NCT00901199

Brief Summary

This is a pilot study looking at the safety and efficacy of giving combination chelation with deferasirox and deferoxamine. The hypothesis is that combination chelation is safe in decreasing overall iron in patients with thalassemia.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
22

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Sep 2007

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

September 1, 2007

Completed
1.7 years until next milestone

First Submitted

Initial submission to the registry

May 12, 2009

Completed
1 day until next milestone

First Posted

Study publicly available on registry

May 13, 2009

Completed
3.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2012

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2012

Completed
8.9 years until next milestone

Results Posted

Study results publicly available

August 12, 2021

Completed
Last Updated

August 12, 2021

Status Verified

July 1, 2021

Enrollment Period

5.1 years

First QC Date

May 12, 2009

Results QC Date

June 14, 2021

Last Update Submit

July 20, 2021

Conditions

ThalassemiaIron Overload

Keywords

ThalassemiaChelationIron OverloadThalassemia patients with Iron overload

Outcome Measures

Primary Outcomes (2)

  • Efficacy of Combined Treatment With Deferasirox and Deferoxamine Over 12 Months

    Change in liver iron concentration from baseline to 12 months with the use of combined chelation therapy. The change was calculated as the liver iron concentration at 12 months minus the value at baseline.

    12 months

  • Change in Serum Creatinine During 12 Months Combined Chelation Therapy

    Comparison of average serum creatinine over 12 months of combined chelation therapy compared with baseline serum creatinine.

    12 months

Study Arms (1)

Deferasirox (Exjade) and Deferoxamine (DFO)

EXPERIMENTAL

All subjects received Deferasirox (Exjade) and Deferoxamine (DFO) dosing based on the iron overload at baseline.

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

Interventions

Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO)DRUG

All subjects will be given Deferasirox 20-30 mg/kg for 7 days per week. All subject will be given Deferoxamine 50 mg/kg for 3-7 days per week. The number of days for Deferoxamine will be determined by liver iron concentration at baseline.

Also known as: Deferasirox, (Exjade), Deferoxamine (DFO)
Deferasirox (Exjade) and Deferoxamine (DFO)

Eligibility Criteria

Age8 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • Participating on another interventional clinical trial

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

CHRCO

Oakland, California, 94609, United States

Location

Related Publications (1)

  • 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.

    PMID: 23151373RESULT

MeSH Terms

Conditions

ThalassemiaIron Overload

Interventions

DeferasiroxDeferoxamine

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesIron Metabolism DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

BenzoatesAcids, CarbocyclicCarboxylic AcidsOrganic ChemicalsBenzene DerivativesHydrocarbons, AromaticHydrocarbons, CyclicHydrocarbonsTriazolesAzolesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsHydroxamic AcidsHydroxylaminesAminesHydroxy Acids

Results Point of Contact

Title
Ashutosh Lal, MD
Organization
Children's Hospital & Research Center at Oakland
ashutosh.lal@ucsf.edu
510-428-3172

Study Officials

  • Elliot Vichinsky, MD

    UCSF Benioff Children's Hospital Oakland

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Director, Hematology/Oncology

Study Record Dates

First Submitted

May 12, 2009

First Posted

May 13, 2009

Study Start

September 1, 2007

Primary Completion

October 1, 2012

Study Completion

October 1, 2012

Last Updated

August 12, 2021

Results First Posted

August 12, 2021

Record last verified: 2021-07

Locations

US(1)