Beta-ThalassemiaPhase 3NCT00061750
Safety & Efficacy of ICL670 vs. Deferoxamine in Beta-thalassemia Patients With Iron Overload Due to Blood Transfusions
A Phase 3 study testing ICL670 and deferoxamine in people with Beta-Thalassemia, run by Novartis Pharmaceuticals at 7 sites in United States.
Trial summary
The purpose of this study is to deterimine if the new orally active iron chelator, ICL670, is as effective and as safe as deferoxamine in preventing accumulation of iron in the body while a patient is undergoing repeated blood transfusions. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Beta-Thalassemia
- What is being tested?
- ICL670 and deferoxamine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 595 participants
- Where
- 7 sitesUnited States
- Study dates
- May 2003to Nov 2004
- Phase
- Phase 3
- 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
- Beta-thalassemia patients already treated with or suitable for treatment with deferoxamine 20 to 40 mg/kg/day
- Liver iron content greater than 2 mg iron/g dw as measured by liver biopsy
- Need for regular transfusions 8 or more times per year
Who cannot join
- Non-transfusional iron overload or transfusion-dependent anemias other than beta-thalassemia.
- Documented toxicity to deferoxamine
- Elevated liver enzymes in the year preceeding enrollment
- Active hepatitis B or hepatitis C
- HIV seropositivity
- Elevated serum creatinine or significant proteinuria
Show all 21Show fewer
- History of nephrotic syndrome
- Uncontrolled systemic hypertension
- Fever and other signs/symptoms of infection within 10 days prior to start of the study
- Presence of clinically relevant cataract or previous history of clinically relevant ocular toxicity related to iron chelation
- Second or third degree AV block, clinically relevant Q-T interval prolongation, or patients requiring digoxin or other drugs that prolong the Q-T interval
- Diseases (cardiovascular, renal, hepatic, etc.)that would prevent the patient from undergoing any of the treatment options
- Psychiatric or additive disorders that would prevent the patient from giving informed consent
- History of drug or alcohol abuse within the 12 months prior to the study
- Pregnant or breast feeding patients
- Patients treated with systemic investigational drugs within 4 weeks or topical investigational drugs within 7 days before the start of the study
- Any surgical or medical condition that might significantly alter the absorption, distribution, metabolism or excretion of any drug, such as gastrointestinal disease or major surgery, renal disease, difficulty voiding or urinary obstruction, or impaired pancreatic function.
- Non-compliant or unreliable patients.
- Patients unable to undergo any study procedures such as the hearing or eye tests, or the liver echocardiography.
- Inability to undergo a liver biopsy.
- Patients that would need a dose of ICL670 less than 125 mg per day.
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
Children's Hospital Boston
Boston, Massachusetts, 02115
Children's Memorial Hospital
Chicago, Illinois, 60614-3394
Children's Hospital of Los Angeles
Los Angeles, California, 90027-6062
Weill Medical College of Cornell University
New York, New York, 10021
Children's Hospital Oakland
Oakland, California, 94609
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104-4318
Stanford Hospital
Stanford, California, 94305-5208
About this study
Official title: A Randomized, Comparative, Open Label Phase III Trial on Efficacy & Safety of Long-term Treatment With ICL670 Compared to Deferoxamine in Beta-thalassemia Patients With Transfusional Hemosiderosis
The purpose of this study is to deterimine if the new orally active iron chelator, ICL670, is as effective and as safe as deferoxamine in preventing accumulation of iron in the body while a patient is undergoing repeated blood transfusions.
Read the detailed description
Patients who require repeated blood transfusions to live accumulate iron in the body as blood cells contain iron and there is no natural body mechanism to eliminate it. After a while the iron levels get high enough to be toxic to the body. The current therapy of choice is deferoxamine, which does a good job of removing excess iron, but is difficult to administer. Deferoxamine requires subcutaneous (under the skin) infusions over 4 to 8 hours nightly 3 to 7 nights per week. In addition to the need to wear an infusion pump nightly, adverse reactions around the site of the injection are frequent.
