Evaluation of the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration of ICL670 Relative to Deferoxamine(DFO).

NCT ID: NCT01090323

Last Updated: 2011-06-06

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

185 participants

Study Classification

INTERVENTIONAL

Study Start Date

2004-07-31

Brief Summary

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The safety, tolerability, effects on liver iron concentration and pharmacokinetics of ICL670 is studied in sickle cell disease patients with transfusional hemosiderosis.

Detailed Description

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The treatment period started once the patient completed the core study and signed informed consent. It is continued for up to 4 years. Safety parameters were assessed every 4 weeks. Eye and Ear examinations were performed on a yearly basis. To further investigate the extent of iron overload, serum ferritin, iron, and transferrin were monitored every four weeks. The Program Safety Board monitored the safety of ICL670 during the study to evaluate and categorize any serious case reported in association with ICL670.

Conditions

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Sickle Cell Disease

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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ICL670

Group Type EXPERIMENTAL

ICL670

Intervention Type DRUG

Daily doses of ICL670 were taken orally 30 minutes before breakfast. The doses range from 5-40 mg/kg and were determined based on the patient's trend in serum ferritin over time during the core study (0109) and on the frequency of blood transfusions the patient received. The treatment duration was up to 4 years.

Interventions

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ICL670

Daily doses of ICL670 were taken orally 30 minutes before breakfast. The doses range from 5-40 mg/kg and were determined based on the patient's trend in serum ferritin over time during the core study (0109) and on the frequency of blood transfusions the patient received. The treatment duration was up to 4 years.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

Patients were included who met the following criteria:

* Completion of the core \[Study 0109\]
* Serum ferritin greater than or equal to 500 µg/L
* Ability to comply with all study-related procedures, medications, and evaluations
* Sexually active post-menarche female patients must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or oophorectomy, tubal ligation or be postmenopausal defined by amenorrhea for at least 12 months.
* Written informed consent and assent by the patient and or their parents or legal guardian.

Exclusion Criteria

Patients who met the following criteria were to be excluded:

* History of non-compliance to medical regimens and patients who are considered potentially unreliable and/or not cooperative
* Serum creatinine above the age-appropriate upper limit of normal within one week prior to entry
* Patients with ALT ≥ 500 U/L within one week prior to entry
* Evidence of chelation-related cataracts or hearing loss within 4 weeks prior to baseline
* Pregnancy (as indicated by serum β-HCG pregnancy test for all female patients with the potential to become pregnant) and patients who are breastfeeding
* Patients treated with systemic investigational drug within 4 weeks prior to or with topical investigational drug within 7 days prior to the baseline visit
Minimum Eligible Age

2 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Novartis Pharmaceuticals

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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University of South Alabama College of Medicine

Mobile, Alabama, United States

Site Status

Loma Linda University Medical Center

Loma Linda, California, United States

Site Status

Children's Hospital Los Angeles

Los Angeles, California, United States

Site Status

Children's Hospital & Research Center at Oakland

Oakland, California, United States

Site Status

University of Colorado Health Science Center

Denver, Colorado, United States

Site Status

Howard University Hospital

Washington D.C., District of Columbia, United States

Site Status

St Joseph Children's Hospital of Tampa

Tampa, Florida, United States

Site Status

Grady Hospital, Georgia Comprehensive Sickle Cell Center

Atlanta, Georgia, United States

Site Status

Medical College of Georgia

Augusta, Georgia, United States

Site Status

University of Illinois at Chicago

Chicago, Illinois, United States

Site Status

Children's Memorial Hospital

Chicago, Illinois, United States

Site Status

Tulane University Medical Center

New Orleans, Louisiana, United States

Site Status

LSUHSC Dept of Pediatrics

Shreveport, Louisiana, United States

Site Status

Children's Hospital Boston

Boston, Massachusetts, United States

Site Status

Karmanos Cancer Institute

Detroit, Michigan, United States

Site Status

New York Methodist Hospital

Brooklyn, New York, United States

Site Status

New York Presbyterian Hospital

New York, New York, United States

Site Status

Montefiore Medical Center

The Bronx, New York, United States

Site Status

Wake Forest University Health Sciences

Winston-Salem, North Carolina, United States

Site Status

Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

University of Cincinnati

Cincinnati, Ohio, United States

Site Status

Milton S. Hershey Medical Center

Hershey, Pennsylvania, United States

Site Status

Yasin

Philadelphia, Pennsylvania, United States

Site Status

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status

Palmetto Health Richland

Columbia, South Carolina, United States

Site Status

Santee Hematology/Oncology

Sumter, South Carolina, United States

Site Status

St. Jude's Children Research Hospital

Memphis, Tennessee, United States

Site Status

Texas Children's Hospital

Houston, Texas, United States

Site Status

The Methodist Hospital

Houston, Texas, United States

Site Status

Scott & White Memorial Hospital

Temple, Texas, United States

Site Status

Children's Hospital of the King's Daughters

Norfolk, Virginia, United States

Site Status

Novartis Investigative Site

Toronto, , Canada

Site Status

Novartis Investigative Site

Créteil, , France

Site Status

Novartis Investigative Site

Paris, , France

Site Status

Novartis Investigative Site

Catania, , Italy

Site Status

Novartis Investigative Site

Genova, , Italy

Site Status

Novartis Investigative Site

Milan, , Italy

Site Status

Novartis Investigative Site

Roma, , Italy

Site Status

Novartis Investigative Site

London, , United Kingdom

Site Status

Countries

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United States Canada France Italy United Kingdom

Other Identifiers

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EudraCT no. 2004-000597-31

Identifier Type: REGISTRY

Identifier Source: secondary_id

CICL670A0109E1

Identifier Type: -

Identifier Source: org_study_id

NCT00171197

Identifier Type: -

Identifier Source: nct_alias

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