Chronic AnemiaPhase 2NCT00631163
Efficacy and Safety of Deferasirox in Patients With Chronic Anemia and Transfusional Hemosiderosis
A Phase 2 study testing Deferasirox (ICL670) in people with Chronic Anemia and Transfusional Hemosiderosis, run by Novartis Pharmaceuticals at 31 sites in 5 countries.
Trial summary
The overall purpose of this trial is to further evaluate the efficacy and safety of deferasirox, dosed initially according to the transfusional iron intake, in patients with transfusion dependant anemia related to disorders other than β-thalassemia and sickle cell disease. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Chronic Anemia, Transfusional Hemosiderosis
- What is being tested?
- Deferasirox (ICL670)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 102 participants
- Where
- 31 sitesin 5 countries
- Study dates
- Oct 2007to Feb 2012
- Phase
- Phase 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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients with transfusional iron overload due to:
- low or intermediate (INT-1) risk Myelodysplastic Syndrome (MDS)determined via International Prognosis Scoring System (IPSS) criteria
- other congenital or acquired anemias excluding B-thalassemia and sickle cell disease
- Lifetime transfusion history of ≥20 unit (approximately 100 mL/kg) of packed red blood cells or showing evidence of iron overload (serum ferritin >1000 µg/L).
- Able to provide written informed consent
- Life expectancy ≥ 12 months If patient was previously treated with deferiprone, a washout period of one month should occur before the first dose of deferasirox
- Patients completing the planned 12-month core study (CICL670A2204).
- Written informed consent obtained from the patient and/or legal guardian on the patient's behalf in accordance with national legislation.
Who cannot join
- Patients with β-thalassemia, sickle cell disease or myelodysplastic syndrome with an IPSS score being Intermediate-2 or High.
- Patients with serum creatinine > ULN
- Patients with ALT(SGPT) levels > 5 x ULN
- Significant proteinuria as indicated by a urinary protein/creatinine ratio >0.5 mg/mg in a non-first void urine sample on two assessments during the screening period.
- History of HIV positive test result , or of clinical or laboratory evidence of active Hepatitis B or Hepatitis C (HBsAg in the absence of HBsAb OR HCV Ab positive with HCV RNA positive and ALT above the normal range)
- Patients on investigational MDS therapies, including lenalidomide, thalidomide, azacitidine and arsenic trioxide, must have a ≥ 4 week washout period prior to the first dose of study drug.
Show all 16Show fewer
- Patients with systemic uncontrolled hypertension
- Patients with unstable cardiac disease not controlled by standard medical therapy
- Systemic disease (cardiovascular, renal, hepatic, etc.) which would prevent study treatment
- Pregnancy (as documented in required screening laboratory test) or breast feeding.
- Patients treated with systemic investigational drug within the past 4 weeks or topical investigational drug within the past 7 days
- Other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of study drug
- Patients being considered by the investigator potentially unreliable and/or not cooperative with regard to the study protocol
- History of hypersensitivity to any of the study drug or excipients
- Sexually active pre-menopausal female patients without adequate contraception. Female patients must use effective contraception or must have undergone clinically documented total hysterectomy and/or oophorectomy, tubal ligation or be postmenopausal defined by amenorrhea for at least 12 months.
- Other protocol defined inclusion/exclusion criteria may apply.
Trial locations and contacts
31 sites in 5 countries.
31 sites in 5 countries
Japan22 sites
Novartis Investigative Site
Bunkyo-ku, Tokyo, 113-8655
Novartis Investigative Site
Cyuo-ku, Tokyo, 104-8560
Novartis Investigative Site
Fukuoka, Fukuoka, 814-0180
Novartis Investigative Site
Hiroshima, 734-8551
Novartis Investigative Site
Kahoku-gun, Ishikawa-ken, 920-0293
Novartis Investigative Site
Kanazawa, Ishikawa-ken, 920-8641
Novartis Investigative Site
Kumamoto, 860-0811
Novartis Investigative Site
Kyoto, 606-8507
Novartis Investigative Site
Minato-ku, Tokyo, 108-8639
Novartis Investigative Site
Nagasaki, 852-8501
Novartis Investigative Site
Nagoya, Aichi-ken, 453-8511
Novartis Investigative Site
Nagoya, Aichi-ken, 466-8560
Novartis Investigative Site
Nishinomiya, Hyōgo, 663-8501
Novartis Investigative Site
Sapporo, Hokkaido, 060-8543
Novartis Investigative Site
Sayama, Osaka, 589-8511
Novartis Investigative Site
Shimotsuka-gun, Tochigi, 321-0293
Novartis Investigative Site
Shinagawa-ku, Tokyo, 141-8625
Novartis Investigative Site
Shinjuku-ku, Tokyo, 160-0023
Novartis Investigative Site
Shinjuku-ku, Tokyo, 162-8666
Novartis Investigative Site
Simotsuke-city, Tochigi, 329-0498
Novartis Investigative Site
Suita, Osaka, 565-0871
Novartis Investigative Site
Toyama, 930-8550
Turkey (Türkiye)4 sites
Novartis Investigative Site
Adana, 01330
Novartis Investigative Site
Ankara, 06100
Novartis Investigative Site
Istanbul, 34093
Novartis Investigative Site
Izmir, 35040
Poland2 sites
Novartis Investigative Site
Warsaw, 02-097
Novartis Investigative Site
Warsaw, 02-776
Spain2 sites
Novartis Investigative Site
Salamanca, Castille and León, 37007
Novartis Investigative Site
Valencia, 46026
About this study
Official title: A Multi-Center, Open-label, Non Comparative, Phase II Trial on Efficacy and Safety of ICL670 Given for 1 Year With Dose Adjustments Based on Serum Ferritin in Patients With Chronic Anemia and Transfusional Hemosiderosis Including an Additional 1 Year Extension.
