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

Novartis

Status and phase: Completed; Phase 4

Conditions: Iron Overload

Treatments: Drug: Desferasirox

Study type: Interventional

Funder types: Industry

Identifiers

NCT01546415

CICL670ACN01

Details and patient eligibility

About

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

Enrollment: 64 patients

Sex: All

Ages: 18+ years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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64 participants in 1 patient group

Desferasirox

Experimental group

Treatment:

Drug: Desferasirox