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

• ClinicalTrials.gov Identifier: NCT00235391
• Recruitment Status: Completed
• First Posted: October 10, 2005
• Results First Posted: May 2, 2011
• Last Update Posted: June 7, 2011
• Sponsor: Novartis Pharmaceuticals
• Information provided by: Novartis

Tracking Information

• First Submitted Date: October 6, 2005
• First Posted Date: October 10, 2005
• Results First Submitted Date: December 17, 2010
• Results First Posted Date: May 2, 2011
• Last Update Posted Date: June 7, 2011
• Study Start Date: October 2005
• Actual Primary Completion Date: October 2008 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: June 3, 2011)

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.

• Original Primary Outcome Measures: Not Provided

Current Secondary Outcome Measures (submitted: June 3, 2011)

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.

• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
• 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
• 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Non-Randomized; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment

Condition

• Thalassemia
• Sickle Cell Disease
• Diamond Blackfan Anemia
• Myelofibrosis

Intervention

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.

Study Arms

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.

Intervention: Drug: Deferasirox

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: March 31, 2011): 1683
• Original Enrollment (submitted: October 6, 2005): 3000
• Actual Study Completion Date: October 2008
• Actual Primary Completion Date: October 2008 (Final data collection date for primary outcome measure)

Eligibility 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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 2 Years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Belgium, Canada, Germany, Greece, Italy, Netherlands, Spain, Taiwan, Thailand, Turkey, United Kingdom, United States
• Removed Location Countries: Australia

Administrative Information

• NCT Number: NCT00235391
• Other Study ID Numbers: CICL670A2203
• Has Data Monitoring Committee: Not Provided
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: External Affairs, Novartis Pharmaceuticals
• Original Responsible Party: Not Provided
• Current Study Sponsor: Novartis Pharmaceuticals
• Original Study Sponsor: Novartis
• Collaborators: Not Provided

Investigators

• Study Director: Novartis Pharmaceuticals; Novartis Pharmaceuticals

• PRS Account: Novartis
• Verification Date: June 2011