Iron OverloadPhase 1/2NCT00395629
Safety and Efficacy of Deferasirox (ICL670) in Patients With Iron Overload Resulting From Hereditary Hemochromatosis
A Phase 1/2 study testing Deferasirox (ICL670) in people with Iron Overload and Hereditary Hemochromatosis, run by Novartis Pharmaceuticals at 18 sites in 6 countries.
Trial summary
Brief Summary: This study was designed to explore a safe dose and characterize the preliminary safety and efficacy of ICL670 in adult patients with previously documented history of homozygous C282Y. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Iron Overload, Hereditary Hemochromatosis
- What is being tested?
- Deferasirox (ICL670)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 49 participants
- Where
- 18 sitesin 6 countries
- Study dates
- Aug 2006to Mar 2009
- Phase
- Phase 1/2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age 18 years of age or older
- Male or female patients homozygous for the C282Y mutation.
- Iron overload as documented by serum ferritin and transferrin saturation
- No known allergy or contraindication to the administration of deferasirox
- Ability to comply with all study-related procedures, medications, and evaluations
- Effective use of birth control measures.
Who cannot join
- Iron overload not due to hereditary hemochromatosis
- Males with hemoglobin <13 mg/dL, females with hemoglobin <12 mg/dL
- Desferal treatment within 1 month of the screening visit
- Patients currently or previously treated with deferiprone or deferasirox
- Significant medical condition interfering with the ability to partake in this study
- Presence of a surgical or medical condition that might significantly alter the absorption, distribution, metabolism or excretion of any study drug
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- Clinical evidence of Active Hepatitis B or C
- Positive HIV serology
- Pregnant or breast feeding patients
- Patients treated with systemic investigational drug within 4 weeks prior or with topical investigational drug within 7 days prior to the screening visit
- Other protocol-defined inclusion/exclusion criteria may apply.
Trial locations and contacts
18 sites in 6 countries.
18 sites in 6 countries
United States9 sites
Carolinas Medical Center
Charlotte, North Carolina, 28203
Three Medical Park
Columbia, South Carolina, 29203
Ohio State University Medical Center
Columbus, Ohio, 43210
Henry Ford Health System
Detroit, Michigan, 48202
University of Connecticut Health Center
Farmington, Connecticut, 06030
UC Irvine/Long Beach
Long Beach, California, 90822
Mayo Clinic
Rochester, Minnesota, 55905
Rochester General Hospital
Rochester, New York, 14625
St. Louis University
St Louis, Missouri, 63110
Germany4 sites
Novartis Investigative Site
Chemnitz
Novartis Investigative Site
Heidelberg
Novartis Investigative Site
Magdeburg
Novartis Investigative Site
Oberhausen
Italy2 sites
Novartis Investigative Site
Modena
Novartis Investigative Site
Monza
About this study
Official title: A Phase I/II Open Label, Dose Escalation Trial and a Six Month Extension to Explore the Safety and Efficacy of ICL670 in Patients With Iron Overload Resulting From Hereditary Hemochromatosis.
Brief Summary: This study was designed to explore a safe dose and characterize the preliminary safety and efficacy of ICL670 in adult patients with previously documented history of homozygous C282Y.
- Conditions
- Iron Overload, Hereditary Hemochromatosis
- Being tested
- Deferasirox (ICL670)
- Keywords
- Deferasirox, ICL670A, Iron chelators, Deferiprone, Transfusional Hemochromatosis
- Medical subject headings
- Iron Overload, Hemochromatosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Deferasirox (ICL670)Drug
| Study group | Role | What they receive |
|---|---|---|
| ICL670 (Deferasirox) | Experimental | Three dose cohorts: 5 mg/kg/day, 10 mg/kg/day, 15 mg/kg/day Receives: Drug: Deferasirox (ICL670) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Absolute Change of Serum Ferritin From Baseline to the End of Extension, by Dose Cohort (Extension Per-protocol Population)
Mean absolute change in serum ferritin from baseline to the end of the extension study.
Measured: 0 to 48 weeks
Secondary outcomes (1)
Trough Concentrations of Deferasirox (ICL670), by Dose Cohort (Per-protocol Population)
A blood sample was collected just prior to administration of the next dose of Deferasirox (pre-dose trough level) or approximately 24 hours after the previous dose at weeks 4, 8, 12, 16, 20 and 24. The mean trough concentration at each time point was calculated.
Measured: 4, 8, 12, 16, 20, and 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Nov 2006 (estimated)
- Study startAug 2006
- Primary completionDec 2007
- Study completionMar 2009
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 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 49 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Novartis Pharmaceuticals, M.D., Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00395629
- Sponsor's protocol ID
- CICL670A2202
- Other IDs
- EudraCT no. 2006-002102-57
- Status last verified
- May 2011
- First submitted
- 1 Nov 2006
- Last updated
- 30 May 2011
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 May 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Open Label, Dose Escalation Trial and a Six Month Extension to Explore the Safety and Efficacy of ICL670 in Patients With Iron Overload Resulting From Hereditary Hemochromatosis.
- ClinicalTrials.gov ID
- NCT00395629
- Acronym / sponsor ID
- CICL670A2202
- Conditions
- Iron Overload, Hereditary Hemochromatosis
- Keywords
- Deferasirox, ICL670A, Iron chelators, Deferiprone, Transfusional Hemochromatosis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 49 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Aug 2006
- Primary completion
- Dec 2007 (actual)
- Study completion
- Mar 2009 (actual)
- First posted
- 3 Nov 2006 (estimated)
- Results
- Posted 18 May 2011
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Brief Summary: This study was designed to explore a safe dose and characterize the preliminary safety and efficacy of ICL670 in adult patients with previously documented history of homozygous C282Y.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ICL670 (Deferasirox) | Experimental | Drug: Deferasirox (ICL670)Three dose cohorts: 5 mg/kg/day, 10 mg/kg/day, 15 mg/kg/day |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Age 18 years of age or older
- Male or female patients homozygous for the C282Y mutation.
- Iron overload as documented by serum ferritin and transferrin saturation
- No known allergy or contraindication to the administration of deferasirox
- Ability to comply with all study-related procedures, medications, and evaluations
Key exclusion criteria (11 listed)
- Iron overload not due to hereditary hemochromatosis
- Males with hemoglobin <13 mg/dL, females with hemoglobin <12 mg/dL
- Desferal treatment within 1 month of the screening visit
- Patients currently or previously treated with deferiprone or deferasirox
- Significant medical condition interfering with the ability to partake in this study
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 (ICL670) | Drug | ICL670 (Deferasirox) |
Primary outcome
Absolute Change of Serum Ferritin From Baseline to the End of Extension, by Dose Cohort (Extension Per-protocol Population)
Time frame: 0 to 48 weeks
Mean absolute change in serum ferritin from baseline to the end of the extension study.
1 secondary outcome
Trough Concentrations of Deferasirox (ICL670), by Dose Cohort (Per-protocol Population)
Time frame: 4, 8, 12, 16, 20, and 24 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 May 2011.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Related InfoLink 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.
Novartis Pharmaceuticals, M.D. study director
Novartis Pharmaceuticals
18 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Deferasirox (ICL670) in people with Iron Overload and Hereditary Hemochromatosis, run by Novartis Pharmaceuticals at 18 sites in 6 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 6 inclusion and 11 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 30 May 2011. 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 United States, Germany, Italy, Australia, Canada and 1 more country. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 18 May 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?
Absolute Change of Serum Ferritin From Baseline to the End of Extension, by Dose Cohort (Extension Per-protocol Population) (time frame: 0 to 48 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 30 May 2011.