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Phase II Study of Safety & Efficacy of Deferasirox Given for 1 Year in Patients With Chronic Anemias and Transfusional Hemosiderosis Unable to be Treated With Deferoxamine


Phase 2
2 Years
N/A
Not Enrolling
Both
Beta-thalassemia, Myelodysplastic Syndromes, Fanconi Syndrome, Anemia, Diamond-Blackfan, Anemia, Aplastic

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Trial Information

Phase II Study of Safety & Efficacy of Deferasirox Given for 1 Year in Patients With Chronic Anemias and Transfusional Hemosiderosis Unable to be Treated With Deferoxamine


Inclusion Criteria:



- Beta-thalassemia patients with documented non-compliance to deferoxamine, defined as
taking less than 50% of prescribed doses in year prior to study, and having a liver
iron content at least 14 mg iron/gm dry weight liver tissue

- Beta-thalassemia patients unable to take deferoxamine because of documented side
effects or contra-indication, or documented poor response despite proper compliance,
with liver iron content at least 2 mg iron/gm dry weight liver tissue

- Patients with chronic anemias with a liver iron content at least 2 mg/gm dry weight
liver tissue.

- Beta-thalassemia or other chronic anemia patients having previously taken
deferiprone, provided that they stop the deferiprone at least 28 days before the
study and have a liver iron content at least 2 mg/gm dry weight liver tissue.

- All patients: Regular transfusions indicated by a requirement of at least 8 blood
transfusions per year.

- Life expectancy of at least one year.

Exclusion Criteria:

- Beta-thalassemia able to be treated with deferoxamine, Sickle Cell Disease or
non-transfusional iron overload

- Elevated liver enzymes in the year preceding enrollment

- Active Hepatitis B or Hepatitis C

- HIV seropositivity

- Elevated serum creatinine or significant proteinuria

- History of nephrotic syndrome

- Uncontrolled systemic hypertension

- Fever and other signs/symptoms of infection within 10 days prior to start of the
study.

- Presence of clinically relevant cataract or previous history of clinically relevant
ocular toxicity related to iron chelation.

- Second or third degree AV block, clinically relevant Q-T interval prolongation, or
patients requiring digoxin or other drugs that prolong the Q-T interval.

- Diseases (cardiovascular, renal, hepatic, etc.) that would prevent the patient from
undergoing any of the treatment options.

- Psychiatric or additive disorders that would prevent the patient from giving informed
consent.

- History of drug or alcohol abuse within the 12 months prior to the study.

- Pregnant or breast feeding patients.

- Patients treated with systemic investigational drugs within 4 weeks or topical
investigational drugs within 7 days before the start of teh study.

- Any surgical or medical condition that might significantly alter the absorption,
distribution, metabolism or excretion of any drug, such as gastrointestinal disease
or major surgery, renal disease, difficulty voiding or urinary obstruction, or
impaired pancreatic function.

- Non-compliant or unreliable patients

- Patients unable to undergo any study procedures such as the hearing or eye tests, or
the liver echocardiography.

- Patients that would need a dose of Deferasirox less than 125 mg per day.

Type of Study:

Interventional

Study Design:

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

Outcome Measure:

To evaluate the effects of treatment on the liver iron content(LIC)

Principal Investigator

Novartis Pharmaceuticals

Investigator Role:

Study Director

Investigator Affiliation:

Novartis Pharmaceuticals

Authority:

United States: Food and Drug Administration

Study ID:

CICL670A0108

NCT ID:

NCT00061763

Start Date:

May 2003

Completion Date:

Related Keywords:

  • Beta-Thalassemia
  • Myelodysplastic Syndromes
  • Fanconi Syndrome
  • Anemia, Diamond-Blackfan
  • Anemia, Aplastic
  • beta-thalassemia
  • iron overload
  • deferoxamine
  • Myelodysplastic Syndromes
  • Fanconi Syndrome
  • Anemia, Diamond-Blackfan
  • Anemia, Aplastic
  • Anemia
  • Anemia, Aplastic
  • Beta-Thalassemia
  • Fanconi Syndrome
  • Fanconi Anemia
  • Myelodysplastic Syndromes
  • Preleukemia
  • Thalassemia
  • Iron Overload
  • Anemia, Diamond-Blackfan

Name

Location

Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania  19104
Weill Medical College of Cornell UniversityNew York, New York  10021
Children's Hospital BostonBoston, Massachusetts  02115
Northwest Medical SpecialistsArlington Heights, Illinois  60004
Children's Hospital OaklandOakland, California  94609
Stanford HospitalStanford, California  94305-5208