Clinical trial NCT00564941: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Myelodysplastic Syndromes
Beta-Thalassemia
Keywords: Myelodysplastic Syndromes
beta-Thalassemia
Iron Overload
Iron Chelating Agents
deferasirox
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2007-11-29
Enrollment: 309
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


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

Primary outcomes:
measure: This study will evaluate the safety and efficacy of deferasirox in transfusion dependent Myelodysplastic Syndrome, Beta-thalassaemia major patients with chronic iron overload
timeFrame: monthly during the therapy and at the end of the treatment (aftr 9 months therapy)
Secondary outcomes:
measure: 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
timeFrame: during the treatment (9 months)
Other outcomes:
Locations:
city: Budapest
country: Hungary
facility: Novartis Investigative Site
city: Debrecen
country: Hungary
facility: Novartis Investigative Site
city: Pecs
country: Hungary
facility: Novartis Investigative Site
city: Szeged
country: Hungary
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: