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Transfusion-dependent Iron OverloadPhase 3NCT00171821

A Study Assessing the Efficacy and Safety of Deferasirox in Patients With Transfusion-dependent Iron Overload

A Phase 3 study testing Deferasirox in people with Transfusion-dependent Iron Overload, run by Novartis Pharmaceuticals at 97 sites in 23 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This study uses a single arm, multi-center, open-label trial design. The study will assess the efficacy and safety of 52 weeks of treatment with deferasirox (ICL670) in patients with evidence of transfusion induced iron overload. Read more about this study

Who is it for?
Ages 2 years and older
Conditions the study is for
Transfusion-dependent Iron Overload
What is being tested?
Deferasirox
Could I receive a placebo?
No, everyone receives an active treatment
How many people
1,784 participants
Where
97 sitesin 23 countries
Study dates
Apr 2005to Jul 2010
Phase
Phase 3
Are there results?
None posted in the registry

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

  • Patients presenting with transfusion-dependent anemias (independent of underlying condition) with transfusional iron overload as shown by a serum ferritin level of ≥ 1000 ng/ml
  • Patients of either gender and aged ≥ 2 years
  • Female patients who have reached menarche and who are sexually active must use an effective method of contraception, or must have undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation.
  • Additional Inclusion Criteria for Adult Patients:
  • Written informed consent by the patient
  • Additional Inclusion Criteria for Pediatric Patients:
  • The definition of the term "pediatric" will be in accordance with local legislation. Parents or legal guardians will be fully informed by the investigator as to the requirements of the study. The pediatric patients themselves will be informed according to their capabilities in a language and terms that they are able to understand. Written informed consent will be obtained from their parents or legal guardians on the patient's behalf in accordance with the national legislation. If capable, all patients should also personally sign their written informed assent.

Who cannot join

  • Non-transfusional hemosiderosis
  • Patients with clinical evidence supporting the need for intensive chelation, based on the investigator's judgment
  • Patients with mean levels of alanine aminotransferase (ALT) > 300 U/l
  • Patients with uncontrolled systemic hypertension
  • Patients with serum creatinine above the upper limit of normal (ULN)
  • Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 (mg/mg) in second-voiding urine samples taken at both visits 1 and 2. A third sample is to be taken from patients in whom one ratio is > 0.5 (mg/mg) and one is ≤ 0.5 (mg/mg) and patients in whom the urinary protein/creatinine ratio is > 0.5 (mg/mg) in two of the three determinations are also to be excluded.
Show all 25Show fewer
  • History of nephrotic syndrome
  • Patients with 3rd atrioventricular (A-V) block, clinically relevant Q-T interval prolongation as well as patients requiring treatment with digoxin and similar compounds or drugs which may induce prolongation of the Q-T interval
  • Patients with a previous history of clinically relevant ocular toxicity related to iron chelation
  • Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent the patient from undergoing study treatment
  • Patients with psychiatric or addictive disorders which prevent them from giving their informed consent or undergoing study treatment
  • Pregnant or breast feeding patients
  • Patients treated with systemic investigational drugs within the past 4 weeks or topical investigational drugs within the past 7 days
  • Any other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of any drug. The investigator should be guided by evidence of any of the following:
  • history of inflammatory bowel syndrome, gastritis, ulcers, gastrointestinal or rectal bleeding;
  • history of major gastrointestinal tract surgery such as gastrectomy, gastroenterostomy, or bowel resection;
  • history of pancreatic injury or pancreatitis; indications of impaired pancreatic function/injury as indicated by abnormal lipase or amylase;
  • history or presence of impaired renal function as indicated by creatinine or blood urea nitrogen (BUN) values equal or above ULN;
  • history of urinary obstruction or difficulty in voiding.
  • History of non-compliance to medical regimens and patients who are considered potentially unreliable and/or not cooperative
  • History of drug or alcohol abuse within the 12 months prior to dosing or evidence of such abuse as indicated by the laboratory assays conducted during the run-in period
  • Patients with positive test to HIV
  • Life expectancy of < 1 year
  • Patient body weight which prevents the use of the smallest tablet strength (i.e. 125 mg) for proper dosing
  • Other protocol-defined inclusion/exclusion criteria may apply.

