Transfusional Iron OverloadPhase 2NCT01044186
A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity
A Phase 2 study testing ICL670 in people with Transfusional Iron Overload, run by Novartis Pharmaceuticals at 18 sites in 3 countries.
Trial summary
The purpose of this open-label, non-comparative, multi-center protocol was to further evaluate safety and to provide treatment with ICL670 to patients who had or were at risk of life threatening complications due to transfusional iron overload with a documented inability to tolerate any of the commercially available… Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Transfusional Iron Overload
- What is being tested?
- ICL670
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 18 sitesin 3 countries
- Study dates
- Jun 2003to Dec 2007
- Phase
- Phase 2
- 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
- Patients had to be at risk of life-threatening complications due to transfusional iron overload and be unable to tolerate therapy with any of the commercially available iron chelators (mainly deferoxamine and/or deferiprone) because of documented severe toxicity.
- Patients with a degree of iron overload which was not immediately life-threatening and who were ineligible for other trials with ICL670 could also be enrolled providing they had a well-documented, sound justification for alternative chelation therapy.
- Serum ferritin ≥ 8000 μg/L.
- Serum ferritin < 8000μg/L and LIC of ≥ 7 mg Fe/g dry weight.
- Patients for whom ≥ 8 blood transfusions per year were required in order to maintain the Hemoglobin level at > 9 g/dL.
- Female patients who have reached menarche and who were sexually active had to use double barrier contraception (oral plus barrier contraception), or had to have undergone total hysterectomy and/or ovariectomy, or tubal ligation.
- Written, voluntary informed consent.
Who cannot join
- Patients with transfusional iron overload who were not experiencing severe toxicities during therapy with other iron chelators (e.g. deferoxamine and/or deferiprone).
- Patients with non-transfusional hemosiderosis.
- Patients with severe liver failure as defined by a score of ≥ 10 points on the Child-Pugh scale.
- Patients with serum creatinine 1.5 times the upper limit of normal (ULN) at screening.
- Patients with a history of nephrotic syndrome.
- Patients with a diagnosis of clinically relevant cataract or a previous history of clinically relevant ocular toxicity related to iron chelation therapy.
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- Patients with severe systemic diseases unrelated to iron overload and which would prevent them from undergoing treatment with ICL670.
- Patients with psychiatric or addictive disorders which prevent them from giving informed consent or undergoing treatment with ICL670.
- Pregnant or breast feeding patients.
- Patients treated with systemic investigational drugs within the past four weeks or topical investigational drugs within the past seven days.
- Any surgical or medical condition which might significantly alter the absorption or excretion of drugs as shown by evidence of any of the following:
- History of inflammatory bowel disease
- History of major gastrointestinal tract surgery such as gastrectomy, gastroenterostomy, or bowel resection
- History of pancreatic injury or pancreatitis; indication of impaired pancreatic function/injury as indicated by abnormal lipase or amylase
- Patients being considered by the investigator as potentially unreliable and/or not cooperative with regard to the protocol.
- History of drug or alcohol abuse within the 12 months prior to dosing.
Trial locations and contacts
18 sites in 3 countries.
18 sites in 3 countries
Italy9 sites
Novartis Investigative Site
Ancona
Novartis Investigative Site
Brindisi
Novartis Investigative Site
Cagliari
Novartis Investigative Site
Cosenza
Novartis Investigative Site
Florence
Novartis Investigative Site
Milan
Novartis Investigative Site
Modena
Novartis Investigative Site
Naples
Novartis Investigative Site
Torino
United States8 sites
Children's Hospital Boston
Boston, Massachusetts, 02115
Cincinnatti Children's Hospital Medical center
Cincinnatti, Ohio, 45229
Novartis Investigative Site
Houston, Texas, 77030
Queens Hospital Center
Jamaica, New York, 11432
New York Presbyterian Hospital/Weill Medical College of Cornell University
New York, New York, 10021
New York Methodist Hospital
New York, New York, 11215
Children's Hospital and Research Center - Oakland
Oakland, California, 94609
Children's Hospital of Orange County
Orange, California, 92868
About this study
The purpose of this open-label, non-comparative, multi-center protocol was to further evaluate safety and to provide treatment with ICL670 to patients who had or were at risk of life threatening complications due to transfusional iron overload with a documented inability to tolerate any of the commercially available iron chelators due to severe toxicity rendering continued therapy either impossible or hazardous. Patients who were also ineligible for all on-going registration trials with ICL670 were included in the study. In exceptional cases, patients with a degree of iron overload which was not immediately life-threatening and who were ineligible for the registration trials were also enrolled provided they had a well-documented, sound justification for alternative chelation therapy.
