A multi center clinical trial of deferasirox in patients with myelodysplastic syndromes and transfusional iron overload

Phase 1

• Conditions: Transfusional iron overload; MedDRA version: 14.1Level: LLTClassification code 10019613Term: HemosiderosisSystem Organ Class: 10027433 - Metabolism and nutrition disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2009-012418-38-NL
• Lead Sponsor: ovartis Pharma Services AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2011-05-11
• Last Update Posted: 11 June 2018

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 630

Inclusion Criteria

-Male or female patients, =18 years of age
-Patient must weigh between 35-135 kg
-Patients 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
-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
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 126
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 504

Exclusion Criteria

-More than 6 months of cumulative iron chelation therapy (such as daily deferasirox (Exjade®) or deferiprone or 5x/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 x ULN at screening
- Serum creatinine will be measured at Screening Visit 1 and Screening Visit 2 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, 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)
-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 cell 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, intending-to-become pregnant, or breast-feeding patients
-History of drug or alcohol abuse within the 12 months prior to enrollment.

Study & Design

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