Trial locations and contacts

97 sites in 23 countries.

97 sites in 23 countries

Italy14 sites

  • Novartis Investigative Site

    Bologna

  • Novartis Investigative Site

    Brindisi

  • Novartis Investigative Site

    Cagliari

  • Novartis Investigative Site

    Cona

  • Novartis Investigative Site

    Genova

  • Novartis Investigative Site

    Milan

  • Novartis Investigative Site

    Naples

  • Novartis Investigative Site

    Orbassano

  • Novartis Investigative Site

    Palermo

  • Novartis Investigative Site

    Pavia

  • Novartis Investigative Site

    Pisa

  • Novartis Investigative Site

    Reggio Calabria

  • Novartis Investigative Site

    Roma

  • Novartis Investigative Site

    Sassari

Other trials in Italy

Germany13 sites

  • Novartis Investigative Site

    Augsburg

  • Novartis Investigative Site

    Braunschweig

  • Novartis Investigative Site

    Dresden

  • Novartis Investigative Site

    Düsseldorf

  • Novartis Investigative Site

    Frankfurt

  • Novartis Investigative Site

    Frankfurt am Main

  • Novartis Investigative Site

    Frieburg

  • Novartis Investigative Site

    Göttingen

  • Novartis Investigative Site

    Greifswald

  • Novartis Investigative Site

    Hanover

  • Novartis Investigative Site

    Mainz

  • Novartis Investigative Site

    München

  • Novartis Investigative Site

    Ulm

Other trials in Germany

France12 sites

  • Novartis Investigative Site

    Angers

  • Novartis Investigative Site

    Avignon

  • Novartis Investigative Site

    Bobigny

  • Novartis Investigative Site

    Créteil

  • Novartis Investigative Site

    Lille

  • Novartis Investigative Site

    Lyon

  • Novartis Investigative Site

    Nice

  • Novartis Investigative Site

    Paris

  • Novartis Investigative Site

    Pessac

  • Novartis Investigative Site

    Rennes

  • Novartis Investigative Site

    Toulouse

  • Novartis Investigative Site

    Vandœuvre-lès-Nancy

Other trials in France

Australia7 sites

  • Novartis Investigative Site

    Adelaide

  • Novartis Investigative Site

    Camperdown

  • Novartis Investigative Site

    Clayton

  • Novartis Investigative Site

    Melbourne

  • Novartis Investigative Site

    Perth

  • Novartis Investigative Site

    South Brisbane

  • Novartis Investigative Site

    Westmead

Other trials in Australia

Belgium5 sites

  • Novartis Investigative Site

    Brussels

  • Novartis Investigative Site

    Ghent

  • Novartis Investigative Site

    Godinne

  • Novartis Investigative Site

    La Louvière

  • Novartis Investigative Site

    Leuven

Other trials in Belgium

Spain5 sites

  • Novartis Investigative Site

    Barakaldo

  • Novartis Investigative Site

    Barcelona

  • Novartis Investigative Site

    Madrid

  • Novartis Investigative Site

    Seville

  • Novartis Investigative Site

    Valencia

Other trials in Spain

United Kingdom5 sites

  • Novartis Investigative Site

    Leeds

  • Novartis Investigative Site

    Leicester

  • Novartis Investigative Site

    London

  • Novartis Investigative Site

    Manchester

  • Novartis Investigative Site

    Sheffield

Other trials in United Kingdom

Denmark4 sites

  • Novartis Investigative Site

    Aarhus

  • Novartis Investigative Site

    Copenhagen

  • Novartis Investigative Site

    Herlev

  • Novartis Investigative Site

    Hillerød

Other trials in Denmark

Greece4 sites

  • Novartis Investigative Site

    Athens

  • Novartis Investigative Site

    Larissa

  • Novartis Investigative Site

    Pátrai

  • Novartis Investigative Site

    Thessaloniki

Other trials in Greece

Turkey (Türkiye)4 sites

  • Novartis Investigative Site

    Adana

  • Novartis Investigative Site

    Ankara

  • Novartis Investigative Site

    Istanbul

  • Novartis Investigative Site

    Izmir

Other trials in Turkey (Türkiye)

