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)
| ISRCTN | ISRCTN52984371 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN52984371 |
| Secondary identifying numbers | TRA-158 (EC09/080) |
- Submission date
- 31/01/2011
- Registration date
- 18/04/2011
- Last edited
- 18/04/2011
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Haematological Disorders
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data
Record updated in last year
Plain English summary of protocol
Not provided at time of registration
Contact information
Dr Pilar Giraldo Castellano
Scientific
Scientific
Servicio de Hematología
Hospital Universitario Miguel Servet
Paseo Isabel La Católica 1-3
Zaragoza
50009
Spain
| Phone | +34 670285339 |
|---|---|
| giraldo.p@gmail.com |
Study information
| Study design | Phase III single-centre prospective randomised clinical study |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Randomised controlled trial |
| Study setting(s) | Hospital |
| Study type | Treatment |
| Participant information sheet | Not available in web format, please use the contact details below to request a patient information sheet |
| Scientific title | 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 |
| Study acronym | QuelaFer |
| Study objectives | The main aim of this study is to evaluate the efficacy of Deferasirox versus Deferoxamine in reducing serum ferritin levels and iron liver deposits in iron overload patients. Excess iron in blood and tissues causes irreversible tissue damage. The removal of excess iron as response to treatment positively influences on the survival of patients with overload. Currently, there are two types of iron chelating agents authorised, each one with different route of administration (Deferoxamine-parenteral and Deferasirox-oral) but the superiority of one over another is not defined in relation to the intensity of the response, time to be reached or the severity and frequency of associated adverse effects or its impact on quality of life. |
| Ethics approval(s) | Clinical Research Ethics Committee of Aragon (Comité Ético de Investigación Clínica de Aragón) (CEICA). Ref: C.I. EC09/080 11/08/2010 |
| Health condition(s) or problem(s) studied | Haemosiderosis |
| Intervention | 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/week for 4 months 3. Patients from both groups will be followed for 1-month period. 4. The following tests should be performed: 4.1. Peripheral blood creatinine (weekly in the 1st month and then monthly), serum transaminases, bilirubin and alkaline phosphatase (on day 1 of treatment, every 2 weeks in the 1st month, then monthly, and at the end of treatment) 4.2. Serum ferritin (at screening, at baseline and at the beginning of each month of treatment) 4.3. Liver magnetic resonance imaging (MRI) and quantification of tissue iron (at screening visit and at the end of 4 months of treatment) 4.4. Computerised tomography (CT), CCL18 and proBNP activity (at screening, before the 2nd month of treatment and after treatment) 4.5. Eastern Cooperative Oncology Group (ECOG) performance status (at screening, and on day 1 of each new cycle of treatment) 4.6. Electrocardiogram (at screening and whenever the investigator deems it appropriate, SF36 quality of life questionnaire (at screening, before the 2nd month of treatment and after treatment). |
| Intervention type | Drug |
| Pharmaceutical study type(s) | |
| Phase | Phase III |
| Drug / device / biological / vaccine name(s) | 1. Deferoxamine 2. Deferasirox |
| Primary outcome measure | 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 | 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 |
| Overall study start date | 01/03/2011 |
| Completion date | 30/09/2012 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Adult |
| Lower age limit | 18 Years |
| Sex | Both |
| Target number of participants | 32 |
| Key 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 |
| Key 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 |
| Date of first enrolment | 01/03/2011 |
| Date of final enrolment | 30/09/2012 |
Locations
Countries of recruitment
- Spain
Study participating centre
Servicio de Hematología
Zaragoza
50009
Spain
50009
Spain
Sponsor information
Aragon Institute of Health Sciences [Instituto Aragonés de Ciencias de la Salud] (Spain)
Government
Government
c/o Esteban de Manuel Kenoy
Instituto Aragonés de Ciencias de la Salud
Avenida Gómez Laguna, 25
Zaragoza
50009
Spain
| emlopezh.iacs@aragon.es | |
| Website | http://www.aragon.es/ |
"ROR" |
https://ror.org/05p0enq35 |
Funders
Funder type
Government
Aragon Institute of Health Sciences [Instituto Aragonés de Ciencias de la Salud] (Spain)
No information available
Results and Publications
| Intention to publish date | |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not provided at time of registration |
| Publication and dissemination plan | Not provided at time of registration |
| IPD sharing plan |
