Study of the efficacy and safety indicators of two different iron chelators in patients with iron overload (Estudio de los indicadores de eficacia y seguridad de dos quelantes del hierro en pacientes con sobrecarga ferrica)
- Conditions
- HaemosiderosisHaematological DisordersDisorders of iron metabolism
- Registration Number
- ISRCTN52984371
- Lead Sponsor
- Aragon Institute of Health Sciences [Instituto Aragonés de Ciencias de la Salud] (Spain)
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- All
- Target Recruitment
- 32
1. Age = 18 years
2. Diagnosis of myelodysplastic syndrome
3. Have received a hematopoietic stem cell transplantation in the last 6 months
4. Gaucher's disease diagnosis
5. Serum ferritin concentration >500 mcg/L
6. To not have received previous iron chelation therapy
7. No severe renal failure (creatinine clearance >30 ml/min/1.73 m2)
8. No severe liver failure (liver enzymes under twice the upper normal limit)
9. Life expectancy of at least 6 months
1. To not accept to use reliable contraception throughout the study and during three months after cessation of treatment
2. Pregnancy or breast-feeding
3. History of cataracts or increasing risk of cataract formation
4. Severe renal failure (creatinine clearance <30 ml/min/1.73 m2)
5. Active chronic disease such as human immunodeficiency virus (HIV) or hepatitis B or C
6. To have received treatment with iron chelators in the last 6 months
7. Suspected or known hypersensitivity to the drug under study or any of the excipients
8. Dependence or current abuse of drugs or alcohol
9. Treatment with another investigational product in the last 6 months prior to baseline
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Treatment efficacy was determined by measuring the serum ferritin at screening, at baseline and at the beginning of each month of treatment
- Secondary Outcome Measures
Name Time Method 1. Identify whether blood biomarkers of macrophage activation (chitotriosidase, CCL-18) are higher in patients with iron overload than in the population with normal serum ferritin levels stratified by age and sex and if they can be used as markers of response to chelation therapy<br>2. To study if the biomarkers concentration correlates with the serum ferritin level, liver MRI, cardiac function assessed by ultrasound and its own changes after 4-month treatment with iron chelators<br>3.To assess the quality of life of patients undergoing both these treatments