Beta-ThalassemiaPhase 3NCT00171171
A Study of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis
A Phase 3 study testing deferasirox in people with Beta-Thalassemia and Hemosiderosis, run by Novartis at 5 sites in 5 countries.
Trial summary
Because patients with beta-thalassemia are unable to actively eliminate iron from the body, toxic and eventually lethal levels of iron can accumulate as a result of repeated blood transfusions. This study will evaluate the efficacy, safety and tolerability of deferasirox. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Beta-Thalassemia, Hemosiderosis
- What is being tested?
- deferasirox
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 252 participants
- Where
- 5 sitesin 5 countries
- Study dates
- May 2004to Nov 2006
- 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
- Beta-thalassemia outpatients with transfusional hemosiderosis and unable to be chelated with deferoxamine (DFO) due to DFO being contra-indicated and/or due to documented unacceptable toxicity of DFO or documented poor response to DFO despite proper compliance, or documented non-compliance to DFO, with serum ferritin ≥ 500 ng/ml and LIC ≥ 2 mg/Fe/g dw liver
- Beta-thalassemia outpatients with transfusional hemosiderosis treated with deferiprone
Who cannot join
- Means levels of ALT > 300 U/L
- Serum creatinine above upper limit of normal
- Active hepatitis C or chronic hepatitis B receiving specific treatment
Trial locations and contacts
5 sites in 5 countries.
5 sites in 5 countries
About this study
Official title: A Study of Efficacy and Safety of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis
Because patients with beta-thalassemia are unable to actively eliminate iron from the body, toxic and eventually lethal levels of iron can accumulate as a result of repeated blood transfusions. This study will evaluate the efficacy, safety and tolerability of deferasirox.
- Conditions
- Beta-Thalassemia, Hemosiderosis
- Being tested
- deferasirox
- Keywords
- Thalassemia (beta-thal. major), Transfusional hemosiderosis, Deferasirox, Beta-thalassemia major patients, Unable to be chelated with deferoxamine or deferiprone
- Medical subject headings
- beta-Thalassemia, Hemosiderosis, Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
deferasiroxDrug
| Study group | Role | What they receive |
|---|---|---|
| Deferasirox | Experimental | Not described Receives: Drug: deferasirox |
What is being measured
The primary outcome is the main question the study is designed to answer.
Liver Iron Concentration (as measured by biopsy)
Measured: at baseline and after 1 yeor of ICL670 treatment
Secondary outcomes (5)
Iron balance, i.e. Total Body Iron Excretion (based on the iron influx as determined by the amount of red cells transfused and the change in total body iron (TBI) stores)
Measured: after 1 year of ICL670 treatment
Putative surrogate markers such as serum ferritin serum iron, serum transferrin and transferrin saturation
Measured: monthly assessments for 1 year
Adverse events
Measured: during 1 year ICL670 treatment
hematology and biochemistry, urinalysis
Measured: monthly assessments for 1 year ICL670 treatment
ECG and Echocardiography
Measured: 6-monthly for 1 year ICL670 treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Sep 2005 (estimated)
- Study startMay 2004
- Primary completionNov 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Beta-ThalassemiaStudies of the same condition, open and under way.
- 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
- 252 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Ali Taher, Ass.Prof., Principal investigator, American University of Beirut Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00171171
- Sponsor's protocol ID
- CICL670A2402
- Status last verified
- Nov 2016
- First submitted
- 12 Sep 2005
- Last updated
- 1 Mar 2017
- Results posted
- No
Publications
Taher A, Al Jefri A, Elalfy MS, Al Zir K, Daar S, Rofail D, Baladi JF, Habr D, Kriemler-Krahn U, El-Beshlawy A. Improved treatment satisfaction and convenience with deferasirox in iron-overloaded patients with beta-Thalassemia: Results from the ESCALATOR Trial. Acta Haematol. 2010;123(4):220-5. doi: 10.1159/000313447. Epub 2010 Apr 27.
PubMed 20424435
Study at a glance
From the registry record, last updated by the sponsor on 1 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study of Efficacy and Safety of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis
- ClinicalTrials.gov ID
- NCT00171171
- Acronym / sponsor ID
- CICL670A2402
- Conditions
- Beta-Thalassemia, Hemosiderosis
- Keywords
- Thalassemia (beta-thal. major), Transfusional hemosiderosis, Deferasirox, Beta-thalassemia major patients, Unable to be chelated with deferoxamine or deferiprone
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 252 participants (actual)
- Lead sponsor
- NovartisIndustry (pharma, biotech, devices)
- Study start
- May 2004
- Primary completion
- Nov 2006 (actual)
- First posted
- 15 Sep 2005 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Because patients with beta-thalassemia are unable to actively eliminate iron from the body, toxic and eventually lethal levels of iron can accumulate as a result of repeated blood transfusions. This study will evaluate the efficacy, safety and tolerability of deferasirox.
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: deferasirox |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Beta-thalassemia outpatients with transfusional hemosiderosis and unable to be chelated with deferoxamine (DFO) due to DFO being contra-indicated and/or due to documented unacceptable toxicity of DFO or documented poor response to DFO despite proper compliance, or documented non-compliance to DFO, with serum ferritin ≥ 500 ng/ml and LIC ≥ 2 mg/Fe/g dw liver
- Beta-thalassemia outpatients with transfusional hemosiderosis treated with deferiprone
Key exclusion criteria (3 listed)
- Means levels of ALT > 300 U/L
- Serum creatinine above upper limit of normal
- Active hepatitis C or chronic hepatitis B receiving specific treatment
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 |
|---|---|---|
| deferasirox | Drug | Deferasirox |
Primary outcome
Liver Iron Concentration (as measured by biopsy)
Time frame: at baseline and after 1 yeor of ICL670 treatment
5 secondary outcomes
Iron balance, i.e. Total Body Iron Excretion (based on the iron influx as determined by the amount of red cells transfused and the change in total body iron (TBI) stores)
Time frame: after 1 year of ICL670 treatment
Putative surrogate markers such as serum ferritin serum iron, serum transferrin and transferrin saturation
Time frame: monthly assessments for 1 year
Adverse events
Time frame: during 1 year ICL670 treatment
hematology and biochemistry, urinalysis
Time frame: monthly assessments for 1 year ICL670 treatment
ECG and Echocardiography
Time frame: 6-monthly for 1 year ICL670 treatment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Mar 2017.
- Results publication: PubMed 20424435Taher A, Al Jefri A, Elalfy MS, Al Zir K, Daar S, Rofail D, Baladi JF, Habr D, Kriemler-Krahn U, El-Beshlawy A. Improved treatment satisfaction and convenience with deferasirox in iron-overloaded patients with beta-Thalassemia: Results from the ESCALATOR Trial. Acta Haematol. 2010;123(4):220-5. doi: 10.1159/000313447. Epub 2010 Apr 27.
- Results for CICL670A2402 from the Novartis Clinical Trials websiteLink provided by the sponsor.
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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.
Ali Taher, Ass.Prof. principal investigator
American University of Beirut Medical Center
5 sites in 5 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 Beta-Thalassemia and Hemosiderosis, run by Novartis at 5 sites in 5 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 2 inclusion and 3 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 1 Mar 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Egypt, Lebanon, Oman, Saudi Arabia, Syria. 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?
Liver Iron Concentration (as measured by biopsy) (time frame: at baseline and after 1 yeor of ICL670 treatment). 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 NCT00171171, 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 1 Mar 2017.