Anemia, Sickle CellPhase 3NCT00981370
Clinical Importance of Treating Iron Overload in Sickle Cell Disease
A Phase 3 study testing deferasirox in people with Anemia, Sickle Cell and Transfusion Hemosiderosis, run by Children's Hospital Los Angeles at 1 site in United States.
Trial summary
Hypothesis: The investigators suspect that significant degrees of iron overload in subjects with SCD result in decreased red cell survival, abnormal endothelial function and markedly dysregulated autonomic function. Read more about this study
- Who is it for?
- Ages 14 years and older
- Conditions the study is for
- Anemia, Sickle Cell, Transfusion Hemosiderosis
- What is being tested?
- deferasirox
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2009to Dec 2010
- Phase
- Phase 3
- 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 14 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or female patients with sickle cell anemia (SS or SB thalassemia) with transfusional iron overload.
- Currently not on chronic or frequent transfusion
- Age equal or greater then 14 years
- Patients with iron overload from repeated blood transfusion, as defined by one of the following:
- For patients greater then 16 years old receiving simple transfusions: estimated lifetime history of receipt of at least 100 ml/kg or 15 adult units of packed red blood cells, OR
- For patients equal to or less then 16 years old receiving simple transfusions: estimated lifetime history of receipt of at least 100 ml/kg of packed red blood cells, OR
Show all 12Show fewer
- For any patient: liver iron content equal/greater then 3 mg Fe/g dw as measured by biopsy or magnetic resonance imaging who have not been adequately chelated since that measurement, OR
- a serum ferritin equal/greater then 1000 ng/mL on at least two occasions, at least two weeks apart, during the prior year. Samples must be obtained in the absence of concomitant infection
- Life expectancy equal/greater then 12 months
- Sexually active women must use an effective method of contraception, or must have undergone clinically documented total hysterectomy and/or oophorectomy, or tubal ligation or be postmenopausal (defined as amenorrhea for at least 12 months)
- Meets all inclusion criteria for screening
- LIC by MRI greater than or equal to 8 mg/g.
Who cannot join
- Blood transfusion within 12 weeks of the day 0 hemolysis labs
- Currently requires blood transfusion more than three times a year.
- Contraindication to MRI, including cardiac pacemaker, brain aneurysm clip, implanted neurostimulator, insulin pump, cochlear implant, metal slivers in the eyes, intrauterine device or any other MRI incompatible metal implants or intractable claustrophobia.
- Serum creatinine above the upper limit of normal
- Concomitant treatment with erythropoietin or its analogs.
- AST or ALT greater then 250 U/L during screening (patients may be re-screened at a later date if the cause of the elevation is known to be due to a transient process).
Show all 17Show fewer
- Patients receiving currently on chelation will be asked to stop for one week before starting or restarting Exjade. (a equal/greater then 1 week washout period prior to first dose of study drug is required)
- History of HIV positive test result (ELISA or Western blot)
- History of drug or alcohol abuse within the 12 months prior to enrollment
- Patients with uncontrolled systemic hypertension
- Severe cardiac insufficiency (NYHA III or IV), with uncontrolled and/or unstable cardiac or coronary artery disease not controlled by standard medical therapy
- Patients with a diagnosis of or history of clinically relevant ocular toxicity related to iron chelation
- Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent study treatment
- Pregnancy (as documented in required screening laboratory test) or breast feeding
- Patients who received treatment with systemic investigational drug within the past 4 weeks or topical investigational drug within the past 7 days or are planning to receive other investigational drugs while participating in the study
- Other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of study drug
- History of non-compliance to medical regimens or patients who are considered potentially unreliable and/or not cooperative
Trial locations and contacts
1 site in 1 country.
United States1 site
Childrens Hospital Los Angeles
Los Angeles, California, 90027
About this study
Hypothesis:
The investigators suspect that significant degrees of iron overload in subjects with SCD result in decreased red cell survival, abnormal endothelial function and markedly dysregulated autonomic function. Furthermore, the investigators anticipate that the magnitude of these effects is proportional not only to the magnitude of total body iron stores but also to the duration of exposure to the high iron levels in tissues.
Read more
Primary objective To determine if red cell survival as assessed by 51Cr red cell survival analysis, hemoglobin level, reticulocyte count, lactic acid dehydrogenase, and plasma hemoglobin in sickle cell patients is related to the degree of iron overload.
Secondary objective(s)
- Determine if the magnitude of endothelial-dependant vasodilation is related to The degree of iron overload.
- Determine if the degree of change in cardiac beat to beat variability in response to hypoxic exposure or to cold exposure ("cold-face-test") is related the magnitude of iron overload.
The primary measure of iron overload will be MRI determination of liver iron concentration.
