Transfusional Iron Overload in β-thalassemiaPhase 3NCT00171210

An Extension Study of Iron Chelation Therapy With Deferasirox (ICL670) in β-thalassemia Patients With Transfusional Iron Overload

A Phase 3 study testing Deferasirox in people with Transfusional Iron Overload in β-thalassemia, run by Novartis Pharmaceuticals at 49 sites in 12 countries.

Completed

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

Official record

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Trial summary

Who is it for?: Ages 2 years and older
Conditions the study is for: Transfusional Iron Overload in β-thalassemia
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: 506 participants
Where: 49 sitesin 12 countries
Study dates: Oct 2004to Apr 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

Patients who completed the 12-month core study (NCT00061750)
Female patients after menarche and who were sexually active, if they used double-barrier contraception, oral contraceptive plus barrier contraceptive, or had undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation
Written informed consent obtained from the patient and/or legal guardian on the patient's behalf in accordance with the national legislation

Who cannot join

Pregnant or breast feeding patients
Patients with a history of non-compliance to medical regimens or those considered to be potentially unreliable

Trial locations and contacts

49 sites in 12 countries.

Country

City, postcode or hospital

49 sites in 12 countries

Italy14 sites

Novartis Investigative Site
Brindisi
Novartis Investigative Site
Cagliari
Novartis Investigative Site
Catania
Novartis Investigative Site
Ferrara
Novartis Investigative Site
Genova
Novartis Investigative Site
Milan
Novartis Investigative Site
Monza
Novartis Investigative Site
Naples
Novartis Investigative Site
Palermo
Novartis Investigative Site
Pavia
Novartis Investigative Site
Roma
Novartis Investigative Site
Sassari
Novartis Investigative Site
Syracuse
Novartis Investigative Site
Turin

Other trials in Italy

United States6 sites

Children's Hospital Boston, Dept of Hematology
Boston, Massachusetts, 02115
Children's Memorial Hospital
Chicago, Illinois, 60614
Children's Hospital Los Angeles
Los Angeles, California, 90027
Children's Hospital and Research Center at Oakland
Oakland, California, 94609-1809
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104-4399
Stanford Hospital, Division of Oncology
Stanford, California, 94305-5208

Other trials in United States

Germany5 sites

Novartis Investigative Site
Berlin
Novartis Investigative Site
Düsseldorf
Novartis Investigative Site
Frankfurt
Novartis Investigative Site
Hamburg
Novartis Investigative Site
Ulm

Other trials in Germany

Belgium4 sites

Novartis Investigative Site
Brussels
Novartis Investigative Site
Laken
Novartis Investigative Site
Liège
Novartis Investigative Site
Mons

Other trials in Belgium

France4 sites

Novartis Investigative Site
Créteil
Novartis Investigative Site
Marseille
Novartis Investigative Site
Paris
Novartis Investigative Site
Pierre-Bénite

Other trials in France

Greece4 sites

Novartis Investigative Site
Athens
Novartis Investigative Site
Ioannina
Novartis Investigative Site
Pátrai
Novartis Investigative Site
Thessaloniki

Other trials in Greece

Turkey (Türkiye)4 sites

Novartis Investigative Site
Adana
Novartis Investigative Site
Isparta
Novartis Investigative Site
Istanbul
Novartis Investigative Site
Izmir

Other trials in Turkey (Türkiye)

Argentina2 sites

Novartis Investigative Site
Buenos Aires
Novartis Investigative Site
Córdoba

Other trials in Argentina

Brazil2 sites

Novartis Investigative Site
Campinas
Novartis Investigative Site
São Paulo

Other trials in Brazil

Canada2 sites

Novartis Investigative Site
Montreal
Novartis Investigative Site
Toronto

Other trials in Canada

Tunisia1 site

Novartis Investigative Site
Tunis

Other trials in Tunisia

United Kingdom1 site

Novartis Investigative Site
London

Other trials in United Kingdom

About this study

Official title: An Extension Study of Iron Chelation Therapy With Deferasirox (ICL670)in β-thalassemia Patients With Transfusional Iron Overload

A 1-year randomized Phase III core trial (NCT00061750) using deferoxamine as the comparator was conducted to investigate the efficacy of deferasirox in regularly transfused patients with β-thalassemia 2 years of age and older. Patients who successfully completed this main trial may continue in this extension trial to receive chelation therapy with deferasirox for an additional 4 years.

The objective of this study is to assess the efficacy and long-term safety of deferasirox in regularly transfused patients with β-thalassemia 2 years of age and older.

Conditions: Transfusional Iron Overload in β-thalassemia
Being tested: Deferasirox
Keywords: β-thalassemia, iron overload, deferasirox
Medical subject headings: Iron Overload

What participants receive

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

DeferasiroxDrug
Tablets taken orally once a day.

