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Study for the Treatment of Transfusional Iron Overload in Myelodysplastic Patients

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00117507
Recruitment Status : Completed
First Posted : July 7, 2005
Last Update Posted : March 1, 2017
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Brief Summary:

Thirty patients will be enrolled into this open-label, single-arm trial designed to assess the safety and tolerability of oral deferasirox in adult transfusion dependent myelodysplastic syndrome (MDS) patients with iron overload. Patients enrolled in this study will have low or intermediate (INT-1) risk MDS per International Prognostic Scoring System (IPSS) criteria. All patients will initiate treatment with 20mg/kg/day deferasirox.

Deferasirox will be administered orally once per day for 12 months.

Condition or disease Intervention/treatment Phase
Myelodysplastic Syndromes Iron Overload Drug: deferasirox Phase 2

Detailed Description:
Patients will be screened for eligibility to determine if they meet all inclusion/exclusion criteria. The screening period will be up to 4 weeks. Patient's baseline LIC will be determined non-invasively by means of MRI R2 analysis. In addition, blood and urine samples will be taken for the determination of baseline safety data.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 24 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Study Start Date : April 2005
Actual Primary Completion Date : January 2008
Actual Study Completion Date : January 2008

Arm Intervention/treatment
Experimental: 20mg/kg/day deferasirox
Deferasirox will be administered orally once per day for 12 months. Surrogate marker findings, including serum ferritin, and LIC in the context of the study results will be monitored on a regular basis for any indications of clinically important over- or under-chelation.
Drug: deferasirox
deferasirox 20 mg/kg/day taken over one year.Deferasirox should be taken every morning 30 minutes before breakfast, if possible consistently around the same time between 7:00 and 9:00 AM. The tablets should be dropped into water or orange juice and gently stirred for 1 to 3 minutes until completely dispersed.
Other Names:
  • cheltor
  • iron chelator

Primary Outcome Measures :
  1. Safety and tolerability in myelodysplastic syndrome (MDS) patients [ Time Frame: 12 months ]

Secondary Outcome Measures :
  1. Efficacy based on serum ferritin and Liver iron concentration (LIC) [ Time Frame: through out the study ]
  2. Changes in liver iron concentration (LIC) [ Time Frame: through out the study ]
  3. Pharmacokinetics (PK) in MDS patients [ Time Frame: through out the study ]
  4. Non-transferrin bound iron (NTBI) & iron metabolism parameters [ Time Frame: through out the study ]
  5. Iron metabolism parameters [ Time Frame: through out the study ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Male or female patients with low or intermediate (INT-1) risk MDS, determined via IPSS criteria, with transfusional iron overload. NOTE: Bone marrow morphology and cytogenetic studies completed within 3 months prior to screening can be used if the patient has been hematologically stable. Every attempt to obtain cytogenetics studies should be made; however, if there is culture failure, repeat marrow aspiration will not be mandated. In this case, RAEB with less than 11% marrow blasts will be accepted.
  • Patients can be EITHER naïve to iron chelation OR have had prior treatment with deferoxamine (DFO). DFO must be discontinued the day prior to starting deferasirox dosing.
  • Age: greater than or equal to 18 years
  • Serum ferritin:

    • For entry into the screening period: serum ferritin greater than or equal to 1000 µg/mL on at least two occasions, at least two weeks apart, during the prior year. Samples must be obtained in the absence of concomitant infection;
    • For enrollment into the study: serum ferritin greater than or equal to 1000 µg/mL at screening (via the central lab) obtained in the absence of concomitant infection
  • A lifetime minimum of 30 previous packed red cell transfusions
  • Life expectancy greater than or equal to 6 months
  • Women must have a negative serum or urine pregnancy test and use an effective method of contraception, or must have undergone clinically documented total hysterectomy and/or oophorectomy, or tubal ligation or be postmenopausal (defined by amenorrhea for at least 12 months).
  • Able to provide written informed consent

Exclusion Criteria:

  • Serum creatinine above the upper limit of normal
  • ALT greater than 250 U/L during screening
  • Clinical or laboratory evidence of active hepatitis B or hepatitis C (HBsAg in the absence of HBsAb -OR- HCV Ab positive with HCV RNA positive and ALT above the normal range)
  • Significant proteinuria as indicated by a urinary protein/creatinine ratio greater than 0.5 mg/mg in a non-first void urine sample during screening (or alternatively in two of three samples obtained for screening)
  • History of HIV positive test result (ELISA or Western blot)
  • ECOG performance status greater than 2
  • Uncontrolled systemic hypertension
  • Unstable cardiac disease not controlled by standard medical therapy
  • Third degree atrioventricular (AV) block or QT interval prolongation above the normal range
  • 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 a systemic investigational drug within the past 4 weeks or a topical investigational drug within the past 7 days.
  • Other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of any drug. The investigator should be guided by evidence of any of the following:

    • inflammatory bowel syndrome, gastritis, ulcers, gastrointestinal or rectal bleeding;
    • major gastrointestinal tract surgery, such as gastrectomy, gastroenterostomy, or bowel resection;
    • pancreatic injury or pancreatitis or indications of impaired pancreatic function/injury, as indicated by abnormal lipase or amylase;
    • urinary obstruction or difficulty in voiding.
  • History of non-compliance to medical regimens or patients who are considered potentially unreliable and/or not cooperative

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00117507

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United States, California
Stanford University Medical Center
Stanford, California, United States, 94305-5821
United States, Michigan
Karmanos Cancer Center
Detroit, Michigan, United States, 48201
United States, Texas
MD Anderson Cancer Center
Houston, Texas, United States, 77030-4009
Sponsors and Collaborators
Novartis Pharmaceuticals
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
Additional Information:
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Responsible Party: Novartis Pharmaceuticals Identifier: NCT00117507    
Other Study ID Numbers: CICL670AUS02
First Posted: July 7, 2005    Key Record Dates
Last Update Posted: March 1, 2017
Last Verified: November 2016
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Myelodysplastic Syndrome
ICL-670 and Myelodysplastic Syndrome
Additional relevant MeSH terms:
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Myelodysplastic Syndromes
Iron Overload
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Iron Metabolism Disorders
Metabolic Diseases
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action