Read the detailed description
Patients with sickle cell anemia often require blood transfusion as part of the treatment for their disease. Since each teaspoon of packed red blood cells contains about 5 mg of iron and humans have no way to get rid of excess iron, the levels of iron in sickle cell patients increase rapidly with each transfusion. Too much iron is extremely dangerous and causes damage to blood vessels, red blood cells, liver, hormone producing glands and heart. It is very difficult to know what damage due to iron overload in sickle cell patients because the sickle cell disease itself causes organ damage to the same organs affected by iron.
The purpose of this project is to demonstrate that iron overload significantly increases the morbidity of sickle cell disease and that treatment of the iron overload with Exjade® prevents or attenuates iron-related morbidity. To accomplish this we will screen sickle cell patients with a history of many blood transfusions to see if they have high iron levels. Then we will treat the patients who have very high iron levels with a drug which will remove the iron. Only patients with a very high iron level will be eligible for the treatment. These patients will have been transfused many times before but cannot currently be on blood transfusions. Before we start the treatment we will test the level of anemia, how fast the red cells are being destroyed, how well their blood vessels work and how well their heart works. When the treatment is over, we will repeat these tests and see if there is an improvement.
To qualify for this study, you must carry the diagnosis of sickle cell anemia and you must have received 10 or more blood transfusions in your life. You also cannot currently be on a regular transfusion program where you are getting blood transfusions regularly planned more than three times a year.
- Conditions
- Anemia, Sickle Cell, Transfusion Hemosiderosis
- Being tested
- deferasirox
- Keywords
- Sickle Cell Disease, Sickle Cell Anemia, Iron Overload, Chelation Therapy, Iron overload from blood transfusions, Heart rate variability, Autonomic dysfunction
- Medical subject headings
- Anemia, Sickle Cell, Iron Overload, Primary Dysautonomias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
deferasiroxDrug
Also called Exjade
Treatment starting dose of 20mg/kg/day based on subjects baseline LIC (liver iron concentration) and gradually escalate the dose to a maximum of 35 mg/kg/day based upon toxicity. Duration is up to a max of 12 months.
| Study group | Role | What they receive |
|---|---|---|
| Single Arm study | Other | Single Arm study, all subjects to receive study medication, deferasirox (Exjade). Receives: Drug: deferasirox |
What is being measured
The primary outcome is the main question the study is designed to answer.
To Determine if Red Cell Survival as Assessed by Hemoglobin Level, Reticulocyte Count, Lactic Acid Dehydrogenase, and Plasma Hemoglobin in Sickle Cell Patients is Related to the Degree of Iron Overload
Measured: Baseline, 6 months and 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Sep 2009 (estimated)
- Study startApr 2009
- Primary completionOct 2010
- Study completionDec 2010
Why it stopped: 1 consented patient never started on study drug, lost to follow up
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 (9)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 Children's Hospital Los AngelesThe 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Los Angeles
- Sponsor type
- University, hospital or other
- Collaborators
- Novartis Pharmaceuticals
- Responsible party
- Principal investigator, Thomas Coates, Children's Hospital Los Angeles
- Investigators
- Thomas D. Coates, M.D., Principal investigator, Children's Hospital Los Angeles
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00981370
- Sponsor's protocol ID
- CICL670AUS30T
- Status last verified
- Dec 2016
- First submitted
- 21 Sep 2009
- Last updated
- 8 Feb 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No: no data entered, no subjects enrolled
Publications
Cappellini MD, Cohen A, Piga A, Bejaoui M, Perrotta S, Agaoglu L, Aydinok Y, Kattamis A, Kilinc Y, Porter J, Capra M, Galanello R, Fattoum S, Drelichman G, Magnano C, Verissimo M, Athanassiou-Metaxa M, Giardina P, Kourakli-Symeonidis A, Janka-Schaub G, Coates T, Vermylen C, Olivieri N, Thuret I, Opitz H, Ressayre-Djaffer C, Marks P, Alberti D. A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemia. Blood. 2006 May 1;107(9):3455-62. doi: 10.1182/blood-2005-08-3430. Epub 2005 Dec 13.
PubMed 16352812Vichinsky 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 17233848Natta C, Creque L, Navarro C. Compartmentalization of iron in sickle cell anemia--an autopsy study. Am J Clin Pathol. 1985 Jan;83(1):76-8. doi: 10.1093/ajcp/83.1.76.
PubMed 3966442Koduri PR. Iron in sickle cell disease: a review why less is better. Am J Hematol. 2003 May;73(1):59-63. doi: 10.1002/ajh.10313.
PubMed 12701123Castro O, Poillon WN, Finke H, Massac E. Improvement of sickle cell anemia by iron-limited erythropoiesis. Am J Hematol. 1994 Oct;47(2):74-81. doi: 10.1002/ajh.2830470203.
