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Safety and Efficacy of Desferasirox in Chinese Patients With Iron Overload and Aplastic Anemia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01546415
Recruitment Status : Completed
First Posted : March 7, 2012
Last Update Posted : October 28, 2014
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Brief Summary:
The study is designed to collect safety and efficacy of Desferasirox in Chinese patients with Iron Overload and Aplastic Anemia.

Condition or disease Intervention/treatment Phase
Iron Overload Drug: Desferasirox Phase 4

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 64 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Single Arm, Multicenter, Open Label Study of Desferasirox in Chinese Patients With Iron Overload and Aplastic Anemia
Study Start Date : October 2011
Actual Primary Completion Date : November 2013
Actual Study Completion Date : November 2013

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: Desferasirox Drug: Desferasirox
Initial Dose: 20 mg/kg/d. Dose modification according to protocol.

Primary Outcome Measures :
  1. Change in serum ferritin from baseline to 52 weeks with desferasirox treatment in Chinese patients with AA presenting with transfusional hemosiderosis and pre-existing serum ferritin levels of ≥ 1000 ng/mL. [ Time Frame: Every 4 Weeks for 52 weeks of treatment ]
    Difference in serum ferritin from baseline vs. 52 weeks of treatment.

Secondary Outcome Measures :
  1. Correlation between serum ferritin (SF) and transferrin saturation (TFS) [ Time Frame: Every 4 Weeks for 52 weeks of treatment ]
    Study will compare SF with TFS level to find degree of relationship measured by correlation.

  2. Number of dispensed, used, partially used or unused packages of study medication to assess drug usage compliance [ Time Frame: Every 4 Weeks for 52 weeks of treatment ]
  3. Correlation between dose adjustment (increase or decrease) regimens and transfusional burden [ Time Frame: Every 3 months for 52 Weeks of treatment ]
    Evaluation of the relationship between dose adjustment regimens (as dictated by both efficacy and safety parameters) and transfusional burden will be undertaken.

  4. Number of patients with adverse events, serious adverse events and death [ Time Frame: Every 4 weeks for 52 Weeks of treatment ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Patients presenting with transfusion-related IOL as shown by a SF level of ≥ 1000 ng/mL at start of study
  • History of transfusion >20 international units or 100 mL/kg of red blood cells
  • Underlying transfusion-dependent illness:

    • AA: Clinically diagnosed AA with bone marrow confirmation of the diagnosis
  • Age ≥ 18 years, male or female
  • Written informed consent obtained from patient prior to any screening procedures.

Exclusion Criteria:

  • Patients with serum creatinine > ULN or with significant proteinuria as indicated by a urinary protein/creatinine ratio (UPCR) ≥ 1.0 mg/mg in a non-first void urine sample at baseline. If serum creatinine is found to be > ULN or UPCR is found to be ≥ 1 mg/mg the test can be repeated after 1 month.
  • Creatinine Clearance <40 ml/min
  • Patients with other than AA transfusion-dependent underlying illnesses
  • Patients with a previous history of clinically relevant ocular and/or auditory toxicity related to iron chelation
  • Any other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of any drug. The investigator should be guided by evidence of any of the following:
  • history of inflammatory bowel syndrome, gastritis, ulcers, gastrointestinal or rectal bleeding
  • history of major gastrointestinal tract surgery such as gastrectomy, gastroenterostomy, or bowel resection
  • history of pancreatic injury or pancreatitis; indications of impaired pancreatic function/injury as indicated by abnormal lipase or amylase
  • history or presence of impaired renal function as indicated by creatinine or blood urea nitrogen (BUN) values equal or above ULN
  • history of urinary obstruction or difficulty in voiding

Other protocol-defined inclusion/exclusion criteria may apply

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01546415

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China, Shanghai
Novartis Investigative Site
Shanghai, Shanghai, China, 200437
China, Sichuan
Novartis Investigative Site
Chengdu, Sichuan, China, 610041
China, Tianjin
Novartis Investigative Site
Tianjin, Tianjin, China, 300020
Novartis Investigative Site
Beijing, China
Novartis Investigative Site
Chengdu, China
Novartis Investigative Site
Shanghai, China
Novartis Investigative Site
Tianjin, China
Sponsors and Collaborators
Novartis Pharmaceuticals
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals

Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Novartis Pharmaceuticals Identifier: NCT01546415     History of Changes
Other Study ID Numbers: CICL670ACN01
First Posted: March 7, 2012    Key Record Dates
Last Update Posted: October 28, 2014
Last Verified: October 2014
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Phase 4
Iron Overload
Aplastic Anemia
Additional relevant MeSH terms:
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Anemia, Aplastic
Iron Overload
Hematologic Diseases
Bone Marrow Diseases
Iron Metabolism Disorders
Metabolic Diseases
Trace Elements
Growth Substances
Physiological Effects of Drugs