Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
NCT ID: NCT00235391
Last Updated: 2011-06-07
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
1683 participants
INTERVENTIONAL
2005-10-31
2008-10-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Deferasirox
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Starting dose was determined by the frequency of blood transfusions and recommended initial daily dose of deferasirox is 20 mg/kg body weight for patients receiving blood transfusion, 10 mg/kg for patients receiving less frequent transfusion/exchange transfusion and 30 mg/kg for patients receiving more frequent blood transfusions.
Deferasirox
125 mg, 250 mg and 500 mg tablets. Dosage was calculated based on participant's body weight. Tablets were dispersed in water, orange or apple juice and taken orally once a day.
Interventions
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Deferasirox
125 mg, 250 mg and 500 mg tablets. Dosage was calculated based on participant's body weight. Tablets were dispersed in water, orange or apple juice and taken orally once a day.
Eligibility Criteria
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Inclusion Criteria
* Documented congenital disorder of red blood cells (e.g., β-thalassemia major, sickle cell anemia, diamond-blackfan anemia) requiring ongoing blood transfusions
* Cannot be adequately treated with a locally approved iron chelator due to one of the following reasons:
* Documented non-compliance, defined as having taken less than 50% of the prescribed chelation therapy doses in the 12 months prior to study entry
* Contraindications, unacceptable toxicities and/or documented poor response to locally approved iron chelators despite proper compliance
* History of at least 20 blood transfusions (equivalent to 100 mL/kg of packed red blood cells (PRBC\])
* Serum ferritin value greater than or equal to 1000 µg/L
* Ability to comply with all study-related procedures, medications, and evaluations
Exclusion Criteria
* Patients who meet the eligibility criteria for any other ongoing Novartis sponsored clinical study protocol with deferasirox and who have geographic access to these sites
* Patients unable to tolerate (or who have unacceptable toxicities to) prior treatment with deferasirox
* Serum creatinine above the upper limit of normal at screening.
* Patients with ALT ≥ 500 U/L at screening.
* Evidence of chelation-related cataracts or hearing loss within 4 weeks prior to baseline
* Pregnancy (as indicated by serum β-HCG pregnancy test at screening 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
2 Years
ALL
No
Sponsors
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Novartis Pharmaceuticals
INDUSTRY
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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Arkansas Children's Hospital, UAMS College of Medicine
Little Rock, Arkansas, United States
Alta Bates Comprehensive Cancer Center
Berkeley, California, United States
Children's Hospital of Orange County
Orange, California, United States
Children's Hospital and Health Center of San Diego
San Diego, California, United States
Stanford University
Stanford, California, United States
Alfred I. Dupong Hospital for Children
Wilmington, Delaware, United States
Osler Medical, Inc.
Melbourne, Florida, United States
Hematalogy Oncology Associates
Pensacola, Florida, United States
Tampa Children's Hospital at St. Joseph's Hospital
Tampa, Florida, United States
James A. Haley Veterans Hospital
Tampa, Florida, United States
Backus Children's Hospital, Memorial Health University Medical Center
Savannah, Georgia, United States
