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Trial record 3 of 8 for:    "Congenital Hypoplastic Anemia" | "Iron"

Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload

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: NCT00235391
Recruitment Status : Completed
First Posted : October 10, 2005
Results First Posted : May 2, 2011
Last Update Posted : June 7, 2011
Information provided by:

Brief Summary:
This is an open-label, non-randomized, multi-center trial designed to provide expanded access of deferasirox to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot adequately be treated with locally approved iron chelators.

Condition or disease Intervention/treatment Phase
Thalassemia Sickle Cell Disease Diamond Blackfan Anemia Myelofibrosis Drug: Deferasirox Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 1683 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Study to Provide Expanded Access of (Exjade®) Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload From Blood Transfusions Who Cannot Adequately be Treated With Other Locally Approved Iron Chelators
Study Start Date : October 2005
Actual Primary Completion Date : October 2008
Actual Study Completion Date : October 2008

Arm Intervention/treatment
Experimental: Deferasirox
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Starting dose was determined by the frequency of blood transfusions and recommended initial daily dose of deferasirox is 20 mg/kg body weight for patients receiving blood transfusion, 10 mg/kg for patients receiving less frequent transfusion/exchange transfusion and 30 mg/kg for patients receiving more frequent blood transfusions.
Drug: Deferasirox
125 mg, 250 mg and 500 mg tablets. Dosage was calculated based on participant's body weight. Tablets were dispersed in water, orange or apple juice and taken orally once a day.

Primary Outcome Measures :
  1. Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events [ Time Frame: Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks) ]
    Safety as assessed by the number of participants with death, serious adverse events (SAE), and/or Adverse Events (AEs) leading to study drug interruption or discontinuation. Note: only treatment emergent AEs are summarized.

Secondary Outcome Measures :
  1. The Change in Serum Ferritin Values From Baseline Through Completion of the Study [ Time Frame: Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks) ]
    The number of participants with Improvement, No Change or Worsening in Serum ferritin category levels at the end of the study compared to baseline. Serum ferritin levels in µg/L were divided into to 6 categories: (<1000), (1000-<2500), (2500-<4000), (4000-<5500), (5500-<7000) and (>=7000). Improvement was defined as a shift to a lower category at the end of study compared to the category at baseline. Worsening was defined as a shift to a higher category at the end of the study compared to the category at baseline. No change was no change in category at end of study from baseline.

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Male or female patients greater than or equal to 2 years of age
  • Documented congenital disorder of red blood cells (e.g., β-thalassemia major, sickle cell anemia, diamond-blackfan anemia) requiring ongoing blood transfusions
  • Cannot be adequately treated with a locally approved iron chelator due to one of the following reasons:

    • Documented non-compliance, defined as having taken less than 50% of the prescribed chelation therapy doses in the 12 months prior to study entry
    • Contraindications, unacceptable toxicities and/or documented poor response to locally approved iron chelators despite proper compliance
  • History of at least 20 blood transfusions (equivalent to 100 mL/kg of packed red blood cells (PRBC])
  • Serum ferritin value greater than or equal to 1000 µg/L
  • Ability to comply with all study-related procedures, medications, and evaluations

Exclusion Criteria:

  • Ongoing treatment with another iron chelator (Any other iron chelation therapy must be discontinued at least 24 hours prior to study entry.)
  • Patients who meet the eligibility criteria for any other ongoing Novartis sponsored clinical study protocol with deferasirox and who have geographic access to these sites
  • Patients unable to tolerate (or who have unacceptable toxicities to) prior treatment with deferasirox
  • Serum creatinine above the upper limit of normal at screening.
  • Patients with ALT ≥ 500 U/L at screening.
  • Evidence of chelation-related cataracts or hearing loss within 4 weeks prior to baseline
  • Pregnancy (as indicated by serum β-HCG pregnancy test at screening for all female patients with the potential to become pregnant) and patients who are breastfeeding
  • Patients treated with systemic investigational drug within 4 weeks prior to or with topical investigational drug within 7 days prior to the baseline visit

Other protocol-defined inclusion/exclusion criteria may apply.

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): NCT00235391

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Sponsors and Collaborators
Novartis Pharmaceuticals
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals

Additional Information:
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Responsible Party: External Affairs, Novartis Pharmaceuticals Identifier: NCT00235391     History of Changes
Other Study ID Numbers: CICL670A2203
First Posted: October 10, 2005    Key Record Dates
Results First Posted: May 2, 2011
Last Update Posted: June 7, 2011
Last Verified: June 2011

Keywords provided by Novartis:
Chronic Iron Overload
Transfusional Iron Overload
Iron Chelators
Oral Iron Chelators
Diamond Blackfan Anemia
Congenital Anemias
Red Blood Cell Disorders
Sickle Cell Disease

Additional relevant MeSH terms:
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Iron Chelating Agents
Anemia, Sickle Cell
Primary Myelofibrosis
Iron Overload
Anemia, Diamond-Blackfan
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Genetic Diseases, Inborn
Myeloproliferative Disorders
Bone Marrow Diseases
Iron Metabolism Disorders
Metabolic Diseases
Anemia, Hypoplastic, Congenital
Anemia, Aplastic
Red-Cell Aplasia, Pure
Chelating Agents
Trace Elements
Growth Substances
Physiological Effects of Drugs
Sequestering Agents
Molecular Mechanisms of Pharmacological Action