Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias
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|ClinicalTrials.gov Identifier: NCT00061763|
Recruitment Status : Completed
First Posted : June 4, 2003
Last Update Posted : August 22, 2017
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|Condition or disease||Intervention/treatment||Phase|
|Beta-thalassemia Myelodysplastic Syndromes Fanconi Syndrome Anemia, Diamond-Blackfan Anemia, Aplastic||Drug: Deferasirox||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||175 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||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|
|Study Start Date :||May 2003|
|Actual Primary Completion Date :||November 2004|
- To evaluate the effects of treatment on the liver iron content(LIC)
- Evaluate tolerability profile
- Estimate the absolute and relative change of LIC and total body iron excretion (TBIE) rate
- Evaluate the relationship between LIC and potential surrogate markers
- Evaluate the relationship between PD and safety variables
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|Ages Eligible for Study:||2 Years and older (Child, Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00061763
|United States, California|
|Children's Hospital Oakland|
|Oakland, California, United States, 94609|
|Stanford, California, United States, 94305-5208|
|United States, Illinois|
|Northwest Medical Specialists|
|Arlington Heights, Illinois, United States, 60004|
|United States, Massachusetts|
|Children's Hospital Boston|
|Boston, Massachusetts, United States, 02115|
|United States, New York|
|Weill Medical College of Cornell University|
|New York, New York, United States, 10021|
|United States, Pennsylvania|
|Children's Hospital of Philadelphia|
|Philadelphia, Pennsylvania, United States, 19104-4318|
|Study Director:||Novartis Pharmaceuticals||Novartis Pharmaceuticals|
|Responsible Party:||External Affairs, Novartis Pharmaceuticals|
|Other Study ID Numbers:||
|First Posted:||June 4, 2003 Key Record Dates|
|Last Update Posted:||August 22, 2017|
|Last Verified:||August 2017|
Bone Marrow Diseases
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Iron Metabolism Disorders
Bone Marrow Failure Disorders
Renal Tubular Transport, Inborn Errors
Female Urogenital Diseases
Female Urogenital Diseases and Pregnancy Complications