- Conditions
- Beta-Thalassemia
- Being tested
- ICL670, deferoxamine
- Keywords
- Thalassemia, iron overload, deferoxamine, hemosiderosis
- Medical subject headings
- beta-Thalassemia, Thalassemia, Iron Overload, Hemosiderosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ICL670Drug
Also called Deferasirox
deferoxamineDrug
| Study group | Role | What they receive |
|---|---|---|
| ICL670 | Experimental | Not described Receives: Drug: ICL670 |
| Deferoxamine | Active comparator | Not described Receives: Drug: deferoxamine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Demonstrate non-inferiority to deferoxamine in its effects on liver iron content (LIC)
Secondary outcomes (4)
Evaluate tolerability profile
Estimate absolute and relative change of LIC and Total body iron excretion
Evaluation relationship between LIC and potential surrogate markers
Evaluate the relationship between pharmacokinetics, pharmacodynamics and safety variable
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jun 2003 (estimated)
- Study startMay 2003
- Primary completionNov 2004
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Beta-ThalassemiaStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 595 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
- NCT00061750
- Sponsor's protocol ID
- CICL670A0107
- Status last verified
- Apr 2012
- First submitted
- 3 Jun 2003
- Last updated
- 19 Apr 2012
- Results posted
- No
Publications
Padhani ZA, Gangwani MK, Sadaf A, Hasan B, Colan S, Alvi N, Das JK. Calcium channel blockers for preventing cardiomyopathy due to iron overload in people with transfusion-dependent beta thalassaemia. Cochrane Database Syst Rev. 2023 Nov 17;11(11):CD011626. doi: 10.1002/14651858.CD011626.pub3.
PubMed 37975597Cohen AR, Glimm E, Porter JB. Effect of transfusional iron intake on response to chelation therapy in beta-thalassemia major. Blood. 2008 Jan 15;111(2):583-7. doi: 10.1182/blood-2007-08-109306. Epub 2007 Oct 19.
PubMed 17951527
Study at a glance
From the registry record, last updated by the sponsor on 19 Apr 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Comparative, Open Label Phase III Trial on Efficacy & Safety of Long-term Treatment With ICL670 Compared to Deferoxamine in Beta-thalassemia Patients With Transfusional Hemosiderosis
- ClinicalTrials.gov ID
- NCT00061750
- Acronym / sponsor ID
- CICL670A0107
- Conditions
- Beta-Thalassemia
- Keywords
- Thalassemia, iron overload, deferoxamine, hemosiderosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 595 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- May 2003
- Primary completion
- Nov 2004 (actual)
- First posted
- 4 Jun 2003 (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 study is to deterimine if the new orally active iron chelator, ICL670, is as effective and as safe as deferoxamine in preventing accumulation of iron in the body while a patient is undergoing repeated blood transfusions.
Detailed description
Patients who require repeated blood transfusions to live accumulate iron in the body as blood cells contain iron and there is no natural body mechanism to eliminate it. After a while the iron levels get high enough to be toxic to the body. The current therapy of choice is deferoxamine, which does a good job of removing excess iron, but is difficult to administer. Deferoxamine requires subcutaneous (under the skin) infusions over 4 to 8 hours nightly 3 to 7 nights per week. In addition to the need to wear an infusion pump nightly, adverse reactions around the site of the injection are frequent.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ICL670 | Experimental | Drug: ICL670 |
| Deferoxamine | Active comparator | Drug: deferoxamine |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Beta-thalassemia patients already treated with or suitable for treatment with deferoxamine 20 to 40 mg/kg/day
- Liver iron content greater than 2 mg iron/g dw as measured by liver biopsy
- Need for regular transfusions 8 or more times per year
Key exclusion criteria (21 listed)
- Non-transfusional iron overload or transfusion-dependent anemias other than beta-thalassemia.
- Documented toxicity to deferoxamine
- Elevated liver enzymes in the year preceeding enrollment
- Active hepatitis B or hepatitis C
- HIV seropositivity
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 |
|---|---|---|
| ICL670Also: Deferasirox | Drug | ICL670 |
| deferoxamine | Drug | Deferoxamine |
Primary outcome
Demonstrate non-inferiority to deferoxamine in its effects on liver iron content (LIC)
4 secondary outcomes
Evaluate tolerability profile
Estimate absolute and relative change of LIC and Total body iron excretion
Evaluation relationship between LIC and potential surrogate markers
Evaluate the relationship between pharmacokinetics, pharmacodynamics and safety variable
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 19 Apr 2012.
- Derived from this study: PubMed 37975597Padhani ZA, Gangwani MK, Sadaf A, Hasan B, Colan S, Alvi N, Das JK. Calcium channel blockers for preventing cardiomyopathy due to iron overload in people with transfusion-dependent beta thalassaemia. Cochrane Database Syst Rev. 2023 Nov 17;11(11):CD011626. doi: 10.1002/14651858.CD011626.pub3.
- Derived from this study: PubMed 17951527Cohen AR, Glimm E, Porter JB. Effect of transfusional iron intake on response to chelation therapy in beta-thalassemia major. Blood. 2008 Jan 15;111(2):583-7. doi: 10.1182/blood-2007-08-109306. Epub 2007 Oct 19.
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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 study director
Novartis Pharmaceuticals
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing ICL670 and deferoxamine in people with Beta-Thalassemia, run by Novartis Pharmaceuticals at 7 sites 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 21 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 19 Apr 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Demonstrate non-inferiority to deferoxamine in its effects on liver iron content (LIC). 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 NCT00061750, which also lists 2 linked publications.
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 19 Apr 2012.