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A Randomized, Open Label, Phase II Study on Safety and Efficacy of Long Term Treatment of ICL670 Relative to Deferoxamine in Sickle Cell Disease Patients With Transfusional Hemosiderosis


Phase 2
2 Years
N/A
Not Enrolling
Both
Anemia, Sickle Cell

Thank you

Trial Information

A Randomized, Open Label, Phase II Study on Safety and Efficacy of Long Term Treatment of ICL670 Relative to Deferoxamine in Sickle Cell Disease Patients With Transfusional Hemosiderosis


Patients who require repeated blood transfusions 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.


Inclusion Criteria:



- Age greater than or equal to 2 years

- Sickle cell disease patients already treated with or suitable for treatment with
deferoxamine 20 to 40 mg/kg/day

- Serum ferritin greater than 1000 mg/ml

- Liver iron content greater than 2 mg iron/g dw assessed by means of superconducting
quantum interference device (SQUID) for patients who receive simple transfusions and
greater than 5 mg iron/ g dw for patients who receive exchange transfusions or who
have a history of intermittent blood transfusion.

- Regular transfusion aimed at maintaining % Hb A above 50% or a previous history of
simple transfusion being the recipient of at least 20 units of packed red blood
cells.

Exclusion Criteria:

- Chronic anemias other than sickle cell disease

- 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 the 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 (other than
beta-adrenergic receptor blocking agents).

- Diseases (cardiovascular, renal, hepatic, etc.) that would prevent the patient from
undergoing any of the treatment options

- Psychiatric or addictive 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

- Patients who require concomitant therapy with hydroxyurea

- 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

- Patients unable to undergo SQUID examination

Type of Study:

Interventional

Study Design:

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Evaluate the safety and tolerability of multiple doses of ICL670

Outcome Time Frame:

1 year

Safety Issue:

No

Principal Investigator

Novartis Pharmaceuticals

Investigator Role:

Study Director

Investigator Affiliation:

Novartis Pharmaceuticals

Authority:

United States: Food and Drug Administration

Study ID:

CICL670A0109

NCT ID:

NCT00067080

Start Date:

May 2003

Completion Date:

Related Keywords:

  • Anemia, Sickle Cell
  • Sickle cell disease
  • iron overload
  • deferoxamine
  • hemosiderosis
  • Anemia
  • Anemia, Sickle Cell
  • Hemosiderosis
  • Iron Overload

Name

Location

Baylor College of MedicineHouston, Texas  77030
Howard University HospitalWashington, District of Columbia  20060
Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania  19104
Loma Linda University Medical CenterLoma Linda, California  92354
Boston Medical CenterBoston, Massachusetts  02118
Children's Hospital Los AngelesLos Angeles, California  90027-0700
Children's Hospital of PittsburghPittsburgh, Pennsylvania  15213
Weill Medical College of Cornell UniversityNew York, New York  10021
University of Illinois at ChicagoChicago, Illinois  60612
Children's Memorial HospitalChicago, Illinois  60614
Santee Hematology/OncologySumter, South Carolina  29150
Karmanos Cancer InstituteDetroit, Michigan  48201
Wake Forest University School of MedicineWinston-Salem, North Carolina  27157-1023
U. of S. Alabama Medical CenterMobile, Alabama  36604
Children's Hospital & Research CenterOakland, California  94609
Colorado Sickle Cell Treatment and Research CenterDenver, Colorado  80262
Tampa Children's Hospital at St Joseph'sTampa, Florida  33607
Georgia Comprehensive Sickle cell Center, Grady HospitalAtlanta, Georgia  30335
Adult Sickle Cell Clinic, Medical College of GeorgiaAugusta, Georgia  30912
Tulane University Sickle Cell CenterNew Orleans, Louisiana  70112
Children's Hospital, Department of Hematology/OncologyNew Orleans, Louisiana  70118
Children's Hospital Boston, Division of Hematology/OncologyBoston, Massachusetts  02115
Sickle Cell Center, Montefiore HospitalBronx, New York  10467
NY Methodist HospitalBrooklyn, New York  11215
U. Of Rochester Medical CenterRochester, New York  14642
Children's Hospital Medical CenterCincinnati, Ohio  45229
Barrett Center, University of CincinnatiCincinnati, Ohio  45219
James Cancer HospitalColumbus, Ohio  43210
Penn State Milton S Hershey Medical CenterHershey, Pennsylvania  17033
Liberty Hematology Oncology CenterColumbia, South Carolina  29203
Palmetto Health Clinical TrialsColumbia, South Carolina  29203
Texas Children's Hospital/Baylor College of MedicineHouston, Texas  77030
Scott and White Memorial Hospital & ClinicsTemple, Texas  76508
Children's Hospital of the King's DaughterNorfolk, Virginia  23507