Pharmacogenetic Study in Patients Received Iron Chelating Agent

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Hyoung Jin Kang, Seoul National University Hospital
ClinicalTrials.gov Identifier:
NCT01623895
First received: June 12, 2012
Last updated: July 11, 2014
Last verified: December 2013
  Purpose

To investigate effect of genetic variations on the toxicities and find optimal target population, the investigators planned to analyze the genetic polymorphisms of UDP-glucuronosyltransferase.


Condition
Hemosiderosis

Study Type: Observational
Study Design: Observational Model: Case-Only
Time Perspective: Prospective
Official Title: Pharmacogenetic Study in Patients Received Iron Chelating Agent

Resource links provided by NLM:


Further study details as provided by Seoul National University Hospital:

Primary Outcome Measures:
  • Genetic polymorphism associated with side effects of deferasirox [ Time Frame: up to 1 year ] [ Designated as safety issue: No ]

    Genetic polymorphism associated with side effects of deferasirox

    - Side effects:

    Increased AST or ALT > 5 x ULN or increased bilirubin > 3 x ULN which was thought to be caused by deferasirox Serum creatinine level increase > 50% above the baseline value.

    • Biospecimen Retention: Samples With DNA
    • Candidate genes exhibit polymorphisms and encodes proteins that are involved in the pharmacokinetics and pharmacodynamics of deferasirox.

    Candidate genes : MRP2, BCRP, UGT1A subfamily



Biospecimen Retention:   Samples With DNA

Candidate genes exhibit polymorphisms and encodes proteins that are involved in the pharmacokinetics and pharmacodynamics of deferasirox.

Candidate genes : MRP2, BCRP, UGT1A subfamily


Estimated Enrollment: 100
Study Start Date: December 2007
Study Completion Date: June 2013
Primary Completion Date: June 2013 (Final data collection date for primary outcome measure)
Detailed Description:

Transfusion-associated iron overload induces systemic toxicity. Recently, deferasirox, a convenient long acting oral agent, has been introduced in clinical practice with promising efficacy. However, some patients experience drug-related toxicities and cannot tolerate it. To investigate effect of genetic variations on the toxicities and find optimal target population, we planned to analyze the genetic polymorphisms of UDP-glucuronosyltransferase 1A (UGT1A) subfamily, multi-drug resistance-associated protein 2 (MRP2) and breast cancer resistance protein (BCRP) among pediatric patients received deferasirox.

  Eligibility

Ages Eligible for Study:   up to 21 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Patients who received deferasirox because of transfusion associated iron overload

Criteria

Inclusion Criteria:

  1. Patients who received deferasirox because of transfusion associated iron overload (Transfusion associated iron overload was defined as ferritin ≥ 1,000 ng/mL in patients who needed over 8 units of RBC transfusions per a year).
  2. Patients with written informed consents

Exclusion Criteria:

Patients or parents refusal

  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: NCT01623895

Locations
Korea, Republic of
Seoul National University Hospital
Seoul, Chongno-gu, Korea, Republic of
Sponsors and Collaborators
Seoul National University Hospital
  More Information

No publications provided

Responsible Party: Hyoung Jin Kang, MD. Ph D., Professor, Seoul National University Hospital
ClinicalTrials.gov Identifier: NCT01623895     History of Changes
Other Study ID Numbers: SNUCH-R-0701
Study First Received: June 12, 2012
Last Updated: July 11, 2014
Health Authority: Korea: Food and Drug Administration

Additional relevant MeSH terms:
Hemosiderosis
Iron Overload
Iron Metabolism Disorders
Metabolic Diseases
Chelating Agents
Iron Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on August 18, 2014