Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia MajorPhase 4NCT01610297

Post Hematopoietic Stem Cell Transplantation

A Phase 4 study testing ICL670 in people with Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major, run by Novartis Pharmaceuticals at 6 sites in Turkey (Türkiye).

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

This was a prospective, single-arm, multicenter, national, phase II clinical study. The purpose of this Phase II study was to examine the safety and efficacy of deferasirox to decrease iron overload (IOL) in the posttransplant period in patients with beta-thalassemia major. Read more about this study

Who is it for?: Ages 2 years and older
Conditions the study is for: Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major
What is being tested?: ICL670
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 27 participants
Where: 6 sitesTurkey (Türkiye)
Study dates: Sep 2013to Oct 2015
Phase: Phase 4
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 had hematopoietic stem cell transplantation (HSCT) for beta-thalassemia major
hematopoietic stem cell transplantation (HSCT) was performed minimum 6 months and maximum 2 years ago
The washout period after the immunosuppressive therapy should be at least 3 months.
Signifacant IOL should be present including:
Serum ferritin >1000 μg/L or B. cardiac MRI <20 ms or C. liver iron concentration ≥ 5 mg/g dry weight measured by R2* MRI

Who cannot join

Patients who had any contraindication for treatment with deferasirox according to the prescribing information
•Patients who depended on transfusion
Patients with clinical symptoms of cardiac dysfunction (shortness of breath at rest or exertion, orthopnea, exercise intolerance, lower extremity edema, arrhythmias)
Patients who were experiencing severe complication of HSCT e.g. acute Graft-versus host disease (GVHD)
Significant proteinuria / Increase in serum creatinine

Trial locations and contacts

6 sites in 1 country.

City, postcode or hospital

6 sites in 1 country

Turkey (Türkiye)6 sites

Novartis Investigative Site
Ankara, Turkey, 06100
Novartis Investigative Site
Ankara, Turkey, 06500
Novartis Investigative Site
Antalya, 07000
Novartis Investigative Site
Antalya, 07070
Novartis Investigative Site
Istanbul
Novartis Investigative Site
Izmir, Turkey, 35040

Other trials in Turkey (Türkiye)

About this study

Official title: A Phase II, Multi-center, Single-arm, Prospective Study to Evaluate the Safety and Efficacy of Deferasirox in Beta-thalassemia Major Patients After Hematopoietic Stem Cell Transplantation.

Conditions: Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major
Being tested: ICL670
Keywords: hematopoietic stem cell transplantation, beta-thalassemia major, deferasirox, iron overload
Medical subject headings: beta-Thalassemia, Iron Overload

What participants receive

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

ICL670Drug
Oral dose of ICL670 at 10 mg/kg daily

Study groups in NCT01610297
Study group	Role	What they receive
ICL670	Experimental	Oral dose of ICL670 at 10 mg/kg daily Receives: Drug: ICL670

What is being measured

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

Number of Participants With Adverse Events, Serious Adverse Events and Deaths as a Measure of Safety and Tolerability
To determine the safety; incidence, type and severity of adverse events including renal, hepatic, biochemistry and hematologic parameters of deferasirox in the treatment of iron overload after hematopoietic stem cell transplantation (HSCT) in patients with beta-thalassemia major in 12 months period
Measured: 12 months

Secondary outcomes (4)

Change in Serum Ferritin Level.
Blood samples were collected and serum levels were assessed at study baseline (BL) and at 12 months.
Measured: Baseline, 12 Months
Change in the Further Parameters of Iron Overload (Liver Iron Concentration by Magnetic Resonance Imaging (MRI Examination)
Liver Iron Concentration (LIC) values between 3 and 7 mg Fe / g dry weight are indicative of mild iron deposition, while values between 7 and 15 mg Fe / g dry weight are indicative of moderate iron deposition which have been associated with liver disease. Values >15 mg Fe/g dry weight are indicative of severe iron deposition which is associated with progressive liver fibrosis, increased morbidity and mortality
Measured: Baseline, 12 month
The Percentage of Patients Reaching Serum Ferritin Levels Lower Than 500 μg/L
Serum Ferritin values between 1000-2500 μg/L are indicative of mild to moderate iron overload while values >2500 μg/L are indicative of severe iron overload and levels constantly higher than 2500 μg/L has been shown to to increase the risk of cardiac complications and endocrine disease. Maintaining levels <1000 μg/L is associated with increased survival and less morbidity.
Measured: Week 28 and Week 52
Change in the Further Parameters of Iron Overload (Cardiac Iron Concentration by Magnetic Resonance Imaging (MRI Examination)
Cardiac MRI values between 10 to 20 milliseconds (ms) are indicative of moderate cardiac iron deposition associated with declining left ventricular ejection fraction and arrhythmias while values <10 ms are indicative of deposition sufficient to risk cardiac decompensation and associated with overt heart failure and mortality.
Measured: Baseline, 12 month

Timeline

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

Sep 2013Oct 2015

First posted4 Jun 2012 (estimated)
Study startSep 2013
Primary completionOct 2015
Study completionOct 2015

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 Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia MajorStudies 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 4
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 27 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01610297
Sponsor's protocol ID: CICL670ATR04
Status last verified: Sep 2016
First submitted: 30 May 2012
Last updated: 24 Oct 2016
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Yesilipek MA, Karasu G, Kaya Z, Kuskonmaz BB, Uygun V, Dag I, Ozudogru O, Ertem M. A Phase II, Multicenter, Single-Arm Study to Evaluate the Safety and Efficacy of Deferasirox after Hematopoietic Stem Cell Transplantation in Children with beta-Thalassemia Major. Biol Blood Marrow Transplant. 2018 Mar;24(3):613-618. doi: 10.1016/j.bbmt.2017.11.006. Epub 2017 Nov 16.
PubMed 29155313

Study at a glance

From the registry record, last updated by the sponsor on 24 Oct 2016. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase II, Multi-center, Single-arm, Prospective Study to Evaluate the Safety and Efficacy of Deferasirox in Beta-thalassemia Major Patients After Hematopoietic Stem Cell Transplantation.
ClinicalTrials.gov ID: NCT01610297
Acronym / sponsor ID: CICL670ATR04
Conditions: Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major
Keywords: hematopoietic stem cell transplantation, beta-thalassemia major, deferasirox, iron overload
Phase: Phase 4
Study type: Interventional
Recruitment status: Completed
Enrolment: 27 participants (actual)
Lead sponsor: Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: Sep 2013
Primary completion: Oct 2015 (actual)
Study completion: Oct 2015 (actual)
First posted: 4 Jun 2012 (estimated)
Results: Posted 21 Jul 2016

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
ICL670	Experimental	Drug: ICL670Oral dose of ICL670 at 10 mg/kg daily

Who may qualify

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

Key inclusion criteria (5 listed)

Patients who had hematopoietic stem cell transplantation (HSCT) for beta-thalassemia major
hematopoietic stem cell transplantation (HSCT) was performed minimum 6 months and maximum 2 years ago
The washout period after the immunosuppressive therapy should be at least 3 months.
Signifacant IOL should be present including:
Serum ferritin >1000 μg/L or B. cardiac MRI <20 ms or C. liver iron concentration ≥ 5 mg/g dry weight measured by R2* MRI

Key exclusion criteria (5 listed)

Patients who had any contraindication for treatment with deferasirox according to the prescribing information
•Patients who depended on transfusion
Patients with clinical symptoms of cardiac dysfunction (shortness of breath at rest or exertion, orthopnea, exercise intolerance, lower extremity edema, arrhythmias)
Patients who were experiencing severe complication of HSCT e.g. acute Graft-versus host disease (GVHD)
Significant proteinuria / Increase in serum creatinine

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
ICL670Oral dose of ICL670 at 10 mg/kg daily	Drug	ICL670

Primary outcome

Number of Participants With Adverse Events, Serious Adverse Events and Deaths as a Measure of Safety and Tolerability
Time frame: 12 months
To determine the safety; incidence, type and severity of adverse events including renal, hepatic, biochemistry and hematologic parameters of deferasirox in the treatment of iron overload after hematopoietic stem cell transplantation (HSCT) in patients with beta-thalassemia major in 12 months period

4 secondary outcomes

Change in Serum Ferritin Level.
Time frame: Baseline, 12 Months
Change in the Further Parameters of Iron Overload (Liver Iron Concentration by Magnetic Resonance Imaging (MRI Examination)
Time frame: Baseline, 12 month
The Percentage of Patients Reaching Serum Ferritin Levels Lower Than 500 μg/L
Time frame: Week 28 and Week 52
Change in the Further Parameters of Iron Overload (Cardiac Iron Concentration by Magnetic Resonance Imaging (MRI Examination)
Time frame: Baseline, 12 month

Results and safety

Results posted to the registry on 21 Jul 2016, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
ICL670	27	26

Primary outcome: Number of Participants With Adverse Events, Serious Adverse Events and Deaths as a Measure of Safety and Tolerability

Time frame: 12 months. Number. Unit: Participants

Measure	ICL670 n = 27
Adverse events	25
Serious adverse events	3
Death	0

Population analysed: The Safety Set (SS) includes all included patients who were included in the study. All statistical analyses of safety and tolerability will be done in the SS.

4 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	3 of 27 (11.1%)	23 of 27 (85.2%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 24 Oct 2016.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 29155313Yesilipek MA, Karasu G, Kaya Z, Kuskonmaz BB, Uygun V, Dag I, Ozudogru O, Ertem M. A Phase II, Multicenter, Single-Arm Study to Evaluate the Safety and Efficacy of Deferasirox after Hematopoietic Stem Cell Transplantation in Children with beta-Thalassemia Major. Biol Blood Marrow Transplant. 2018 Mar;24(3):613-618. doi: 10.1016/j.bbmt.2017.11.006. Epub 2017 Nov 16.

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

6 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

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 5 inclusion and 5 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 24 Oct 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites in Turkey (Türkiye). 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

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 21 Jul 2016). 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?

Number of Participants With Adverse Events, Serious Adverse Events and Deaths as a Measure of Safety and Tolerability (time frame: 12 months). 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 NCT01610297, 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 24 Oct 2016.