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

ß-thalassemia Major Clinical Trial

— MACS2163  

Official title:

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

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT01724138
• Other study ID #: CICL670ACN02
• Status: Withdrawn
• Phase: Phase 4
• First received: October 24, 2012
• Last updated: April 19, 2017
• Start date: June 2013
• Est. completion date: October 2014

Study information

• Verified date: August 2014
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: October 2014
• Est. primary completion date: April 2014
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 71 Years

Eligibility

Inclusion Criteria:

• Pediatric patients aged from 2 to less than 6 years old.

• Patients with transfusion dependent ß-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.

Related Conditions & MeSH terms

• beta-Thalassemia
• ß-thalassemia Major
• Thalassemia

Intervention

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

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	the PK profile of deferasirox in pediatric ß-thalassemia major patients aged from 2 to less than 6 years old.	Area under the plasma concentration-time curve from time zero to the end of the dosing interval.	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.
Primary	the PK profile of deferasirox in pediatric ß-thalassemia major patients aged from 2 to less than 6 years old: Cmax	The maximum plasma concentration of study medication.	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.
Primary	the PK profile of deferasirox in pediatric ß-thalassemia major patients aged from 2 to less than 6 years old: Tmax	Tmax was directly determined from the raw plasma concentration-time data.	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.
Secondary	The safety and tolerability of deferasirox following multiple dosing in pediatric ß-thalassemia major patients.	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.	Baseline, every 4 weeks until 48 weeks after taking the drug
Secondary	The efficacy of deferasirox in pediatric ß-thalassemia patients as measured by change of serum ferritin.	Changes in serum ferritin from baseline to every 4 weeks are collected for the measurement of efficacy.	Baseline, every 4 weeks until 48 weeks after taking the drug