Combo Chelation Trial

• ClinicalTrials.gov Identifier: NCT00901199
• Recruitment Status: Completed
• First Posted: May 13, 2009
• Last Update Posted: August 7, 2013
• Sponsor: UCSF Benioff Children's Hospital Oakland
• Information provided by (Responsible Party): Elliott Vichinsky, UCSF Benioff Children's Hospital Oakland

Tracking Information

• First Submitted Date: May 12, 2009
• First Posted Date: May 13, 2009
• Last Update Posted Date: August 7, 2013
• Study Start Date: September 2007
• Actual Primary Completion Date: October 2012 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: May 12, 2009): Monitoring kidney function with urine protein/creatine levels [ Time Frame: with transfusion ]
• Original Primary Outcome Measures: Same as current
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Combo Chelation Trial
• Official Title: Safety of Deferasirox (ICL670) and Deferoxamine (Desferal or DFO) Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload
• Brief Summary: This is a small pilot study looking at the safety of giving combination chelation with Exjade and Desferal to 15 patients. The hypothesis is that combination chelation is safe in decreasing overall iron in patients with thalassemia.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 4
• Study Design: Allocation: Non-Randomized; Intervention Model: Parallel Assignment; Masking: None (Open Label); Primary Purpose: Treatment

Condition

• Thalassemia
• Iron Overload

Intervention

Drug: Combo Chelation with Deferasirox (Exjade) and Desferal (DFO)

All subjects will be given Exjade 7 days per week 20-30mg/kg and Deferoxamine 3-7 days per week 50mg/kg. Days per week are dependent on how much iron overload subject has.

Other Names:

• Exjade, ICL670 (deferisirox)
• DFO, Desferal (deferoxamine)

Study Arms

• Child Cohort
  This is the cohort in the study for children ages 8-18 years old. All subjects in this arm must have Liver Iron by SQUID of between 5-15mg/g dry liver and have a documented endocrinopathy or cardiac finding (low T2* or decreased cardiac function). All subjects in this arm will receive 7 days per week of Exjade and 3-5 days per week of Desferal.
  Intervention: Drug: Combo Chelation with Deferasirox (Exjade) and Desferal (DFO)
• Moderate Adult Cohort
  Adults in this arm will have moderate iron overload,defined as SQUID of 5-15mg/g dry weight. They will also have to have a documented endocrinopathy or cardiac finding (low T2*). All subjects in this cohort will receive 7 days per week of Exjade (20-30mg/kg) and Desferal (50mg/kg)3-5 days per week.
  Intervention: Drug: Combo Chelation with Deferasirox (Exjade) and Desferal (DFO)
• Adults cohort with high iron overload
  Adults with high iron overload defined as over 15mg/g dry liver. No cardiac finding or endocrinopathy necessary. Subjects in this cohort will receive Exjade 20-30mg/kg 7 days per week and Desferal (50mg/kg)5-7 days per week.
  Intervention: Drug: Combo Chelation with Deferasirox (Exjade) and Desferal (DFO)

• Publications *: Lal A, Porter J, Sweeters N, Ng V, Evans P, Neumayr L, Kurio G, Harmatz P, Vichinsky E. Combined chelation therapy with deferasirox and deferoxamine in thalassemia. Blood Cells Mol Dis. 2013 Feb;50(2):99-104. doi: 10.1016/j.bcmd.2012.10.006. Epub 2012 Nov 11.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: August 5, 2013): 22
• Original Estimated Enrollment (submitted: May 12, 2009): 15
• Actual Study Completion Date: October 2012
• Actual Primary Completion Date: October 2012 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Transfusion Dependent Thalassemia
• If iron between 5-15 mg/g dry liver by SQUID, subject must have a documented endocrinopathy or cardiac finding
• Older than 8 years

Exclusion Criteria:

• Participating on another interventional clinical trial

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 8 Years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT00901199
• Other Study ID Numbers: CICL670AUS24T
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Responsible Party: Elliott Vichinsky, UCSF Benioff Children's Hospital Oakland
• Study Sponsor: UCSF Benioff Children's Hospital Oakland
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: UCSF Benioff Children's Hospital Oakland
• Verification Date: August 2013