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)

A single-centre, prospective, randomised, phase III clinical study to evaluate the efficacy and safety indicators of two different iron chelators in patients with iron overload

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

ISRCTN

Registry ID

ISRCTN52984371

Enrollment

Registered

2011-04-18

Start date

2011-03-01

Completion date

Unknown

Last updated

2015-01-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Haemosiderosis Haematological Disorders Disorders of iron metabolism

Interventions

1. Group A: Deferasirox 20 mg/kg/day oral capsules for 4 months 2. Group B: Desferoxamine 30 mg/kg/day, slow Subcutaneous (SC) infusion administered over 8 hours by a portable infusion pump 3 times/we

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 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

Exclusion criteria

Exclusion criteria: 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

Design outcomes

Primary

Measure	Time frame
Treatment efficacy was determined by measuring the serum ferritin at screening, at baseline and at the beginning of each month of treatment	—

Secondary

Measure	Time frame
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 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 3.To assess the quality of life of patients undergoing both these treatments	—

Measure

Time frame

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 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 3.To assess the quality of life of patients undergoing both these treatments

—

Countries

Spain

Outcome results

None listed

Source: ISRCTN (via WHO ICTRP) · Data processed: Feb 4, 2026