Transfusional Iron OverloadPhase 2NCT00390858

A 4-year Extension Study to Core 1-year Study of Iron Chelation Therapy With Deferasirox in β-thalassemia Major Pediatric Patients With Transfusional Iron Overload.

A Phase 2 study testing Deferasirox in people with Transfusional Iron Overload and β-thalassemia Major, run by Novartis Pharmaceuticals at 4 sites in 2 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

In this 4-year extension study the safety, efficacy and and pharmacokinetics of deferasirox in regularly transfused pediatric patients with β-thalassemia major was assessed. Patients who successfully completed the main 1 year trial (NCT00390858) were eligible to continue in this extension trial and receive chelation… Read more about this study

Who is it for?: Ages 1 year to 18 years
Conditions the study is for: Transfusional Iron Overload, β-thalassemia Major, Pediatric Rare 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: 40 participants
Where: 4 sitesin 2 countries
Study dates: Sep 2003to Feb 2008
Phase: Phase 2
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 1 year to 18 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

Completion of the planned 12-month core trial, (NCT00390858).
Female patients who have reached menarche and who were sexually active were to use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation.
Written informed consent obtained from the patient, and/or from the parent or legal guardian in accordance with the national legislation.

Who cannot join

Pregnant or breast feeding patients
Patients with a history of non-compliance to medical regimens and patients who are considered by the investigator as potentially unreliable.
Other protocol-defined exclusion criteria may apply.

Trial locations and contacts

4 sites in 2 countries.

Country

City, postcode or hospital

4 sites in 2 countries

Italy3 sites

Novartis Investigative Site
Cagliari
Novartis Investigative Site
Genova
Novartis Investigative Site
Torino

Other trials in Italy

France1 site

Novartis Investigative Site
Lyon

Other trials in France

About this study

Official title: A 4-year Extension to a Phase II a Multicenter Study Evaluating Long-term Safety, Tolerability, Pharmacokinetics and Effects on Liver Iron Concentration of Repeated Doses of 10 mg/kg/Day of Deferasirox in Pediatric Patients With Transfusion Dependent β-thalassemia Major.

Conditions: Transfusional Iron Overload, β-thalassemia Major, Pediatric Rare Anemia
Being tested: Deferasirox
Keywords: β-thalassemia major, iron overload, deferasirox, pediatric rare anemia
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
Also called ICL670
Deferasirox in children from 1 to 18 years old was given orally once daily, 30 minutes prior to breakfast. An initial daily dose of 10 mg/kg was used during the 1-year core study. In this 4-year extension study dose modifications of ± 5 or 10 mg/kg were based on safety parameters and on increasing or decreasing Liver Iron Concentration (LIC), and serum ferritin. Deferasirox was available as 125 mg, 250 mg and 500 mg tablets.

Study groups in NCT00390858
Study group	Role	What they receive
Deferasirox	Experimental	Initial dose of 10 mg/kg, dose modifications of ± 5 or 10 mg/kg were based on participant response. Receives: Drug: Deferasirox

What is being measured

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

Participants With Adverse Events by Primary System Organ Class (SOC)
Safety parameters were measured by the number and type of adverse events (AEs). An adverse event is any untoward medical occurence in a patient administered a medicinal product that does not necessarily have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign ( for example, an abnormal laboratory finding), symptom or disease temporally associated with the use of the medicinal product, whether or not this is associated with the use of this medicinal product.
Measured: 4 year extension + core 1 year
Change in Liver Iron Concentration (LIC)
Change in Liver Iron Concentration [LIC] measured by means of SQUID (Superconducting Quantum Interference Device). LIC is expressed in milligrams of iron per gram of liver dry weight (mg Fe/g dw)
Measured: Baseline of Core Study to End of Extension Study, up to 5 years.

Secondary outcomes (2)

Total Body Iron Elimination (TBIE) Rate (mg/kg/Day)
Total Iron Body Elimination (TBIE) Rate [mg/kg/Day] was calculated for each patient based on SQUID ( Superconducting Quantum Interference Device) results.
Measured: Baseline of Core Study to End of Extension Study, up to 5 years
Relative Change in Serum Ferritin Level
Serum levels were drawn at the baseline of the Core Study up to 18 months of the Extension Study. Levels were analyzed for serum ferritin measured in micrograms per Liter. Relative change (%) in serum ferritin level was assessed from Baseline to Extension 18 months. Relative Change = 1 - (Change in ferritin level from Baseline/Baseline level) x 100.
Measured: Baseline of Core Study to Extension 18 months, up to 2.5 years.

Timeline

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

Sep 2003Feb 2008

First posted20 Oct 2006 (estimated)
Study startSep 2003
Primary completionFeb 2008
Study completionFeb 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 Transfusional Iron OverloadStudies 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 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 40 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00390858
Sponsor's protocol ID: CICL670A0106E1
Status last verified: Feb 2017
First submitted: 18 Oct 2006
Last updated: 20 Mar 2017
Results posted: Yes, on ClinicalTrials.gov

Study at a glance

From the registry record, last updated by the sponsor on 20 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A 4-year Extension to a Phase II a Multicenter Study Evaluating Long-term Safety, Tolerability, Pharmacokinetics and Effects on Liver Iron Concentration of Repeated Doses of 10 mg/kg/Day of Deferasirox in Pediatric Patients With Transfusion Dependent β-thalassemia Major.
ClinicalTrials.gov ID: NCT00390858
Acronym / sponsor ID: CICL670A0106E1
Conditions: Transfusional Iron Overload, β-thalassemia Major, Pediatric Rare Anemia
Keywords: β-thalassemia major, iron overload, deferasirox, pediatric rare anemia
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 40 participants (actual)
Lead sponsor: Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: Sep 2003
Primary completion: Feb 2008 (actual)
Study completion: Feb 2008 (actual)
First posted: 20 Oct 2006 (estimated)
Results: Posted 15 Aug 2011

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Deferasirox	Experimental	Drug: DeferasiroxInitial dose of 10 mg/kg, dose modifications of ± 5 or 10 mg/kg were based on participant response.

