Clinical trial NCT00235391: Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
Official title: A Study to Provide Expanded Access of (Exjade®) Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload From Blood Transfusions Who Cannot Adequately be Treated With Other Locally Approved Iron Chelators
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Thalassemia
Sickle Cell Disease
Diamond Blackfan Anemia
Myelofibrosis
Keywords: 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
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2005-10-10
Enrollment: 1683
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events
timeFrame: Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks)
Secondary outcomes:
measure: The Change in Serum Ferritin Values From Baseline Through Completion of the Study
timeFrame: Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks)
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Arkansas Children's Hospital, UAMS College of Medicine
state: Arkansas
city: Berkeley
country: United States
facility: Alta Bates Comprehensive Cancer Center
state: California
city: Orange
country: United States
facility: Children's Hospital of Orange County
state: California
city: San Diego
country: United States
facility: Children's Hospital and Health Center of San Diego
state: California
city: Stanford
country: United States
facility: Stanford University
state: California
city: Wilmington
country: United States
facility: Alfred I. Dupong Hospital for Children
state: Delaware
city: Melbourne
country: United States
facility: Osler Medical, Inc.
state: Florida
city: Pensacola
country: United States
facility: Hematalogy Oncology Associates
state: Florida
city: Tampa
country: United States
facility: Tampa Children's Hospital at St. Joseph's Hospital
state: Florida
city: Tampa
country: United States
facility: James A. Haley Veterans Hospital
state: Florida
city: Savannah
country: United States
facility: Backus Children's Hospital, Memorial Health University Medical Center
state: Georgia
city: Baton Rouge
country: United States
facility: Hematalogy Oncology Clinic
state: Louisiana
city: Kalamazoo
country: United States
facility: Borgess Hospital
state: Michigan
city: Minneapolis
country: United States
facility: Children's Hospitals and Clinics of Minnesota
state: Minnesota
city: Jackson
country: United States
facility: University of Mississippi Medical Center
state: Mississippi
city: Kansas City
country: United States
facility: Children's Mercy Hospital
state: Missouri
city: New Brunswick
country: United States
facility: The Cancer Institute of New Jersey
state: New Jersey
city: New Hyde Park
country: United States
facility: Schneider Children's Hospital
state: New York
city: Columbus
country: United States
facility: PCTI
state: Ohio
city: Dayton
country: United States
facility: The Children's Medical Center of Dayton
state: Ohio
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Hershey
country: United States
facility: Hershey Medical Center
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Children's Hospitals of Pittsburgh
state: Pennsylvania
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Norfolk
country: United States
facility: Children's Hospital of the Kings Daughters
state: Virginia
city: Richmond
country: United States
facility: VCU Pediatric Hematology/Oncology
state: Virginia
city: Seattle
country: United States
facility: University of Washington Seattle Cancer Care Alliance
state: Washington
city: Antwerpen
country: Belgium
facility: Ziekenhuisnetwerk Antwerpen-AZ Middelheim
city: Charleroi
country: Belgium
facility: Centre Hospitalier Notre Dame et Reine
city: Liege
country: Belgium
facility: CHR de la Citadelle
city: Montegnee
country: Belgium
facility: Centre Hospitalier Chretien-Clinique Saint-Joseph
city: Ottawa
country: Canada
facility: The Ottawa Hospital-General Campus
state: Ontario
city: Edmonton
country: Canada
