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Efficacy and Safety of Deferasirox in Patients With Myelodysplastic Syndrome and Transfusion-dependent Iron Overload

Primary Purpose

Myelodysplastic Syndromes, Transfusion Dependent Iron Overload

Status

Completed

Phase

Phase 4

Locations

Germany

Study Type

Interventional

Intervention

ICL670/Deferasirox

About this trial

This is an interventional treatment trial for Myelodysplastic Syndromes focused on measuring Myelodysplastic Syndrome, MDS, iron overload, chelation, chelators, ICL670, Deferasirox, serum ferritin, LIC, transfusional hemosiderosis, Refractory Anemia, RA, Refractory Anemia with Ringed Sideroblasts, RARS, blood transfusions, Mydelodysplastic Syndrome(s)

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

MDS patients presenting with low or intermediate-1 IPSS risk and transfusional iron overload
Patients of either gender and age ≥ 18 years
History of at least 20 units of red blood cell transfusions or 100mL/kg of prepacked red blood cells (PRBCs)
Patients can be either naïve to iron chelation or have had prior treatment with deferoxamine (DFO) or deferiprone (L1)
Females of childbearing potential must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or oophorectomy, tubal ligation or be postmenopausal defined by amenorrhea for at least 12 months.

Exclusion Criteria:

Non-transfusion related iron overload
Treatment with deferasirox (ICL670) before study start
Patients with a concomitant malignant disease
Patients with out of range lab values
History of nephrotic syndrome
Patients with a previous history of clinically relevant ocular toxicity related to iron chelation
Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent the patient from undergoing study treatment
Patients treated with systemic investigational drugs within the past 4 weeks or topical investigational drug within the past 7 days
Any other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of any drug.
Patients with active uncontrolled infectious disease
Pregnancy or breast feeding

Other protocol-defined inclusion/exclusion criteria may apply.

Sites / Locations

Novartis Investigative Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

deferasirox

Arm Description

Outcomes

Primary Outcome Measures

To assess iron chelation by comparing serum ferritin values at baseline vs. 52 weeks of treatment with deferasirox

Secondary Outcome Measures

Safety and tolerability of deferasirox assessed by monitoring and recording all adverse events (AEs) and serious adverse events (SAEs)

Full Information

NCT ID

NCT00481143

First Posted

May 31, 2007

Last Updated

May 30, 2017

Sponsor

Novartis Pharmaceuticals

1. Study Identification

Unique Protocol Identification Number

NCT00481143

Brief Title

Efficacy and Safety of Deferasirox in Patients With Myelodysplastic Syndrome and Transfusion-dependent Iron Overload

Official Title

A One-year, Open-label, Single Arm, Multi-center Trial Evaluating the Efficacy and Safety of Oral ICL670 in Patients Diagnosed With Low and INT-1 Risk Myelodysplastic Syndrome (MDS) and Transfusion-dependent Iron Overload

Study Type

Interventional

2. Study Status

Record Verification Date

November 2016

Overall Recruitment Status

Completed

Study Start Date

May 2007 (undefined)

Primary Completion Date

November 2010 (Actual)

Study Completion Date

undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Novartis Pharmaceuticals

4. Oversight

5. Study Description

Brief Summary

The purpose of this study is to investigate the effects of iron chelation using deferasirox in low and INT-1 risk (referring to the international prognostic scoring system, IPSS) MDS patients who show signs of iron overload due to repeated blood transfusions. This trial is not recruiting patients in the United States.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Myelodysplastic Syndromes, Transfusion Dependent Iron Overload

Keywords

Myelodysplastic Syndrome, MDS, iron overload, chelation, chelators, ICL670, Deferasirox, serum ferritin, LIC, transfusional hemosiderosis, Refractory Anemia, RA, Refractory Anemia with Ringed Sideroblasts, RARS, blood transfusions, Mydelodysplastic Syndrome(s)

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 4

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

63 (Actual)

8. Arms, Groups, and Interventions

Arm Title

deferasirox

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

ICL670/Deferasirox

Other Intervention Name(s)

ICL670

Primary Outcome Measure Information:

Title

To assess iron chelation by comparing serum ferritin values at baseline vs. 52 weeks of treatment with deferasirox

Time Frame

52 weeks

Secondary Outcome Measure Information:

Title

Safety and tolerability of deferasirox assessed by monitoring and recording all adverse events (AEs) and serious adverse events (SAEs)

Time Frame

52 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: MDS patients presenting with low or intermediate-1 IPSS risk and transfusional iron overload Patients of either gender and age ≥ 18 years History of at least 20 units of red blood cell transfusions or 100mL/kg of prepacked red blood cells (PRBCs) Patients can be either naïve to iron chelation or have had prior treatment with deferoxamine (DFO) or deferiprone (L1) Females of childbearing potential must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or oophorectomy, tubal ligation or be postmenopausal defined by amenorrhea for at least 12 months. Exclusion Criteria: Non-transfusion related iron overload Treatment with deferasirox (ICL670) before study start Patients with a concomitant malignant disease Patients with out of range lab values History of nephrotic syndrome Patients with a previous history of clinically relevant ocular toxicity related to iron chelation Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent the patient from undergoing study treatment Patients treated with systemic investigational drugs within the past 4 weeks or topical investigational drug within the past 7 days Any other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of any drug. Patients with active uncontrolled infectious disease Pregnancy or breast feeding Other protocol-defined inclusion/exclusion criteria may apply.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Novartis Pharmaceuticals

Organizational Affiliation

Novartis Pharmeceuticals

Official's Role

Study Director

Facility Information:

Facility Name

Novartis Investigative Site

City

Berlin

ZIP/Postal Code

12200

Country

Germany

12. IPD Sharing Statement

Citations:

PubMed Identifier

23073603

Citation

Nolte F, Hochsmann B, Giagounidis A, Lubbert M, Platzbecker U, Haase D, Luck A, Gattermann N, Taupitz M, Baier M, Leismann O, Junkes A, Schumann C, Hofmann WK, Schrezenmeier H. Results from a 1-year, open-label, single arm, multi-center trial evaluating the efficacy and safety of oral Deferasirox in patients diagnosed with low and int-1 risk myelodysplastic syndrome (MDS) and transfusion-dependent iron overload. Ann Hematol. 2013 Jan;92(2):191-8. doi: 10.1007/s00277-012-1594-z. Epub 2012 Oct 17.

Results Reference

result

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Efficacy and Safety of Deferasirox in Patients With Myelodysplastic Syndrome and Transfusion-dependent Iron Overload

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