Study of Deferasirox for Treatment of Transfusional Iron Overload in Myelodysplastic Patients - Full Text View

Sponsor:	Novartis Pharmaceuticals
Information provided by:	Novartis
ClinicalTrials.gov Identifier:	NCT00110266

Purpose

The purpose of this trial is to examine the safety and efficacy of deferasirox in patients with Myelodysplastic Syndrome (MDS) and chronic iron overload from blood transfusions.

Condition	Intervention	Phase
Myelodysplastic Syndrome Iron Overload	Drug: Deferasirox	Phase II

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Official Title:	An Open Label, Safety and Tolerability Study of Deferasirox for Treatment of Transfusional Iron Overload in Low-risk and INT-1, Myelodysplastic Patients Using Serum Ferritin Monitoring

Resource links provided by NLM:

Drug Information available for: Deferoxamine Deferasirox Deferoxamine mesylate

U.S. FDA Resources

Further study details as provided by Novartis:

Primary Outcome Measures:

12 month safety in patients with myelodysplastic syndrome (MDS) [ Time Frame: throughout the study ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

Changes in serum ferritin from baseline to 3, 6, 9 and 12 months after initiating treatment [ Time Frame: throughout the study ] [ Designated as safety issue: Yes ]
Effect of non-transferrin bound iron (NTBI), serum iron, transferrin and transferrin saturation on the safe administration of deferasirox [ Time Frame: throughout the study ] [ Designated as safety issue: Yes ]
Changes in transfusion requirements, serum erythropoietin levels, and estimated frequency of hematologic improvement in patients who are not receiving growth factors or chemotherapy for their underlying MDS. [ Time Frame: throughout the study ] [ Designated as safety issue: Yes ]
Trough pharmacokinetic parameters of deferasirox in patients with MDS [ Time Frame: throughout the study ] [ Designated as safety issue: No ]
Drug accountability [ Time Frame: through out the study ] [ Designated as safety issue: No ]

Enrollment:	176
Study Start Date:	May 2005
Estimated Study Completion Date:	April 2007
Primary Completion Date:	March 2008 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
1: Experimental Evaluate the safety and tolerability of deferasirox 20 mg/kg/day over one year in patients with MDS	Drug: Deferasirox 20 mg/kg/day over one year in patients with MDS

Detailed Description:

Study entry requires a diagnosis of low or intermediate (INT-1) risk MDS per International Prognostic Scoring System (IPSS) criteria and serum ferritin ≥ 1000 ng/mL. Patients must have had at least 30 prior red blood cell transfusions. Deferasirox will be administered at an initial dose of 20 mg/kg orally once per day. Patient transfusion history and at least three complete blood count (CBC) values must be available for the 12 weeks prior to study registration for patients with MDS and chronic iron overload from blood transfusions.

Eligibility

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

Criteria

Inclusion Criteria:

Male or female patients with low or intermediate (INT-1) risk MDS
Patients can be EITHER naïve to iron chelation OR have had prior treatment with deferoxamine (DFO).
Age greater than or equal to 18 years
Availability of transfusion records for the 12 weeks prior to registration
A lifetime minimum of 30 previous packed red blood cell transfusions
Availability of at least three CBC values (pretransfusion) during the 12 weeks prior to registration
Serum Ferritin:

For entry into the screening period, serum ferritin ≥ 1000 ng/mL on at least two occasions, at least two weeks apart, during the prior year.

Serum ferritin ≥ 1000 ng/mL at screening via the central lab.

Life expectancy ≥ 6 months
Sexually active women must use an effective method of contraception, or must have undergone clinically documented total hysterectomy and/or oophorectomy, or tubal ligation or be postmenopausal (defined as amenorrhea for at least 12 months)
Able to provide written informed consent

Exclusion Criteria:

Serum creatinine above the upper limit of normal
ALT > 500 U/L during screening
Clinical or laboratory evidence of active Hepatitis B or C
Urinary protein/creatinine ratio > 0.5 mg/mg
History of HIV positive test result (ELISA or Western blot)
ECOG Performance Status > 2
Patients with uncontrolled systemic hypertension
Unstable cardiac disease not controlled by standard medical therapy
Patients with a diagnosis of or history of clinically relevant ocular toxicity related to iron chelation
Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent study treatment
Pregnancy or breast feeding
Treatment with systemic investigational drug within the past 4 weeks or topical investigational drug within the past 7 days
Other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of study drug
History of non-compliance to medical regimens or patients who are considered potentially unreliable and/or not cooperative

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00110266

Show 37 Study Locations

Sponsors and Collaborators

Novartis Pharmaceuticals

Investigators

Study Director:

Novartis Pharmaceuticals

More Information

Additional Information:

Visit NovartisClinicalTrials.com: Pre-qualify for a trial, and view a list of trials and participating study centers. This link exits the ClinicalTrials.gov site

This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party:	Novartis ( External Affairs )
Study ID Numbers:	CICL670AUS03
Study First Received:	May 4, 2005
Last Updated:	November 18, 2009
ClinicalTrials.gov Identifier:	NCT00110266 History of Changes
Health Authority:	United States: Food and Drug Administration

Keywords provided by Novartis:

ICL670
Deferasirox
Iron chelation
Chelator
Desferal

Additional relevant MeSH terms:

Disease
Metabolic Diseases
Molecular Mechanisms of Pharmacological Action
Precancerous Conditions
Deferasirox
Hematologic Diseases
Growth Substances
Physiological Effects of Drugs
Myelodysplastic Syndromes
Iron Chelating Agents
Trace Elements

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

ClinicalTrials.gov processed this record on November 22, 2009