Safety & Efficacy of ICL670 vs. Deferoxamine in Beta-thalassemia Patients With Iron Overload Due to Blood Transfusions
This study has been completed.
Sponsor:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT00061750
First received: June 3, 2003
Last updated: April 18, 2012
Last verified: April 2012
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Purpose
The purpose of this study is to deterimine if the new orally active iron chelator, ICL670, is as effective and as safe as deferoxamine in preventing accumulation of iron in the body while a patient is undergoing repeated blood transfusions.
| Condition | Intervention | Phase |
|---|---|---|
|
Beta-Thalassemia |
Drug: ICL670 Drug: deferoxamine |
Phase 3 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | A Randomized, Comparative, Open Label Phase III Trial on Efficacy & Safety of Long-term Treatment With ICL670 Compared to Deferoxamine in Beta-thalassemia Patients With Transfusional Hemosiderosis |
Resource links provided by NLM:
Further study details as provided by Novartis:
Primary Outcome Measures:
- Demonstrate non-inferiority to deferoxamine in its effects on liver iron content (LIC)
Secondary Outcome Measures:
- Evaluate tolerability profile
- Estimate absolute and relative change of LIC and Total body iron excretion
- Evaluation relationship between LIC and potential surrogate markers
- Evaluate the relationship between pharmacokinetics, pharmacodynamics and safety variable
| Enrollment: | 595 |
| Study Start Date: | May 2003 |
| Primary Completion Date: | November 2004 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: ICL670 |
Drug: ICL670
Other Name: Deferasirox
|
| Active Comparator: Deferoxamine | Drug: deferoxamine |
Detailed Description:
Patients who require repeated blood transfusions to live accumulate iron in the body as blood cells contain iron and there is no natural body mechanism to eliminate it. After a while the iron levels get high enough to be toxic to the body. The current therapy of choice is deferoxamine, which does a good job of removing excess iron, but is difficult to administer. Deferoxamine requires subcutaneous (under the skin) infusions over 4 to 8 hours nightly 3 to 7 nights per week. In addition to the need to wear an infusion pump nightly, adverse reactions around the site of the injection are frequent.
Eligibility| Ages Eligible for Study: | 2 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Beta-thalassemia patients already treated with or suitable for treatment with deferoxamine 20 to 40 mg/kg/day
- Liver iron content greater than 2 mg iron/g dw as measured by liver biopsy
- Need for regular transfusions 8 or more times per year
Exclusion Criteria:
- Non-transfusional iron overload or transfusion-dependent anemias other than beta-thalassemia.
- Documented toxicity to deferoxamine
- Elevated liver enzymes in the year preceeding 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 the 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.
- Inability to undergo a liver biopsy.
- Patients that would need a dose of ICL670 less than 125 mg per day.
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00061750
Locations
| United States, California | |
| Children's Hospital of Los Angeles | |
| Los Angeles, California, United States, 90027-6062 | |
| Children's Hospital Oakland | |
| Oakland, California, United States, 94609 | |
| Stanford Hospital | |
| Stanford, California, United States, 94305-5208 | |
| United States, Illinois | |
| Children's Memorial Hospital | |
| Chicago, Illinois, United States, 60614-3394 | |
| 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 | |
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
| Study Director: | Novartis Pharmaceuticals | Novartis Pharmaceuticals |
More Information
No publications provided by Novartis
Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Novartis ( Novartis Pharmaceuticals ) |
| ClinicalTrials.gov Identifier: | NCT00061750 History of Changes |
| Other Study ID Numbers: | CICL670A0107 |
| Study First Received: | June 3, 2003 |
| Last Updated: | April 18, 2012 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by Novartis:
|
Thalassemia, iron overload, deferoxamine, hemosiderosis |
Additional relevant MeSH terms:
|
Beta-Thalassemia Hemosiderosis Thalassemia Iron Overload Anemia, Hemolytic, Congenital Anemia, Hemolytic Anemia Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn |
Iron Metabolism Disorders Metabolic Diseases Deferoxamine Deferasirox Siderophores Iron Chelating Agents Chelating Agents Molecular Mechanisms of Pharmacological Action Pharmacologic Actions |
ClinicalTrials.gov processed this record on May 16, 2013