Anemia, Sickle CellPhase 2NCT00067080
Safety of ICL670 vs. Deferoxamine in Sickle Cell Disease Patients With Iron Overload Due to Blood Transfusions
A Phase 2 study testing ICL670, deferoxamine in people with Anemia, Sickle Cell, run by Novartis Pharmaceuticals at 34 sites in United States.
Trial summary
The purpose of this study is to determine if the new orally active iron chelator, ICL670, is 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
- Anemia, Sickle Cell
- What is being tested?
- ICL670, deferoxamine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 195 participants
- Where
- 34 sitesUnited States
- Study dates
- May 2003to Jul 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
- Age greater than or equal to 2 years
- Sickle cell disease patients already treated with or suitable for treatment with deferoxamine 20 to 40 mg/kg/day
- Serum ferritin greater than 1000 mg/ml
- Liver iron content greater than 2 mg iron/g dw assessed by means of superconducting quantum interference device (SQUID) for patients who receive simple transfusions and greater than 5 mg iron/ g dw for patients who receive exchange transfusions or who have a history of intermittent blood transfusion.
- Regular transfusion aimed at maintaining % Hb A above 50% or a previous history of simple transfusion being the recipient of at least 20 units of packed red blood cells.
Who cannot join
- Chronic anemias other than sickle cell disease
- 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 the 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 (other than beta-adrenergic receptor blocking agents).
- Diseases (cardiovascular, renal, hepatic, etc.) that would prevent the patient from undergoing any of the treatment options
- Psychiatric or addictive 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
- Patients who require concomitant therapy with hydroxyurea
- 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
- Patients unable to undergo SQUID examination
Trial locations and contacts
34 sites in 1 country.
34 sites in 1 country
United States34 sites
Georgia Comprehensive Sickle cell Center, Grady Hospital
Atlanta, Georgia, 30335
Adult Sickle Cell Clinic, Medical College of Georgia
Augusta, Georgia, 30912
Children's Hospital Boston, Division of Hematology/Oncology
Boston, Massachusetts, 02115
Boston Medical Center
Boston, Massachusetts, 02118
NY Methodist Hospital
Brooklyn, New York, 11215
University of Illinois at Chicago
Chicago, Illinois, 60612
Children's Memorial Hospital
Chicago, Illinois, 60614
Barrett Center, University of Cincinnati
Cincinnati, Ohio, 45219
Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Liberty Hematology Oncology Center
Columbia, South Carolina, 29203
Palmetto Health Clinical Trials
Columbia, South Carolina, 29203
James Cancer Hospital
Columbus, Ohio, 43210
Colorado Sickle Cell Treatment and Research Center
Denver, Colorado, 80262
Karmanos Cancer Institute
Detroit, Michigan, 48201
Penn State Milton S Hershey Medical Center
Hershey, Pennsylvania, 17033
Baylor College of Medicine
Houston, Texas, 77030
Texas Children's Hospital/Baylor College of Medicine
Houston, Texas, 77030
Loma Linda University Medical Center
Loma Linda, California, 92354
Children's Hospital Los Angeles
Los Angeles, California, 90027
U. of S. Alabama Medical Center
Mobile, Alabama, 36604
Tulane University Sickle Cell Center
New Orleans, Louisiana, 70112
Children's Hospital, Department of Hematology/Oncology
New Orleans, Louisiana, 70118
Weill Medical College of Cornell University
New York, New York, 10021
Children's Hospital of the King's Daughter
Norfolk, Virginia, 23507
Children's Hospital & Research Center
Oakland, California, 94609
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15213
U. Of Rochester Medical Center
Rochester, New York, 14642
Santee Hematology/Oncology
Sumter, South Carolina, 29150
Tampa Children's Hospital at St Joseph's
Tampa, Florida, 33607
Scott and White Memorial Hospital & Clinics
Temple, Texas, 76508
Sickle Cell Center, Montefiore Hospital
The Bronx, New York, 10467
Howard University Hospital
Washington D.C., District of Columbia, 20059
Wake Forest University School of Medicine
Winston-Salem, North Carolina, 27106
About this study
Official title: A Randomized, Open Label, Phase II Study on Safety and Efficacy of Long Term Treatment of ICL670 Relative to Deferoxamine in Sickle Cell Disease Patients With Transfusional Hemosiderosis
The purpose of this study is to determine if the new orally active iron chelator, ICL670, is 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 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
- Anemia, Sickle Cell
- Being tested
- ICL670, deferoxamine
- Keywords
- Sickle cell disease, iron overload, deferoxamine, hemosiderosis
- Medical subject headings
- Anemia, Sickle Cell, Iron Overload, Hemosiderosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ICL670, deferoxamineDrug
Also called deferasirox
| Study group | Role | What they receive |
|---|---|---|
| ICL670 + deferoxamine | Experimental | Not described Receives: Drug: ICL670, deferoxamine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate the safety and tolerability of multiple doses of ICL670
Measured: 1 year
Secondary outcomes (4)
Estimate the absolute and relative change of liver iron content (LIC) and total body iron excretion (TBIE)
Measured: at baseline, after 24 weeks and at 1year (end of study)
Evaluate the pharmacokinetics
Measured: 24 hours post-dose @ 4, 12, 24 and 52 weeks
Evaluate the relationship between pharmacokinetics, pharmacodynamics and safety variables
Measured: at 24 and 52 weks pre-dose
Evaluate the relationship between hepatic iron and potential surrogate markers
Measured: at screen, at washout, then every 2 weeks for the first 12 weeks followed by every 4 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Aug 2003 (estimated)
- Study startMay 2003
- Primary completionJul 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Anemia, Sickle CellStudies 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 195 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00067080
- Sponsor's protocol ID
- CICL670A0109
- Status last verified
- Aug 2017
- First submitted
- 11 Aug 2003
- Last updated
- 22 Aug 2017
- Results posted
- No
Publications
Vichinsky E, Onyekwere O, Porter J, Swerdlow P, Eckman J, Lane P, Files B, Hassell K, Kelly P, Wilson F, Bernaudin F, Forni GL, Okpala I, Ressayre-Djaffer C, Alberti D, Holland J, Marks P, Fung E, Fischer R, Mueller BU, Coates T; Deferasirox in Sickle Cell Investigators. A randomised comparison of deferasirox versus deferoxamine for the treatment of transfusional iron overload in sickle cell disease. Br J Haematol. 2007 Feb;136(3):501-8. doi: 10.1111/j.1365-2141.2006.06455.x.