- Conditions
- Transfusional Iron Overload
- Being tested
- ICL670
- Keywords
- Deferasirox, ICL670A, Iron chelators, Deferiprone, Transfusional hemosiderosis, Congenital aplastic anemia (Diamond Blackfan anemia), Red cell aplasia, Thalassemia, β thalassemia
- Medical subject headings
- Cystinosis, Anemia, Diamond-Blackfan, Red-Cell Aplasia, Pure, Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ICL670Drug
| Study group | Role | What they receive |
|---|---|---|
| ICL670 | Experimental | Not described Receives: Drug: ICL670 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the safety profile and to provide treatment with ICL670 for patients with or at risk of life-threatening complications due to transfusional iron overload who are unable to tolerate other iron chelators because of documented severe toxicity.
Measured: 0 - 163 weeks
Secondary outcomes (3)
To estimate the absolute and relative change of liver iron concentration (LIC), to be measured using appropriate methodology available at individual centers.
Measured: Yearly
To evaluate the role of serum ferritin, serum iron, transferrin and transferrin saturation in monitoring iron burden in these patients.
Measured: Quarterly
To evaluate the relationship between changes in LIC and serum ferritin, transferring saturation and serum iron.
Measured: Yearly
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jan 2010 (estimated)
- Study startJun 2003
- Primary completionDec 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01044186
- Sponsor's protocol ID
- CICL670A0117
- Other IDs
- 2004-002303-32 EudraCT number, 2004
- Status last verified
- Feb 2017
- First submitted
- 5 Jan 2010
- Last updated
- 23 Feb 2017
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Feb 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity
- ClinicalTrials.gov ID
- NCT01044186
- Acronym / sponsor ID
- CICL670A0117
- Conditions
- Transfusional Iron Overload
- Keywords
- Deferasirox, ICL670A, Iron chelators, Deferiprone, Transfusional hemosiderosis, Congenital aplastic anemia (Diamond Blackfan anemia), Red cell aplasia, Thalassemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Jun 2003
- Primary completion
- Dec 2007 (actual)
- First posted
- 7 Jan 2010 (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.
The purpose of this open-label, non-comparative, multi-center protocol was to further evaluate safety and to provide treatment with ICL670 to patients who had or were at risk of life threatening complications due to transfusional iron overload with a documented inability to tolerate any of the commercially available iron chelators due to severe toxicity rendering continued therapy either impossible or hazardous. Patients who were also ineligible for all on-going registration trials with ICL670 were included in the study. In exceptional cases, patients with a degree of iron overload which was not immediately life-threatening and who were ineligible for the registration trials were also enrolled provided they had a well-documented, sound justification for alternative chelation therapy.
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: ICL670 |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Patients had to be at risk of life-threatening complications due to transfusional iron overload and be unable to tolerate therapy with any of the commercially available iron chelators (mainly deferoxamine and/or deferiprone) because of documented severe toxicity.
- Patients with a degree of iron overload which was not immediately life-threatening and who were ineligible for other trials with ICL670 could also be enrolled providing they had a well-documented, sound justification for alternative chelation therapy.
- Serum ferritin ≥ 8000 μg/L.
- Serum ferritin < 8000μg/L and LIC of ≥ 7 mg Fe/g dry weight.
- Patients for whom ≥ 8 blood transfusions per year were required in order to maintain the Hemoglobin level at > 9 g/dL.
Key exclusion criteria (16 listed)
- Patients with transfusional iron overload who were not experiencing severe toxicities during therapy with other iron chelators (e.g. deferoxamine and/or deferiprone).
- Patients with non-transfusional hemosiderosis.
- Patients with severe liver failure as defined by a score of ≥ 10 points on the Child-Pugh scale.
- Patients with serum creatinine 1.5 times the upper limit of normal (ULN) at screening.
- Patients with a history of nephrotic syndrome.
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 |
|---|---|---|
| ICL670 | Drug | ICL670 |
Primary outcome
To evaluate the safety profile and to provide treatment with ICL670 for patients with or at risk of life-threatening complications due to transfusional iron overload who are unable to tolerate other iron chelators because of documented severe toxicity.
Time frame: 0 - 163 weeks
3 secondary outcomes
To estimate the absolute and relative change of liver iron concentration (LIC), to be measured using appropriate methodology available at individual centers.
Time frame: Yearly
To evaluate the role of serum ferritin, serum iron, transferrin and transferrin saturation in monitoring iron burden in these patients.
Time frame: Quarterly
To evaluate the relationship between changes in LIC and serum ferritin, transferring saturation and serum iron.
Time frame: Yearly
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 23 Feb 2017.
- Related InfoLink provided by the sponsor.
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
18 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ICL670 in people with Transfusional Iron Overload, run by Novartis Pharmaceuticals at 18 sites in 3 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 7 inclusion and 16 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 23 Feb 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in Italy, United States, Greece. 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?
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?
To evaluate the safety profile and to provide treatment with ICL670 for patients with or at risk of life-threatening complications due to transfusional iron overload who are unable to tolerate other iron chelators because of documented severe toxicity. (time frame: 0 - 163 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 23 Feb 2017.