The overall purpose of this trial is to further evaluate the efficacy and safety of deferasirox, dosed initially according to the transfusional iron intake, in patients with transfusion dependant anemia related to disorders other than β-thalassemia and sickle cell disease.
During the study, the dose will be adjusted based on serum Ferritin.The overall purpose of the extension is to allow further treatment of patients who have already completed the core study, and to enable collection of long term efficacy and safety data. Patients will continue to receive Deferasirox at the dose they received at the end of the core study.
- Conditions
- Chronic Anemia, Transfusional Hemosiderosis
- Being tested
- Deferasirox (ICL670)
- Keywords
- Iron Overload,, Chelation,, MDS,, Chronic Anemia,, Deferasirox,, Transfusional hemosiderosis,
- Medical subject headings
- Iron Overload
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Deferasirox (ICL670)Drug
Also called ICL670
The recommended initial daily dose of Deferasirox is 20 mg/kg body weight.
| Study group | Role | What they receive |
|---|---|---|
| Deferasirox | Experimental | Participants received initial dose of 20 milligrams per kilogram (mg/kg) Deferasirox tablets was administered orally once daily (OD) based on the Participants body weight. The dose of Deferasirox was adjusted to either 10 mg/kg or 30 mg/kg based on the volumes of blood transfusions being administered. Receives: Drug: Deferasirox (ICL670) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Absolute Change From Baseline in Liver Iron Concentration (LIC) to Year 1
Liver iron concentration (LIC), a predictor of iron burden, was measured using relaxation rate magnetic resonance imaging (R2-MRI) technique. Relaxation rate was determined as R2 = 1/relaxation time (T2). The baseline value of LIC of participants was categorized as < 7, ≥ 7 to < 15, and ≥ 15 milligram of iron/tissue dry weight (mg Fe/g dw). A negative change from baseline favored study treatment in reducing LIC.
Measured: From the Baseline, Year 1 (End of core study)
Secondary outcomes (8)
Absolute Change From Baseline in Liver Iron Concentration (LIC) to End of Year 2
Liver Iron Concentration (LIC), a predictor of iron burden, was measured using R2-MRI technique. Relaxation rate was determined as R2 = 1/T2. The baseline value of LIC of participants was categorized as < 7, ≥ 7 to < 15, and ≥ 15 mg Fe/g dw. A negative change from baseline favoured study treatment in reducing LIC.
Measured: From the Baseline to End of Year 2 (End of extension study)
Absolute Change From Baseline in Liver Iron Concentration (LIC) in Japanese Subgroup
Liver Iron Concentration (LIC), a predictor of iron burden, was measured using R2-MRI technique. Relaxation rate was determined as R2 = 1/T2. The baseline value of LIC was < 7, ≥ 7 to < 15, and ≥ 15 mg Fe/g dw. A negative change from baseline favoured study treatment in reducing LIC.
Measured: From the Baseline, End of Year 1 (End of core study), End of Year 2 (End of extension study)
Absolute Change From Baseline in Serum Ferritin Levels to Year 2
Serum ferritin was a marker for the monitoring of chelation therapy. Ferritin protein stores iron and provides overall iron levels, higher ferritin in blood showed more iron content.
Measured: From the Baseline up to Year 2 (End of extension study)
Absolute Serum Ferritin Levels Over 2 Years
Serum ferritin was a marker for the monitoring of chelation therapy. Ferritin protein stores iron and provides overall iron levels, higher ferritin in blood showed more iron content.
