Efficacy and safety of iron chelation therapy with deferasirox compared toplacebo in patients with myelodysplastic syndromes and transfusional ironoverload
- Conditions
- Myelodysplastic syndrome, transfusional iron overloadMedDRA version: 18.1Level: LLTClassification code 10019613Term: HemosiderosisSystem Organ Class: 100000004861
- Registration Number
- EUCTR2009-012418-38-DK
- Lead Sponsor
- ovartis Pharma Services AG
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 210
• Male or female , =18 years of age, with low or intermediate (int-1) risk MDS, as determined by IPSS score. This must be confirmed by a bone marrow examination within 6 months prior to study entry and must be hematologically stable.
• Weight between 35-135 kg
• Ferritin > 1000 mcg/L at screening
• History of transfusion of 15 to 75 pRBC units
• Anticipated to be transfused with at least 8 units of PRBCs annually during the study. Sexually active pre-menopausal female patients must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or oophorectomy, tubal ligation
Additional inclusion criteria as per full protocol may apply.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range
• More than 6 months of cumulative iron chelation therapy (such as daily
deferasirox (Exjade®) or deferiprone or 5×/week deferoxamine)
- Intermittent deferoxamine doses in association with blood
transfusions are not exclusionary regardless of duration of such
treatment
• More than 3 years since patient began receiving regular transfusions
(2 units per 8 weeks or 4 units received in a 3 month period)
• Creatinine Clearance <40 ml/min
• Serum creatinine > 1.5 × ULN at screening
- Serum creatinine will be measured at Screening Visit 1 and Screening
Visit 2 (at least 14 days apart) and the mean value will be used for
eligibility criteria.
• Significant proteinuria as indicated by a urinary protein/creatinine
ratio > 0.5 mg/mg in a non-first void urine sample at Visit 1 or Visit 2
(or alternatively in two of three samples obtained for screening)
• ECOG performance status > 2
• Left ventricular ejection fraction < 50% by echocardiography as per
the central reading assessment
• A history of hospitalization for congestive heart failure
• Systemic diseases which would prevent study treatment (e.g.
uncontrolled hypertension, cardiovascular, renal, hepatic (including
Child-Pugh Class B and C), metabolic, etc.)
• Clinical or laboratory evidence of active Hepatitis B or Hepatitis C
(HBsAg in the absence of HBsAb OR HCV Ab positive with HCV RNA
positive)
• History of HIV positive test result (ELISA or Western blot)
• Treatment with systemic investigational drug within 4 weeks or topical
investigational drug within 7 days of study start
• ALT or AST > 3.5 × ULN at screening
• Total bilirubin > 1.5 × ULN at screening
• Diagnosis of liver cirrhosis (either established diagnosis or diagnosis
by liver biopsy or central ultrasound reading)
• Patients participating in another clinical trial other than an
observational registry study
• Patients with a history of another malignancy within the past five
years, with the exception of basal skin carcinoma or cervical carcinoma
in situ or completely resected colonic polyps carcinoma in situ.
• History of non-compliance to medical regimens, or patients who are
considered potentially unreliable and/or not cooperative
• Presence of a surgical or medical condition which might significantly
alter the absorption,
distribution, metabolism or excretion of study drug
• Pregnant, or breast-feeding patients, or patients of child-bearing
potential not employing an effective method of birth control (see Women
of child-bearing potential, below, for further details regarding effective
methods of birth control).
• History of drug or alcohol abuse within the 12 months prior to
enrollment
Additional exclusion criteria as per full protocol may apply.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method