Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias
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
1 other identifier
interventional
175
1 country
6
Brief Summary
The purpose of this study is to determine the effects of the oral iron chelator Deferasirox on liver iron content after one year of treatment in patients with iron overload from repeated blood transfusions. Beta-thalassemia patients unable to be treated with deferoxamine or patients with rare chronic anemias such as Myelodysplastic Syndrome, Fanconi's Syndrome, Blackfan-Diamond Syndrome, and Pure Red Blood Cell Anemia are eligible for this study. Liver iron content will be measured by liver biopsy at the beginning of the study and after one year of treatment. However, those patients living in the San Francisco/Oakland area may have a SQUID in place of the liver biopsy if the biopsy is not medically possible for them. The SQUID is a non-invasive magnetic means to measure liver iron content.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
6 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
May 1, 2003
CompletedFirst Submitted
Initial submission to the registry
June 3, 2003
CompletedFirst Posted
Study publicly available on registry
June 4, 2003
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 1, 2004
CompletedAugust 22, 2017
August 1, 2017
1.5 years
June 3, 2003
August 17, 2017
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
To evaluate the effects of treatment on the liver iron content(LIC)
Secondary Outcomes (4)
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
Interventions
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
- +4 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (6)
Children's Hospital Oakland
Oakland, California, 94609, United States
Stanford Hospital
Stanford, California, 94305-5208, United States
Northwest Medical Specialists
Arlington Heights, Illinois, 60004, United States
Children's Hospital Boston
Boston, Massachusetts, 02115, United States
Weill Medical College of Cornell University
New York, New York, 10021, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104-4318, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Novartis Pharmaceuticals
Novartis Pharmaceuticals
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
Study Record Dates
First Submitted
June 3, 2003
First Posted
June 4, 2003
Study Start
May 1, 2003
Primary Completion
November 1, 2004
Last Updated
August 22, 2017
Record last verified: 2017-08