Evaluation of the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration of ICL670 Relative to Deferoxamine(DFO).
A One Year Open Label, Non-comparative Extension to a Randomized, Multicenter, Phase II Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration (LIC) of Repeated Doses of 5-30mg/kg/Day ICL670 Relative to Deferoxamine (DFO) in Sickle Cell Disease (SCD) Patients With Transfusional Hemosideresis (THS) [Amendment 3: Extension Prolonged to 4-years]
2 other identifiers
interventional
185
5 countries
39
Brief Summary
The safety, tolerability, effects on liver iron concentration and pharmacokinetics of ICL670 is studied in sickle cell disease patients with transfusional hemosiderosis.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
39 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
July 1, 2004
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 1, 2009
CompletedFirst Submitted
Initial submission to the registry
March 15, 2010
CompletedFirst Posted
Study publicly available on registry
March 19, 2010
CompletedResults Posted
Study results publicly available
January 20, 2011
CompletedJune 6, 2011
May 1, 2011
5 years
March 15, 2010
December 21, 2010
May 3, 2011
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Number of Participants With Adverse Events After Start of ICL670
Safety as assessed by the number of participants with adverse event or death after the start of ICL670.
0 - 60 months
Secondary Outcomes (1)
Change in Serum Ferritin From Start of ICL670 to End of Study
0 - 60 months
Study Arms (1)
ICL670
EXPERIMENTALInterventions
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.
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (39)
University of South Alabama College of Medicine
Mobile, Alabama, 36604, United States
Loma Linda University Medical Center
Loma Linda, California, 92354, United States
Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
Children's Hospital & Research Center at Oakland
Oakland, California, 90609, United States
University of Colorado Health Science Center
Denver, Colorado, 80262, United States
Howard University Hospital
Washington D.C., District of Columbia, 20059, United States
St Joseph Children's Hospital of Tampa
Tampa, Florida, 33607, United States
Grady Hospital, Georgia Comprehensive Sickle Cell Center
Atlanta, Georgia, 30303, United States
Medical College of Georgia
Augusta, Georgia, 30912, United States
University of Illinois at Chicago
Chicago, Illinois, 60612, United States
Children's Memorial Hospital
Chicago, Illinois, 60614, United States
Tulane University Medical Center
New Orleans, Louisiana, 70112, United States
LSUHSC Dept of Pediatrics
Shreveport, Louisiana, 71130, United States
Children's Hospital Boston
Boston, Massachusetts, 02118, United States
Karmanos Cancer Institute
Detroit, Michigan, 48201, United States
New York Methodist Hospital
Brooklyn, New York, 11215, United States
New York Presbyterian Hospital
New York, New York, 10021, United States
Montefiore Medical Center
The Bronx, New York, 10467, United States
Wake Forest University Health Sciences
Winston-Salem, North Carolina, 27157, United States
Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
University of Cincinnati
Cincinnati, Ohio, 45267, United States
Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033, United States
Yasin
Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15213, United States
Palmetto Health Richland
Columbia, South Carolina, 29203, United States
Santee Hematology/Oncology
Sumter, South Carolina, 29150, United States
St. Jude's Children Research Hospital
Memphis, Tennessee, 38105, United States
Texas Children's Hospital
Houston, Texas, 77030, United States
The Methodist Hospital
Houston, Texas, 77030, United States
Scott & White Memorial Hospital
Temple, Texas, 76508, United States
Children's Hospital of the King's Daughters
Norfolk, Virginia, 23507, United States
Novartis Investigative Site
Toronto, Canada
Novartis Investigative Site
Créteil, France
Novartis Investigative Site
Paris, France
Novartis Investigative Site
Catania, Italy
Novartis Investigative Site
Genova, Italy
Novartis Investigative Site
Milan, Italy
Novartis Investigative Site
Roma, Italy
Novartis Investigative Site
London, United Kingdom
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Study Director
- Organization
- Novartis Pharmaceuticals
Study Officials
- STUDY DIRECTOR
Novartis Pharmaceuticals
Novartis Pharmaceuticals
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
Study Record Dates
First Submitted
March 15, 2010
First Posted
March 19, 2010
Study Start
July 1, 2004
Primary Completion
July 1, 2009
Last Updated
June 6, 2011
Results First Posted
January 20, 2011
Record last verified: 2011-05