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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT00235391
Other study ID # CICL670A2203
Secondary ID
Status Completed
Phase Phase 3
First received October 6, 2005
Last updated June 3, 2011
Start date October 2005
Est. completion date October 2008

Study information

Verified date June 2011
Source Novartis
Contact n/a
Is FDA regulated No
Health authority United States: Food and Drug Administration
Study type Interventional

Clinical Trial 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.


Recruitment information / eligibility

Status Completed
Enrollment 1683
Est. completion date October 2008
Est. primary completion date October 2008
Accepts healthy volunteers No
Gender Both
Age group 2 Years and older
Eligibility Inclusion Criteria:

- Male or female patients greater than or equal to 2 years of age

- Documented congenital disorder of red blood cells (e.g., ß-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 ß-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

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment


Intervention

Drug:
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.

Locations

Country Name City State
Belgium Ziekenhuisnetwerk Antwerpen-AZ Middelheim Antwerpen
Belgium Centre Hospitalier Notre Dame et Reine Charleroi
Belgium CHR de la Citadelle Liege
Belgium Centre Hospitalier Chretien-Clinique Saint-Joseph Montegnee
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 The Ottawa Hospital-General Campus Ottawa Ontario
Canada Hopital de l'Enfant-Jesus Quebec
Canada The Hospital for Sick Children, Toronto
Canada Toronto General Hospital-Hemoglobinopathy Toronto
Canada Burrard Medical Building Vancouver
Germany Charite-Universitatsmedizin Berlin Berlin
Germany Universitaetsklinik Dusseldorf Duesseldorf
Germany Johann Wolfgang von Goethe Universitat Frankfurt am Main
Germany Georg-August-Universitat Gottingen Gottingen
Germany Universitatskrankenhaus Hamburg-Eppendorf Hamburg
Germany Medizinische Hochschule Hannover Hannover
Germany Universitatsklinikum Koln Köln
Germany Klinikum Stuttgart Bismarckstrasse 8 Stuttgart
Germany Universitatsklinikum Ulm Ulm
Greece Agia Sofia Hospital of Athens Athens
Greece General Hospital of Athens Athens
Greece Ippokration Hospital of Athens Athens
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 Athens Kerkyra
Greece General Hospital of Korinthos Korinthos
Greece General Hospital of Larisa Tsakalof 1 Larisa
Greece General Hospital of Mytilini Vostaneio Mytilini
Greece General State Hospital of Nikaia St. Panteleimon Nikaia
Greece University Hospital of Patras Patra
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 Xanthi
Italy 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 Firenze
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 Milano
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
Netherlands AMC Amsterdam
Netherlands Haga Ziekenhuis Den Haag
Netherlands Catharina-ziekenhuis Eindhoven
Netherlands Erasmus MC Rotterdam
Netherlands Erasmus Medisch Centrum, locatie Sophia Rotterdam
Spain Hospital de Torrecardenas Almeria
Spain Hospital Infanta Cristina Badajoz
Spain Hospital Cruces Baracaldo
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
Taiwan Kaohsiung Medical University Hospital Kaohsiung
Taiwan Mackay Memorial Hospital Taipei
Taiwan National Taiwan University Hospital Taipei
Taiwan Chang Gung Children's Hospital Taoyuan
Taiwan Tao-yuan General Hospital Tau-Yuan County
Thailand Phramongkutklao Hospital Bangkok
Thailand Ramathibodi Hospital Bangkok
Thailand Songklanagarind Hospital Bangkok
Thailand Srinagarind Hospital Khon Kaen
Turkey Cukurova Universitesi Adana
Turkey Hacettepe Universitesi Ankara
Turkey Akdeniz Universitesi Antalya
Turkey Gaziantep Universitesi Gaziantep
Turkey Suleyman Demirel Isparta
Turkey Istanbul Universitesi Istanbul
Turkey Ege Universitesi Tip Fakultesi Izmir
Turkey Erciyes Universitesi Kayseri
United Kingdom 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 States Hematalogy Oncology Clinic Baton Rouge Louisiana
United States Alta Bates Comprehensive Cancer Center Berkeley California
United States PCTI Columbus Ohio
United States The Children's Medical Center of Dayton Dayton Ohio
United States Hershey Medical Center Hershey Pennsylvania
United States Texas Children's Hospital Houston Texas
United States University of Mississippi Medical Center Jackson Mississippi
United States Borgess Hospital Kalamazoo Michigan
United States Children's Mercy Hospital Kansas City Missouri
United States Arkansas Children's Hospital, UAMS College of Medicine Little Rock Arkansas
United States Osler Medical, Inc. Melbourne Florida
United States Children's Hospitals and Clinics of Minnesota Minneapolis Minnesota
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 Children's Hospital of the Kings Daughters Norfolk Virginia
United States Children's Hospital of Orange County Orange California
United States Hematalogy Oncology Associates Pensacola Florida
United States Children's Hospitals of Pittsburgh Pittsburgh Pennsylvania
United States Oregon Health & Science University Portland Oregon
United States VCU Pediatric Hematology/Oncology Richmond Virginia
United States Children's Hospital and Health Center of San Diego San Diego California
United States Backus Children's Hospital, Memorial Health University Medical Center Savannah Georgia
United States University of Washington Seattle Cancer Care Alliance Seattle Washington
United States Stanford University Stanford California
United States James A. Haley Veterans Hospital Tampa Florida
United States Tampa Children's Hospital at St. Joseph's Hospital Tampa Florida
United States Alfred I. Dupong Hospital for Children Wilmington Delaware

Sponsors (1)

Lead Sponsor Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

United States,  Belgium,  Canada,  Germany,  Greece,  Italy,  Netherlands,  Spain,  Taiwan,  Thailand,  Turkey,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events 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. Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks) No
Secondary The Change in Serum Ferritin Values From Baseline Through Completion of the Study 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. Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks) No
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