NCT00171171

Brief Summary

Because patients with beta-thalassemia are unable to actively eliminate iron from the body, toxic and eventually lethal levels of iron can accumulate as a result of repeated blood transfusions. This study will evaluate the efficacy, safety and tolerability of deferasirox.

Trial Health

90
On Track

Trial Health Score

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

Enrollment
252

participants targeted

Target at P50-P75 for phase_3

Geographic Reach
5 countries

5 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, 2004

Completed
1.4 years until next milestone

First Submitted

Initial submission to the registry

September 12, 2005

Completed
3 days until next milestone

First Posted

Study publicly available on registry

September 15, 2005

Completed
1.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2006

Completed
Last Updated

March 1, 2017

Status Verified

November 1, 2016

Enrollment Period

2.5 years

First QC Date

September 12, 2005

Last Update Submit

February 28, 2017

Conditions

Beta-ThalassemiaHemosiderosis

Keywords

Thalassemia (beta-thal. major)Transfusional hemosiderosisDeferasiroxBeta-thalassemia major patientsUnable to be chelated with deferoxamine or deferiprone

Outcome Measures

Primary Outcomes (1)

  • Liver Iron Concentration (as measured by biopsy)

    at baseline and after 1 yeor of ICL670 treatment

Secondary Outcomes (5)

  • Iron balance, i.e. Total Body Iron Excretion (based on the iron influx as determined by the amount of red cells transfused and the change in total body iron (TBI) stores)

    after 1 year of ICL670 treatment

  • Putative surrogate markers such as serum ferritin serum iron, serum transferrin and transferrin saturation

    monthly assessments for 1 year

  • Adverse events

    during 1 year ICL670 treatment

  • hematology and biochemistry, urinalysis

    monthly assessments for 1 year ICL670 treatment

  • ECG and Echocardiography

    6-monthly for 1 year ICL670 treatment

Study Arms (1)

Deferasirox

EXPERIMENTAL
Drug: deferasirox

Interventions

deferasiroxDRUG
Deferasirox

Eligibility Criteria

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

You may qualify if:

  • Beta-thalassemia outpatients with transfusional hemosiderosis and unable to be chelated with deferoxamine (DFO) due to DFO being contra-indicated and/or due to documented unacceptable toxicity of DFO or documented poor response to DFO despite proper compliance, or documented non-compliance to DFO, with serum ferritin ≥ 500 ng/ml and LIC ≥ 2 mg/Fe/g dw liver
  • Beta-thalassemia outpatients with transfusional hemosiderosis treated with deferiprone

You may not qualify if:

  • Means levels of ALT \> 300 U/L
  • Serum creatinine above upper limit of normal
  • Active hepatitis C or chronic hepatitis B receiving specific treatment

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (5)

Novartis Investigative Site

Cairo, Egypt

Location

Ali Taher

Beirut, Riad El Solh 1107 2020, Lebanon

Location

Novartis Investigative Site

Muscat, Oman

Location

Novartis Investigative Site

Riyadh, Saudi Arabia

Location

Novartis Investigative Site

Damascus, Syria

Location

Related Publications (1)

  • Taher A, Al Jefri A, Elalfy MS, Al Zir K, Daar S, Rofail D, Baladi JF, Habr D, Kriemler-Krahn U, El-Beshlawy A. Improved treatment satisfaction and convenience with deferasirox in iron-overloaded patients with beta-Thalassemia: Results from the ESCALATOR Trial. Acta Haematol. 2010;123(4):220-5. doi: 10.1159/000313447. Epub 2010 Apr 27.

    PMID: 20424435RESULT

Related Links

MeSH Terms

Conditions

beta-ThalassemiaHemosiderosisThalassemia

Interventions

Deferasirox

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

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

Study Officials

  • Ali Taher, Ass.Prof.

    American University of Beirut Medical Center

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY

Study Record Dates

First Submitted

September 12, 2005

First Posted

September 15, 2005

Study Start

May 1, 2004

Primary Completion

November 1, 2006

Last Updated

March 1, 2017

Record last verified: 2016-11

Locations

EG(1)SA(1)OM(1)SY(1)LE(1)