Efficacy and Safety of Deferasirox in Patients With Chronic Anemia and Transfusional Hemosiderosis

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT00631163
First received: February 27, 2008
Last updated: February 9, 2013
Last verified: February 2013
  Purpose

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.


Condition Intervention Phase
Chronic Anemia
Transfusional Hemosiderosis
Drug: Deferasirox (ICL670)
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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.

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • Change of Liver iron concentration measured by R2 MRI over a period of 1 year treatment. [ Time Frame: 12 months, 24 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Change of serum ferritin concentration over a period of 1 year treatment. [ Time Frame: 12 months, 24 months ] [ Designated as safety issue: No ]
  • Evaluation of safety and tolerability of deferasirox in Japanese patients. [ Time Frame: 12 months, 24 months ] [ Designated as safety issue: Yes ]
  • Evaluation of Iron balance after 1 year of treatment [ Time Frame: 12 months, 24 months ] [ Designated as safety issue: No ]
  • Evaluation of serum ferritin as a marker for accurate monitoring of chelation therapy [ Time Frame: 12 months, 24 months ] [ Designated as safety issue: No ]
  • Evaluation of ophthalmologic safety of deferasirox [ Time Frame: 12 months, 24 months ] [ Designated as safety issue: Yes ]

Enrollment: 114
Study Start Date: October 2007
Study Completion Date: February 2012
Primary Completion Date: January 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Deferasirox

The recommended initial daily dose of Deferasirox is 20 mg/kg body weight for most patients.

An initial daily dose of 30 mg/kg or 10mg/kg should be considered for patients requiring more intensive or less intensive chelation, respectively

Drug: Deferasirox (ICL670)
The recommended initial daily dose of Deferasirox is 20 mg/kg body weight.
Other Name: ICL670

  Eligibility

Ages Eligible for Study:   2 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion criteria (Core):

  • 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

Inclusion criteria (Extension):

  • 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.

Exclusion criteria (Core and Extension):

  • 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.
  • 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.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00631163

Locations
Japan
Novartis Investigative Site
Nagoya, Aichi, Japan, 453-8511
Novartis Investigative Site
Nagoya, Aichi, Japan, 466-8560
Novartis Investigative Site
Fukuoka-city, Fukuoka, Japan, 814-0180
Novartis Investigative Site
Sapporo-city, Hokkaido, Japan, 060-8543
Novartis Investigative Site
Nishinomiya, Hyogo, Japan, 663-8501
Novartis Investigative Site
Kahoku-gun, Ishikawa, Japan, 920-0293
Novartis Investigative Site
Kanazawa, Ishikawa, Japan, 920-8641
Novartis Investigative Site
OsakaSayama, Osaka, Japan, 589-8511
Novartis Investigative Site
Suita-city, Osaka, Japan, 565-0871
Novartis Investigative Site
Shimotsuka-gun, Tochigi, Japan, 321-0293
Novartis Investigative Site
Simotsuke-city, Tochigi, Japan, 329-0498
Novartis Investigative Site
Bunkyo-ku, Tokyo, Japan, 113-8655
Novartis Investigative Site
Cyuo-ku, Tokyo, Japan, 104-8560
Novartis Investigative Site
Minato-ku, Tokyo, Japan, 108-8639
Novartis Investigative Site
Shinagawa-ku, Tokyo, Japan, 141-8625
Novartis Investigative Site
Shinjuku-ku, Tokyo, Japan, 160-0023
Novartis Investigative Site
Shinjuku-ku, Tokyo, Japan, 162-8666
Novartis Investigative Site
Hiroshima, Japan, 734-8551
Novartis Investigative Site
Kumamoto, Japan, 860-0811
Novartis Investigative Site
Kyoto, Japan, 606-8507
Novartis Investigative Site
Nagasaki, Japan, 852-8501
Novartis Investigative Site
Toyama, Japan, 930-8550
Poland
Novartis Investigative Site
Warszawa, Poland, 02-097
Novartis Investigative Site
Warszawa, Poland, 02-776
Singapore
Novartis Investigative Site
Singapore, Singapore, 169608
Spain
Novartis Investigative Site
Salamanca, Castilla y Leon, Spain, 37007
Novartis Investigative Site
Valencia, Spain, 46026
Turkey
Novartis Investigative Site
Adana, Turkey, 01330
Novartis Investigative Site
Ankara, Turkey, 06100
Novartis Investigative Site
Istanbul, Turkey, 34093
Novartis Investigative Site
Izmir, Turkey, 35040
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
Study Director: Novartis Pharmaceuticals Novartis Pharmeceuticals
  More Information

No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT00631163     History of Changes
Obsolete Identifiers: NCT01199003
Other Study ID Numbers: CICL670A2204, 2006-003337-32
Study First Received: February 27, 2008
Last Updated: February 9, 2013
Health Authority: United States: Food and Drug Administration
Spain: Ministry of Health
Japan: Ministry of Health, Labor and Welfare
Poland: Ministry of Health
Singapore: Ministry of Health

Keywords provided by Novartis:
Iron Overload,
Chelation,
MDS,
Chronic Anemia,
Deferasirox,
Transfusional hemosiderosis,

Additional relevant MeSH terms:
Anemia
Hemosiderosis
Hematologic Diseases
Iron Overload
Iron Metabolism Disorders
Metabolic Diseases
Deferasirox
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on August 18, 2014