PubMed 17233848
Study at a glance
From the registry record, last updated by the sponsor on 22 Aug 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Open Label, Phase II Study on Safety and Efficacy of Long Term Treatment of ICL670 Relative to Deferoxamine in Sickle Cell Disease Patients With Transfusional Hemosiderosis
- ClinicalTrials.gov ID
- NCT00067080
- Acronym / sponsor ID
- CICL670A0109
- Conditions
- Anemia, Sickle Cell
- Keywords
- Sickle cell disease, iron overload, deferoxamine, hemosiderosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 195 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- May 2003
- Primary completion
- Jul 2007 (actual)
- First posted
- 13 Aug 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 determine if the new orally active iron chelator, ICL670, is 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 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
- 1
| Arm | Type | Receives |
|---|---|---|
| ICL670 + deferoxamine | Experimental | Drug: ICL670, deferoxamine |
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)
- Age greater than or equal to 2 years
- Sickle cell disease patients already treated with or suitable for treatment with deferoxamine 20 to 40 mg/kg/day
- Serum ferritin greater than 1000 mg/ml
- Liver iron content greater than 2 mg iron/g dw assessed by means of superconducting quantum interference device (SQUID) for patients who receive simple transfusions and greater than 5 mg iron/ g dw for patients who receive exchange transfusions or who have a history of intermittent blood transfusion.
- Regular transfusion aimed at maintaining % Hb A above 50% or a previous history of simple transfusion being the recipient of at least 20 units of packed red blood cells.
Key exclusion criteria (21 listed)
- Chronic anemias other than sickle cell disease
- 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 |
|---|---|---|
| ICL670, deferoxamineAlso: deferasirox | Drug | ICL670 + deferoxamine |
Primary outcome
Evaluate the safety and tolerability of multiple doses of ICL670
Time frame: 1 year
4 secondary outcomes
Estimate the absolute and relative change of liver iron content (LIC) and total body iron excretion (TBIE)
Time frame: at baseline, after 24 weeks and at 1year (end of study)
Evaluate the pharmacokinetics
Time frame: 24 hours post-dose @ 4, 12, 24 and 52 weeks
Evaluate the relationship between pharmacokinetics, pharmacodynamics and safety variables
Time frame: at 24 and 52 weks pre-dose
Evaluate the relationship between hepatic iron and potential surrogate markers
Time frame: at screen, at washout, then every 2 weeks for the first 12 weeks followed by every 4 weeks
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 22 Aug 2017.
- Results publication: PubMed 17233848Vichinsky E, Onyekwere O, Porter J, Swerdlow P, Eckman J, Lane P, Files B, Hassell K, Kelly P, Wilson F, Bernaudin F, Forni GL, Okpala I, Ressayre-Djaffer C, Alberti D, Holland J, Marks P, Fung E, Fischer R, Mueller BU, Coates T; Deferasirox in Sickle Cell Investigators. A randomised comparison of deferasirox versus deferoxamine for the treatment of transfusional iron overload in sickle cell disease. Br J Haematol. 2007 Feb;136(3):501-8. doi: 10.1111/j.1365-2141.2006.06455.x.
- Results for CICL670A0109 from the Novartis Clinical Trials websiteLink 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 study director
Novartis Pharmaceuticals
34 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ICL670, deferoxamine in people with Anemia, Sickle Cell, run by Novartis Pharmaceuticals at 34 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 5 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 22 Aug 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 34 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 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?
Evaluate the safety and tolerability of multiple doses of ICL670 (time frame: 1 year). 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 NCT00067080, 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 22 Aug 2017.