Austria3 sites

  • Novartis Investigative Site

    Graz

  • Novartis Investigative Site

    Linz

  • Novartis Investigative Site

    Vienna

Other trials in Austria

China3 sites

  • Novartis Investigative Site

    Guangzhou

  • Novartis Investigative Site

    Nanjing

  • Novartis Investigative Site

    Shanghai

Other trials in China

Israel3 sites

  • Novartis Investigative Site

    Afula

  • Novartis Investigative Site

    Jerusalem

  • Novartis Investigative Site

    Petah Tikva

Other trials in Israel

Malaysia2 sites

  • Novartis Investigative Site

    Kota Bharu

  • Novartis Investigative Site

    Kuala Lumpur

Other trials in Malaysia

South Africa2 sites

  • Novartis Investigative Site

    Johannesburg

  • Novartis Investigative Site

    Parktown

Other trials in South Africa

Switzerland2 sites

  • Novartis Investigative Site

    Geneva

  • Novartis Investigative Site

    Zurich

Other trials in Switzerland

Taiwan2 sites

  • Novartis Investigative Site

    Taichung

  • Novartis Investigative Site

    Taipei

Other trials in Taiwan

Thailand2 sites

  • Novartis Investigative Site

    Bangkok

  • Novartis Investigative Site

    Chaingmai

Other trials in Thailand

Egypt1 site

  • Novartis Investigative Site

    Cairo

Other trials in Egypt

Hong Kong1 site

  • Novartis Investigative Site

    Hong Kong

Other trials in Hong Kong

Lebanon1 site

  • Novartis Investigative Site

    Hazmiyeh

Other trials in Lebanon

Netherlands1 site

  • Novartis Investigative Site

    Nijmegen

Other trials in Netherlands

South Korea1 site

  • Novartis Investigative Site

    Seoul

Other trials in South Korea

About this study

Official title: A One Year, Open-label, Single-arm, Multi-center Trial Evaluating the Efficacy and Safety of Oral ICL670 (20 mg/kg/Day) in Patients Diagnosed With Transfusion-dependent Iron Overload

This study uses a single arm, multi-center, open-label trial design. The study will assess the efficacy and safety of 52 weeks of treatment with deferasirox (ICL670) in patients with evidence of transfusion induced iron overload.

Being tested
Deferasirox
Keywords
Thalassemia, Myelodysplastic Syndrome, MDS, Sickle cell, Diamond-Blackfan anemia, Transfusion, Anemia, Fanconi, Chelation, Deferasirox
Medical subject headings
Thalassemia, Myelodysplastic Syndromes, Anemia, Diamond-Blackfan, Anemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • DeferasiroxDrug

    Also called ICL670

Study groups in NCT00171821
Study groupRoleWhat they receive
ICL670 (Deferasirox)ExperimentalNot described
Receives: Drug: Deferasirox

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. To evaluate if fixed starting doses of ICL670, based on transfusion history and subsequent dose titration can provide clinically acceptable chelation as measured by serum ferritin

    Measured: at baseline and at 52 weeks

Secondary outcomes (3)

  1. To evaluate the safety and tolerability profile of in patients treated for up to 52 weeks

    Measured: Monthly

  2. Evaluate efficacy, tolerabilty and safety in the subgroup of patients with baseline LIC < 7 mg Fe/g dw

    Measured: Monthly

  3. Evaluate the relationship between serum ferritin and potential surrogate markers

    Measured: Monthly

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 Sep 2005 (estimated)
  2. Study startApr 2005
  3. Primary completionMay 2009
  4. Study completionJul 2010

