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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00235391
• Date of registration: 06/10/2005
• Prospective Registration: No
• Primary sponsor: Novartis Pharmaceuticals
• Public title: Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
• Scientific 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
• Date of first enrolment: October 2005
• Target sample size: 1683
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00235391
• Study type: Interventional
• Phase: Phase 3

Countries of recruitment

Australia, Belgium, Canada, Germany, Greece, Italy, Netherlands, Spain, Taiwan, Thailand, Turkey, United Kingdom, United States

Contacts

• Name: Novartis Pharmaceuticals
• Affiliation: Novartis Pharmaceuticals

Key inclusion & exclusion 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., ß-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 ß-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.



Age minimum: 2 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Thalassemia

Myelofibrosis

Sickle Cell Disease

Diamond Blackfan Anemia

Intervention(s)

Drug: Deferasirox

Primary Outcome(s)

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)]

Secondary Outcome(s)

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)]

Secondary ID(s)

CICL670A2203

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 02/05/2011
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00235391