Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
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| ClinicalTrials.gov Identifier: NCT00235391 |
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Recruitment Status :
Completed
First Posted : October 10, 2005
Results First Posted : May 2, 2011
Last Update Posted : June 7, 2011
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Sponsor:
Novartis Pharmaceuticals
Information provided by:
Novartis
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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 |
| 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 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Primary myelofibrosis
Sickle cell disease
Diamond-Blackfan anemia
Drug Information available for:
Deferasirox
| Arm | Intervention/treatment |
|---|---|
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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.
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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 :
- 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 :
- 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.
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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 |
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
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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 ClinicalTrials.gov identifier (NCT number): NCT00235391
Locations
Show 141 study locations
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
| Study Director: | Novartis Pharmaceuticals | Novartis Pharmaceuticals |
Additional Information:
| Responsible Party: | External Affairs, Novartis Pharmaceuticals |
| ClinicalTrials.gov Identifier: | NCT00235391 |
| 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:
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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 ICL670A |
Additional relevant MeSH terms:
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Anemia Anemia, Sickle Cell Primary Myelofibrosis Thalassemia Anemia, Diamond-Blackfan Iron Overload Hematologic Diseases Anemia, Hemolytic, Congenital Anemia, Hemolytic Hemoglobinopathies Genetic Diseases, Inborn Myeloproliferative Disorders Bone Marrow Diseases |
Iron Metabolism Disorders Metabolic Diseases Anemia, Hypoplastic, Congenital Anemia, Aplastic Red-Cell Aplasia, Pure Congenital Bone Marrow Failure Syndromes Bone Marrow Failure Disorders Deferasirox Iron Chelating Agents Chelating Agents Sequestering Agents Molecular Mechanisms of Pharmacological Action |