Study groups in NCT00171210
Study group	Role	What they receive
Deferasirox	Experimental	All participants received Deferasirox (ICL670) orally once a day. Dosage based on body weight. Receives: Drug: Deferasirox

What is being measured

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

Long Term Safety and Tolerability Profile of ICL670 Based on the Number of Participants Who Experienced Any Adverse Event
Adverse events results are based on preferred terms with at least 7% of participants in any group.
Measured: up to 5 years

Secondary outcomes (11)

Long-term Effect of ICL670 on Hepatic Iron Stores Measured by Means of Liver Iron Content (LIC) as Assessed by Liver Biopsy
Mean absolute change of LIC from start of Deferasirox (ICL670) treatment to the end of study assessed by liver biopsy. Reported in milligrams of Iron per gram dry weight (mg Fe/g dw).
Measured: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Long-term Effect of ICL670 on Hepatic Iron Stores Measured by Means of Liver Iron Content (LIC) as Assessed by SQUID
Mean absolute change in LIC from start of Deferasirox (ICL670) treatment to the end of the study assessed by Superconducting Quantum Interfering Device (SQUID) measurement used as a non-invasive alternative to Biopsy for pediatric participants. Reported in milligrams of Iron per gram dry weight (mg Fe/g dw).
Measured: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Long-term Effect of Treatment With ICL670 on the Changes in Serum Ferritin Levels From Start of ICL670 Treatment to End of Study
Mean Absolute Change in serum ferritin (ug/L) from start of treatment with Deferasirox (ICL670) to end of study taking into account the therapeutic goal which will either be to maintain iron balance or to induce negative iron balance. End of study taken as the mean of, at most, the last three available results after start of treatment with ICL670.
Measured: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Change in Surrogate Marker: Serum Transferrin From Start of Treatment With ICL670 to End of Study
Measurement of the relative change in percent of potential surrogate marker: Serum Transferrin (g/L) from start of treatment with Deferasirox (ICL670) to end of study.
(Serum Transferrin at the End of Study-Serum Transferrin at Start of ICL670)/Serum Transferrin at Start of ICL670*100.
Measured: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Change in Surrogate Marker: Serum Iron From Start of Treatment With ICL670 to End of Study
Measurement of the relative change of potential surrogate markers: Serum Iron (µmol/L) from start of treatment with Deferasirox (ICL670) to end of study.
(Serum Iron at the End of Study-Serum Iron at Start of ICL670)/Serum Iron at Start of ICL670*100.
Measured: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Change in Surrogate Marker: Transferrin Saturation From Start of Treatment With ICL670 to End of Study
Measurement of the relative change of potential surrogate marker: Transferrin Saturation (Percent) from start of treatment with Deferasirox (ICL670) to end of study.
(Transferrin Saturation at the End of Study-Tranferrin Saturation at Start of ICL670)/Transferrin Saturation at Start of ICL670*100.
Measured: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Absolute Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by Biopsy
Measurement of median absolute change in liver iron content (LIC) from start of treatment with Deferasirox (ICL670) to end of study obtained through biopsy. Absolute change = End of study value - start of treatment value. LIC is expressed in mg of iron per gram of liver dry weight (mg Fe/g dw).
Measured: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Relative Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by Biopsy
Relative change in liver iron content (LIC) as measured by biopsy and calculated by: End of study value - Start of ICL670 treatment value (absolute change) / Start of ICL670 treatment value.
Measured: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Absolute Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by SQUID
Measurement of the median absolute change in liver iron content (LIC) from start of treatment with Deferasirox (ICL670) to end of study obtained through Superconducting Quantum Interfering Device (SQUID). Absolute change = End of study value - start of treatment value. LIC is expressed in mg of iron per gram of liver dry weight (mg Fe/g dw).
Measured: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Relative Change in Liver Iron Content From Start of ICL670 Treatment to End of Study as Measured by SQUID
Relative change in liver iron content (LIC) measured by Superconducting Quantum Interfering Device (SQUID), calculated by: End of study value - Start of ICL670 treatment value (absolute change) / Start of ICL670 treatment value.
Measured: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Change of Total Body Iron Excretion Rate (TBIE) From Start of ICL670 Treatment to the End of Study
Median change in TBIE (mg/kg/day) from start of treatment with Deferasirox (ICL670) to end of study.
Measured: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

Timeline

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

Oct 2004Apr 2008

First posted15 Sep 2005 (estimated)
Study startOct 2004
Primary completionApr 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.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Transfusional Iron Overload in β-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: 506 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00171210
Sponsor's protocol ID: CICL670A0107E1
Status last verified: May 2011
First submitted: 12 Sep 2005
Last updated: 30 May 2011
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Cappellini MD, Bejaoui M, Agaoglu L, Canatan D, Capra M, Cohen A, Drelichman G, Economou M, Fattoum S, Kattamis A, Kilinc Y, Perrotta S, Piga A, Porter JB, Griffel L, Dong V, Clark J, Aydinok Y. Iron chelation with deferasirox in adult and pediatric patients with thalassemia major: efficacy and safety during 5 years' follow-up. Blood. 2011 Jul 28;118(4):884-93. doi: 10.1182/blood-2010-11-316646. Epub 2011 May 31.
PubMed 21628399

Study at a glance

From the registry record, last updated by the sponsor on 30 May 2011. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Extension Study of Iron Chelation Therapy With Deferasirox (ICL670)in β-thalassemia Patients With Transfusional Iron Overload
ClinicalTrials.gov ID: NCT00171210
Acronym / sponsor ID: CICL670A0107E1
Conditions: Transfusional Iron Overload in β-thalassemia
Keywords: β-thalassemia, iron overload, deferasirox
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 506 participants (actual)
Lead sponsor: Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: Oct 2004
Primary completion: Apr 2008 (actual)
First posted: 15 Sep 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.

