Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
- Conditions
- Diamond Blackfan AnemiaThalassemiaSickle Cell DiseaseMyelofibrosis
- Interventions
- Registration Number
- NCT00235391
- Lead Sponsor
- Novartis Pharmaceuticals
- Brief Summary
This is an open-label, non-randomized, multi-center trial designed to provide expanded access of deferasirox to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot adequately be treated with locally approved iron chelators.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 1683
-
Male or female patients greater than or equal to 2 years of age
-
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
- Ongoing treatment with another iron chelator (Any other iron chelation therapy must be discontinued at least 24 hours prior to study entry.)
- 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
Other protocol-defined inclusion/exclusion criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Deferasirox 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.
- Primary Outcome Measures
Name Time Method Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks) Safety as assessed by the number of participants with death, serious adverse events (SAE), and/or Adverse Events (AEs) leading to study drug interruption or discontinuation. Note: only treatment emergent AEs are summarized.
- Secondary Outcome Measures
Name Time Method The Change in Serum Ferritin Values From Baseline Through Completion of the Study Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks) The number of participants with Improvement, No Change or Worsening in Serum ferritin category levels at the end of the study compared to baseline. Serum ferritin levels in µg/L were divided into to 6 categories: (\<1000), (1000-\<2500), (2500-\<4000), (4000-\<5500), (5500-\<7000) and (\>=7000). Improvement was defined as a shift to a lower category at the end of study compared to the category at baseline. Worsening was defined as a shift to a higher category at the end of the study compared to the category at baseline. No change was no change in category at end of study from baseline.
Trial Locations
- Locations (138)
Azienda Ospedali Vittorio Emanuele, Ferrarotto e San Bambino
🇮🇹Catania, Italy
PresidioOspedaliero S. Luigi Curro
🇮🇹Catania, Italy
PCTI
🇺🇸Columbus, Ohio, United States
Children's Hospitals of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
Ziekenhuisnetwerk Antwerpen-AZ Middelheim
🇧🇪Antwerpen, Belgium
Centre Hospitalier Notre Dame et Reine
🇧🇪Charleroi, Belgium
General Hospital of Athens
🇬🇷Kerkyra, Greece
General Hospital of Thessaloniki Agios Pavlos
🇬🇷Thessaloniki, Greece
General Hospital of Kalamata
🇬🇷Kalamata, Greece
University Hospital of Patras
🇬🇷Patra, Greece
General Hospital Thessalonikis Hippokratio
🇬🇷Thessaloniki, Greece
General Hospital of Volos
🇬🇷Volos, Greece
General Hospital of Larisa Tsakalof 1
🇬🇷Larisa, Greece
General Hospital of Mytilini Vostaneio
🇬🇷Mytilini, Greece
General Hospital of Xanthi
🇬🇷Xanthi, Greece
Haga Ziekenhuis
🇳🇱Den Haag, Netherlands
Erasmus MC
🇳🇱Rotterdam, Netherlands
Srinagarind Hospital
🇹🇭Khon Kaen, Thailand
Texas Children's Hospital
🇺🇸Houston, Texas, United States
Children's Hospital and Health Center of San Diego
🇺🇸San Diego, California, United States
AMC
🇳🇱Amsterdam, Netherlands
Children's Hospitals and Clinics of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Oregon Health & Science University
🇺🇸Portland, Oregon, United States
University of Washington Seattle Cancer Care Alliance
🇺🇸Seattle, Washington, United States
Osler Medical, Inc.
