Study on gastrointestinal (GI) tolerability of deferasirox in low or intermediate (int-1) risk myelodysplastic syndrome (MDS) patients with transfusional iron overload

• Conditions: Iron overload in low or intermediate (int-1) risk MDS patients withferritin > 1000 mcg/L at screening or a history of transfusion of at least20 PBRC units; MedDRA version: 14.1Level: HLTClassification code 10022979Term: Iron excessSystem Organ Class: 10027433 - Metabolism and nutrition disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2011-001077-13-IT
• Lead Sponsor: OVARTIS FARMA

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2012-02-23
• Last Update Posted: 10 December 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 172

Inclusion Criteria

Written informed consent obtained prior to any screening procedures
• Male or female patients = 18 years of age
• Patient must weigh between 45-135 kg
• Patients with low or intermediate (int-1) risk MDS, as determined by IPSS score or RA,
RARS by WHO criteria. IPSS must be confirmed by a bone marrow examination within 6
months prior to study entry and must be hematologically stable
• Ferritin > 1000 mcg/L at screening or a history of transfusion of at least 20 PBRC units
• Deferasirox naïve patients
• 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
Are the trial subjects under 18? no
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 64
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 110

Exclusion Criteria

Creatinine clearance < 40 mL/min except in cases where guidance is already given in the
local deferasirox label stating that creatinine clearance should be < 60 mL/min
• Serum creatinine >1. 2x ULN at screening
• Significant proteinurea 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 echo cardiography
• Systemic diseases which would prevent study treatment (e.g. uncontrolled hypertension,
cardiovascular, renal, hepatic, 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
• Platelet counts < 25x 109/L except in cases where guidance is already given in the local
deferasirox label stating that the platelet counts should be < 50x 109/L
• ALT or AST > 2.5xULN at screening
• Total bilirubin > ULN at screening
• Diagnosis of liver cirrhosis
• 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
• Patients with known hypersensitivity to deferasirox or any of its components
History of non-compliance to medical regimens, or patients who are considered
potentially unreliable and/or uncooperative
• Presence of a surgical or medical condition which might significantly alter the absorption,
distribution, metabolism or excretion of study drug
• History of drug or alcohol abuse within the 12 months prior to enrollment
• History of major gastrointestinal tract surgery such as gastrectomy, gastroenterostomy, or
bowel resection
• History or current GI disease
• Patients with GI sensitivities e.g. lactose intolerance
• Patients who are found to be ineligible after screening procedures will have this
documented on the screening log. These patients will not need to complete the end of
study of study assessments
• Pregnant or intending-to-become pregnant or breastfeeding patients
• Women of child-bearing potential, defined as all women physiologically capable of
becoming pregnant, including women whose career, lifestyle, or sexual orientation
precludes intercourse with a male partner and women whose partners have been sterilized
by vasectomy or other means, UNLESS they are using two birth control methods. The two
methods can be a double barrier method or a barrier method plus a hormonal method.
Reliable contraception should be maintained throughout the study and for 28 days after
study drug discontinuation

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified