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

A Single Arm, Multicenter, Open Label Study of Desferasirox in Chinese Patients With Iron Overload and Aplastic Anemia

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

Study Overview

• Status: Completed
• Conditions: Iron Overload
• Intervention / Treatment: Drug: Desferasirox

• Study Type: Interventional
• Enrollment (Actual): 64
• Phase: Phase 4

Contacts and Locations

Study Locations

• China
  • Beijing, China
    • Novartis Investigative Site
  • Chengdu, China
    • Novartis Investigative Site
  • Shanghai, China
    • Novartis Investigative Site
  • Tianjin, China
    • Novartis Investigative Site
  • Shanghai
    • Shanghai, Shanghai, China, 200437
      • Novartis Investigative Site
  • Sichuan
    • Chengdu, Sichuan, China, 610041
      • Novartis Investigative Site
  • Tianjin
    • Tianjin, Tianjin, China, 300020
      • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NON_RANDOMIZED
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Desferasirox	Drug: Desferasirox; Initial Dose: 20 mg/kg/d. Dose modification according to protocol.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	Difference in serum ferritin from baseline vs. 52 weeks of treatment.	Every 4 Weeks for 52 weeks of treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Correlation between serum ferritin (SF) and transferrin saturation (TFS)	Study will compare SF with TFS level to find degree of relationship measured by correlation.	Every 4 Weeks for 52 weeks of treatment
Number of dispensed, used, partially used or unused packages of study medication to assess drug usage compliance		Every 4 Weeks for 52 weeks of treatment
Correlation between dose adjustment (increase or decrease) regimens and transfusional burden	Evaluation of the relationship between dose adjustment regimens (as dictated by both efficacy and safety parameters) and transfusional burden will be undertaken.	Every 3 months for 52 Weeks of treatment
Number of patients with adverse events, serious adverse events and death		Every 4 weeks for 52 Weeks of treatment

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals

Publications and helpful links

General Publications

• Shi J, Chang H, Zhang L, Shao Y, Nie N, Zhang J, Huang J, Zhang L, Tang X, Quan R, Zheng C, Xiao H, Hu D, Hu L, Liu F, Zhou Y, Zheng Y, Zhang F. [Efficacy and safety of deferasirox in aplastic anemia patients with iron overload: a single arm, multi-center,prospective study in China]. Zhonghua Xue Ye Xue Za Zhi. 2016 Jan;37(1):1-6. doi: 10.3760/cma.j.issn.0253-2727.2016.01.001. Chinese.

Study record dates

Study Major Dates

• Study Start: October 1, 2011
• Primary Completion (ACTUAL): November 1, 2013
• Study Completion (ACTUAL): November 1, 2013

Study Registration Dates

• First Submitted: November 10, 2011
• First Submitted That Met QC Criteria: March 1, 2012
• First Posted (ESTIMATE): March 7, 2012

Study Record Updates

• Last Update Posted (ESTIMATE): October 28, 2014
• Last Update Submitted That Met QC Criteria: October 27, 2014
• Last Verified: October 1, 2014

More Information

Terms related to this study

• Keywords: Iron Overload; Aplastic Anemia; Phase 4; Desferasirox
• Additional Relevant MeSH Terms: Metabolic Diseases; Bone Marrow Diseases; Hematologic Diseases; Iron Metabolism Disorders; Bone Marrow Failure Disorders; Iron Overload; Anemia; Anemia, Aplastic
• Other Study ID Numbers: CICL670ACN01