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Deferasirox BID (Twice a Day) in Transfusion Dependent Thalassemia Patients With Inadequate Response to High Doses

Primary Purpose

Focus on Transfusion Dependent Thalassemia Patients Who Are Inadequate Responders to Deferasirox > 35mg/kgQD

Status
Withdrawn
Phase
Phase 2
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 Focus on Transfusion Dependent Thalassemia Patients Who Are Inadequate Responders to Deferasirox > 35mg/kgQD focused on measuring Thalassemia

Eligibility Criteria

2 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Written informed consent must be obtained prior to any screening procedures
  • Male or female aged ≥ 2 years at screening
  • Patients with transfusion dependent thalassemia
  • Patients confirmed as inadequate- responders to deferasirox > 35 mg/kg QD and treated with QD for at least 6 months
  • Regular transfusion indicated by a blood requirement ≥ 8 blood transfusions per year at screening
  • Serum ferritin level > 2,500 ng/mL at screening (two consecutive values at least 2 weeks apart from each other)
  • One SF measurement > 2,500 ng/mL between 6 and 9 months prior to study enrollment
  • Three SF measurements > 2,500 ng/mL, performed at least 3 weeks apart from each other, during the 6 month treatment with QD dosing of deferasirox prior to study enrollment
  • The average of the two screening SF values (collected 2 weeks apart from each other) must not show a decrease from the 6 to 9 month SF value taken prior to study enrollment
  • The average of the two screening SF values (collected 2 weeks apart from each other) must not show a decrease from each of the three SF values obtained during the 6 months of deferasirox QD treatment prior to study enrollment
  • LIC ≥ 7 mg Fe/g dw measured at the screening visit, (this value will be used as a baseline measurement

Exclusion Criteria:

  • Patients who are intolerant to > 35 mg/kg/day QD of deferasirox in the 6 months prior to study enrollment
  • Patients with mean levels of ALT >5 x ULN
  • Patients with serum creatinine above the upper limit of normal (ULN)
  • Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 (mg/mg)
  • Creatinine clearance ≤ 60 ml/min
  • Chronic hepatitis B infection, active hepatitis C infection
  • History of a positive HIV test
  • Uncontrolled systemic hypertension
  • Patients participating in another clinical trial or receiving a systemic investigational drug within the past 4 weeks or topical investigational drug within the past 7 days of screening
  • History of non-compliance with medical regimens or patients who are considered potentially unreliable and/or not cooperative, unwilling or unable to comply with the protocol
  • History of hypersensitivity to any of the study drug or excipients
  • Significant medical condition interfering with the ability to partake in this study (e.g. systemic uncontrolled hypertension, unstable cardiac disease not controlled by standard medical therapy, systemic disease (cardiovascular, renal, hepatic etc.)
  • History of drug or alcohol abuse within the 12 months prior to enrollment.

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    Deferasirox

    Arm Description

    Deferasirox 20mg/kg taken BID

    Outcomes

    Primary Outcome Measures

    Mean relative change in SF (Serum Ferritin)
    To evaluate the efficacy of a twice-daily dose regimen of deferasirox in inadequate-responders to a once daily dose regimen as measured by relative change in serum ferritin from baseline to 24 weeks of treatment

    Secondary Outcome Measures

    Mean Absolute/relative change in LIC
    To evaluate the efficacy of BID deferasirox in liver iron removal as measured by MRI by absolute/relative change from baseline to 24 weeks of treatment

    Full Information

    First Posted
    September 19, 2013
    Last Updated
    April 19, 2017
    Sponsor
    Novartis Pharmaceuticals
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    1. Study Identification