Hematalogy Oncology Clinic
Baton Rouge, Louisiana, United States
Borgess Hospital
Kalamazoo, Michigan, United States
Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, United States
University of Mississippi Medical Center
Jackson, Mississippi, United States
Children's Mercy Hospital
Kansas City, Missouri, United States
The Cancer Institute of New Jersey
New Brunswick, New Jersey, United States
Schneider Children's Hospital
New Hyde Park, New York, United States
PCTI
Columbus, Ohio, United States
The Children's Medical Center of Dayton
Dayton, Ohio, United States
Oregon Health & Science University
Portland, Oregon, United States
Hershey Medical Center
Hershey, Pennsylvania, United States
Children's Hospitals of Pittsburgh
Pittsburgh, Pennsylvania, United States
Texas Children's Hospital
Houston, Texas, United States
Children's Hospital of the Kings Daughters
Norfolk, Virginia, United States
VCU Pediatric Hematology/Oncology
Richmond, Virginia, United States
University of Washington Seattle Cancer Care Alliance
Seattle, Washington, United States
Ziekenhuisnetwerk Antwerpen-AZ Middelheim
Antwerp, , Belgium
Centre Hospitalier Notre Dame et Reine
Charleroi, , Belgium
CHR de la Citadelle
Liège, , Belgium
Centre Hospitalier Chretien-Clinique Saint-Joseph
Montegnée, , Belgium
The Ottawa Hospital-General Campus
Ottawa, Ontario, Canada
University of Alberta
Edmonton, , Canada
CHUM-Hopital-Notre-Dame
Montreal, , Canada
MUHC- Montreal Children's Hospital
Montreal, , Canada
MUHC- Royal Victoria Hospital
Montreal, , Canada
Hopital de l'Enfant-Jesus
Québec, , Canada
The Hospital for Sick Children,
Toronto, , Canada
Toronto General Hospital-Hemoglobinopathy
Toronto, , Canada
Burrard Medical Building
Vancouver, , Canada
Charite-Universitatsmedizin Berlin
Berlin, , Germany
Universitatsklinikum Koln
Cologne, , Germany
Universitaetsklinik Dusseldorf
Düsseldorf, , Germany
Johann Wolfgang von Goethe Universitat
Frankfurt am Main, , Germany
Georg-August-Universitat Gottingen
Göttingen, , Germany
Universitatskrankenhaus Hamburg-Eppendorf
Hamburg, , Germany
Medizinische Hochschule Hannover
Hanover, , Germany
Klinikum Stuttgart Bismarckstrasse 8
Stuttgart, , Germany
Universitatsklinikum Ulm
Ulm, , Germany
Agia Sofia Hospital of Athens
Athens, , Greece
General Hospital of Athens
Athens, , Greece
Ippokration Hospital of Athens
Athens, , Greece
General Hospital of Athens
Corfu, , Greece
General Hospital of Korinthos
Corinth, , Greece
General Hospital of Heraklion Benizeleio-Pananeio
Heraklion, , Greece
University Hospital of Ioannina
Ioannina, , Greece
General Hospital of Kalamata
Kalamata, , Greece
Agia Sofia Hospital of Athens
Karditsa, , Greece
General Hospital of Karditsa
Karditsa, , Greece
General Hospital of Larisa Tsakalof 1
Larissa, , Greece
General Hospital of Mytilini Vostaneio
Mytilene, , Greece
General State Hospital of Nikaia St. Panteleimon
Nikaia, , Greece
University Hospital of Patras
Pátrai, , Greece
General Hospital of Thessaloniki Agios Pavlos
Thessaloniki, , Greece
General Hospital Thessalonikis Hippokratio
Thessaloniki, , Greece
General Hospital of Volos
Volos, , Greece
General Hospital of Xanthi
Xánthi, , Greece
Presidio Ospedale Muscatello
Augusta, , Italy
A.O. Ospedale Policlinico Consorziale di Bari
Bari, , Italy
Presidio Ospedaliero Antonio Perrino
Brindisi, , Italy
Ospedale Regionale Microcitemie
Cagliari, , Italy
Ospedale Di Venere
Carbonara di Bari, , Italy
Azienda Ospedali Vittorio Emanuele, Ferrarotto e San Bambino
Catania, , Italy
Presidio Ospedaliero S. Bambino
Catania, , Italy
PresidioOspedaliero S. Luigi Curro
Catania, , Italy
Azienda Ospedaliera Pugliese Cicaccio
Catanzaro, , Italy
