks) and 1 year ] [ Designated as safety issue: Yes ]
  • Measure the preservation of hematopoietic stem cell reserve in patients with FA [ Time Frame: 4 months (16 weeks) and 1 year ] [ Designated as safety issue: Yes ]
  • To measure the impact of quercetin therapy on changes in insulin sensitivity/glucose tolerance. [ Time Frame: 4 month (16 weeks) and 1 year ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 12
Study Start Date: July 2012
Estimated Study Completion Date: October 2017
Estimated Primary Completion Date: September 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 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.
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.

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

  Eligibility

Ages Eligible for Study:   Child, Adult, Senior
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of FA proven by DEB test
  • Able to take enteral medication
  • All age groups, including adults

Exclusion Criteria:

  • 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01720147

Contacts
Contact: Stephanie Edwards, BSN 513-636-9292 stephanieL.edwards@cchmc.org
Contact: Sara Loveless 513-803-7656 Sara.Loveless@cchmc.org

Locations
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Principal Investigator: Parinda Mehta, MD         
Sponsors and Collaborators
Children's Hospital Medical Center, Cincinnati
Investigators
Principal Investigator: Parinda Mehta, MD Children's Hospital Medical Center, Cincinnati
  More Information

Responsible Party: Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier: NCT01720147     History of Changes
Other Study ID Numbers: 2011-2049  1R01FD004383-01A1 
Study First Received: July 25, 2012
Last Updated: August 19, 2016
Health Authority: United States: Food and Drug Administration
Individual Participant Data  
Plan to Share IPD: No

Keywords provided by Children's Hospital Medical Center, Cincinnati:
Fanconi Anemia
FA
Quercetin

Additional relevant MeSH terms:
Anemia
Fanconi Anemia
Anemia, Hypoplastic, Congenital
Anemia, Aplastic
Fanconi Syndrome
Hematologic Diseases
Bone Marrow Diseases
Genetic Diseases, Inborn
DNA Repair-Deficiency Disorders
Metabolic Diseases
Renal Tubular Transport, Inborn Errors
Kidney Diseases
Urologic Diseases
Metabolism, Inborn Errors
Quercetin
Antioxidants
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on December 05, 2016