PubMed 7522396Romero Mestre JC, Hernandez A, Agramonte O, Hernandez P. Cardiovascular autonomic dysfunction in sickle cell anemia: a possible risk factor for sudden death? Clin Auton Res. 1997 Jun;7(3):121-5. doi: 10.1007/BF02308838.
PubMed 9232355Wood JC, Ghugre N. Magnetic resonance imaging assessment of excess iron in thalassemia, sickle cell disease and other iron overload diseases. Hemoglobin. 2008;32(1-2):85-96. doi: 10.1080/03630260701699912.
PubMed 18274986Cheung YF, Chan GC, Ha SY. Arterial stiffness and endothelial function in patients with beta-thalassemia major. Circulation. 2002 Nov 12;106(20):2561-6. doi: 10.1161/01.cir.0000037225.92759.a7.
PubMed 12427652Sangkatumvong S, Khoo MC, Coates TD. Abnormal cardiac autonomic control in sickle cell disease following transient hypoxia. Annu Int Conf IEEE Eng Med Biol Soc. 2008;2008:1996-9. doi: 10.1109/IEMBS.2008.4649581.
PubMed 19163084
Study at a glance
From the registry record, last updated by the sponsor on 8 Feb 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Importance of Treating Iron Overload in Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT00981370
- Acronym / sponsor ID
- CICL670AUS30T
- Conditions
- Anemia, Sickle Cell, Transfusion Hemosiderosis
- Keywords
- Sickle Cell Disease, Sickle Cell Anemia, Iron Overload, Chelation Therapy, Iron overload from blood transfusions, Heart rate variability, Autonomic dysfunction
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: 1 consented patient never started on study drug, lost to follow up
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Children's Hospital Los AngelesUniversity, hospital or other
- Collaborators
- Novartis Pharmaceuticals
- Study start
- Apr 2009
- Primary completion
- Oct 2010 (actual)
- Study completion
- Dec 2010 (actual)
- First posted
- 22 Sep 2009 (estimated)
- Results
- Posted 8 Feb 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Hypothesis:
The investigators suspect that significant degrees of iron overload in subjects with SCD result in decreased red cell survival, abnormal endothelial function and markedly dysregulated autonomic function. Furthermore, the investigators anticipate that the magnitude of these effects is proportional not only to the magnitude of total body iron stores but also to the duration of exposure to the high iron levels in tissues.
Primary objective To determine if red cell survival as assessed by 51Cr red cell survival analysis, hemoglobin level, reticulocyte count, lactic acid dehydrogenase, and plasma hemoglobin in sickle cell patients is related to the degree of iron overload.
Secondary objective(s)
- Determine if the magnitude of endothelial-dependant vasodilation is related to The degree of iron overload.
- Determine if the degree of change in cardiac beat to beat variability in response to hypoxic exposure or to cold exposure ("cold-face-test") is related the magnitude of iron overload.
The primary measure of iron overload will be MRI determination of liver iron concentration.
Detailed description
Patients with sickle cell anemia often require blood transfusion as part of the treatment for their disease. Since each teaspoon of packed red blood cells contains about 5 mg of iron and humans have no way to get rid of excess iron, the levels of iron in sickle cell patients increase rapidly with each transfusion. Too much iron is extremely dangerous and causes damage to blood vessels, red blood cells, liver, hormone producing glands and heart. It is very difficult to know what damage due to iron overload in sickle cell patients because the sickle cell disease itself causes organ damage to the same organs affected by iron.
The purpose of this project is to demonstrate that iron overload significantly increases the morbidity of sickle cell disease and that treatment of the iron overload with Exjade® prevents or attenuates iron-related morbidity. To accomplish this we will screen sickle cell patients with a history of many blood transfusions to see if they have high iron levels. Then we will treat the patients who have very high iron levels with a drug which will remove the iron. Only patients with a very high iron level will be eligible for the treatment. These patients will have been transfused many times before but cannot currently be on blood transfusions. Before we start the treatment we will test the level of anemia, how fast the red cells are being destroyed, how well their blood vessels work and how well their heart works. When the treatment is over, we will repeat these tests and see if there is an improvement.
To qualify for this study, you must carry the diagnosis of sickle cell anemia and you must have received 10 or more blood transfusions in your life. You also cannot currently be on a regular transfusion program where you are getting blood transfusions regularly planned more than three times a year.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single Arm study | Other | Drug: deferasiroxSingle Arm study, all subjects to receive study medication, deferasirox (Exjade). |
Who may qualify
- Ages
- 14 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (12 listed)
- Male or female patients with sickle cell anemia (SS or SB thalassemia) with transfusional iron overload.
