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A Randomized, Open-label, Multi-center, Phase II Study to Evaluate the Safety and Efficacy of Deferasirox (ICL670) 20 mg/kg/Day Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients With Iron Overload From Repeated Blood Transfusions


Phase 2
2 Years
N/A
Not Enrolling
Both
Sickle Cell Disease, Iron Overload, Hemolytic Anemia

Thank you

Trial Information

A Randomized, Open-label, Multi-center, Phase II Study to Evaluate the Safety and Efficacy of Deferasirox (ICL670) 20 mg/kg/Day Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients With Iron Overload From Repeated Blood Transfusions


Inclusion Criteria:



- Age greater than or equal to 2 years

- Male or female patients with sickle cell disease (SS, SC, SD, Sβo or Sβ+ thalassemia)

- Iron overload from repeated blood transfusion, as defined by one of the following:

1. For patients > 16 years old receiving simple transfusions: lifetime history of
receipt of at least 120 ml/kg or 30 adult units of packed red blood cells, OR

2. For patients ≤ 16 years old receiving simple transfusions: lifetime history of
receipt of at least 120 ml/kg of packed red blood cells, OR

3. For all patients receiving exchange transfusions in the absence of a previous
attempt to achieve negative iron balance: lifetime performance of at least 20
procedures, OR

4. For all patients: liver iron content ≥ 7 mg Fe/g dry weight as measured by
biopsy, Magnetic Resonance Imaging (MRI), or magnetic susceptibility performed
within 3 months prior to entry into screening

- For entry into the screening period: serum ferritin of ≥ 1000 µg/mL on at least two
occasions during the prior year obtained in the absence of concomitant infection.

- Body weight > 10 kg

- No known allergy or contraindication to the administration of deferoxamine

- Ability to comply with all study-related procedures, medications, and evaluations

- Sexually active pre-menopausal 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 by the patient or for pediatric patient's consent of the
patient's legal guardian. The definition of the term 'pediatric' for enrollment and
study conduct will be in accordance with the local legislation.

Exclusion Criteria:

- Serum creatinine above the upper limit of normal

- Significant proteinuria

- History of nephrotic syndrome

- Alanine aminotransferase (ALT) ≥ 250 U/L at screening

- Clinical evidence of active hepatitis B or hepatitis C

- History of HIV

- Fever or other signs/symptoms of infection within 10 days prior to the screening
visit

- Uncontrolled systemic hypertension

- History of Myocardial Infarction, Congestive Heart Failure or unstable cardiac
disease not controlled by standard medical therapy

- Clinically relevant cataract or a previous history of clinically relevant ocular
toxicity related to iron chelation

- Presence of a surgical or medical condition that might significantly alter the
absorption, distribution, metabolism or excretion of any study drug

- History of drug or alcohol abuse within the 12 months prior to enrollment

- Pregnant or breast feeding patients

- Patients treated with systemic investigational drug within 4 weeks prior or with
topical investigational drug 7 days prior to the screening visit

- Randomization in a previous clinical trial involving ICL670

Other protocol-related inclusion / exclusion criteria may apply.

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:

The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment

Outcome Description:

The number of participants with Adverse Events (AEs) overall and according to Medical Dictionary for Regulatory Activities (MedDRA) preferred term greater than or equal to 5% participants in any group by treatment in the first 24 weeks.

Outcome Time Frame:

24 Weeks

Safety Issue:

Yes

Principal Investigator

Novartis Pharmaceuticals

Investigator Role:

Study Director

Investigator Affiliation:

Novartis Pharmaceuticals

Authority:

United States: Food and Drug Administration

Study ID:

CICL670A2201

NCT ID:

NCT00110617

Start Date:

May 2005

Completion Date:

April 2008

Related Keywords:

  • Sickle Cell Disease
  • Iron Overload
  • Hemolytic Anemia
  • Sickle Cell Disease
  • Iron Overload from Repeated Blood Transfusions
  • Iron Overload
  • Blood Transfusions
  • Anemia
  • Anemia, Hemolytic
  • Anemia, Sickle Cell
  • Iron Overload
  • Hemolysis

Name

Location

Howard University HospitalWashington, District of Columbia  20060
University of MichiganAnn Arbor, Michigan  48109-0624
Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania  19104
Washington University School of MedicineSaint Louis, Missouri  63110
Loma Linda University Medical CenterLoma Linda, California  92354
New York Methodist HospitalBrooklyn, New York  11215-3609
Children's National Medical CenterWashington, District of Columbia  20010-2970
Children's Hospital of PittsburghPittsburgh, Pennsylvania  15213
University of South Alabama Medical CenterMobile, Alabama  36604
Miami Children's HospitalMiami, Florida  33155-4069
Weill Medical College of Cornell UniversityNew York, New York  10021
University of Illinois at ChicagoChicago, Illinois  60612
SUNY Downstate Medical CenterBrooklyn, New York  11203
Emory University School of MedicineAtlanta, Georgia  30322
Columbia UniversityNew York, New York  10032-3784
Children's Hospital BostonBoston, Massachusetts  02115
Medical College of VirginiaRichmond, Virginia  23298-0341
Children's Memorial HospitalChicago, Illinois  60614
Santee Hematology/OncologySumter, South Carolina  29150
Virginia Commonwealth UniversityRichmond, Virginia  
Karmanos Cancer InstituteDetroit, Michigan  48201
Children's Hospital OaklandOakland, California  94609
Drexel University College of MedicinePhiladelphia, Pennsylvania  19129
University of CincinnatiCincinnati, Ohio  45267-0502
Tampa Children's Hospital at St Joseph'sTampa, Florida  33607
Tulane University Sickle Cell CenterNew Orleans, Louisiana  70112
Sickle Cell Center, Montefiore HospitalBronx, New York  10467
Children's Hospital Medical CenterCincinnati, Ohio  45229
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
Hillman Cancer CenterPittsburg, Pennsylvania  15232
University of South AlabamaMobile, Alabama  36693
University of Alabama Pediatric Hematology/OncologyBirmingham, Alabama  35233
University of Alabama Medical centerBirmingham, Alabama  35294
Center for Cancer and Blood DisordersWashington, District of Columbia  20010-2970
Tampa Children's Hospital at St. Joseph'sTampa, Florida  33607-6387
H. Lee Muffit Cancer Center and Research Institute/James A. Haley Veterans HospitalTampa, Florida  33612
Children's Healthcare of Atlanta at Scottish RiteAtlanta, Georgia  30342
Adult Sickle Cell ClinicAugusta, Georgia  30912-3128
Backus Children's HospitalSavannah, Georgia  31403
Pediatric Sickle Cell Program/James Whitcomb Riley Hospital for ChildrenIndianapolis, Indiana  46202
St. Jude Children's Hospital AffiliateBaton Rouge, Louisiana  70808
Children's HospitalNew Orleans, Louisiana  70118
LSU Health Sciences Center/Carroll W. Feist Professor of Cancer ResearchShreveport, Louisiana  71130
Children's HospitalBoston, Massachusetts  02115
Brigham and Woman's Hospital/Harvard Medical SchoolBoston, Massachusetts  02115
Carolinas Medical Transplant CenterCharlotte, North Carolina  28232
The University of OklahomaOklahoma City, Oklahoma  73104
Jefferson UniversityPhiladelphia, Pennsylvania  19107
Pennsylvania Oncology/HematologyPhiladelphia, Pennsylvania  19106
St Jude's Children's Research HospitalMemphis, Tennessee  38105-2794
St. Jude's Children Research HospitalMemphis, Tennessee  38105
Cooks Children's HospitalFort Worth, Texas  76104-2724