facility: University of Alberta
city: Montreal
country: Canada
facility: CHUM-Hopital-Notre-Dame
city: Montreal
country: Canada
facility: MUHC- Montreal Children's Hospital
city: Montreal
country: Canada
facility: MUHC- Royal Victoria Hospital
city: Quebec
country: Canada
facility: Hopital de l'Enfant-Jesus
city: Toronto
country: Canada
facility: The Hospital for Sick Children,
city: Toronto
country: Canada
facility: Toronto General Hospital-Hemoglobinopathy
city: Vancouver
country: Canada
facility: Burrard Medical Building
city: Berlin
country: Germany
facility: Charite-Universitatsmedizin Berlin
city: Duesseldorf
country: Germany
facility: Universitaetsklinik Dusseldorf
city: Frankfurt am Main
country: Germany
facility: Johann Wolfgang von Goethe Universitat
city: Gottingen
country: Germany
facility: Georg-August-Universitat Gottingen
city: Hamburg
country: Germany
facility: Universitatskrankenhaus Hamburg-Eppendorf
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover
city: Köln
country: Germany
facility: Universitatsklinikum Koln
city: Stuttgart
country: Germany
facility: Klinikum Stuttgart Bismarckstrasse 8
city: Ulm
country: Germany
facility: Universitatsklinikum Ulm
city: Athens
country: Greece
facility: Agia Sofia Hospital of Athens
city: Athens
country: Greece
facility: General Hospital of Athens
city: Athens
country: Greece
facility: Ippokration Hospital of Athens
city: Heraklion
country: Greece
facility: General Hospital of Heraklion Benizeleio-Pananeio
city: Ioannina
country: Greece
facility: University Hospital of Ioannina
city: Kalamata
country: Greece
facility: General Hospital of Kalamata
city: Karditsa
country: Greece
facility: Agia Sofia Hospital of Athens
city: Karditsa
country: Greece
facility: General Hospital of Karditsa
city: Kerkyra
country: Greece
facility: General Hospital of Athens
city: Korinthos
country: Greece
facility: General Hospital of Korinthos
city: Larisa
country: Greece
facility: General Hospital of Larisa Tsakalof 1
city: Mytilini
country: Greece
facility: General Hospital of Mytilini Vostaneio
city: Nikaia
country: Greece
facility: General State Hospital of Nikaia St. Panteleimon
city: Patra
country: Greece
facility: University Hospital of Patras
city: Thessaloniki
country: Greece
facility: General Hospital of Thessaloniki Agios Pavlos
city: Thessaloniki
country: Greece
facility: General Hospital Thessalonikis Hippokratio
city: Volos
country: Greece
facility: General Hospital of Volos
city: Xanthi
country: Greece
facility: General Hospital of Xanthi
city: Augusta
country: Italy
facility: Presidio Ospedale Muscatello
city: Bari
country: Italy
facility: A.O. Ospedale Policlinico Consorziale di Bari
city: Brindisi
country: Italy
facility: Presidio Ospedaliero Antonio Perrino
city: Cagliari
country: Italy
facility: Ospedale Regionale Microcitemie
city: Carbonara di Bari
country: Italy
facility: Ospedale Di Venere
city: Catania
country: Italy
facility: Azienda Ospedali Vittorio Emanuele, Ferrarotto e San Bambino
city: Catania
country: Italy
facility: Presidio Ospedaliero S. Bambino
city: Catania
country: Italy
facility: PresidioOspedaliero S. Luigi Curro
city: Catanzaro
country: Italy
facility: Azienda Ospedaliera Pugliese Cicaccio
city: Cosenza
country: Italy
facility: Ospedale Civile dell'Annunziata
city: Empoli
country: Italy
facility: Ospedale San Giuseppe
city: Ferrara
country: Italy
facility: Azienda Ospedaliera Universitaria di Ferrara
city: Firenze
country: Italy
facility: Azienda Ospedaliera A. Meyer
city: Genova
country: Italy
facility: E.O. Ospedali Galliera
city: Itala
country: Italy
facility: Ospedale Regionale Microcitemie
city: Matera
country: Italy
facility: Ospedale Madonna delle Grazie
city: Messina
country: Italy
facility: Az. Ospedaliera Universitaria Policlinico G. Martino
city: Milano
country: Italy
facility: Fondazione Ospedale
city: Modena
country: Italy
facility: Azienda Ospedaliero-Universitaria di Modena
