Study for the Treatment of Transfusional Iron Overload in Myelodysplastic Patients
Thirty patients will be enrolled into this open-label, single-arm trial designed to assess the safety and tolerability of oral deferasirox in adult transfusion dependent myelodysplastic syndrome (MDS) patients with iron overload. Patients enrolled in this study will have low or intermediate (INT-1) risk MDS per International Prognostic Scoring System (IPSS) criteria. All patients will initiate treatment with 20mg/kg/day deferasirox.
Deferasirox will be administered orally once per day for 12 months.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||An Open Label, Safety and Tolerability Study of Deferasirox for Treatment of Transfusional Iron Overload in Low-Risk and INT-1 Myelodysplastic Patients|
- Safety and tolerability in myelodysplastic syndrome (MDS) patients [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
- Efficacy based on serum ferritin and Liver iron concentration (LIC) [ Time Frame: through out the study ] [ Designated as safety issue: No ]
- Changes in liver iron concentration (LIC) [ Time Frame: through out the study ] [ Designated as safety issue: Yes ]
- Pharmacokinetics (PK) in MDS patients [ Time Frame: through out the study ] [ Designated as safety issue: No ]
- Non-transferrin bound iron (NTBI) & iron metabolism parameters [ Time Frame: through out the study ] [ Designated as safety issue: Yes ]
- Iron metabolism parameters [ Time Frame: through out the study ] [ Designated as safety issue: Yes ]
|Study Start Date:||April 2005|
|Study Completion Date:||January 2008|
|Primary Completion Date:||January 2008 (Final data collection date for primary outcome measure)|
Experimental: 20mg/kg/day deferasirox
Deferasirox will be administered orally once per day for 12 months. Surrogate marker findings, including serum ferritin, and LIC in the context of the study results will be monitored on a regular basis for any indications of clinically important over- or under-chelation.
deferasirox 20 mg/kg/day taken over one year.Deferasirox should be taken every morning 30 minutes before breakfast, if possible consistently around the same time between 7:00 and 9:00 AM. The tablets should be dropped into water or orange juice and gently stirred for 1 to 3 minutes until completely dispersed.
Patients will be screened for eligibility to determine if they meet all inclusion/exclusion criteria. The screening period will be up to 4 weeks. Patient's baseline LIC will be determined non-invasively by means of MRI R2 analysis. In addition, blood and urine samples will be taken for the determination of baseline safety data.
|United States, California|
|Stanford University Medical Center|
|Stanford, California, United States, 94305-5821|
|United States, Michigan|
|Karmanos Cancer Center|
|Detroit, Michigan, United States, 48201|
|United States, Texas|
|MD Anderson Cancer Center|
|Houston, Texas, United States, 77030-4009|
|Study Director:||Novartis Pharmaceuticals||Novartis Pharmaceuticals|