Measured: From the Baseline, Year 1 (End of core study), Year 2 (End of extension study)
Total Body Iron Elimination Rate (TBIE), Iron Intake, Iron Excretion/Iron Intake and Chelation Efficiency After 2 Years
Total body iron excretion (TBIE)was used to investigate the chelation efficacy of Deferasirox therapy. TBIE rate was estimated based on the iron influx as determined by the amount of red cells transfused and the change in total body iron (TBI) stores.
Measured: From the Baseline, Year 2 (End of extension study)
Correlation of LIC and Serum Ferritin at Core and Extension Study
LIC, a predictor of iron burden, was measured using R2-MRI technique. Relaxation rate was determined as R2 = 1/T2. The baseline value of LIC was < 7, ≥ 7 to < 15, and ≥ 15 mg Fe/g dw. Serum ferritin was a marker for the monitoring of chelation therapy. Ferritin protein stores iron and provides overall iron levels, higher ferritin in blood showed more iron content. The correlation between absolute change in LIC and absolute change in serum ferritin was determined.
Measured: From the Baseline, Year 1 (End of core study), Year 2 (End of extension study)
Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Adverse Event of Special Interest (AESI), Discontinuation and Interruption
Adverse events (AEs) were defined as any unfavorable and unintended diagnosis, symptom, sign (including an abnormal laboratory finding), syndrome or disease which either occurs during study, having been absent at baseline, or, if present at baseline, appears to worsen. Serious adverse events (SAEs) were defined as any untoward medical occurrences that result in death, are life threatening, require hospitalisation, cause persistent or significant disability/incapacity, result in congenital anomalies or birth defects, or are other conditions which in judgement of investigators represent significant hazards. Death was defined as a fatal event leading to permanent cessation of all vital functions of the body.
Measured: From the Baseline up to Year 2 (End of extension study)
Number of Participants With Clinically Significant Ophthalmological Abnormalities
Clinically significant changes in left eye and right eye were assessed by the investigator based on methods like visual acuity, slit lamp examination, tonometry and fundus oculi.
Measured: At 2 years (End of extension study)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Mar 2008 (estimated)
- Study startOct 2007
- Primary completionJan 2011
- Study completionFeb 2012
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 (1)Papers the sponsor has linked to this study.
- Other trials for Chronic AnemiaStudies 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
- 102 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis PharmaceuticalsNovartis Pharmaceuticals, Study director, Novartis Pharmeceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00631163
- Sponsor's protocol ID
- CICL670A2204
- Other IDs
- 2006-003337-32
- Status last verified
- Jun 2021
- First submitted
- 27 Feb 2008
- Last updated
- 29 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Kohgo Y, Urabe A, Kilinc Y, Agaoglu L, Warzocha K, Miyamura K, Lim LC, Glaser S, Wang C, Wiktor-Jedrzejczak W. Deferasirox Decreases Liver Iron Concentration in Iron-Overloaded Patients with Myelodysplastic Syndromes, Aplastic Anemia and Other Rare Anemias. Acta Haematol. 2015;134(4):233-42. doi: 10.1159/000381893.
PubMed 26138856
Study at a glance
From the registry record, last updated by the sponsor on 29 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-Center, Open-label, Non Comparative, Phase II Trial on Efficacy and Safety of ICL670 Given for 1 Year With Dose Adjustments Based on Serum Ferritin in Patients With Chronic Anemia and Transfusional Hemosiderosis Including an Additional 1 Year Extension.
- ClinicalTrials.gov ID
- NCT00631163
- Acronym / sponsor ID
- CICL670A2204
- Conditions
- Chronic Anemia, Transfusional Hemosiderosis
- Keywords
- Iron Overload,, Chelation,, MDS,, Chronic Anemia,, Deferasirox,, Transfusional hemosiderosis,
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 102 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Oct 2007
- Primary completion
- Jan 2011 (actual)
- Study completion
- Feb 2012 (actual)
- First posted
- 7 Mar 2008 (estimated)
- Results
- Posted 29 Jun 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The overall purpose of this trial is to further evaluate the efficacy and safety of deferasirox, dosed initially according to the transfusional iron intake, in patients with transfusion dependant anemia related to disorders other than β-thalassemia and sickle cell disease.
