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
Verified date | June 2011 |
Source | Novartis |
Contact | n/a |
Is FDA regulated | No |
Health authority | United States: Food and Drug Administration |
Study type | Interventional |
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.
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. |
Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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 |
Lead Sponsor | Collaborator |
---|---|
Novartis Pharmaceuticals |
United States, Belgium, Canada, Germany, Greece, Italy, Netherlands, Spain, Taiwan, Thailand, Turkey, United Kingdom,
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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