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

• ClinicalTrials.gov Identifier: NCT00171171
• Recruitment Status: Completed
• First Posted: September 15, 2005
• Last Update Posted: March 1, 2017
• Sponsor: Novartis
• Information provided by: Novartis

Tracking Information

• First Submitted Date: September 12, 2005
• First Posted Date: September 15, 2005
• Last Update Posted Date: March 1, 2017
• Study Start Date: May 2004
• Actual Primary Completion Date: November 2006 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: May 5, 2010): Liver Iron Concentration (as measured by biopsy) [ Time Frame: at baseline and after 1 yeor of ICL670 treatment ]
• Original Primary Outcome Measures (submitted: September 14, 2005): Efficacy of ICL670 treatment based on LIC

Current Secondary Outcome Measures (submitted: November 17, 2016)

• 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 ]
• Putative surrogate markers such as serum ferritin serum iron, serum transferrin and transferrin saturation [ Time Frame: monthly assessments for 1 year ]
• Adverse events [ Time Frame: during 1 year ICL670 treatment ]
• hematology and biochemistry, urinalysis [ Time Frame: monthly assessments for 1 year ICL670 treatment ]
• ECG and Echocardiography [ Time Frame: 6-monthly for 1 year ICL670 treatment ]

Original Secondary Outcome Measures (submitted: September 14, 2005)

• Tolerability profile of ICL670
• Absolute + relative change of LIC and Total Body Iron Excretion (TBIE) rate for subgroups
• Relationship between LIC and potential surrogate markers
• Potential of Magnetic Resonance Imaging (MRI)for LIC
• Quality of life and patient global assessment
• Drug usage
• Dose adjustment regimens as dictated by efficacy and safety parameters in comparison to transfusional burdens

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Study of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis
• Official Title: A Study of Efficacy and Safety of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis
• 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Non-Randomized; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment

Condition

• Beta-Thalassemia
• Hemosiderosis

• Intervention: Drug: deferasirox

Study Arms

Experimental: Deferasirox

Intervention: Drug: deferasirox

• Publications *: 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.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: April 30, 2010): 252
• Original Enrollment (submitted: September 14, 2005): 250
• Study Completion Date: Not Provided
• Actual Primary Completion Date: November 2006 (Final data collection date for primary outcome measure)

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 2 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: Egypt, Lebanon, Oman, Saudi Arabia, Syrian Arab Republic

Administrative Information

• NCT Number: NCT00171171
• Other Study ID Numbers: CICL670A2402
• Has Data Monitoring Committee: Not Provided
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: External Affairs, Novartis Pharmaceuticals
• Original Responsible Party: Not Provided
• Current Study Sponsor: Novartis
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Principal Investigator: Ali Taher, Ass.Prof.; American University of Beirut Medical Center

• PRS Account: Novartis
• Verification Date: November 2016