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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.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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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

Other trials in Japan

Turkey (Türkiye)4 sites

  • Novartis Investigative Site

    Adana, 01330

  • Novartis Investigative Site

    Ankara, 06100

  • Novartis Investigative Site

    Istanbul, 34093

  • Novartis Investigative Site

    Izmir, 35040

Other trials in Turkey (Türkiye)

Poland2 sites

  • Novartis Investigative Site

    Warsaw, 02-097

  • Novartis Investigative Site

    Warsaw, 02-776

Other trials in Poland

Spain2 sites

  • Novartis Investigative Site

    Salamanca, Castille and León, 37007

  • Novartis Investigative Site

    Valencia, 46026

Other trials in Spain

Singapore1 site

  • Novartis Investigative Site

    Singapore, 169608

Other trials in Singapore

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.

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 groups in NCT00631163
Study groupRoleWhat they receive
DeferasiroxExperimentalParticipants 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.

  1. 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)

  1. 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)

  2. 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)

  3. 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)

  4. 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)

  5. 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)

  6. 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)

  7. 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)

  8. 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.

  1. First posted7 Mar 2008 (estimated)
  2. Study startOct 2007
  3. Primary completionJan 2011
  4. Study completionFeb 2012

Resources

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)
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
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

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.

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