Who may qualify

Ages: 1 year to 18 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (3 listed)

Completion of the planned 12-month core trial, (NCT00390858).
Female patients who have reached menarche and who were sexually active were to use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation.
Written informed consent obtained from the patient, and/or from the parent or legal guardian in accordance with the national legislation.

Key exclusion criteria (3 listed)

Pregnant or breast feeding patients
Patients with a history of non-compliance to medical regimens and patients who are considered by the investigator as potentially unreliable.
Other protocol-defined exclusion criteria may apply.

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
DeferasiroxAlso: ICL670Deferasirox in children from 1 to 18 years old was given orally once daily, 30 minutes prior to breakfast. An initial daily dose of 10 mg/kg was used during the 1-year core study. In this 4-year extension study dose modifications of ± 5 or 10 mg/kg were based on safety parameters and on increasing or decreasing Liver Iron Concentration (LIC), and serum ferritin. Deferasirox was available as 125 mg, 250 mg and 500 mg tablets.	Drug	Deferasirox

Primary outcome

Participants With Adverse Events by Primary System Organ Class (SOC)
Time frame: 4 year extension + core 1 year
Safety parameters were measured by the number and type of adverse events (AEs). An adverse event is any untoward medical occurence in a patient administered a medicinal product that does not necessarily have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign ( for example, an abnormal laboratory finding), symptom or disease temporally associated with the use of the medicinal product, whether or not this is associated with the use of this medicinal product.
Change in Liver Iron Concentration (LIC)
Time frame: Baseline of Core Study to End of Extension Study, up to 5 years.
Change in Liver Iron Concentration [LIC] measured by means of SQUID (Superconducting Quantum Interference Device). LIC is expressed in milligrams of iron per gram of liver dry weight (mg Fe/g dw)

2 secondary outcomes

Total Body Iron Elimination (TBIE) Rate (mg/kg/Day)
Time frame: Baseline of Core Study to End of Extension Study, up to 5 years
Relative Change in Serum Ferritin Level
Time frame: Baseline of Core Study to Extension 18 months, up to 2.5 years.

Results and safety

Results posted to the registry on 15 Aug 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
Children ( < 12 Years)	20	11
Adolescents ( ≧12 Years)	20	13

Primary outcome: Participants With Adverse Events by Primary System Organ Class (SOC)

Time frame: 4 year extension + core 1 year. Number. Unit: participants

Measure	Children (<12 Years) n = 20	Adolescents ( ≧12 Years) n = 20
Patients with at least one Adverse Event (AE)	20	20
General disorders & administration site conditions	19	20
Respiratory, thoracic & mediastinal disorders	19	17
Gastrointestinal disorders	18	17
Infections & infestations	18	17
Musculoskeletal & connective tissue disorders	12	15
Investigations	13	10
Nervous system disorders	9	13

Population analysed: The safety set comprising of all the 40 patients who received at least one dose of deferasirox during the core or extension study was used in all analyses.

Primary outcome: Change in Liver Iron Concentration (LIC)

Time frame: Baseline of Core Study to End of Extension Study, up to 5 years.. Mean (Standard Deviation). Unit: mg Fe/g dw

Measure	Children (<12 Years) n = 20	Adolescents ( ≧12 Years) n = 20
Core Baseline LIC (n = 20, 20)	6.25 (2.507)	5.73 (2.185)
End of Extension LIC (n=19, 20)	5.46 (3.192)	4.66 (3.533)
Change from Baseline LIC (n=19, 20)	-0.9 (3.85)	-1.10 (3.03)

Population analysed: The safety set was used for all analyses. This comprised of all 40 patients who received at least one dose of deferasirox during the core or extension study.

2 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse Events were collected from Core Study Baseline to Extension End of Study, up to 5 years.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Children (<12 Years)	4 of 20 (20%)	20 of 20 (100%)	Not reported
Adolescents ( ≧12 Years)	8 of 20 (40%)	20 of 20 (100%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 20 Mar 2017.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

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.

Dr. Gianluca Forni study director

Novartis Pharmaceuticals

Prof. Renzo Galanello study director

Novartis Pharmaceuticals

Prof. Antonio Piga study director

Novartis Pharmaceuticals

4 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Deferasirox in people with Transfusional Iron Overload and β-thalassemia Major, run by Novartis Pharmaceuticals at 4 sites in 2 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 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 20 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 4 sites in Italy, France. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 15 Aug 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?

Participants With Adverse Events by Primary System Organ Class (SOC) (time frame: 4 year extension + core 1 year). The study lists 2 primary outcomes in all. 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 NCT00390858.

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 20 Mar 2017; AzTrials last refreshed its copy on 27 Oct 2026.