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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01546415
First received: November 10, 2011
Last updated: December 24, 2013
Last verified: December 2013
  Purpose

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


Condition Intervention Phase
Iron Overload
Drug: Desferasirox
Phase 4

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]
    Difference in serum ferritin from baseline vs. 52 weeks of treatment.


Secondary Outcome Measures:
  • Correlation between serum ferritin (SF) and transferrin saturation (TFS) [ Time Frame: Every 4 Weeks for 52 weeks of treatment ] [ Designated as safety issue: No ]
    Study will compare SF with TFS level to find degree of relationship measured by correlation.

  • 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 ] [ Designated as safety issue: No ]
  • Correlation between dose adjustment (increase or decrease) regimens and transfusional burden [ Time Frame: Every 3 months for 52 Weeks of treatment ] [ Designated as safety issue: No ]
    Evaluation of the relationship between dose adjustment regimens (as dictated by both efficacy and safety parameters) and transfusional burden will be undertaken.

  • Number of patients with adverse events, serious adverse events and death [ Time Frame: Every 4 weeks for 52 Weeks of treatment ] [ Designated as safety issue: No ]

Enrollment: 64
Study Start Date: October 2011
Study Completion Date: November 2013
Primary Completion Date: November 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Desferasirox Drug: Desferasirox
Initial Dose: 20 mg/kg/d. Dose modification according to protocol.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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

  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01546415

Locations
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
China
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
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01546415     History of Changes
Other Study ID Numbers: CICL670ACN01
Study First Received: November 10, 2011
Last Updated: December 24, 2013
Health Authority: United States: Food and Drug Administration
China: Food and Drug Administration

Keywords provided by Novartis:
Phase 4
Desferasirox
Iron Overload
Aplastic Anemia

Additional relevant MeSH terms:
Anemia
Anemia, Aplastic
Iron Overload
Hematologic Diseases
Bone Marrow Diseases
Iron Metabolism Disorders
Metabolic Diseases

ClinicalTrials.gov processed this record on April 23, 2014