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

NCT01546415 | Completed Phase 4

• 1 other identifier: CICL670ACN01
• Study Type: interventional
• Target: 64
• Locations: 1 country
• Sites: 7

Brief Summary

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

Conditions

Iron Overload

Keywords

Phase 4; Desferasirox; Iron Overload; Aplastic Anemia

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (4)

• Correlation between serum ferritin (SF) and transferrin saturation (TFS)

  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

  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

Study Arms (1)

Desferasirox

EXPERIMENTAL

Drug: Desferasirox

Interventions

Desferasirox DRUG

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

Desferasirox

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (7)

Novartis Investigative Site

Shanghai, Shanghai Municipality, 200437, China

Location

Novartis Investigative Site

Chengdu, Sichuan, 610041, China

Location

Novartis Investigative Site

Tianjin, Tianjin Municipality, 300020, China

Location

Novartis Investigative Site

Beijing, China

Location

Novartis Investigative Site

Chengdu, China

Location

Novartis Investigative Site

Shanghai, China

Location

Novartis Investigative Site

Tianjin, China

Location

Related Publications (1)

• 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.

  PMID: 26876245 DERIVED

MeSH Terms

Conditions

Iron Overload; Anemia, Aplastic

Condition Hierarchy (Ancestors)

Iron Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 10, 2011
• First Posted: March 7, 2012
• Study Start: October 1, 2011
• Primary Completion: November 1, 2013
• Study Completion: November 1, 2013
• Last Updated: October 28, 2014

Record last verified: 2014-10

Locations

CN (7)