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

This study has been completed.
Sponsor:
Information provided by:
Novartis
ClinicalTrials.gov Identifier:
NCT00235391
First received: October 6, 2005
Last updated: June 3, 2011
Last verified: June 2011
  Purpose

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 Intervention Phase
Thalassemia
Sickle Cell Disease
Diamond Blackfan Anemia
Myelofibrosis
Drug: Deferasirox
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • 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) ] [ Designated as safety issue: No ]
    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:
  • 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) ] [ Designated as safety issue: No ]
    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.


Enrollment: 1683
Study Start Date: October 2005
Study Completion Date: October 2008
Primary Completion Date: October 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.

  Eligibility

Ages Eligible for Study:   2 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.

  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00235391

  Show 141 Study Locations
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

Additional Information:
No publications provided

Responsible Party: External Affairs, Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00235391     History of Changes
Other Study ID Numbers: CICL670A2203
Study First Received: October 6, 2005
Results First Received: December 17, 2010
Last Updated: June 3, 2011
Health Authority: United States: Food and Drug Administration

Keywords provided by Novartis:
Diamond Blackfan Anemia
Deferasirox
Congenital Anemias
Anemias
Red Blood Cell Disorders
Chronic Iron Overload
Transfusional Iron Overload
Iron Chelators
Oral Iron Chelators
Thalassemia
Sickle Cell Disease
Myelofibrosis
ICL670A

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

ClinicalTrials.gov processed this record on July 20, 2014