Status:
COMPLETED
Study for the Treatment of Transfusional Iron Overload in Myelodysplastic Patients
Lead Sponsor:
Novartis Pharmaceuticals
Conditions:
Myelodysplastic Syndromes
Iron Overload
Eligibility:
All Genders
18+ years
Phase:
PHASE4
Brief Summary
Thirty patients were to be enrolled and 24 patients were actually enrolled into this open-label, single-arm trial designed to assess the safety and tolerability of oral deferasirox in adult transfusio...
Detailed Description
Patients were screened for eligibility to determine if they meet all inclusion/exclusion criteria. The screening period were up to 4 weeks. Patient's baseline LIC will be determined non-invasively by ...
Eligibility Criteria
Inclusion
- Male or female patients with low or intermediate (INT-1) risk MDS, determined via IPSS criteria, with transfusional iron overload. NOTE: Bone marrow morphology and cytogenetic studies completed within 3 months prior to screening can be used if the patient has been hematologically stable. Every attempt to obtain cytogenetics studies should be made; however, if there is culture failure, repeat marrow aspiration will not be mandated. In this case, RAEB with less than 11% marrow blasts will be accepted.
- Patients on chelation therapy at the time of screening required a 1-day wash out prior to the first dose of study drug.
- Age: greater than or equal to 18 years
- Serum ferritin:
- For entry into the screening period: serum ferritin greater than or equal to 1000 µg/mL on at least two occasions, at least two weeks apart, during the prior year. Samples must be obtained in the absence of concomitant infection;
- For enrollment into the study: serum ferritin greater than or equal to 1000 µg/mL at screening (via the central lab) obtained in the absence of concomitant infection
- A lifetime minimum of 20 previous packed red cell transfusions
- Life expectancy greater than or equal to 6 months
- Women must have a negative serum or urine pregnancy test and use an effective method of contraception, or must have undergone clinically documented total hysterectomy and/or oophorectomy, or tubal ligation or be postmenopausal (defined by amenorrhea for at least 12 months).
- Able to provide written informed consent
Exclusion
- Serum creatinine greater than 2 × upper limit of normal (ULN)
- ALT or AST greater than 5 × ULN.
- 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 and ALT above the normal range)
- Significant proteinuria as indicated by a urinary protein/creatinine ratio greater than 0.5 mg/mg in a non-first void urine sample during screening (or alternatively in two of three samples obtained for screening)
- History of HIV positive test result (ELISA or Western blot)
- ECOG performance status greater than 2
- Uncontrolled systemic hypertension
- Unstable cardiac disease not controlled by standard medical therapy
- Third degree atrioventricular (AV) block or QT interval prolongation above the normal range
- History of clinically relevant ocular toxicity related to iron chelation
- Pregnancy or breast feeding
- Treatment with a systemic investigational drug within the past 4 weeks or a topical investigational drug within the past 7 days.
- 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:
- inflammatory bowel syndrome, gastritis, ulcers, gastrointestinal or rectal bleeding;
- major gastrointestinal tract surgery, such as gastrectomy, gastroenterostomy, or bowel resection;
- pancreatic injury or pancreatitis or indications of impaired pancreatic function/injury, as indicated by abnormal lipase or amylase;
- urinary obstruction or difficulty in voiding.
- History of non-compliance to medical regimens or patients who are considered potentially unreliable and/or not cooperative
Key Trial Info
Start Date :
September 1 2005
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 1 2008
Estimated Enrollment :
24 Patients enrolled
Trial Details
Trial ID
NCT00117507
Start Date
September 1 2005
End Date
January 1 2008
Last Update
June 24 2021
Active Locations (3)
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1
Stanford University Medical Center
Stanford, California, United States, 94305-5821
2
Karmanos Cancer Center
Detroit, Michigan, United States, 48201
3
MD Anderson Cancer Center
Houston, Texas, United States, 77030-4009