A one-year, open-label, single arm, multi-center trial evaluating the efficacy and safety of oral ICL670 in patients diagnosed with Low and INT-1 risk Myelodysplastic Syndrome (MDS) and transfusion-dependent iron overload
- Conditions
- myelodysplastic syndrome and transfusion-dependent iron overloadMedDRA version: 8.1Level: LLTClassification code 10028533Term: Myelodysplastic syndrome
- Registration Number
- EUCTR2006-003971-11-DE
- Lead Sponsor
- ovartis Pharma GmbH
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- Not specified
•MDS patients presenting with low or intermediate-1 IPSS risk and transfusional iron overload as shown by a serum ferritin level of > 1000 ng/ml. This level should have been verified at least at two occasions within 6 months before giving informed consent and the time window between these two measurements must have been at least 4 weeks. Samples must be obtained in the absence of concomitant infection
•Patients of either gender and age > 18 years
•Life expectancy > 12 months
•History of at least 20 units of red blood cell transfusions or 100mL/kg of prepacked red blood cells (PRBCs)
•Patients can be either naïve to iron chelation or have had prior treatment with deferoxamine (DFO) or deferiprone (L1)
•Females of childbearing potential must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or oophorectomy, tubal ligation or be postmenopausal defined by amenorrhea for at least 12 months. Only contraception with a pearl-index below 1% should be considered.
•Written informed consent by the patient
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
•Non-transfusion related hemosiderosis
•Treatment with ICL670 before study start
•Patients with a concomitant malignant disease
•Patients with mean levels of alanine aminotransferase (ALT) > 5x ULN
•Patients with uncontrolled systemic hypertension
•Patients with serum creatinine > 1.5x the upper limit of normal (ULN)
•History of nephrotic syndrome
•Patients with a previous history of clinically relevant ocular toxicity related to iron chelation
•Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent the patient from undergoing study treatment
•Patients with psychiatric or addictive disorders which prevent them from giving their informed consent or undergoing study treatment
•Patients treated with systemic investigational drugs within the past 4 weeks or topical investigational drug within the past 7 days
•Any other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of any drug. The investigator should be guided by evidence of any of the following:
•history of inflammatory bowel syndrome, gastritis, ulcers, gastrointestinal or rectal bleeding;
•history of major gastrointestinal tract surgery such as gastrectomy, gastroenterostomy, or bowel resection;
•history of pancreatic injury or pancreatitis; indications of impaired pancreatic function/injury as indicated by abnormal lipase or amylase;
•history of urinary obstruction or difficulty in voiding
•History of non-compliance to medical regimens and patients who are considered potentially unreliable and/or not cooperative
•History of drug or alcohol abuse within the 12 months prior to dosing or evidence of such abuse as indicated by the laboratory assays conducted during the run-in period
•Patients with active uncontrolled infectious disease
•Pregnancy or breast feeding
•QT > 470 msec on screening ECG
•Patients with a history of Torsades de Pointes
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