    Unique Protocol Identification Number
    NCT01948817
    Brief Title
    Deferasirox BID (Twice a Day) in Transfusion Dependent Thalassemia Patients With Inadequate Response to High Doses
    Official Title
    A Prospective Single Arm Study to Assess the Efficacy and Safety of Deferasirox 20 mg/kg BID in Transfusion Dependent Thalassemia Patients Inadequately Responding to Current Treatment With Doses > 35mg/kg QD (Once a Day).
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    January 2014
    Overall Recruitment Status
    Withdrawn
    Study Start Date
    February 2014 (undefined)
    Primary Completion Date
    February 2016 (Anticipated)
    Study Completion Date
    February 2016 (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
    This is an open label, single arm, and multicenter study. The study will include the following phases. A screening phase which lasts for 4 weeks to determine patient eligibility. This phase will be followed by a 24 week Open label treatment phase. The study treatment is defined as deferasirox 20mg/kg BID 9Twice a day). Serum Ferritin Levels and MRI (Magmetic Resonance Imaging) LIC (Liver Iron Concentration) will be measured to evaluate the response to BID.
    Detailed Description
    Study treatment is defined as deferasirox 20 mg/kg BID. The study treatment duration is 24 weeks. After the baseline visit, patient visits will occur weekly during the first month because key safety parameters need to be performed weekly in the first month of treatment and then every 4 weeks thereafter until week 24 End of Treatment (EOT). Patients will have their first dose of study treatment at Visit 3. Safety assessments are routinely performed including collection of AEs (Adverse Events), SAEs (Serious ADverse Events<, vital signs, physical examination, ECG (Electrocardiograph), hematological and biochemistry assessments. Patients will continue therapy until intolerable toxicity, patient decision or after 24 weeks treatment duration at which point and End of Treatment (EOT) visit will be performed and the End of Treatment CRF( Case Report Form) will be completed. 30 day Safety follow-up Patients who discontinue study drug before completing the study should be scheduled for a visit as soon as possible, at which time all of the assessments listed for the final visit will be performed. At a minimum, all patients who discontinue study treatment, including those who refuse to return for a final visit, will be contacted for safety evaluations during the 30 days following the last dose of study drug. All patients must be followed for AEs and SAEs that may have occurred after discontinuation from the study treatment. The safety follow-up visit will take place 30 days after the last dose of study drug.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Focus on Transfusion Dependent Thalassemia Patients Who Are Inadequate Responders to Deferasirox > 35mg/kgQD
    Keywords
    Thalassemia

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 2
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    Non-Randomized
    Enrollment
    0 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    Deferasirox
    Arm Type
    Experimental
    Arm Description
    Deferasirox 20mg/kg taken BID
    Intervention Type
    Drug
    Intervention Name(s)
    Deferasirox
    Intervention Description
    Deferasirox 20mg/kg taken BID
    Primary Outcome Measure Information:
    Title
    Mean relative change in SF (Serum Ferritin)
    Description
    To evaluate the efficacy of a twice-daily dose regimen of deferasirox in inadequate-responders to a once daily dose regimen as measured by relative change in serum ferritin from baseline to 24 weeks of treatment
    Time Frame
    Baseline, 24 Weeks
    Secondary Outcome Measure Information:
    Title
    Mean Absolute/relative change in LIC
    Description
    To evaluate the efficacy of BID deferasirox in liver iron removal as measured by MRI by absolute/relative change from baseline to 24 weeks of treatment
    Time Frame
    Baseline, 24 Weeks

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    2 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: Written informed consent must be obtained prior to any screening procedures Male or female aged ≥ 2 years at screening Patients with transfusion dependent thalassemia Patients confirmed as inadequate- responders to deferasirox > 35 mg/kg QD and treated with QD for at least 6 months Regular transfusion indicated by a blood requirement ≥ 8 blood transfusions per year at screening Serum ferritin level > 2,500 ng/mL at screening (two consecutive values at least 2 weeks apart from each other) One SF measurement > 2,500 ng/mL between 6 and 9 months prior to study enrollment Three SF measurements > 2,500 ng/mL, performed at least 3 weeks apart from each other, during the 6 month treatment with QD dosing of deferasirox prior to study enrollment The average of the two screening SF values (collected 2 weeks apart from each other) must not show a decrease from the 6 to 9 month SF value taken prior to study enrollment The average of the two screening SF values (collected 2 weeks apart from each other) must not show a decrease from each of the three SF values obtained during the 6 months of deferasirox QD treatment prior to study enrollment LIC ≥ 7 mg Fe/g dw measured at the screening visit, (this value will be used as a baseline measurement Exclusion Criteria: Patients who are intolerant to > 35 mg/kg/day QD of deferasirox in the 6 months prior to study enrollment Patients with mean levels of ALT >5 x ULN Patients with serum creatinine above the upper limit of normal (ULN) Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 (mg/mg) Creatinine clearance ≤ 60 ml/min Chronic hepatitis B infection, active hepatitis C infection History of a positive HIV test Uncontrolled systemic hypertension Patients participating in another clinical trial or receiving a systemic investigational drug within the past 4 weeks or topical investigational drug within the past 7 days of screening History of non-compliance with medical regimens or patients who are considered potentially unreliable and/or not cooperative, unwilling or unable to comply with the protocol History of hypersensitivity to any of the study drug or excipients Significant medical condition interfering with the ability to partake in this study (e.g. systemic uncontrolled hypertension, unstable cardiac disease not controlled by standard medical therapy, systemic disease (cardiovascular, renal, hepatic etc.) History of drug or alcohol abuse within the 12 months prior to enrollment.
    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

    Deferasirox BID (Twice a Day) in Transfusion Dependent Thalassemia Patients With Inadequate Response to High Doses

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