Clinical trial NCT01724138: 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
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
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE4
Conditions: β-thalassemia Major
Keywords: deferasirox
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2012-11-09
Enrollment: 0
Sex: ALL
Minimum age (years): 2
Maximum age (years): 71
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: the PK profile of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old.
timeFrame: 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.
measure: the PK profile of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old: Cmax
timeFrame: 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.
measure: the PK profile of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old: Tmax
timeFrame: 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 outcomes:
measure: The safety and tolerability of deferasirox following multiple dosing in pediatric β-thalassemia major patients.
timeFrame: Baseline, every 4 weeks until 48 weeks after taking the drug
measure: The efficacy of deferasirox in pediatric β-thalassemia patients as measured by change of serum ferritin.
timeFrame: Baseline, every 4 weeks until 48 weeks after taking the drug
Other outcomes:
Locations:
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: