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

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Locations

United States, Alabama
Univ of Alabama Birmingham
Birmingham, Alabama, United States, 35294
United States, Arizona
Mayo Clinic
Scottsdale, Arizona, United States, 85259
United States, California
Cedars-Sinai Medical Center, UCLA School of Medicine
Los Angeles, California, United States, 90048
UCSF
San Francisco, California, United States, 94143-0324
City of Hope National Medical Center
Duarte, California, United States, 91010
Bay Area Cancer Research Group
Concord, California, United States, 94520
UCSF
San Francisco, California, United States, 94143
UCLA Medical Center
Los Angeles, California, United States, 90095-1678
United States, Colorado
Rocky Mountain Cancer Centers
Aurora, Colorado, United States, 80012
United States, Florida
Mayo Clinic
Jacksonville, Florida, United States, 32224
United States, Georgia
Winship Cancer Institute
Atlanta, Georgia, United States, 30322
United States, Hawaii
Straub Clinic and Hospital
Honolulu, Hawaii, United States, 96813
United States, Illinois
University of Chicago Hospital
Chicago, Illinois, United States, 60637-1470
United States, Kansas
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
United States, Kentucky
University of Kentucky College of Medicine, Markey Cancer Center
Lexington, Kentucky, United States, 40536-0093
United States, Louisiana
Cabrini Center for Cancer Care
Alexandria, Louisiana, United States, 71301
United States, Maryland
St. Agnes HealthCare
Baltimore, Maryland, United States, 21231-1000
United States, Massachusetts
Rush Cancer Institute Univ. of Massachussets Medical Center
Worchester, Massachusetts, United States, 01605
United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
United States, Missouri
The Center for Cancer Care & Research (TCCCR)
St. Louis, Missouri, United States, 63110
United States, Nebraska
Oncology Hematology West, PC
Omaha, Nebraska, United States, 68124-2346
United States, New Hampshire
Dartmouth Hitchcock Medical Center
Lebanon, New Hampshire, United States, 03756-0001
United States, New Jersey
Local Institution
Hackensack, New Jersey, United States, 07601
United States, New Mexico
NMOHC
Albuquerque, New Mexico, United States, 87109
United States, New York
Roswell Park Cancer Center
Buffalo, New York, United States, 14623
Rochester General Hospital
Rochester, New York, United States, 14621
United States, North Carolina
Wake Forest University
Winston Salem, North Carolina, United States, 27157-1082
Cancer Care of WNC
Asheville, North Carolina, United States, 28801
United States, Ohio
The Ohio State University
Columbus, Ohio, United States, 43210
The Cleveland Clinic Foundation
Cleveland, Ohio, United States, 44195
United States, Pennsylvania
Thomas Jefferson University; Jefferson Medical College, Kimmel Cancer Center
Philadelphia, Pennsylvania, United States, 19107
Western Pennsylvania Hospital; Cancer Institute
Pittsburgh, Pennsylvania, United States, 15224
United States, Tennessee
The West Cancer Clinic
Memphis, Tennessee, United States, 38120
United States, Texas
Baylor/Methodist
Houston, Texas, United States, 77030
United States, Utah
Utah Cancer Specialists
Salt Lake City, Utah, United States, 84106
United States, Virginia
Arlington Fairfax Hematology Oncology PC
Arlington, Virginia, United States, 22205
United States, Wisconsin
Medical College of Wisconsin
Milwaukee, Wisconsin, United States, 53226

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 27, 2009