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

Iron Overload Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01546415
• Other study ID #: CICL670ACN01
• Status: Completed
• Phase: Phase 4
• First received: November 10, 2011
• Last updated: October 27, 2014
• Start date: October 2011
• Est. completion date: November 2013

Study information

• Verified date: October 2014
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationChina: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 64
• Est. completion date: November 2013
• Est. primary completion date: November 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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 Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Anemia, Aplastic
• Iron Overload

Intervention

Drug:
• Desferasirox
Initial Dose: 20 mg/kg/d. Dose modification according to protocol.

Locations

Country	Name	City	State
China	Novartis Investigative Site	Beijing
China	Novartis Investigative Site	Chengdu
China	Novartis Investigative Site	Chengdu	Sichuan
China	Novartis Investigative Site	Shanghai	Shanghai
China	Novartis Investigative Site	Shanghai
China	Novartis Investigative Site	Tianjin
China	Novartis Investigative Site	Tianjin	Tianjin

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	No
Secondary	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	No
Secondary	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	No
Secondary	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	No
Secondary	Number of patients with adverse events, serious adverse events and death		Every 4 weeks for 52 Weeks of treatment	No