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Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
This study is ongoing, but not recruiting participants.
First Received: October 6, 2005   Last Updated: May 1, 2008   History of Changes
Sponsored by: Novartis
Information provided by: Novartis
ClinicalTrials.gov Identifier: NCT00235391
  Purpose

This is an open-label, non-randomized, six month, 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 due to documented non-compliance, contraindications, unacceptable toxicities and/or documented poor response.


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

Study Type: Interventional
Study Design: Treatment, Non-Randomized, Open Label, Uncontrolled, Safety Study
Official Title: Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • Provide expanded access of deferasirox (ICL670) to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions
  • Further evaluate the safety profile of ICL670

Secondary Outcome Measures:
  • Evaluate the effect of treatment with ICL670 on changes in serum ferritin

Estimated Enrollment: 1396
Study Start Date: October 2005
  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 within one week prior to baseline
  • Patients with ALT ≥ 500 U/L within one week prior to baseline
  • Evidence of chelation-related cataracts or hearing loss within 4 weeks prior to baseline
  • Pregnancy (as indicated by serum β-HCG pregnancy test within 7 days of baseline 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
Please refer to this study by its ClinicalTrials.gov identifier: NCT00235391

  Show 27 Study Locations
Sponsors and Collaborators
Novartis
Investigators
Study Chair: Novartis Novartis
  More Information

Additional Information:
No publications provided

Study ID Numbers: CICL670A2203
Study First Received: October 6, 2005
Last Updated: May 1, 2008
ClinicalTrials.gov Identifier: NCT00235391     History of Changes
Health Authority: United States: Food and Drug Administration

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

Study placed in the following topic categories:
Diamond-Blackfan Anemia
Aplastic Anemia
Iron Metabolism Disorders
Red-Cell Aplasia, Pure
Aase Syndrome
Metaplasia
Anemia, Diamond-Blackfan
Anemia, Aplastic
Hemoglobinopathy
Anemia, Sickle Cell
Metabolic Disorder
Myelofibrosis
Metabolic Diseases
Deferasirox
Hematologic Diseases
Myeloproliferative Disorders
Anemia
Anemia, Hemolytic
Thalassemia
Myeloid Metaplasia
Lymphatic Diseases
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Hemoglobinopathies
Sickle Cell Anemia
Chelating Agents
Iron Overload
Bone Marrow Diseases
Iron

Additional relevant MeSH terms:
Molecular Mechanisms of Pharmacological Action
Iron Metabolism Disorders
Red-Cell Aplasia, Pure
Anemia, Diamond-Blackfan
Anemia, Aplastic
Anemia, Sickle Cell
Myelofibrosis
Metabolic Diseases
Hematologic Diseases
Deferasirox
Myeloproliferative Disorders
Anemia
Iron Chelating Agents
Anemia, Hemolytic
Thalassemia
Pharmacologic Actions
Myeloid Metaplasia
Anemia, Hemolytic, Congenital
Lymphatic Diseases
Anemia, Hypoplastic, Congenital
Genetic Diseases, Inborn
Hemoglobinopathies
Iron Overload
Chelating Agents
Bone Marrow Diseases
Splenic Diseases

ClinicalTrials.gov processed this record on July 02, 2009