Clinical trial NCT01546415: 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
Overall status: COMPLETED
Phase: PHASE4
Conditions: Iron Overload
Keywords: Phase 4
Desferasirox
Iron Overload
Aplastic Anemia
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2012-03-07
Enrollment: 64
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


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

Primary outcomes:
measure: 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.
timeFrame: Every 4 Weeks for 52 weeks of treatment
Secondary outcomes:
measure: Correlation between serum ferritin (SF) and transferrin saturation (TFS)
timeFrame: Every 4 Weeks for 52 weeks of treatment
measure: Number of dispensed, used, partially used or unused packages of study medication to assess drug usage compliance
timeFrame: Every 4 Weeks for 52 weeks of treatment
measure: Correlation between dose adjustment (increase or decrease) regimens and transfusional burden
timeFrame: Every 3 months for 52 Weeks of treatment
measure: Number of patients with adverse events, serious adverse events and death
timeFrame: Every 4 weeks for 52 Weeks of treatment
Other outcomes:
Locations:
city: Shanghai
country: China
facility: Novartis Investigative Site
state: Shanghai
city: Chengdu
country: China
facility: Novartis Investigative Site
state: Sichuan
city: Tianjin
country: China
facility: Novartis Investigative Site
state: Tianjin
city: Beijing
country: China
facility: Novartis Investigative Site
city: Chengdu
country: China
facility: Novartis Investigative Site
city: Shanghai
country: China
facility: Novartis Investigative Site
city: Tianjin
country: China
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
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.