🇺🇸Melbourne, Florida, United States
Hematalogy Oncology Associates
🇺🇸Pensacola, Florida, United States
The Children's Medical Center of Dayton
🇺🇸Dayton, Ohio, United States
CHR de la Citadelle
🇧🇪Liege, Belgium
Medizinische Hochschule Hannover
🇩🇪Hannover, Germany
Agia Sofia Hospital of Athens
🇬🇷Karditsa, Greece
University Hospital of Ioannina
🇬🇷Ioannina, Greece
Klinikum Stuttgart Bismarckstrasse 8
🇩🇪Stuttgart, Germany
General Hospital of Korinthos
🇬🇷Korinthos, Greece
General State Hospital of Nikaia St. Panteleimon
🇬🇷Nikaia, Greece
Presidio Ospedaliero Antonio Perrino
🇮🇹Brindisi, Italy
Azienda Ospedaliera Pugliese Cicaccio
🇮🇹Catanzaro, Italy
Ospedale San Giuseppe
🇮🇹Empoli, Italy
Azienda Ospedaliera A. Meyer
🇮🇹Firenze, Italy
Fondazione Ospedale
🇮🇹Milano, Italy
Azienda Ospedaliera Universitaria Federico II
🇮🇹Napoli, Italy
Azienda Ospedaliera V. Cervello
🇮🇹Palermo, Italy
Azienda Ospedaliera Civile- Maria Paterno
🇮🇹Ragusa, Italy
Ospedale S. Eugenio
🇮🇹Roma, Italy
Azienda Ospedaliera Villa Sofia-CTO
🇮🇹Palermo, Italy
Presidio Ospedaliero di Sassari-Ospedale SS
🇮🇹Sassari, Italy
Presidio Ospedaliero Giovanni di Cristina
🇮🇹Palermo, Italy
IRCCS Policlinico San Matteo
🇮🇹Pavia, Italy
Kaohsiung Medical University Hospital
🇨🇳Kaohsiung, Taiwan
Azienda Ospedaliero Universitaria Pisana
🇮🇹Pisa, Italy
Azienda Ospedaliera Ospedali Civili Riuniti di Sciacca
🇮🇹Sciacca, Italy
Tao-yuan General Hospital
🇨🇳Tau-Yuan County, Taiwan
Hacettepe Universitesi
🇹🇷Ankara, Turkey
Ege Universitesi Tip Fakultesi
🇹🇷Izmir, Turkey
Hospital Xeral de Vigo
🇪🇸Vigo, Spain
Hospital Universitario Marques de Valdecilla
🇪🇸Santander, Spain
Hospital Universitario La Fe
🇪🇸Valencia, Spain
Catharina-ziekenhuis
🇳🇱Eindhoven, Netherlands
Althaia : Xarxa Assistencial de Manresa
🇪🇸Manresa, Spain
Hospital Puerta del Mar
🇪🇸Cadiz, Spain
Chang Gung Children's Hospital
🇨🇳Taoyuan, Taiwan
Suleyman Demirel
🇹🇷Isparta, Turkey
Mackay Memorial Hospital
🇨🇳Taipei, Taiwan
Hospital Virgen de la Salud
🇪🇸Toledo, Spain
Ramathibodi Hospital
🇹🇭Bangkok, Thailand
Phramongkutklao Hospital
🇹🇭Bangkok, Thailand
St. George's Hospital
🇬🇧Tooting, United Kingdom
North Middlesex University Hospital
🇬🇧London, United Kingdom
Songklanagarind Hospital
🇹🇭Bangkok, Thailand
Cukurova Universitesi
🇹🇷Adana, Turkey
Evelina Hospital St. Thomas' Hospital
🇬🇧London, United Kingdom
The Royal Hospital London
🇬🇧London, United Kingdom
Whittington Hospital
🇬🇧London, United Kingdom
Azienda Ospedaliera San Salvatore
🇮🇹Pesaro, Italy
Azienda Ospedaliera Bianchi-Melacrino-Morelli
🇮🇹Reggio Calabria, Italy
Ospedale nostra Signora di Bonaria
🇮🇹San Gavino Monreale- CA, Italy
Presidio Ospedaliero Centrale
🇮🇹Taranto, Italy
Ospedale Infantile Regina Margherita
🇮🇹Torino, Italy
Akdeniz Universitesi
🇹🇷Antalya, Turkey
Gaziantep Universitesi
🇹🇷Gaziantep, Turkey
Istanbul Universitesi
🇹🇷Istanbul, Turkey
Erciyes Universitesi
🇹🇷Kayseri, Turkey
University of Alberta
🇨🇦Edmonton, Canada
Hospital De Gran Canaria
🇪🇸Canara, Spain
Hospital Universitario La Paz
🇪🇸Madrid, Spain
University of Mississippi Medical Center
🇺🇸Jackson, Mississippi, United States