During the study, the dose will be adjusted based on serum Ferritin.The overall purpose of the extension is to allow further treatment of patients who have already completed the core study, and to enable collection of long term efficacy and safety data. Patients will continue to receive Deferasirox at the dose they received at the end of the core study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Deferasirox | Experimental | Drug: Deferasirox (ICL670)Participants received initial dose of 20 milligrams per kilogram (mg/kg) Deferasirox tablets was administered orally once daily (OD) based on the Participants body weight. The dose of Deferasirox was adjusted to either 10 mg/kg or 30 mg/kg based on the volumes of blood transfusions being administered. |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- Patients with transfusional iron overload due to:
- low or intermediate (INT-1) risk Myelodysplastic Syndrome (MDS)determined via International Prognosis Scoring System (IPSS) criteria
- other congenital or acquired anemias excluding B-thalassemia and sickle cell disease
- Lifetime transfusion history of ≥20 unit (approximately 100 mL/kg) of packed red blood cells or showing evidence of iron overload (serum ferritin >1000 µg/L).
- Able to provide written informed consent
Key exclusion criteria (16 listed)
- Patients with β-thalassemia, sickle cell disease or myelodysplastic syndrome with an IPSS score being Intermediate-2 or High.
- Patients with serum creatinine > ULN
- Patients with ALT(SGPT) levels > 5 x ULN
- Significant proteinuria as indicated by a urinary protein/creatinine ratio >0.5 mg/mg in a non-first void urine sample on two assessments during the screening period.
- History of HIV positive test result , or of clinical or laboratory evidence of active Hepatitis B or Hepatitis C (HBsAg in the absence of HBsAb OR HCV Ab positive with HCV RNA positive and ALT above the normal range)
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)Also: ICL670The recommended initial daily dose of Deferasirox is 20 mg/kg body weight. | Drug | Deferasirox |
Primary outcome
Absolute Change From Baseline in Liver Iron Concentration (LIC) to Year 1
Time frame: From the Baseline, Year 1 (End of core study)
Liver iron concentration (LIC), a predictor of iron burden, was measured using relaxation rate magnetic resonance imaging (R2-MRI) technique. Relaxation rate was determined as R2 = 1/relaxation time (T2). The baseline value of LIC of participants was categorized as < 7, ≥ 7 to < 15, and ≥ 15 milligram of iron/tissue dry weight (mg Fe/g dw). A negative change from baseline favored study treatment in reducing LIC.
8 secondary outcomes
Absolute Change From Baseline in Liver Iron Concentration (LIC) to End of Year 2
Time frame: From the Baseline to End of Year 2 (End of extension study)
Absolute Change From Baseline in Liver Iron Concentration (LIC) in Japanese Subgroup
Time frame: From the Baseline, End of Year 1 (End of core study), End of Year 2 (End of extension study)
Absolute Change From Baseline in Serum Ferritin Levels to Year 2
Time frame: From the Baseline up to Year 2 (End of extension study)
Absolute Serum Ferritin Levels Over 2 Years
Time frame: From the Baseline, Year 1 (End of core study), Year 2 (End of extension study)
Total Body Iron Elimination Rate (TBIE), Iron Intake, Iron Excretion/Iron Intake and Chelation Efficiency After 2 Years
Time frame: From the Baseline, Year 2 (End of extension study)
Correlation of LIC and Serum Ferritin at Core and Extension Study
Time frame: From the Baseline, Year 1 (End of core study), Year 2 (End of extension study)
Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Adverse Event of Special Interest (AESI), Discontinuation and Interruption
Time frame: From the Baseline up to Year 2 (End of extension study)
Number of Participants With Clinically Significant Ophthalmological Abnormalities
Time frame: At 2 years (End of extension study)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jun 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 26138856Kohgo Y, Urabe A, Kilinc Y, Agaoglu L, Warzocha K, Miyamura K, Lim LC, Glaser S, Wang C, Wiktor-Jedrzejczak W. Deferasirox Decreases Liver Iron Concentration in Iron-Overloaded Patients with Myelodysplastic Syndromes, Aplastic Anemia and Other Rare Anemias. Acta Haematol. 2015;134(4):233-42. doi: 10.1159/000381893.
- CICL670A2204 Results at Novartis Clinical Trials Results 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
Novartis Pharmaceuticals study director
Novartis Pharmeceuticals
31 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Deferasirox (ICL670) in people with Chronic Anemia and Transfusional Hemosiderosis, run by Novartis Pharmaceuticals at 31 sites in 5 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 8 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 29 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 31 sites in Japan, Turkey (Türkiye), Poland, Spain, Singapore. 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?
Yes. Results are posted in the registry (first posted 29 Jun 2021). 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 From Baseline in Liver Iron Concentration (LIC) to Year 1 (time frame: From the Baseline, Year 1 (End of core study)). 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 NCT00631163, 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 29 Jun 2021.