Safety, Tolerability, and Efficacy of Deferasirox in MDS

November 21, 2016 updated by: Gruppo Italiano Malattie EMatologiche dell'Adulto

Open Label, Multicenter Study to Evaluate Safety/Tolerability and Efficacy of Deferasirox (ICL670) in Myelodysplastic Syndrome Patients With Chronic Transfusional Hemosiderosis.

Open label, single arm study on Deferasirox treatment in MDS patients with chronic transfusional hemosiderosis.

Patients receive daily oral dosis of Deferasirox in order to eliminate the quantity of iron administered during transfusions and, if needed, to reduce the overload of already present iron.

After an screening phase in which patients are evaluated according to eligibility criteria, a one year treatment phase foresees monthly visits to evaluate safety and efficacy signs.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

It has been widely shown that an appropriate chelating therapy in chronic anemias transfusion dependent can prevent the overstock of iron and can reduce the already existing overstock reducing, then, the co-morbidity and improving survival.

In particular, some authors have shown in MDS affected patients undergoing intensive chelating therapy with deferoxamine haematological recovery with a reduction of the need of transfusions.

With the present study, we plan to evaluate the safety and efficacy of a therapy with the new oral chelating Deferasirox in MDS patients with transfusional hemosiderosis.

This is an open label, single arm study on Deferasirox treatment in MDS patients with chronic transfusional hemosiderosis.

Patients will receive daily oral dosis of Deferasirox in order to eliminate the quantity of iron administered during transfusions and, if needed, to reduce the overload of already present iron.

After an screening phase in which patients are evaluated according to eligibility criteria, a one year treatment phase foresees monthly visits to evaluate safety and efficacy signs.

Study Type

Interventional

Enrollment (Actual)

158

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Italy
      • Alessandria, Italy
        • SOC EMATOLOGIA ASO SS Antonio e Biagio
      • Bologna, Italy
        • Ist.Ematologia e Oncologia Medica L.e A. Seragnoli
      • Cagliari, Italy
        • CTMO-Ematologia Ospedale Binaghi
      • Cagliari, Italy
        • Ospedale "A. Businco"
      • Caserta, Italy
        • Oncoematologia "A.O.R.N. S'Anna e S.Sebastiano"
      • Castelfranco Veneto, Italy
        • US Dipartimentale Centro per le Malattie del Sangue
      • Catania, Italy
        • Università di Catania - Cattedra di Ematologia - Ospedale "Ferrarotto"
      • Firenze, Italy
        • Policlinico di Careggi, Università delgi studi di Firenze
      • Genova, Italy
        • Clinica Ematologica - Università degli Studi
      • Napoli, Italy
        • Divisione di Ematologia e TMO - Ospedale "A. Cardarelli "- Azienda Ospedaliera di Rilievo Nazionale "A. Cardarelli"
      • Perugia, Italy
        • Clinica Ematol Università di Perugia, Policlinico Monteluce
      • Roma, Italy
        • Università Cattolica del Sacro Cuore
      • Roma, Italy
        • Ematologia- Università degli Studi "La Sapienza"
      • Roma, Italy
        • Università degli Studi di Tor Vergata
      • San Giovanni Rotondo, Italy
        • Istituto di Ematologia - IRCCS Ospedale Casa Sollievo della Sofferenza
      • Siena, Italy
        • U.O. Ematologia, Azienda Ospedaliera Universitaria Senese
      • Venezia, Italy
        • Ospedale Civile SS. Giovanni e Paolo

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Patients, both males and females, with low and intermediate I risk (IPSS score) Myelodysplastic syndrome and transfusion-induced hemosiderosis.
  • Age >=18 years
  • Patients who never received chelation therapy or who received a therapy with Desferal after a day of wash out
  • Medical history of at least 20 blood transfusions (equivalent to 100 ml/kg of red cells concentrate).
  • Availability of data concerning blood transfusions during the 12 weeks before screening
  • Serum ferritin >= 1000 µg/L at least twice (at least 2 week interval between the 2 analysis) during the year before the screening
  • Life expectancy > 12 months
  • Availability of at least 3 complete blood counts (before transfusions) during the 12 weeks before the screening

Exclusion Criteria:

  • Diagnosis different from MDS (i.e. myelofibrosis)
  • Severe renal impairment (creatinine clearance < 60 ml/min)
  • ALT/AST > 500 U/L
  • Active B and/or C hepatitis
  • Patients treated during the past 4 weeks with experimental drugs for MDS (including thalidomide, azacitidine, arsenic trioxide). These patients become eligible after a "wash out" of at least 4 weeks
  • Concomitant treatment with another iron-chelating agent

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: Deferasirox
Drug: Deferasirox

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
To evaluate the tolerability and safety profile of Deferasirox in pts with MDS with post-transfusional hemosiderosis
Time Frame: On a monthly basis thereafter from baseline assessment.
On a monthly basis thereafter from baseline assessment.

Secondary Outcome Measures

Outcome Measure
Time Frame
To evaluate Deferasirox efficacy as chelation therapy in terms of reduction of serum ferritin levels compared to basal levels
Time Frame: At 3, 6, 9, and 12 months from baseline assessment.
At 3, 6, 9, and 12 months from baseline assessment.
To evaluate the impact Deferasirox iron chelating therapy vs the normal demand of transfusions in a subgroup of pts that will not receive growth factors or chemotherapy according to their basal characteristics.
Time Frame: On a monthly basis thereafter from baseline assessment.
On a monthly basis thereafter from baseline assessment.
Quality of Life evaluation.
Time Frame: At 3, 6, 9, and 12 months from baseline assessment.
At 3, 6, 9, and 12 months from baseline assessment.
Compliance to chelating therapy evaluation.
Time Frame: On a monthly basis thereafter from baseline assessment.
On a monthly basis thereafter from baseline assessment.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Gruppo Italiano Malattie EMatologiche dell'Adulto

Investigators

  • Principal Investigator: Emanuele ANGELUCCI, Pr., Ospedale "A. Businco", Cagliari

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2007

Primary Completion (ACTUAL)

March 1, 2010

Study Completion (ACTUAL)

November 1, 2013

Study Registration Dates

First Submitted

May 3, 2007

First Submitted That Met QC Criteria

May 3, 2007

First Posted (ESTIMATE)

May 4, 2007

Study Record Updates

Last Update Posted (ESTIMATE)

November 22, 2016

Last Update Submitted That Met QC Criteria

November 21, 2016

Last Verified

November 1, 2016

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MDS0306

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Myelodysplastic Syndromes

Clinical Trials on Deferasirox

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Greece

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe