ThalassemiaPhase 3NCT00235391
Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
A Phase 3 study testing Deferasirox in people with Thalassemia and Sickle Cell Disease, run by Novartis Pharmaceuticals at 141 sites in 12 countries.
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. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia
- What is being tested?
- Deferasirox
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1,683 participants
- Where
- 141 sitesin 12 countries
- Study dates
- Oct 2005to Oct 2008
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 9Show fewer
- 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.
Trial locations and contacts
141 sites in 12 countries.
141 sites in 12 countries
Italy36 sites
Presidio Ospedale Muscatello
Augusta
A.O. Ospedale Policlinico Consorziale di Bari
Bari
Presidio Ospedaliero Antonio Perrino
Brindisi
Ospedale Regionale Microcitemie
Cagliari
Ospedale Di Venere
Carbonara di Bari
Azienda Ospedali Vittorio Emanuele, Ferrarotto e San Bambino
Catania
Presidio Ospedaliero S. Bambino
Catania
PresidioOspedaliero S. Luigi Curro
Catania
Azienda Ospedaliera Pugliese Cicaccio
Catanzaro
Ospedale Civile dell'Annunziata
Cosenza
Ospedale San Giuseppe
Empoli
Azienda Ospedaliera Universitaria di Ferrara
Ferrara
Azienda Ospedaliera A. Meyer
Florence
E.O. Ospedali Galliera
Genova
Ospedale Regionale Microcitemie
Itala
Ospedale Madonna delle Grazie
Matera
Az. Ospedaliera Universitaria Policlinico G. Martino
Messina
Fondazione Ospedale
Milan
Azienda Ospedaliero-Universitaria di Modena
Modena
AORN A. Cardarelli
Naples
Azienda Ospedaliera Universitaria Federico II
Naples
Azienda Ospedaliera V. Cervello
Palermo
Azienda Ospedaliera Villa Sofia-CTO
Palermo
Presidio Ospedaliero Giovanni di Cristina
Palermo
IRCCS Policlinico San Matteo
Pavia
Azienda Ospedaliera San Salvatore
Pesaro
Azienda Ospedaliero Universitaria Pisana
Pisa
Azienda Ospedaliera Civile- Maria Paterno
Ragusa
Azienda Ospedaliera Bianchi-Melacrino-Morelli
Reggio Calabria
Ospedale S. Eugenio
Roma
Ospedale nostra Signora di Bonaria
San Gavino Monreale- CA
Presidio Ospedaliero di Sassari-Ospedale SS
Sassari
Azienda Ospedaliera Ospedali Civili Riuniti di Sciacca
Sciacca
Ospedale Umberto I
Talassemie
Presidio Ospedaliero Centrale
Taranto
Ospedale Infantile Regina Margherita
Torino
United States27 sites
Hematalogy Oncology Clinic
Baton Rouge, Louisiana, 70808
Alta Bates Comprehensive Cancer Center
Berkeley, California, 94704
PCTI
Columbus, Ohio, 43205
The Children's Medical Center of Dayton
Dayton, Ohio, 45404
Hershey Medical Center
Hershey, Pennsylvania, 17033-0850
Texas Children's Hospital
Houston, Texas, 77030
University of Mississippi Medical Center
Jackson, Mississippi, 39762
Borgess Hospital
Kalamazoo, Michigan, 49048
Children's Mercy Hospital
Kansas City, Missouri, 64108
Arkansas Children's Hospital, UAMS College of Medicine
Little Rock, Arkansas, 72202
Osler Medical, Inc.
Melbourne, Florida, 32901
Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, 55405
The Cancer Institute of New Jersey
New Brunswick, New Jersey, 08903
Schneider Children's Hospital
New Hyde Park, New York, 11040
Children's Hospital of the Kings Daughters
Norfolk, Virginia, 23507
Children's Hospital of Orange County
Orange, California, 92868
Hematalogy Oncology Associates
Pensacola, Florida, 32501
Children's Hospitals of Pittsburgh
Pittsburgh, Pennsylvania, 15213
Oregon Health & Science University
Portland, Oregon, 97239
VCU Pediatric Hematology/Oncology
Richmond, Virginia, 23219
Children's Hospital and Health Center of San Diego
San Diego, California, 92123
Backus Children's Hospital, Memorial Health University Medical Center
Savannah, Georgia, 31403
University of Washington Seattle Cancer Care Alliance
Seattle, Washington, 98195
Stanford University
Stanford, California, 94305
Tampa Children's Hospital at St. Joseph's Hospital
Tampa, Florida, 33607
James A. Haley Veterans Hospital
Tampa, Florida, 33612
Alfred I. Dupong Hospital for Children
Wilmington, Delaware, 19803
Greece18 sites
Agia Sofia Hospital of Athens
Athens
General Hospital of Athens
Athens
Ippokration Hospital of Athens
Athens
General Hospital of Athens
Corfu
General Hospital of Korinthos
Corinth
General Hospital of Heraklion Benizeleio-Pananeio
Heraklion
University Hospital of Ioannina
Ioannina
General Hospital of Kalamata
Kalamata
Agia Sofia Hospital of Athens
Karditsa
General Hospital of Karditsa
Karditsa
General Hospital of Larisa Tsakalof 1
Larissa
General Hospital of Mytilini Vostaneio
Mytilene
General State Hospital of Nikaia St. Panteleimon
Nikaia
University Hospital of Patras
Pátrai
General Hospital of Thessaloniki Agios Pavlos
Thessaloniki
General Hospital Thessalonikis Hippokratio
Thessaloniki
General Hospital of Volos
Volos
General Hospital of Xanthi
Xánthi
Spain11 sites
Hospital de Torrecardenas
Almería
Hospital Infanta Cristina
Badajoz
Hospital Cruces
Barakaldo
Hospital Puerta del Mar
Cadiz
Hospital De Gran Canaria
Canara
Hospital Universitario La Paz
Madrid
Althaia : Xarxa Assistencial de Manresa
Manresa
Hospital Universitario Marques de Valdecilla
Santander
Hospital Virgen de la Salud
Toledo
Hospital Universitario La Fe
Valencia
Hospital Xeral de Vigo
Vigo
Canada9 sites
University of Alberta
Edmonton
CHUM-Hopital-Notre-Dame
Montreal
MUHC- Montreal Children's Hospital
Montreal
MUHC- Royal Victoria Hospital
Montreal
The Ottawa Hospital-General Campus
Ottawa, Ontario
Hopital de l'Enfant-Jesus
Québec
The Hospital for Sick Children,
Toronto
Toronto General Hospital-Hemoglobinopathy
Toronto
Burrard Medical Building
Vancouver
Germany9 sites
Charite-Universitatsmedizin Berlin
Berlin
Universitatsklinikum Koln
Cologne
Universitaetsklinik Dusseldorf
Düsseldorf
Johann Wolfgang von Goethe Universitat
Frankfurt am Main
Georg-August-Universitat Gottingen
Göttingen
Universitatskrankenhaus Hamburg-Eppendorf
Hamburg
Medizinische Hochschule Hannover
Hanover
Klinikum Stuttgart Bismarckstrasse 8
Stuttgart
Universitatsklinikum Ulm
Ulm
Turkey (Türkiye)8 sites
Cukurova Universitesi
Adana
Hacettepe Universitesi
Ankara
Akdeniz Universitesi
Antalya
Gaziantep Universitesi
Gaziantep
Suleyman Demirel
Isparta
Istanbul Universitesi
Istanbul
Ege Universitesi Tip Fakultesi
Izmir
Erciyes Universitesi
Kayseri
Netherlands5 sites
AMC
Amsterdam
Catharina-ziekenhuis
Eindhoven
Erasmus MC
Rotterdam
Erasmus Medisch Centrum, locatie Sophia
Rotterdam
Haga Ziekenhuis
The Hague
Taiwan5 sites
Kaohsiung Medical University Hospital
Kaohsiung City
Mackay Memorial Hospital
Taipei
National Taiwan University Hospital
Taipei
Chang Gung Children's Hospital
Taoyuan
Tao-yuan General Hospital
Tau-Yuan County
United Kingdom5 sites
Evelina Hospital St. Thomas' Hospital
London
North Middlesex University Hospital
London
The Royal Hospital London
London
Whittington Hospital
London
St. George's Hospital
Tooting
Belgium4 sites
Ziekenhuisnetwerk Antwerpen-AZ Middelheim
Antwerp
Centre Hospitalier Notre Dame et Reine
Charleroi
CHR de la Citadelle
Liège
Centre Hospitalier Chretien-Clinique Saint-Joseph
Montegnée
Thailand4 sites
Phramongkutklao Hospital
Bangkok
Ramathibodi Hospital
Bangkok
Songklanagarind Hospital
Bangkok
Srinagarind Hospital
Khon Kaen
About this study
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
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.
- Being tested
- Deferasirox
- 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
- Medical subject headings
- Thalassemia, Anemia, Sickle Cell, Anemia, Diamond-Blackfan, Primary Myelofibrosis, Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DeferasiroxDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Deferasirox | Experimental | 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. Receives: Drug: Deferasirox |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks)
Secondary outcomes (1)
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.
Measured: Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Oct 2005 (estimated)
- Study startOct 2005
- Primary completionOct 2008
- Study completionOct 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for ThalassemiaStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1,683 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00235391
- Sponsor's protocol ID
- CICL670A2203
- Status last verified
- Jun 2011
- First submitted
- 6 Oct 2005
- Last updated
- 7 Jun 2011
- Results posted
- Yes, on ClinicalTrials.gov
Study at a glance
From the registry record, last updated by the sponsor on 7 Jun 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00235391
- Acronym / sponsor ID
- CICL670A2203
- Keywords
- Deferasirox, Congenital Anemias, Anemias, Red Blood Cell Disorders, Chronic Iron Overload, Transfusional Iron Overload, Iron Chelators, Oral Iron Chelators
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 1,683 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Oct 2005
- Primary completion
- Oct 2008 (actual)
- Study completion
- Oct 2008 (actual)
- First posted
- 10 Oct 2005 (estimated)
- Results
- Posted 2 May 2011
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Deferasirox | Experimental | Drug: DeferasiroxDeferasirox 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. |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- 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:
- 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
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Deferasirox125 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. | Drug | Deferasirox |
Primary outcome
Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events
Time frame: Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks)
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.
1 secondary outcome
The Change in Serum Ferritin Values From Baseline Through Completion of the Study
Time frame: Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jun 2011.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Visit NovartisClinicalTrials.com: Pre-qualify for a trial, and view a list of trials and participating study centers.Link provided by the sponsor.
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Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Transfusion-dependent Beta-Thalassemia, Gene Therapy
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Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
Beta-Thalassemia
RecruitingPhase 1/2
β-Thalassemia Treatment With KL003 Cell Injection
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A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
141 sites in 12 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Deferasirox in people with Thalassemia and Sickle Cell Disease, run by Novartis Pharmaceuticals at 141 sites in 12 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Jun 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 141 sites in Italy, United States, Greece, Spain, Canada and 7 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 2 May 2011). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events (time frame: Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Jun 2011.