A phase 2 study of the efficacy and safety of Deferasirox administered at early iron loading in patients with transfusion-dependent myelodysplastic syndromes
- Conditions
- Topic: National Cancer Research Network, BloodSubtopic: Haematological Oncology, Blood (all Subtopics)Disease: Unknown Primary Site, Non-malignant haematologyCancerMyelodysplastic syndromes
- Registration Number
- ISRCTN62162141
- Lead Sponsor
- niversity of Birmingham (UK)
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- All
- Target Recruitment
- 13
1. At least 18 years old
2. Written informed consent
3. MDS with:
3.1. Baseline haemoglobin concentration < 11 g/dl and clinically requiring red cell transfusion with a frequency of at least 2 units every 6 weeks for the receding 12 week period.
3.2. Serum ferritin > 300 ug/l but < 1000 ug/l in absence of ongoing inflammation (CRP < 3 x ULN)
3.3. Serum creatinine < 1.2 x ULN and/or creatinine clearance > 40 ml/min
3.4. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) < 2.5 ULN
3.5. International Prognostic Scoring System (IPSS) Low/INT-1 previously untreated or having failed a therapeutic trail of erythropoetic stimulating agents (ESA) or other active MDS drug therapy, or alternatively lost their response to such therapy
3.6. IPSS INT-2 with <10% blasts and lacking a complex karyotype or monosomy 7 (and with stable blood counts from diagnosis to study entry)
Target gender: male & female
1. Active treatment for MDS, including erythropoetic stimulating agents (ESA), 5-azacitidine, antilymphocyte globulin and low dose chemotherapy such as cytarabine during the trial and within the last 8 weeks
2. Life expectancy of less than 1 year
3. Known HIV positive
4. Active infection
5. Use of prior investigational agents within 6 weeks
6. Pregnancy or lactation
7. Other severe concurrent medical illness that limit the patient?s prognosis to <1 year, or psychiatric disorders
8. Concurrent active or previous malignancy, within the last 3 years, except controlled, localised prostate cancer on hormone therapy or basal cell carcinoma or cervical carcinoma in situ or completely resected colonic polyps carcinoma in situ
9. Ongoing inflammation as measured by C-reactive protein (CRP) > 3 x ULN
10. Serum creatinine > 1.2 x ULN and/or creatinine clearance <40 mls/min
11. ALT or AST >2.5 ULN
12. History of drug/alcohol abuse or non-compliance
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Time to reach a ferritin of 1500 ug/l; Timepoint(s): Treatment is for 12 months and follow up for 24 months
- Secondary Outcome Measures
Name Time Method ot provided at time of registration