search
Back to results

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

Primary Purpose

Iron Overload

Status
Completed
Phase
Phase 4
Locations
China
Study Type
Interventional
Intervention
Desferasirox
Sponsored by
Novartis Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Iron Overload focused on measuring Phase 4, Desferasirox, Iron Overload, Aplastic Anemia

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Desferasirox

Arm Description

Outcomes

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.
Difference in serum ferritin from baseline vs. 52 weeks of treatment.

Secondary Outcome Measures

Correlation between serum ferritin (SF) and transferrin saturation (TFS)
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
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.
Number of patients with adverse events, serious adverse events and death

Full Information

First Posted
November 10, 2011
Last Updated
October 27, 2014
Sponsor
Novartis Pharmaceuticals
search

1. Study Identification

Unique Protocol Identification Number
NCT01546415
Brief Title
Safety and Efficacy of Desferasirox in Chinese Patients With Iron Overload and Aplastic Anemia
Official Title
A Single Arm, Multicenter, Open Label Study of Desferasirox in Chinese Patients With Iron Overload and Aplastic Anemia
Study Type
Interventional

2. Study Status

Record Verification Date
October 2014
Overall Recruitment Status
Completed
Study Start Date
October 2011 (undefined)
Primary Completion Date
November 2013 (Actual)
Study Completion Date
November 2013 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals

4. Oversight

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Iron Overload
Keywords
Phase 4, Desferasirox, Iron Overload, Aplastic Anemia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
64 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Desferasirox
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Desferasirox
Intervention Description
Initial Dose: 20 mg/kg/d. Dose modification according to protocol.
Primary Outcome Measure Information:
Title
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.
Description
Difference in serum ferritin from baseline vs. 52 weeks of treatment.
Time Frame
Every 4 Weeks for 52 weeks of treatment
Secondary Outcome Measure Information:
Title
Correlation between serum ferritin (SF) and transferrin saturation (TFS)
Description
Study will compare SF with TFS level to find degree of relationship measured by correlation.
Time Frame
Every 4 Weeks for 52 weeks of treatment
Title
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
Title
Correlation between dose adjustment (increase or decrease) regimens and transfusional burden
Description
Evaluation of the relationship between dose adjustment regimens (as dictated by both efficacy and safety parameters) and transfusional burden will be undertaken.
Time Frame
Every 3 months for 52 Weeks of treatment
Title
Number of patients with adverse events, serious adverse events and death
Time Frame
Every 4 weeks for 52 Weeks of treatment

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis Pharmaceuticals
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Novartis Investigative Site
City
Shanghai
State/Province
Shanghai
ZIP/Postal Code
200437
Country
China
Facility Name
Novartis Investigative Site
City
Chengdu
State/Province
Sichuan
ZIP/Postal Code
610041
Country
China
Facility Name
Novartis Investigative Site
City
Tianjin
State/Province
Tianjin
ZIP/Postal Code
300020
Country
China
Facility Name
Novartis Investigative Site
City
Beijing
Country
China
Facility Name
Novartis Investigative Site
City
Chengdu
Country
China
Facility Name
Novartis Investigative Site
City
Shanghai
Country
China
Facility Name
Novartis Investigative Site
City
Tianjin
Country
China

12. IPD Sharing Statement

Citations:
PubMed Identifier
26876245
Citation
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.
Results Reference
derived

Learn more about this trial

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

We'll reach out to this number within 24 hrs