Resources

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
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
1,784 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00171821
Sponsor's protocol ID
CICL670A2409
Other IDs
2004-003953-16, 2004
Status last verified
Jun 2013
First submitted
13 Sep 2005
Last updated
11 Feb 2020
Results posted
No

Publications

  • Porter JB, El-Alfy M, Viprakasit V, Giraudier S, Chan LL, Lai Y, El-Ali A, Han J, Cappellini MD. Utility of labile plasma iron and transferrin saturation in addition to serum ferritin as iron overload markers in different underlying anemias before and after deferasirox treatment. Eur J Haematol. 2016 Jan;96(1):19-26. doi: 10.1111/ejh.12540. Epub 2015 Jun 23.

    PubMed 25691036

  • Lee JW, Yoon SS, Shen ZX, Ganser A, Hsu HC, El-Ali A, Habr D, Martin N, Porter JB. Hematologic responses in patients with aplastic anemia treated with deferasirox: a post hoc analysis from the EPIC study. Haematologica. 2013 Jul;98(7):1045-8. doi: 10.3324/haematol.2012.077669. Epub 2013 Apr 12.

    PubMed 23585526

  • Pennell DJ, Porter JB, Cappellini MD, Chan LL, El-Beshlawy A, Aydinok Y, Ibrahim H, Li CK, Viprakasit V, Elalfy MS, Kattamis A, Smith G, Habr D, Domokos G, Roubert B, Taher A. Continued improvement in myocardial T2* over two years of deferasirox therapy in beta-thalassemia major patients with cardiac iron overload. Haematologica. 2011 Jan;96(1):48-54. doi: 10.3324/haematol.2010.031468. Epub 2010 Nov 11.

    PubMed 21071497

  • Lee JW, Yoon SS, Shen ZX, Ganser A, Hsu HC, Habr D, Domokos G, Roubert B, Porter JB; EPIC study investigators. Iron chelation therapy with deferasirox in patients with aplastic anemia: a subgroup analysis of 116 patients from the EPIC trial. Blood. 2010 Oct 7;116(14):2448-54. doi: 10.1182/blood-2010-01-261289. Epub 2010 Jun 21.

    PubMed 20566896

  • Pennell DJ, Porter JB, Cappellini MD, El-Beshlawy A, Chan LL, Aydinok Y, Elalfy MS, Sutcharitchan P, Li CK, Ibrahim H, Viprakasit V, Kattamis A, Smith G, Habr D, Domokos G, Roubert B, Taher A. Efficacy of deferasirox in reducing and preventing cardiac iron overload in beta-thalassemia. Blood. 2010 Mar 25;115(12):2364-71. doi: 10.1182/blood-2009-04-217455. Epub 2009 Dec 8.

    PubMed 19996412

  • Cappellini MD, Porter J, El-Beshlawy A, Li CK, Seymour JF, Elalfy M, Gattermann N, Giraudier S, Lee JW, Chan LL, Lin KH, Rose C, Taher A, Thein SL, Viprakasit V, Habr D, Domokos G, Roubert B, Kattamis A; EPIC Study Investigators. Tailoring iron chelation by iron intake and serum ferritin: the prospective EPIC study of deferasirox in 1744 patients with transfusion-dependent anemias. Haematologica. 2010 Apr;95(4):557-66. doi: 10.3324/haematol.2009.014696. Epub 2009 Nov 30.

    PubMed 19951979

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Deferasirox in people with Transfusion-dependent Iron Overload, run by Novartis Pharmaceuticals at 97 sites in 23 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 7 inclusion and 25 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 11 Feb 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 97 sites in Italy, Germany, France, Australia, Belgium and 18 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?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

To evaluate if fixed starting doses of ICL670, based on transfusion history and subsequent dose titration can provide clinically acceptable chelation as measured by serum ferritin (time frame: at baseline and at 52 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00171821, which also lists 6 linked publications.

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 11 Feb 2020.

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