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

This study is currently recruiting participants.

Verified by Novartis, June 2008

First Received: May 31, 2007 Last Updated: June 30, 2008 History of Changes

Sponsored by:	Novartis
Information provided by:	Novartis
ClinicalTrials.gov Identifier:	NCT00481143

Purpose

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.

Condition	Intervention	Phase
Myelodysplastic Syndromes Transfusion Dependent Iron Overload	Drug: ICL670/Deferasirox	Phase IV

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
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

Resource links provided by NLM:

MedlinePlus related topics: Blood Transfusion and Donation

Drug Information available for: Deferasirox

U.S. FDA Resources

Further study details as provided by Novartis:

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)

Estimated Enrollment:	120
Study Start Date:	May 2007

Eligibility

Ages Eligible for Study:	18 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00481143

Contacts

Contact: Novartis

41 61 324 1111

Locations

Germany
	Recruiting
Berlin, Germany, 12200

Sponsors and Collaborators

Novartis

Investigators

Study Chair:

Novartis

More Information

No publications provided

Responsible Party:	Novartis ( External Affairs )
Study ID Numbers:	CICL670ADE03
Study First Received:	May 31, 2007
Last Updated:	June 30, 2008
ClinicalTrials.gov Identifier:	NCT00481143 History of Changes
Health Authority:	Germany: Federal Institute for Drugs and Medical Devices

Keywords provided by Novartis:

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)

Study placed in the following topic categories:

Metabolic Diseases
Precancerous Conditions
Deferasirox
Hematologic Diseases
Myelodysplastic Syndromes
Anemia
Iron Metabolism Disorders
Refractory Anemia

Preleukemia
Anemia, Refractory
Hemosiderosis
Chelating Agents
Iron Overload
Bone Marrow Diseases
Iron
Metabolic Disorder

Additional relevant MeSH terms:

Disease
Metabolic Diseases
Molecular Mechanisms of Pharmacological Action
Precancerous Conditions
Hematologic Diseases
Deferasirox
Myelodysplastic Syndromes
Iron Chelating Agents
Iron Metabolism Disorders

Pharmacologic Actions
Preleukemia
Neoplasms
Pathologic Processes
Syndrome
Chelating Agents
Iron Overload
Bone Marrow Diseases

ClinicalTrials.gov processed this record on September 11, 2009