A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity
![]() |
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT01044186 |
Recruitment Status :
Completed
First Posted : January 7, 2010
Last Update Posted : February 23, 2017
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Transfusional Iron Overload | Drug: ICL670 | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 30 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity |
Study Start Date : | June 2003 |
Actual Primary Completion Date : | December 2007 |

Arm | Intervention/treatment |
---|---|
Experimental: ICL670 |
Drug: ICL670 |
- To evaluate the safety profile and to provide treatment with ICL670 for patients with or at risk of life-threatening complications due to transfusional iron overload who are unable to tolerate other iron chelators because of documented severe toxicity. [ Time Frame: 0 - 163 weeks ]
- To estimate the absolute and relative change of liver iron concentration (LIC), to be measured using appropriate methodology available at individual centers. [ Time Frame: Yearly ]
- To evaluate the role of serum ferritin, serum iron, transferrin and transferrin saturation in monitoring iron burden in these patients. [ Time Frame: Quarterly ]
- To evaluate the relationship between changes in LIC and serum ferritin, transferring saturation and serum iron. [ Time Frame: Yearly ]

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 2 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Patients had to be at risk of life-threatening complications due to transfusional iron overload and be unable to tolerate therapy with any of the commercially available iron chelators (mainly deferoxamine and/or deferiprone) because of documented severe toxicity.
- Patients with a degree of iron overload which was not immediately life-threatening and who were ineligible for other trials with ICL670 could also be enrolled providing they had a well-documented, sound justification for alternative chelation therapy.
- Serum ferritin ≥ 8000 μg/L.
- Serum ferritin < 8000μg/L and LIC of ≥ 7 mg Fe/g dry weight.
- Patients for whom ≥ 8 blood transfusions per year were required in order to maintain the Hemoglobin level at > 9 g/dL.
- Female patients who have reached menarche and who were sexually active had to use double barrier contraception (oral plus barrier contraception), or had to have undergone total hysterectomy and/or ovariectomy, or tubal ligation.
- Written, voluntary informed consent.
Exclusion Criteria:
- Patients with transfusional iron overload who were not experiencing severe toxicities during therapy with other iron chelators (e.g. deferoxamine and/or deferiprone).
- Patients with non-transfusional hemosiderosis.
- Patients with severe liver failure as defined by a score of ≥ 10 points on the Child-Pugh scale.
- Patients with serum creatinine 1.5 times the upper limit of normal (ULN) at screening.
- Patients with a history of nephrotic syndrome.
- Patients with a diagnosis of clinically relevant cataract or a previous history of clinically relevant ocular toxicity related to iron chelation therapy.
- Patients with severe systemic diseases unrelated to iron overload and which would prevent them from undergoing treatment with ICL670.
- Patients with psychiatric or addictive disorders which prevent them from giving informed consent or undergoing treatment with ICL670.
- Pregnant or breast feeding patients.
- Patients treated with systemic investigational drugs within the past four weeks or topical investigational drugs within the past seven days.
-
Any surgical or medical condition which might significantly alter the absorption or excretion of drugs as shown by evidence of any of the following:
- History of inflammatory bowel disease
- History of major gastrointestinal tract surgery such as gastrectomy, gastroenterostomy, or bowel resection
- History of pancreatic injury or pancreatitis; indication of impaired pancreatic function/injury as indicated by abnormal lipase or amylase
- Patients being considered by the investigator as potentially unreliable and/or not cooperative with regard to the protocol.
- History of drug or alcohol abuse within the 12 months prior to dosing.

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): NCT01044186
United States, California | |
Children's Hospital and Research Center - Oakland | |
Oakland, California, United States, 94609 | |
Children's Hospital of Orange County | |
Orange, California, United States, 92868 | |
United States, Massachusetts | |
Children's Hospital Boston | |
Boston, Massachusetts, United States, 02115 | |
United States, New York | |
Queens Hospital Center | |
Jamaica, New York, United States, 11432 | |
New York Presbyterian Hospital/Weill Medical College of Cornell University | |
New York, New York, United States, 10021 | |
New York Methodist Hospital | |
New York, New York, United States, 11215 | |
United States, Ohio | |
Cincinnatti Children's Hospital Medical center | |
Cincinnatti, Ohio, United States, 45229 | |
United States, Texas | |
Novartis Investigative Site | |
Houston, Texas, United States, 77030 | |
Greece | |
Novartis investigative Site | |
Athens, Greece | |
Italy | |
Novartis Investigative Site | |
Ancona, Italy | |
Novartis Investigative Site | |
Brindisi, Italy | |
Novartis Investigative Site | |
Cagliari, Italy | |
Novartis Investigative Site | |
Cosenza, Italy | |
Novartis Investigative Site | |
Firenze, Italy | |
Novartis Investigative Site | |
Milano, Italy | |
Novartis Investigative Site | |
Modena, Italy | |
Novartis Investigative Site | |
Napoli, Italy | |
Novartis Investigative Site | |
Torino, Italy |
Study Director: | Novartis Pharmaceuticals | Novartis Pharmaceuticals |
Responsible Party: | Novartis Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT01044186 |
Other Study ID Numbers: |
CICL670A0117 2004-002303-32 EudraCT number ( Registry Identifier: 2004-002303-32 ) |
First Posted: | January 7, 2010 Key Record Dates |
Last Update Posted: | February 23, 2017 |
Last Verified: | February 2017 |
Deferasirox ICL670A Iron chelators Deferiprone Transfusional hemosiderosis |
Congenital aplastic anemia (Diamond Blackfan anemia) Red cell aplasia Thalassemia β thalassemia |
Iron Overload Iron Metabolism Disorders Metabolic Diseases Deferasirox |
Iron Chelating Agents Chelating Agents Sequestering Agents Molecular Mechanisms of Pharmacological Action |