NCT00061750

Brief Summary

The purpose of this study is to deterimine if the new orally active iron chelator, ICL670, is as effective and as safe as deferoxamine in preventing accumulation of iron in the body while a patient is undergoing repeated blood transfusions.

Trial Health

80
On Track

Trial Health Score

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

Enrollment
595

participants targeted

Target at P75+ for phase_3

Geographic Reach
1 country

7 active sites

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

May 1, 2003

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

June 3, 2003

Completed
1 day until next milestone

First Posted

Study publicly available on registry

June 4, 2003

Completed
1.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2004

Completed
Last Updated

April 19, 2012

Status Verified

April 1, 2012

Enrollment Period

1.5 years

First QC Date

June 3, 2003

Last Update Submit

April 18, 2012

Conditions

Beta-Thalassemia

Keywords

Thalassemia, iron overload, deferoxamine, hemosiderosis

Outcome Measures

Primary Outcomes (1)

  • Demonstrate non-inferiority to deferoxamine in its effects on liver iron content (LIC)

Secondary Outcomes (4)

  • Evaluate tolerability profile

  • Estimate absolute and relative change of LIC and Total body iron excretion

  • Evaluation relationship between LIC and potential surrogate markers

  • Evaluate the relationship between pharmacokinetics, pharmacodynamics and safety variable

Study Arms (2)

ICL670

EXPERIMENTAL
Drug: ICL670

Deferoxamine

ACTIVE COMPARATOR
Drug: deferoxamine

Interventions

ICL670DRUG
Also known as: Deferasirox
ICL670
deferoxamineDRUG
Deferoxamine

Eligibility Criteria

Age2 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Beta-thalassemia patients already treated with or suitable for treatment with deferoxamine 20 to 40 mg/kg/day
  • Liver iron content greater than 2 mg iron/g dw as measured by liver biopsy
  • Need for regular transfusions 8 or more times per year

You may not qualify if:

  • Non-transfusional iron overload or transfusion-dependent anemias other than beta-thalassemia.
  • Documented toxicity to deferoxamine
  • Elevated liver enzymes in the year preceeding enrollment
  • Active hepatitis B or hepatitis C
  • HIV seropositivity
  • Elevated serum creatinine or significant proteinuria
  • History of nephrotic syndrome
  • Uncontrolled systemic hypertension
  • Fever and other signs/symptoms of infection within 10 days prior to start of the study
  • Presence of clinically relevant cataract or previous history of clinically relevant ocular toxicity related to iron chelation
  • Second or third degree AV block, clinically relevant Q-T interval prolongation, or patients requiring digoxin or other drugs that prolong the Q-T interval
  • Diseases (cardiovascular, renal, hepatic, etc.)that would prevent the patient from undergoing any of the treatment options
  • Psychiatric or additive disorders that would prevent the patient from giving informed consent
  • History of drug or alcohol abuse within the 12 months prior to the study
  • Pregnant or breast feeding patients
  • +6 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

Children's Hospital of Los Angeles

Los Angeles, California, 90027-6062, United States

Location

Children's Hospital Oakland

Oakland, California, 94609, United States

Location

Stanford Hospital

Stanford, California, 94305-5208, United States

Location

Children's Memorial Hospital

Chicago, Illinois, 60614-3394, United States

Location

Children's Hospital Boston

Boston, Massachusetts, 02115, United States

Location

Weill Medical College of Cornell University

New York, New York, 10021, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104-4318, United States

Location

Related Publications (2)

  • Padhani ZA, Gangwani MK, Sadaf A, Hasan B, Colan S, Alvi N, Das JK. Calcium channel blockers for preventing cardiomyopathy due to iron overload in people with transfusion-dependent beta thalassaemia. Cochrane Database Syst Rev. 2023 Nov 17;11(11):CD011626. doi: 10.1002/14651858.CD011626.pub3.

    PMID: 37975597DERIVED

  • Cohen AR, Glimm E, Porter JB. Effect of transfusional iron intake on response to chelation therapy in beta-thalassemia major. Blood. 2008 Jan 15;111(2):583-7. doi: 10.1182/blood-2007-08-109306. Epub 2007 Oct 19.

    PMID: 17951527DERIVED

MeSH Terms

Conditions

beta-ThalassemiaThalassemiaIron OverloadHemosiderosis

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

Study Officials

  • Novartis Pharmaceuticals

    Novartis Pharmaceuticals

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 3, 2003

First Posted

June 4, 2003

Study Start

May 1, 2003

Primary Completion

November 1, 2004

Last Updated

April 19, 2012

Record last verified: 2012-04

Locations

US(7)