Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload

Sickle Cell Disease Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00235391
• Other study ID #: CICL670A2203
• 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

Related Conditions & MeSH terms

• Anemia, Diamond-Blackfan
• Anemia, Sickle Cell
• Diamond Blackfan Anemia
• Iron Overload
• Myelofibrosis
• Primary Myelofibrosis
• Sickle Cell Disease
• Thalassemia

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

External Links

•   Visit NovartisClinicalTrials.com: Pre-qualify for a trial, and view a list of trials and participating study centers.