city: Napoli
country: Italy
facility: AORN A. Cardarelli
city: Napoli
country: Italy
facility: Azienda Ospedaliera Universitaria Federico II
city: Palermo
country: Italy
facility: Azienda Ospedaliera V. Cervello
city: Palermo
country: Italy
facility: Azienda Ospedaliera Villa Sofia-CTO
city: Palermo
country: Italy
facility: Presidio Ospedaliero Giovanni di Cristina
city: Pavia
country: Italy
facility: IRCCS Policlinico San Matteo
city: Pesaro
country: Italy
facility: Azienda Ospedaliera San Salvatore
city: Pisa
country: Italy
facility: Azienda Ospedaliero Universitaria Pisana
city: Ragusa
country: Italy
facility: Azienda Ospedaliera Civile- Maria Paterno
city: Reggio Calabria
country: Italy
facility: Azienda Ospedaliera Bianchi-Melacrino-Morelli
city: Roma
country: Italy
facility: Ospedale S. Eugenio
city: San Gavino Monreale- CA
country: Italy
facility: Ospedale nostra Signora di Bonaria
city: Sassari
country: Italy
facility: Presidio Ospedaliero di Sassari-Ospedale SS
city: Sciacca
country: Italy
facility: Azienda Ospedaliera Ospedali Civili Riuniti di Sciacca
city: Talassemie
country: Italy
facility: Ospedale Umberto I
city: Taranto
country: Italy
facility: Presidio Ospedaliero Centrale
city: Torino
country: Italy
facility: Ospedale Infantile Regina Margherita
city: Amsterdam
country: Netherlands
facility: AMC
city: Den Haag
country: Netherlands
facility: Haga Ziekenhuis
city: Eindhoven
country: Netherlands
facility: Catharina-ziekenhuis
city: Rotterdam
country: Netherlands
facility: Erasmus MC
city: Rotterdam
country: Netherlands
facility: Erasmus Medisch Centrum, locatie Sophia
city: Almeria
country: Spain
facility: Hospital de Torrecardenas
city: Badajoz
country: Spain
facility: Hospital Infanta Cristina
city: Baracaldo
country: Spain
facility: Hospital Cruces
city: Cadiz
country: Spain
facility: Hospital Puerta del Mar
city: Canara
country: Spain
facility: Hospital De Gran Canaria
city: Madrid
country: Spain
facility: Hospital Universitario La Paz
city: Manresa
country: Spain
facility: Althaia : Xarxa Assistencial de Manresa
city: Santander
country: Spain
facility: Hospital Universitario Marques de Valdecilla
city: Toledo
country: Spain
facility: Hospital Virgen de la Salud
city: Valencia
country: Spain
facility: Hospital Universitario La Fe
city: Vigo
country: Spain
facility: Hospital Xeral de Vigo
city: Kaohsiung
country: Taiwan
facility: Kaohsiung Medical University Hospital
city: Taipei
country: Taiwan
facility: Mackay Memorial Hospital
city: Taipei
country: Taiwan
facility: National Taiwan University Hospital
city: Taoyuan
country: Taiwan
facility: Chang Gung Children's Hospital
city: Tau-Yuan County
country: Taiwan
facility: Tao-yuan General Hospital
city: Bangkok
country: Thailand
facility: Phramongkutklao Hospital
city: Bangkok
country: Thailand
facility: Ramathibodi Hospital
city: Bangkok
country: Thailand
facility: Songklanagarind Hospital
city: Khon Kaen
country: Thailand
facility: Srinagarind Hospital
city: Adana
country: Turkey
facility: Cukurova Universitesi
city: Ankara
country: Turkey
facility: Hacettepe Universitesi
city: Antalya
country: Turkey
facility: Akdeniz Universitesi
city: Gaziantep
country: Turkey
facility: Gaziantep Universitesi
city: Isparta
country: Turkey
facility: Suleyman Demirel
city: Istanbul
country: Turkey
facility: Istanbul Universitesi
city: Izmir
country: Turkey
facility: Ege Universitesi Tip Fakultesi
city: Kayseri
country: Turkey
facility: Erciyes Universitesi
city: London
country: United Kingdom
facility: Evelina Hospital St. Thomas' Hospital
city: London
country: United Kingdom
facility: North Middlesex University Hospital
city: London
country: United Kingdom
facility: The Royal Hospital London
city: London
country: United Kingdom
facility: Whittington Hospital
city: Tooting
country: United Kingdom
facility: St. George's Hospital
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: