ThalassemiaPhase 2NCT00901199

Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload

A Phase 2 study testing Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO) in people with Thalassemia and Iron Overload, run by Elliott Vichinsky at 1 site in United States.

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 is a pilot study looking at the safety and efficacy of giving combination chelation with deferasirox and deferoxamine. The hypothesis is that combination chelation is safe in decreasing overall iron in patients with thalassemia. Read more about this study

Who is it for?: Ages 8 years and older
Conditions the study is for: Thalassemia, Iron Overload
What is being tested?: Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO)
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 22 participants
Where: 1 siteUnited States
Study dates: Sep 2007to Oct 2012
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 8 years and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Transfusion Dependent Thalassemia
If iron between 5-15 mg/g dry liver by SQUID, subject must have a documented endocrinopathy or cardiac finding
Older than 8 years

Who cannot join

Participating on another interventional clinical trial

Trial locations and contacts

1 site in 1 country.

United States1 site

CHRCO
Oakland, California, 94609

Other trials in United States

About this study

Official title: Safety of Deferasirox (ICL670) and Deferoxamine (Desferal or DFO) Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload

Read the detailed description

This was a phase two pilot clinical trial designed to evaluate the safety and efficacy of the combination of deferasirox and deferoxamine in transfusion dependent thalassemia with a range of systemic iron burden. The duration of combined therapy was 12 months. Changes in liver iron concentration, ferritin, myocardial iron, and serum creatinine was monitored.

Conditions: Thalassemia, Iron Overload
Being tested: Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO)
Keywords: Thalassemia, Chelation, Iron Overload, Thalassemia patients with Iron overload
Medical subject headings: Thalassemia, Iron Overload

What participants receive

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

Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO)Drug
Also called Deferasirox, (Exjade), Deferoxamine (DFO)
All subjects will be given Deferasirox 20-30 mg/kg for 7 days per week. All subject will be given Deferoxamine 50 mg/kg for 3-7 days per week. The number of days for Deferoxamine will be determined by liver iron concentration at baseline.

Study groups in NCT00901199
Study group	Role	What they receive
Deferasirox (Exjade) and Deferoxamine (DFO)	Experimental	All subjects received Deferasirox (Exjade) and Deferoxamine (DFO) dosing based on the iron overload at baseline. Receives: Drug: Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO)

What is being measured

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

Efficacy of Combined Treatment With Deferasirox and Deferoxamine Over 12 Months
Change in liver iron concentration from baseline to 12 months with the use of combined chelation therapy. The change was calculated as the liver iron concentration at 12 months minus the value at baseline.
Measured: 12 months
Change in Serum Creatinine During 12 Months Combined Chelation Therapy
Comparison of average serum creatinine over 12 months of combined chelation therapy compared with baseline serum creatinine.
Measured: 12 months

Timeline

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

Sep 2007Oct 2012

First posted13 May 2009 (estimated)
Study startSep 2007
Primary completionOct 2012
Study completionOct 2012

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 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 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 22 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00901199
Sponsor's protocol ID: CICL670AUS24T
Status last verified: Jul 2021
First submitted: 12 May 2009
Last updated: 12 Aug 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Publications

Lal A, Porter J, Sweeters N, Ng V, Evans P, Neumayr L, Kurio G, Harmatz P, Vichinsky E. Combined chelation therapy with deferasirox and deferoxamine in thalassemia. Blood Cells Mol Dis. 2013 Feb;50(2):99-104. doi: 10.1016/j.bcmd.2012.10.006. Epub 2012 Nov 11.
PubMed 23151373

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.
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RecruitingObservational study
Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Transfusion-dependent Beta-Thalassemia, Gene Therapy
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Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
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Active, not recruitingPhase 3
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia

All Thalassemia trials

Study at a glance

From the registry record, last updated by the sponsor on 12 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Safety of Deferasirox (ICL670) and Deferoxamine (Desferal or DFO) Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload
ClinicalTrials.gov ID: NCT00901199
Acronym / sponsor ID: CICL670AUS24T
Conditions: Thalassemia, Iron Overload
Keywords: Thalassemia, Chelation, Iron Overload, Thalassemia patients with Iron overload
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 22 participants (actual)
Lead sponsor: Elliott VichinskyUniversity, hospital or other
Study start: Sep 2007
Primary completion: Oct 2012 (actual)
Study completion: Oct 2012 (actual)
First posted: 13 May 2009 (estimated)
Results: Posted 12 Aug 2021

Clinical summary

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

Detailed description

Study design

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

Arm	Type	Receives
Deferasirox (Exjade) and Deferoxamine (DFO)	Experimental	Drug: Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO)All subjects received Deferasirox (Exjade) and Deferoxamine (DFO) dosing based on the iron overload at baseline.

Who may qualify

Ages: 8 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (3 listed)

Transfusion Dependent Thalassemia
If iron between 5-15 mg/g dry liver by SQUID, subject must have a documented endocrinopathy or cardiac finding
Older than 8 years

Key exclusion criteria (1 listed)

Participating on another interventional clinical trial

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
Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO)Also: Deferasirox, (Exjade), Deferoxamine (DFO)All subjects will be given Deferasirox 20-30 mg/kg for 7 days per week. All subject will be given Deferoxamine 50 mg/kg for 3-7 days per week. The number of days for Deferoxamine will be determined by liver iron concentration at baseline.	Drug	Deferasirox (Exjade) and Deferoxamine (DFO)

Primary outcome

Efficacy of Combined Treatment With Deferasirox and Deferoxamine Over 12 Months
Time frame: 12 months
Change in liver iron concentration from baseline to 12 months with the use of combined chelation therapy. The change was calculated as the liver iron concentration at 12 months minus the value at baseline.
Change in Serum Creatinine During 12 Months Combined Chelation Therapy
Time frame: 12 months
Comparison of average serum creatinine over 12 months of combined chelation therapy compared with baseline serum creatinine.

Results and safety

Results posted to the registry on 12 Aug 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Deferasirox (Exjade) and Deferoxamine (DFO)	22	18

Primary outcome: Efficacy of Combined Treatment With Deferasirox and Deferoxamine Over 12 Months

Time frame: 12 months. Median (Full Range). Unit: mg/g

Measure	Deferasirox (Exjade) and Desferal (DFO) n = 18
Value	-6.2 (-23.1 to 2.7)

Population analysed: Eighteen subjects completed one year of therapy. Four subjects did not complete the study for the following reasons: lost to follow-up (1), abdominal pain (1), death (1), and non-compliance (1).

Primary outcome: Change in Serum Creatinine During 12 Months Combined Chelation Therapy

Time frame: 12 months. Mean (95% Confidence Interval). Unit: mg/dl

Measure	Deferasirox (Exjade) and Desferal (DFO) n = 22
Value	0.09 (0.05 to 0.12)

Adverse events

Time frame: 1 year. Other adverse events reported at 2.5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Deferasirox (Exjade) and Deferoxamine (DFO)	2 of 22 (9.1%)	1 of 22 (4.5%)	1 of 22

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 12 Aug 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Results publication: PubMed 23151373Lal A, Porter J, Sweeters N, Ng V, Evans P, Neumayr L, Kurio G, Harmatz P, Vichinsky E. Combined chelation therapy with deferasirox and deferoxamine in thalassemia. Blood Cells Mol Dis. 2013 Feb;50(2):99-104. doi: 10.1016/j.bcmd.2012.10.006. Epub 2012 Nov 11.

Related trials for Thalassemia

Active, not recruiting
Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.
Transfusion-dependent Beta-Thalassemia
RecruitingObservational study
Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Transfusion-dependent Beta-Thalassemia, Gene Therapy
Not yet recruiting
Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
Beta-Thalassemia
RecruitingPhase 1/2
β-Thalassemia Treatment With KL003 Cell Injection
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Active, not recruitingPhase 3
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia

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.

Elliot Vichinsky, MD principal investigator

UCSF Benioff Children's Hospital Oakland

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

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO) in people with Thalassemia and Iron Overload, run by Elliott Vichinsky at 1 site in United States.

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 1 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 12 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. 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 12 Aug 2021). 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?

Efficacy of Combined Treatment With Deferasirox and Deferoxamine Over 12 Months (time frame: 12 months). 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 NCT00901199, 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 12 Aug 2021.