Status:
COMPLETED
Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
Lead Sponsor:
Novartis Pharmaceuticals
Conditions:
Thalassemia
Sickle Cell Disease
Eligibility:
All Genders
2+ years
Phase:
PHASE3
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 blo...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
October 1 2005
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 1 2008
Estimated Enrollment :
1683 Patients enrolled
Trial Details
Trial ID
NCT00235391
Start Date
October 1 2005
End Date
October 1 2008
Last Update
June 7 2011
Active Locations (141)
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1
Arkansas Children's Hospital, UAMS College of Medicine
Little Rock, Arkansas, United States, 72202
2
Alta Bates Comprehensive Cancer Center
Berkeley, California, United States, 94704
3
Children's Hospital of Orange County
Orange, California, United States, 92868
4
Children's Hospital and Health Center of San Diego
San Diego, California, United States, 92123