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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 (MACS2163)

Primary Purpose

β-thalassemia Major

Status
Withdrawn
Phase
Phase 4
Locations
Study Type
Interventional
Intervention
Deferasirox
Sponsored by
Novartis Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for β-thalassemia Major focused on measuring deferasirox

Eligibility Criteria

2 Years - 71 Years (Child, Adult, Older Adult)All SexesDoes not accept 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.

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    Deferasirox

    Arm Description

    The study will provide PK, safety, tolerability and efficacy data collected during 48 weeks of treatment with deferasirox in Chinese pediatric patients with transfusion dependent -thalassemia major, aged 2 to <6 years at enrollment. The target patient pool consists of 20 patients with evidence of iron overload measured by serum ferritin level at the start of study. Patients will start their deferasirox treatment with a dose of 20 mg/kg/day. Serum ferritin will be monitored every month and the dose of deferasirox will be adjusted if necessary every 3 months based on the trends in serum ferritin. Other possible dose adjustments will be based on the patient's safety assessments.

    Outcomes

    Primary Outcome Measures

    the PK profile of deferasirox in pediatric β-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.
    the PK profile of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old: Cmax
    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
    Tmax was directly determined from the raw plasma concentration-time data.

    Secondary Outcome Measures

    The safety and tolerability of deferasirox following multiple dosing in pediatric β-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.
    The efficacy of deferasirox in pediatric β-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.

    Full Information

    First Posted
    October 24, 2012
    Last Updated
    April 19, 2017
    Sponsor
    Novartis Pharmaceuticals
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    1. Study Identification

    Unique Protocol Identification Number
    NCT01724138
    Brief 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
    Acronym
    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
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    August 2014
    Overall Recruitment Status
    Withdrawn
    Study Start Date
    June 2013 (undefined)
    Primary Completion Date
    April 2014 (Anticipated)
    Study Completion Date
    October 2014 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Novartis Pharmaceuticals

    4. Oversight

    Data Monitoring Committee
    No

    5. Study Description

    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    β-thalassemia Major
    Keywords
    deferasirox

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 4
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    Randomized
    Enrollment
    0 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    Deferasirox
    Arm Type
    Experimental
    Arm Description
    The study will provide PK, safety, tolerability and efficacy data collected during 48 weeks of treatment with deferasirox in Chinese pediatric patients with transfusion dependent -thalassemia major, aged 2 to <6 years at enrollment. The target patient pool consists of 20 patients with evidence of iron overload measured by serum ferritin level at the start of study. Patients will start their deferasirox treatment with a dose of 20 mg/kg/day. Serum ferritin will be monitored every month and the dose of deferasirox will be adjusted if necessary every 3 months based on the trends in serum ferritin. Other possible dose adjustments will be based on the patient's safety assessments.
    Intervention Type
    Drug
    Intervention Name(s)
    Deferasirox
    Intervention Description
    Patients will start their deferasirox treatment with a dose of 20 mg/kg/day.
    Primary Outcome Measure Information:
    Title
    the PK profile of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old.
    Description
    Area under the plasma concentration-time curve from time zero to the end of the dosing interval.
    Time Frame
    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.
    Title
    the PK profile of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old: Cmax
    Description
    The maximum plasma concentration of study medication.
    Time Frame
    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.
    Title
    the PK profile of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old: Tmax
    Description
    Tmax was directly determined from the raw plasma concentration-time data.
    Time Frame
    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 Outcome Measure Information:
    Title
    The safety and tolerability of deferasirox following multiple dosing in pediatric β-thalassemia major patients.
    Description
    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.
    Time Frame
    Baseline, every 4 weeks until 48 weeks after taking the drug
    Title
    The efficacy of deferasirox in pediatric β-thalassemia patients as measured by change of serum ferritin.
    Description
    Changes in serum ferritin from baseline to every 4 weeks are collected for the measurement of efficacy.
    Time Frame
    Baseline, every 4 weeks until 48 weeks after taking the drug

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    2 Years
    Maximum Age & Unit of Time
    71 Years
    Accepts Healthy Volunteers
    No
    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.
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Novartis Pharmaceuticals
    Organizational Affiliation
    Novartis Pharmaceuticals
    Official's Role
    Study Director

    12. IPD Sharing Statement

    Learn more about this trial

    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

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