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

NCT ID: NCT01044186

Last Updated: 2017-02-23

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2003-06-30

Brief Summary

The purpose of this open-label, non-comparative, multi-center protocol was to further evaluate safety and to provide treatment with ICL670 to patients who had or were at risk of life threatening complications due to transfusional iron overload with a documented inability to tolerate any of the commercially available iron chelators due to severe toxicity rendering continued therapy either impossible or hazardous. Patients who were also ineligible for all on-going registration trials with ICL670 were included in the study. In exceptional cases, patients with a degree of iron overload which was not immediately life-threatening and who were ineligible for the registration trials were also enrolled provided they had a well-documented, sound justification for alternative chelation therapy.

Conditions

Transfusional Iron Overload

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

ICL670

Group Type: EXPERIMENTAL

ICL670

Intervention Type: DRUG

Interventions

ICL670

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 2 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Novartis Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

Children's Hospital and Research Center - Oakland

Oakland, California, United States

Children's Hospital of Orange County

Orange, California, United States

Children's Hospital Boston

Boston, Massachusetts, United States

Queens Hospital Center

Jamaica, New York, United States

New York Presbyterian Hospital/Weill Medical College of Cornell University

New York, New York, United States

New York Methodist Hospital

New York, New York, United States

Cincinnatti Children's Hospital Medical center

Cincinnatti, Ohio, United States

Novartis Investigative Site

Houston, Texas, United States

Novartis investigative Site

Athens, , Greece

Novartis Investigative Site

Ancona, , Italy

Novartis Investigative Site

Brindisi, , Italy

Novartis Investigative Site

Cagliari, , Italy

Novartis Investigative Site

Cosenza, , Italy

Novartis Investigative Site

Florence, , Italy

Novartis Investigative Site

Milan, , Italy

Novartis Investigative Site

Modena, , Italy

Novartis Investigative Site

Napoli, , Italy

Novartis Investigative Site

Torino, , Italy

Countries

United States; Greece; Italy

Related Links

http://www.novartisclinicaltrials.com/

Related Info

Other Identifiers

2004-002303-32 EudraCT number

Identifier Type: REGISTRY

Identifier Source: secondary_id

CICL670A0117

Identifier Type: -

Identifier Source: org_study_id