Evaluating the Efficacy of Deferasirox in Transfusion Dependent Chronic Anaemias (Myelodysplastic Syndrome, Beta-thalassaemia Patients) With Chronic Iron Overload

• ClinicalTrials.gov Identifier: NCT00564941
• Recruitment Status: Completed
• First Posted: November 29, 2007
• Last Update Posted: February 24, 2017
• Sponsor: Novartis Pharmaceuticals
• Information provided by (Responsible Party): Novartis ( Novartis Pharmaceuticals )

Tracking Information

• First Submitted Date: November 28, 2007
• First Posted Date: November 29, 2007
• Last Update Posted Date: February 24, 2017
• Study Start Date: December 2007
• Actual Primary Completion Date: July 2011 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: January 9, 2009): This study will evaluate the safety and efficacy of deferasirox in transfusion dependent Myelodysplastic Syndrome, Beta-thalassaemia major patients with chronic iron overload [ Time Frame: monthly during the therapy and at the end of the treatment (aftr 9 months therapy) ]
• Original Primary Outcome Measures (submitted: November 28, 2007): This study will evaluate the safety and efficacy of deferasirox in transfusion dependent Myelodysplastic Syndrome, Beta-thalassaemia major patients with chronic iron overload
• Current Secondary Outcome Measures (submitted: January 9, 2009): patient's compliance during the study assessed by the number of the unused tablets returned by the patient safety assessed by patient laboratory data, adverse events, serious adverse events [ Time Frame: during the treatment (9 months) ]
• Original Secondary Outcome Measures (submitted: November 28, 2007): patient's compliance during the study assessed by the number of the unused tablets returned by the patient safety assessed by patient laboratory data, adverse events, serious adverse events
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Evaluating the Efficacy of Deferasirox in Transfusion Dependent Chronic Anaemias (Myelodysplastic Syndrome, Beta-thalassaemia Patients) With Chronic Iron Overload
• Official Title: Evaluating the Efficacy of Deferasirox in Transfusion Dependent Chronic Anaemias (Myelodysplastic Syndrome, Beta-thalassaemia Patients) With Chronic Iron Overload
• Brief Summary: This study will evaluate the safety and efficacy of deferasirox in transfusion dependent Myelodysplastic Syndrome, Beta-thalassaemia major patients with chronic iron overload
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 4
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment

Condition

• Myelodysplastic Syndromes
• Beta-Thalassemia

Intervention

Drug: deferasirox

Other Name: ICL670

Study Arms

Experimental: Deferasirox

Intervention: Drug: deferasirox

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Estimated Enrollment (submitted: November 28, 2007): 309
• Original Estimated Enrollment: Same as current
• Actual Study Completion Date: July 2011
• Actual Primary Completion Date: July 2011 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion criteria:

• In- or outpatients with myelodysplasia / with risk of low or intermedier-1 according to the International Prognostic Scoring System (IPSS) confirmed by bone marrow evaluation within 3 month/ or beta thalassaemia major patients, who have chronic iron overload, as a consequence of frequent blood transfusion
• Serum ferritin> 1800 µg/L
• Age: 18-80 years
• men and women
• Chronic iron overload caused by at least 30 units and maximum 100 units of blood of packed red blood cells
• Deferoxamin therapy is contraindicated or inadequate or unable to use in the recommended dose due to intolerability or other reason
• Eastern Cooperative Oncology Group (ECOG) performance status score between 0-2
• written informed consent

Exclusion criteria:

• beta thalassaemia minor,
• haemosiderosis caused by other than chronic transfusional iron overload,
• patients with impaired renal function (Creatinin clearance< 60 ml/ min),
• pregnancy,
• lactation,
• patient of childbearing potential unwilling to use contraceptive precautions
• known hypersensitivity to deferasirox or any ingredients,
• impaired hepatic function (SGOT,SGPT 5x above UNL).
• Patients severely ill due to underlying disease progression or other severe concomitant disease.
• Patients with poor prognosis of karyotype
• patients with malabsorption caused by inflammatory bowel disease, gastrectomy, pancreatitis or other medical condition
• History of nephrotic syndrome
• Significant proteinuria
• Patients with a previous history of clinically relevant ocular toxicity related to iron chelation
• Patients with positive test to HIV

Other protocol-defined inclusion/exclusion criteria may apply

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 80 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Hungary

Administrative Information

• NCT Number: NCT00564941
• Other Study ID Numbers: CICL670AHU02
• Has Data Monitoring Committee: Not Provided
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Novartis ( Novartis Pharmaceuticals )
• Original Responsible Party: Not Provided
• Current Study Sponsor: Novartis Pharmaceuticals
• Original Study Sponsor: Novartis
• Collaborators: Not Provided

Investigators

• Study Director: Novartis Pharmaceuticals; Novartis Pharmaceuticals

• PRS Account: Novartis
• Verification Date: February 2017