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

Myelodysplastic Syndromes Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00564941
• Other study ID #: CICL670AHU02
• Status: Completed
• Phase: Phase 4
• First received: November 28, 2007
• Last updated: February 22, 2017
• Start date: December 2007
• Est. completion date: July 2011

Study information

• Verified date: February 2017
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This study will evaluate the safety and efficacy of deferasirox in transfusion dependent Myelodysplastic Syndrome, Beta-thalassaemia major patients with chronic iron overload

Recruitment information / eligibility

• Status: Completed
• Enrollment: 309
• Est. completion date: July 2011
• Est. primary completion date: July 2011
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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

Related Conditions & MeSH terms

• Beta-Thalassemia
• Iron Overload
• Myelodysplastic Syndromes
• Preleukemia
• Syndrome
• Thalassemia

Intervention

Drug:
• deferasirox

Locations

Country	Name	City	State
Hungary	Novartis Investigative Site	Budapest
Hungary	Novartis Investigative Site	Debrecen
Hungary	Novartis Investigative Site	Pecs
Hungary	Novartis Investigative Site	Szeged

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Country where clinical trial is conducted

Hungary, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	This study will evaluate the safety and efficacy of deferasirox in transfusion dependent Myelodysplastic Syndrome, Beta-thalassaemia major patients with chronic iron overload		monthly during the therapy and at the end of the treatment (aftr 9 months therapy)
Secondary	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		during the treatment (9 months)

External Links

•   Results of CICL670AHU02 from Novartis Clinical Trials website