- Currently not on chronic or frequent transfusion
- Age equal or greater then 14 years
- Patients with iron overload from repeated blood transfusion, as defined by one of the following:
- Life expectancy equal/greater then 12 months
Key exclusion criteria (17 listed)
- Blood transfusion within 12 weeks of the day 0 hemolysis labs
- Currently requires blood transfusion more than three times a year.
- Contraindication to MRI, including cardiac pacemaker, brain aneurysm clip, implanted neurostimulator, insulin pump, cochlear implant, metal slivers in the eyes, intrauterine device or any other MRI incompatible metal implants or intractable claustrophobia.
- Serum creatinine above the upper limit of normal
- Concomitant treatment with erythropoietin or its analogs.
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 |
|---|---|---|
| deferasiroxAlso: ExjadeTreatment starting dose of 20mg/kg/day based on subjects baseline LIC (liver iron concentration) and gradually escalate the dose to a maximum of 35 mg/kg/day based upon toxicity. Duration is up to a max of 12 months. | Drug | Single Arm study |
Primary outcome
To Determine if Red Cell Survival as Assessed by Hemoglobin Level, Reticulocyte Count, Lactic Acid Dehydrogenase, and Plasma Hemoglobin in Sickle Cell Patients is Related to the Degree of Iron Overload
Time frame: Baseline, 6 months and 12 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Feb 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 16352812Cappellini MD, Cohen A, Piga A, Bejaoui M, Perrotta S, Agaoglu L, Aydinok Y, Kattamis A, Kilinc Y, Porter J, Capra M, Galanello R, Fattoum S, Drelichman G, Magnano C, Verissimo M, Athanassiou-Metaxa M, Giardina P, Kourakli-Symeonidis A, Janka-Schaub G, Coates T, Vermylen C, Olivieri N, Thuret I, Opitz H, Ressayre-Djaffer C, Marks P, Alberti D. A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemia. Blood. 2006 May 1;107(9):3455-62. doi: 10.1182/blood-2005-08-3430. Epub 2005 Dec 13.
- Background: 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.
- Background: PubMed 3966442Natta C, Creque L, Navarro C. Compartmentalization of iron in sickle cell anemia--an autopsy study. Am J Clin Pathol. 1985 Jan;83(1):76-8. doi: 10.1093/ajcp/83.1.76.
- Background: PubMed 12701123Koduri PR. Iron in sickle cell disease: a review why less is better. Am J Hematol. 2003 May;73(1):59-63. doi: 10.1002/ajh.10313.
- Background: PubMed 7522396Castro O, Poillon WN, Finke H, Massac E. Improvement of sickle cell anemia by iron-limited erythropoiesis. Am J Hematol. 1994 Oct;47(2):74-81. doi: 10.1002/ajh.2830470203.
- Background: PubMed 9232355Romero Mestre JC, Hernandez A, Agramonte O, Hernandez P. Cardiovascular autonomic dysfunction in sickle cell anemia: a possible risk factor for sudden death? Clin Auton Res. 1997 Jun;7(3):121-5. doi: 10.1007/BF02308838.
- Background: PubMed 18274986Wood JC, Ghugre N. Magnetic resonance imaging assessment of excess iron in thalassemia, sickle cell disease and other iron overload diseases. Hemoglobin. 2008;32(1-2):85-96. doi: 10.1080/03630260701699912.
- Background: PubMed 12427652Cheung YF, Chan GC, Ha SY. Arterial stiffness and endothelial function in patients with beta-thalassemia major. Circulation. 2002 Nov 12;106(20):2561-6. doi: 10.1161/01.cir.0000037225.92759.a7.
- Background: PubMed 19163084Sangkatumvong S, Khoo MC, Coates TD. Abnormal cardiac autonomic control in sickle cell disease following transient hypoxia. Annu Int Conf IEEE Eng Med Biol Soc. 2008;2008:1996-9. doi: 10.1109/IEMBS.2008.4649581.
- Individual participant dataSharing planned: No. no data entered, no subjects enrolled
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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.
Thomas D. Coates, M.D. principal investigator
Children's Hospital Los Angeles
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing deferasirox in people with Anemia, Sickle Cell and Transfusion Hemosiderosis, run by Children's Hospital Los Angeles 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 12 inclusion and 17 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: stopped early, as of the sponsor's last update on 8 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 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 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?
Yes. Results are posted in the registry (first posted 8 Feb 2017). 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?
To Determine if Red Cell Survival as Assessed by Hemoglobin Level, Reticulocyte Count, Lactic Acid Dehydrogenase, and Plasma Hemoglobin in Sickle Cell Patients is Related to the Degree of Iron Overload (time frame: Baseline, 6 months and 12 months). 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 NCT00981370, which also lists 9 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 8 Feb 2017.