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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


Phase 3
2 Years
N/A
Not Enrolling
Both
Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis

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Trial Information

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


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.

Type of Study:

Interventional

Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events

Outcome Description:

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.

Outcome Time Frame:

Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks)

Safety Issue:

No

Principal Investigator

Novartis Pharmaceuticals

Investigator Role:

Study Director

Investigator Affiliation:

Novartis Pharmaceuticals

Authority:

United States: Food and Drug Administration

Study ID:

CICL670A2203

NCT ID:

NCT00235391

Start Date:

October 2005

Completion Date:

October 2008

Related Keywords:

  • Thalassemia
  • Sickle Cell Disease
  • Diamond Blackfan Anemia
  • Myelofibrosis
  • 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
  • Primary Myelofibrosis
  • Anemia
  • Anemia, Sickle Cell
  • Thalassemia
  • Iron Overload
  • Anemia, Diamond-Blackfan

Name

Location

University of Mississippi Medical CenterJackson, Mississippi  39216-4505
Stanford UniversityStanford, California  94305
Children's Hospital of Orange CountyOrange, California  92668
Children's Mercy HospitalKansas City, Missouri  64108
Schneider Children's HospitalNew Hyde Park, New York  11042
Children's Hospitals and Clinics of MinnesotaSaint Paul, Minnesota  55102
Alta Bates Comprehensive Cancer CenterBerkeley, California  94704
Oregon Health & Science UniversityPortland, Oregon  97201
Texas Children's HospitalHouston, Texas  
The Cancer Institute of New JerseyNew Brunswick, New Jersey  08901
Hershey Medical CenterHershey, Pennsylvania  17033
James A. Haley Veterans HospitalTampa, Florida  33612
Arkansas Children's Hospital, UAMS College of MedicineLittle Rock, Arkansas  72202
Children's Hospital and Health Center of San DiegoSan Diego, California  92123
Alfred I. Dupong Hospital for ChildrenWilmington, Delaware  19803
Osler Medical, Inc.Melbourne, Florida  32901
Hematalogy Oncology AssociatesPensacola, Florida  32501
Tampa Children's Hospital at St. Joseph's HospitalTampa, Florida  33607
Backus Children's Hospital, Memorial Health University Medical CenterSavannah, Georgia  31403
Hematalogy Oncology ClinicBaton Rouge, Louisiana  70808
Borgess HospitalKalamazoo, Michigan  49048
PCTIColumbus, Ohio  43205
The Children's Medical Center of DaytonDayton, Ohio  45404
Children's Hospitals of PittsburghPittsburgh, Pennsylvania  15213
Children's Hospital of the Kings DaughtersNorfolk, Virginia  23507
VCU Pediatric Hematology/OncologyRichmond, Virginia  23219
University of Washington Seattle Cancer Care AllianceSeattle, Washington  98195