Alta Bates Comprehensive Cancer Center
🇺🇸Berkeley, California, United States
Backus Children's Hospital, Memorial Health University Medical Center
🇺🇸Savannah, Georgia, United States
Stanford University
🇺🇸Stanford, California, United States
Borgess Hospital
🇺🇸Kalamazoo, Michigan, United States
Tampa Children's Hospital at St. Joseph's Hospital
🇺🇸Tampa, Florida, United States
James A. Haley Veterans Hospital
🇺🇸Tampa, Florida, United States
Children's Mercy Hospital
🇺🇸Kansas City, Missouri, United States
Schneider Children's Hospital
🇺🇸New Hyde Park, New York, United States
Hershey Medical Center
🇺🇸Hershey, Pennsylvania, United States
Children's Hospital of the Kings Daughters
🇺🇸Norfolk, Virginia, United States
VCU Pediatric Hematology/Oncology
🇺🇸Richmond, Virginia, United States
The Ottawa Hospital-General Campus
🇨🇦Ottawa, Ontario, Canada
Centre Hospitalier Chretien-Clinique Saint-Joseph
🇧🇪Montegnee, Belgium
MUHC- Montreal Children's Hospital
🇨🇦Montreal, Canada
CHUM-Hopital-Notre-Dame
🇨🇦Montreal, Canada
Hopital de l'Enfant-Jesus
🇨🇦Quebec, Canada
MUHC- Royal Victoria Hospital
🇨🇦Montreal, Canada
The Hospital for Sick Children,
🇨🇦Toronto, Canada
Toronto General Hospital-Hemoglobinopathy
🇨🇦Toronto, Canada
Universitatskrankenhaus Hamburg-Eppendorf
🇩🇪Hamburg, Germany
Georg-August-Universitat Gottingen
🇩🇪Gottingen, Germany
Charite-Universitatsmedizin Berlin
🇩🇪Berlin, Germany
Johann Wolfgang von Goethe Universitat
🇩🇪Frankfurt am Main, Germany
Universitaetsklinik Dusseldorf
🇩🇪Duesseldorf, Germany
Burrard Medical Building
🇨🇦Vancouver, Canada
Universitatsklinikum Koln
🇩🇪Köln, Germany
Universitatsklinikum Ulm
🇩🇪Ulm, Germany
Ippokration Hospital of Athens
🇬🇷Athens, Greece
General Hospital of Heraklion Benizeleio-Pananeio
🇬🇷Heraklion, Greece
Presidio Ospedaliero S. Bambino
🇮🇹Catania, Italy
General Hospital of Karditsa
🇬🇷Karditsa, Greece
Presidio Ospedale Muscatello
🇮🇹Augusta, Italy
A.O. Ospedale Policlinico Consorziale di Bari
🇮🇹Bari, Italy
Ospedale Di Venere
🇮🇹Carbonara di Bari, Italy
Ospedale Regionale Microcitemie
🇮🇹Itala, Italy
Ospedale Civile dell'Annunziata
🇮🇹Cosenza, Italy
E.O. Ospedali Galliera
🇮🇹Genova, Italy
Azienda Ospedaliera Universitaria di Ferrara
🇮🇹Ferrara, Italy
AORN A. Cardarelli
🇮🇹Napoli, Italy
Ospedale Madonna delle Grazie
🇮🇹Matera, Italy
Az. Ospedaliera Universitaria Policlinico G. Martino
🇮🇹Messina, Italy
Azienda Ospedaliero-Universitaria di Modena
🇮🇹Modena, Italy
Ospedale Umberto I
🇮🇹Talassemie, Italy
Erasmus Medisch Centrum, locatie Sophia
🇳🇱Rotterdam, Netherlands
Hospital Infanta Cristina
🇪🇸Badajoz, Spain
Hospital de Torrecardenas
🇪🇸Almeria, Spain
Hospital Cruces
🇪🇸Baracaldo, Spain
Hematalogy Oncology Clinic
🇺🇸Baton Rouge, Louisiana, United States
The Cancer Institute of New Jersey
🇺🇸New Brunswick, New Jersey, United States
National Taiwan University Hospital
🇨🇳Taipei, Taiwan
Arkansas Children's Hospital, UAMS College of Medicine
🇺🇸Little Rock, Arkansas, United States
Children's Hospital of Orange County
🇺🇸Orange, California, United States
Alfred I. Dupong Hospital for Children
🇺🇸Wilmington, Delaware, United States