Ospedale Civile dell'Annunziata
Cosenza, , Italy
Ospedale San Giuseppe
Empoli, , Italy
Azienda Ospedaliera Universitaria di Ferrara
Ferrara, , Italy
Azienda Ospedaliera A. Meyer
Florence, , Italy
E.O. Ospedali Galliera
Genova, , Italy
Ospedale Regionale Microcitemie
Itala, , Italy
Ospedale Madonna delle Grazie
Matera, , Italy
Az. Ospedaliera Universitaria Policlinico G. Martino
Messina, , Italy
Fondazione Ospedale
Milan, , Italy
Azienda Ospedaliero-Universitaria di Modena
Modena, , Italy
AORN A. Cardarelli
Napoli, , Italy
Azienda Ospedaliera Universitaria Federico II
Napoli, , Italy
Azienda Ospedaliera V. Cervello
Palermo, , Italy
Azienda Ospedaliera Villa Sofia-CTO
Palermo, , Italy
Presidio Ospedaliero Giovanni di Cristina
Palermo, , Italy
IRCCS Policlinico San Matteo
Pavia, , Italy
Azienda Ospedaliera San Salvatore
Pesaro, , Italy
Azienda Ospedaliero Universitaria Pisana
Pisa, , Italy
Azienda Ospedaliera Civile- Maria Paterno
Ragusa, , Italy
Azienda Ospedaliera Bianchi-Melacrino-Morelli
Reggio Calabria, , Italy
Ospedale S. Eugenio
Roma, , Italy
Ospedale nostra Signora di Bonaria
San Gavino Monreale- CA, , Italy
Presidio Ospedaliero di Sassari-Ospedale SS
Sassari, , Italy
Azienda Ospedaliera Ospedali Civili Riuniti di Sciacca
Sciacca, , Italy
Ospedale Umberto I
Talassemie, , Italy
Presidio Ospedaliero Centrale
Taranto, , Italy
Ospedale Infantile Regina Margherita
Torino, , Italy
AMC
Amsterdam, , Netherlands
Catharina-ziekenhuis
Eindhoven, , Netherlands
Erasmus MC
Rotterdam, , Netherlands
Erasmus Medisch Centrum, locatie Sophia
Rotterdam, , Netherlands
Haga Ziekenhuis
The Hague, , Netherlands
Hospital de Torrecardenas
Almería, , Spain
Hospital Infanta Cristina
Badajoz, , Spain
Hospital Cruces
Barakaldo, , Spain
Hospital Puerta del Mar
Cadiz, , Spain
Hospital De Gran Canaria
Canara, , Spain
Hospital Universitario La Paz
Madrid, , Spain
Althaia : Xarxa Assistencial de Manresa
Manresa, , Spain
Hospital Universitario Marques de Valdecilla
Santander, , Spain
Hospital Virgen de la Salud
Toledo, , Spain
Hospital Universitario La Fe
Valencia, , Spain
Hospital Xeral de Vigo
Vigo, , Spain
Kaohsiung Medical University Hospital
Kaohsiung City, , Taiwan
Mackay Memorial Hospital
Taipei, , Taiwan
National Taiwan University Hospital
Taipei, , Taiwan
Chang Gung Children's Hospital
Taoyuan District, , Taiwan
Tao-yuan General Hospital
Tau-Yuan County, , Taiwan
Phramongkutklao Hospital
Bangkok, , Thailand
Ramathibodi Hospital
Bangkok, , Thailand
Songklanagarind Hospital
Bangkok, , Thailand
Srinagarind Hospital
Khon Kaen, , Thailand
Cukurova Universitesi
Adana, , Turkey (Türkiye)
Hacettepe Universitesi
Ankara, , Turkey (Türkiye)
Akdeniz Universitesi
Antalya, , Turkey (Türkiye)
Gaziantep Universitesi
Gaziantep, , Turkey (Türkiye)
Suleyman Demirel
Isparta, , Turkey (Türkiye)
Istanbul Universitesi
Istanbul, , Turkey (Türkiye)
Ege Universitesi Tip Fakultesi
Izmir, , Turkey (Türkiye)
Erciyes Universitesi
Kayseri, , Turkey (Türkiye)
Evelina Hospital St. Thomas' Hospital
London, , United Kingdom
North Middlesex University Hospital
London, , United Kingdom
The Royal Hospital London
London, , United Kingdom
Whittington Hospital
London, , United Kingdom
St. George's Hospital
Tooting, , United Kingdom
Countries
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Related Links
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Visit NovartisClinicalTrials.com: Pre-qualify for a trial, and view a list of trials and participating study centers.
Other Identifiers
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CICL670A2203
Identifier Type: -
Identifier Source: org_study_id
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