The objective of this study is to assess the efficacy and long-term safety of deferasirox in regularly transfused patients with β-thalassemia 2 years of age and older.

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: DeferasiroxAll participants received Deferasirox (ICL670) orally once a day. Dosage based on body weight.

Who may qualify

Ages: 2 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (3 listed)

Patients who completed the 12-month core study (NCT00061750)
Female patients after menarche and who were sexually active, if they used double-barrier contraception, oral contraceptive plus barrier contraceptive, or had undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation
Written informed consent obtained from the patient and/or legal guardian on the patient's behalf in accordance with the national legislation

Key exclusion criteria (2 listed)

Pregnant or breast feeding patients
Patients with a history of non-compliance to medical regimens or those considered to be potentially unreliable

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
DeferasiroxTablets taken orally once a day.	Drug	Deferasirox

Primary outcome

Long Term Safety and Tolerability Profile of ICL670 Based on the Number of Participants Who Experienced Any Adverse Event
Time frame: up to 5 years
Adverse events results are based on preferred terms with at least 7% of participants in any group.

11 secondary outcomes

Long-term Effect of ICL670 on Hepatic Iron Stores Measured by Means of Liver Iron Content (LIC) as Assessed by Liver Biopsy
Time frame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Long-term Effect of ICL670 on Hepatic Iron Stores Measured by Means of Liver Iron Content (LIC) as Assessed by SQUID
Time frame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Long-term Effect of Treatment With ICL670 on the Changes in Serum Ferritin Levels From Start of ICL670 Treatment to End of Study
Time frame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Change in Surrogate Marker: Serum Transferrin From Start of Treatment With ICL670 to End of Study
Time frame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Change in Surrogate Marker: Serum Iron From Start of Treatment With ICL670 to End of Study
Time frame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Change in Surrogate Marker: Transferrin Saturation From Start of Treatment With ICL670 to End of Study
Time frame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Absolute Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by Biopsy
Time frame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Relative Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by Biopsy
Time frame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Absolute Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by SQUID
Time frame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Relative Change in Liver Iron Content From Start of ICL670 Treatment to End of Study as Measured by SQUID
Time frame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Change of Total Body Iron Excretion Rate (TBIE) From Start of ICL670 Treatment to the End of Study
Time frame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

Results and safety

Results posted to the registry on 2 May 2011, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Crossover	259	190
ICL670	296	181

Primary outcome: Long Term Safety and Tolerability Profile of ICL670 Based on the Number of Participants Who Experienced Any Adverse Event

Time frame: up to 5 years. Number. Unit: Participants

Measure	Crossover n = 259	ICL670 n = 296
Pyrexia	109	113
Cough	85	110
Headache	81	84
Diarrhoea	73	69
Vomiting	61	75
Influenza	65	70
Abdominal pain	59	75
Nasopharyngitis	56	73

Population analysed: All patients enrolled into core study and who consented to participate in extension contributed to the pool of data on long term safety follow-up. Analyses included all patients who received at least 1 dose of ICL670 in the core/extension study, that is, analyses also included ICL670 patients from the core group who did not continue into extension.

11 secondary outcomes are reported in the full results.

Adverse events

Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
ICL670	102 of 296 (34.5%)	266 of 296 (89.9%)	Not reported
Crossover	61 of 259 (23.6%)	236 of 259 (91.1%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 30 May 2011.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 21628399Cappellini MD, Bejaoui M, Agaoglu L, Canatan D, Capra M, Cohen A, Drelichman G, Economou M, Fattoum S, Kattamis A, Kilinc Y, Perrotta S, Piga A, Porter JB, Griffel L, Dong V, Clark J, Aydinok Y. Iron chelation with deferasirox in adult and pediatric patients with thalassemia major: efficacy and safety during 5 years' follow-up. Blood. 2011 Jul 28;118(4):884-93. doi: 10.1182/blood-2010-11-316646. Epub 2011 May 31.

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

49 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 Transfusional Iron Overload in β-thalassemia, run by Novartis Pharmaceuticals at 49 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 3 inclusion and 2 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 30 May 2011. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 49 sites in Italy, United States, Germany, Belgium, France 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?

Long Term Safety and Tolerability Profile of ICL670 Based on the Number of Participants Who Experienced Any Adverse Event (time frame: up to 5 years). 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 NCT00171210, which also lists 1 linked publication.

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 30 May 2011.