A Study of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis

This study has been completed.
Sponsor:
Information provided by:
Novartis
ClinicalTrials.gov Identifier:
NCT00171171
First received: September 12, 2005
Last updated: May 5, 2010
Last verified: May 2010
  Purpose

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.


Condition Intervention Phase
Beta-Thalassemia
Hemosiderosis
Drug: deferasirox
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Study of Efficacy and Safety of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • Liver Iron Concentration (as measured by biopsy) [ Time Frame: at baseline and after 1 yeor of ICL670 treatment ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • 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) [ Time Frame: after 1 year of ICL670 treatment ] [ Designated as safety issue: No ]
  • Putative surrogate markers such as serum ferritin serum iron, serum transferrin and transferrin saturation [ Time Frame: monthy assessments for 1 year ] [ Designated as safety issue: No ]
  • Adverse events [ Time Frame: during 1 year ICL670 treatment ] [ Designated as safety issue: Yes ]
  • hematology and biochemistry, urinalysis [ Time Frame: monthly assessments for 1 year ICL670 treatment ] [ Designated as safety issue: Yes ]
  • ECG and Echocardiography [ Time Frame: 6-monthly for 1 year ICL670 treatment ] [ Designated as safety issue: Yes ]

Enrollment: 252
Study Start Date: May 2004
Primary Completion Date: November 2006 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Deferasirox Drug: deferasirox

  Eligibility

Ages Eligible for Study:   2 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • Means levels of ALT > 300 U/L
  • Serum creatinine above upper limit of normal
  • Active hepatitis C or chronic hepatitis B receiving specific treatment
  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: NCT00171171

Locations
Egypt
Novartis Investigative Site
Cairo, Egypt
Lebanon
Ali Taher
Beirut, Lebanon, Riad El Solh 1107 2020
Oman
Novartis Investigative Site
Muscat, Oman
Saudi Arabia
Novartis Investigative Site
Riyadh, Saudi Arabia
Syrian Arab Republic
Novartis Investigative Site
Damascus, Syrian Arab Republic
Sponsors and Collaborators
Novartis
Investigators
Principal Investigator: Ali Taher, Ass.Prof. American University of Beirut Medical Center
  More Information

No publications provided

Responsible Party: External Affairs, Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00171171     History of Changes
Other Study ID Numbers: CICL670A2402
Study First Received: September 12, 2005
Last Updated: May 5, 2010
Health Authority: Lebanon: Institutional Review Board

Keywords provided by Novartis:
Thalassemia (beta-thal. major)
Transfusional hemosiderosis
Deferasirox
Beta-thalassemia major patients
Unable to be chelated with deferoxamine or deferiprone

Additional relevant MeSH terms:
Thalassemia
Beta-Thalassemia
Hemosiderosis
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Iron Overload
Iron Metabolism Disorders
Metabolic Diseases
Deferasirox
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on October 19, 2014