Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia
About this trial
This is an interventional treatment trial for Thalassemia focused on measuring Deferasirox, Congenital Anemias, Anemias, Red Blood Cell Disorders, Chronic Iron Overload, Transfusional Iron Overload, Iron Chelators, Oral Iron Chelators, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis, ICL670A
Eligibility Criteria
Inclusion Criteria: 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 Exclusion Criteria: 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.
Sites / Locations
- Arkansas Children's Hospital, UAMS College of Medicine
- Alta Bates Comprehensive Cancer Center
- Children's Hospital of Orange County
- Children's Hospital and Health Center of San Diego
- Stanford University
- Alfred I. Dupong Hospital for Children
- Osler Medical, Inc.
- Hematalogy Oncology Associates
- Tampa Children's Hospital at St. Joseph's Hospital
- James A. Haley Veterans Hospital
- Backus Children's Hospital, Memorial Health University Medical Center
- Hematalogy Oncology Clinic
- Borgess Hospital
- Children's Hospitals and Clinics of Minnesota
- University of Mississippi Medical Center
- Children's Mercy Hospital
- The Cancer Institute of New Jersey
- Schneider Children's Hospital
- PCTI
- The Children's Medical Center of Dayton
- Oregon Health & Science University
- Hershey Medical Center
- Children's Hospitals of Pittsburgh
- Texas Children's Hospital
- Children's Hospital of the Kings Daughters
- VCU Pediatric Hematology/Oncology
- University of Washington Seattle Cancer Care Alliance
- Ziekenhuisnetwerk Antwerpen-AZ Middelheim
- Centre Hospitalier Notre Dame et Reine
- CHR de la Citadelle
- Centre Hospitalier Chretien-Clinique Saint-Joseph
- The Ottawa Hospital-General Campus
- University of Alberta
- CHUM-Hopital-Notre-Dame
- MUHC- Montreal Children's Hospital
- MUHC- Royal Victoria Hospital
- Hopital de l'Enfant-Jesus
- The Hospital for Sick Children,
- Toronto General Hospital-Hemoglobinopathy
- Burrard Medical Building
- Charite-Universitatsmedizin Berlin
- Universitaetsklinik Dusseldorf
- Johann Wolfgang von Goethe Universitat
- Georg-August-Universitat Gottingen
- Universitatskrankenhaus Hamburg-Eppendorf
- Medizinische Hochschule Hannover
- Universitatsklinikum Koln
- Klinikum Stuttgart Bismarckstrasse 8
- Universitatsklinikum Ulm
- Agia Sofia Hospital of Athens
- General Hospital of Athens
- Ippokration Hospital of Athens
- General Hospital of Heraklion Benizeleio-Pananeio
- University Hospital of Ioannina
- General Hospital of Kalamata
- Agia Sofia Hospital of Athens
- General Hospital of Karditsa
- General Hospital of Athens
- General Hospital of Korinthos
- General Hospital of Larisa Tsakalof 1
- General Hospital of Mytilini Vostaneio
- General State Hospital of Nikaia St. Panteleimon
- University Hospital of Patras
- General Hospital of Thessaloniki Agios Pavlos
- General Hospital Thessalonikis Hippokratio
- General Hospital of Volos
- General Hospital of Xanthi
- Presidio Ospedale Muscatello
- A.O. Ospedale Policlinico Consorziale di Bari
- Presidio Ospedaliero Antonio Perrino
- Ospedale Regionale Microcitemie
- Ospedale Di Venere
- Azienda Ospedali Vittorio Emanuele, Ferrarotto e San Bambino
- Presidio Ospedaliero S. Bambino
- PresidioOspedaliero S. Luigi Curro
- Azienda Ospedaliera Pugliese Cicaccio
- Ospedale Civile dell'Annunziata
- Ospedale San Giuseppe
- Azienda Ospedaliera Universitaria di Ferrara
- Azienda Ospedaliera A. Meyer
- E.O. Ospedali Galliera
- Ospedale Regionale Microcitemie
- Ospedale Madonna delle Grazie
- Az. Ospedaliera Universitaria Policlinico G. Martino
- Fondazione Ospedale
- Azienda Ospedaliero-Universitaria di Modena
- AORN A. Cardarelli
- Azienda Ospedaliera Universitaria Federico II
- Azienda Ospedaliera V. Cervello
- Azienda Ospedaliera Villa Sofia-CTO
- Presidio Ospedaliero Giovanni di Cristina
- IRCCS Policlinico San Matteo
- Azienda Ospedaliera San Salvatore
- Azienda Ospedaliero Universitaria Pisana
- Azienda Ospedaliera Civile- Maria Paterno
- Azienda Ospedaliera Bianchi-Melacrino-Morelli
- Ospedale S. Eugenio
- Ospedale nostra Signora di Bonaria
- Presidio Ospedaliero di Sassari-Ospedale SS
- Azienda Ospedaliera Ospedali Civili Riuniti di Sciacca
- Ospedale Umberto I
- Presidio Ospedaliero Centrale
- Ospedale Infantile Regina Margherita
- AMC
- Haga Ziekenhuis
- Catharina-ziekenhuis
- Erasmus MC
- Erasmus Medisch Centrum, locatie Sophia
- Hospital de Torrecardenas
- Hospital Infanta Cristina
- Hospital Cruces
- Hospital Puerta del Mar
- Hospital De Gran Canaria
- Hospital Universitario La Paz
- Althaia : Xarxa Assistencial de Manresa
- Hospital Universitario Marques de Valdecilla
- Hospital Virgen de la Salud
- Hospital Universitario La Fe
- Hospital Xeral de Vigo
- Kaohsiung Medical University Hospital
- Mackay Memorial Hospital
- National Taiwan University Hospital
- Chang Gung Children's Hospital
- Tao-yuan General Hospital
- Phramongkutklao Hospital
- Ramathibodi Hospital
- Songklanagarind Hospital
- Srinagarind Hospital
- Cukurova Universitesi
- Hacettepe Universitesi
- Akdeniz Universitesi
- Gaziantep Universitesi
- Suleyman Demirel
- Istanbul Universitesi
- Ege Universitesi Tip Fakultesi
- Erciyes Universitesi
- Evelina Hospital St. Thomas' Hospital
- North Middlesex University Hospital
- The Royal Hospital London
- Whittington Hospital
- St. George's Hospital
Arms of the Study
Arm 1
Experimental
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.