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

NCT00564941 | Completed Phase 4

• 1 other identifier: CICL670AHU02
• Study Type: interventional
• Target: 309
• Locations: 1 country
• Sites: 4

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

Conditions

Myelodysplastic Syndromes; Beta-Thalassemia

Keywords

Myelodysplastic Syndromes; beta-Thalassemia; Iron Overload; Iron Chelating Agents; deferasirox

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• 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)

Study Arms (1)

Deferasirox

EXPERIMENTAL

Drug: deferasirox

Interventions

deferasirox DRUG

Also known as: ICL670

Deferasirox

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (4)

Novartis Investigative Site

Budapest, Hungary

Location

Novartis Investigative Site

Debrecen, Hungary

Location

Novartis Investigative Site

Pécs, Hungary

Location

Novartis Investigative Site

Szeged, Hungary

Location

MeSH Terms

Conditions

Myelodysplastic Syndromes; beta-Thalassemia; Iron Overload

Interventions

Deferasirox

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Iron Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Benzoates; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Triazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 28, 2007
• First Posted: November 29, 2007
• Study Start: December 1, 2007
• Primary Completion: July 1, 2011
• Study Completion: July 1, 2011
• Last Updated: February 24, 2017

Record last verified: 2017-02

Locations

HU (4)