An Open Label Study to Evaluate the Pharmacokinetics, Safety, Tolerability and Efficacy of Deferasirox Administered to Chinese Patients With β-thalassemia Major Aged From 2 to Less Than 6 Years Old

Status

Withdrawn

Phases

Phase 4

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01724138

Acronym

MACS2163

Enrollment

Registered

2012-11-09

Start date

2013-06-30

Completion date

2014-10-31

Last updated

2017-04-20

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

β-thalassemia Major

Keywords

deferasirox

Brief summary

To characterize the PK of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old, when administrated with a fixed starting dose of 20 mg/kg/day.

Interventions

DRUGDeferasirox

Patients will start their deferasirox treatment with a dose of 20 mg/kg/day.

Study design

Allocation

RANDOMIZED

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

2 Years to 71 Years

Healthy volunteers

Inclusion criteria

* Pediatric patients aged from 2 to less than 6 years old. * Patients with transfusion dependent β-thalassemia major. * Serum ferritin values ≥ 1000 ng/ml at screening. * Written informed consent must be obtained from the patient's legal guardian in accordance with local law and regulation prior to any screening procedures.

Exclusion criteria

* Non-transfusion-dependent thalassemia. * Systemic diseases which would prevent study treatment (e.g. uncontrolled hypertension, cardiovascular, renal, hepatic, metabolic, etc.) * Serum creatinine \> age adjusted ULN. * Significant proteinuria as indicated by a urinary protein/creatinine ratio (UPCR) ≥ 0.5mg/mg in a non-first void urine sample at screening. If UPCR is found to be ≥ 0.5 mg/mg the test can be repeated after 1 month. * ALT/AST \> 2.5xULN and total bilirubin \> 1×ULN. * Left ventricular ejection fraction \< 56% by echocardiography. * Patient has a known history of HIV seropositivity or history of active/treated hepatitis B or C (a test for screening is not required). * A history of clinically relevant ocular and/or auditory toxicity related to iron chelation therapy * Any surgical or medical conditions which will significantly alter the absorption, distribution, metabolism or excretion of the drug(e.g. ulcerative disease, uncontrolled nausea, vomiting, malabsorption syndrome, obstruction, or stomach and/or small bowel resection). * Other conditions which investigator deems potential harm to patients if participate the study.

Design outcomes

Primary

Measure	Time frame	Description
the PK profile of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old.	PK sampling times are 0.5, 2.5, 6, 10h in Day 1 postdose; Day 2, 3, 4 and 5 predose and 0.5, 2.5, 6, 10h post dose on Day 4, then pre-dose at each subsequent visit until Week 49. Day -1 PK sample will be treated as Day 1 predose sample.	Area under the plasma concentration-time curve from time zero to the end of the dosing interval.
the PK profile of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old: Cmax	PK sampling times are 0.5, 2.5, 6, 10h in Day 1 postdose; Day 2, 3, 4 and 5 predose and 0.5, 2.5, 6, 10h post dose on Day 4, then pre-dose at each subsequent visit until Week 49. Day -1 PK sample will be treated as Day 1 predose sample.	The maximum plasma concentration of study medication.
the PK profile of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old: Tmax	PK sampling times are 0.5, 2.5, 6, 10h in Day 1 postdose; Day 2, 3, 4 and 5 predose and 0.5, 2.5, 6, 10h post dose on Day 4, then pre-dose at each subsequent visit until Week 49. Day -1 PK sample will be treated as Day 1 predose sample.	Tmax was directly determined from the raw plasma concentration-time data.

Secondary

Measure	Time frame	Description
The safety and tolerability of deferasirox following multiple dosing in pediatric β-thalassemia major patients.	Baseline, every 4 weeks until 48 weeks after taking the drug	The adverse events and abnormal measurements of hematology, blood chemistry, urinalysis, urinary protein/creatinine ratio, physical examination, auditory and ocular examinations, ECG, ECHO, and growth development are collected for the measurement of safety and tolerability.
The efficacy of deferasirox in pediatric β-thalassemia patients as measured by change of serum ferritin.	Baseline, every 4 weeks until 48 weeks after taking the drug	Changes in serum ferritin from baseline to every 4 weeks are collected for the measurement of efficacy.

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026