An Open Label, Multi-center, Efficacy and Safety Study of Deferasirox in Iron Overloaded Patients With Non-transfusion Dependent Thalassemia. (MACS1935)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01709838
First received: October 16, 2012
Last updated: May 1, 2014
Last verified: May 2014

October 16, 2012
May 1, 2014
December 2012
January 2020   (final data collection date for primary outcome measure)
Change in liver iron concentration [ Time Frame: baseline, 52 weeks ] [ Designated as safety issue: No ]
Absolute change in liver iron concentration (MRI) from baseline after 52 weeks of treatment
Same as current
Complete list of historical versions of study NCT01709838 on ClinicalTrials.gov Archive Site
  • Medical Outcomes Study Short Form-36 [ Time Frame: baseline, up to 156 weeks ] [ Designated as safety issue: No ]
    Absolute change from baseline to 52 weeks. The SF-36 is a self-administered questionnaire for adults (from 18 years of age) and contains 36 items which measure: Physical functioning, Role limitation due to physical health problems, Bodily pain, Role limitations due to emotional problems, General mental health and Health change over the past year.
  • Pediatric Quality of Life Questionnaires [ Time Frame: baseline, up to 156 weeks ] [ Designated as safety issue: No ]
    The PedsOL is a modular approach to measuring health-related quality of life in pediatrics (13 to 18 years). The approach includes both a child self-report and a parent proxy-report. The 23-item PedsQL Generic Core Scales encompass the essential core domains for pediatric health related quality of life measurement: Physical functioning, emotional functioning, social functioning and school functioning.
  • Change in liver iron concentration [ Time Frame: baseline, up to 260 weeks ] [ Designated as safety issue: No ]
    Absolute change in liver iron concentration (MRI) from baseline over time (every 6 months approximately)
  • serum ferritin vs liver iron concentration [ Time Frame: baseline, up to 260 weeks ] [ Designated as safety issue: No ]
    change of ferritin vs liver iron concentration from baseline, and end of study
  • change in serum ferritin [ Time Frame: baseline, up to 260 weeks ] [ Designated as safety issue: No ]
    Absolute change in serum ferritin from baseline over time
  • endocrine laboratory parameters [ Time Frame: baseline, up to 260 weeks ] [ Designated as safety issue: No ]
    Absolute change over time from baseline in Total and free testosterone (males), LH and FSH (females), TSH, total and free T4, total and free T3, fasting plasma glucose, insulin, insulin resistance and cortisol.
  • pk parameters [ Time Frame: up to 24 weeks ] [ Designated as safety issue: No ]
    in subset of patients-PAS will be used in all pharmacokinetic data analysis and PK summary statistics. Th Pk parameters, AUCtau, Cmas and tmax may be determined using non-compartmental methods for deferasirox and its iron complex. Biofluid concentrations will be expressed in mass per volume units.
  • adverse events [ Time Frame: up to 260 weeks ] [ Designated as safety issue: Yes ]
    number of participants with adverse events as a measure of safety and tolerability
  • Change in liver iron concentration [ Time Frame: every 4 weeks following baseline to week 260 ] [ Designated as safety issue: No ]
    response rate in patients with baseline LIC >15 mg Fe/g dw defined as proportion of patients achieving LIC <5mg Fe/g dw and time to achieving LIC <5mg Fe/g dw
  • Change in liver iron concentration [ Time Frame: every 4 weeks from week 52 to week 260 ] [ Designated as safety issue: Yes ]
    long term efficacy and safety of treatment to target LIC of 3mg Fe/g dw followed by one or more treatment holidays until the LIC is ≥5 mg Fe/g dw
  • Change in liver iron concentration [ Time Frame: baseline, 52 weeks ] [ Designated as safety issue: No ]
    change in Liver iron concentration from baseline after 52 weks of treatment by non-transfusion dependent thalassemia (NTDT) syndrome
  • Medical Outcomes Study Short Form-36 [ Time Frame: baseline, 52 weeks ] [ Designated as safety issue: No ]
    Absolute change from baseline to 52 weeks. The SF-36 is a self-administered questionnaire for adults (> 18 years of age) and contains 36 items which measure: Physical functioning, Role limitation due to physical health problems, Bodily pain, Role limitations due to emothioal problems, General mental health and Health change over the past year.
  • Pediatric Quality of Life Questionnaires [ Time Frame: baseline, 52 weeks ] [ Designated as safety issue: No ]
    The PedsOL is a modular approach to measuring health-related quality of life in pediatrics (>10 but </= 18 years of age). The approach includes both a child self-report and a parent proxy-report. The 23-item PedsQL Generic Core Scales encompass the essential core domains for pediatric health related quality of life measurement: Physical functioning, emothioal functioning, social functioning and school functioning.
  • Change in liver iron concentration [ Time Frame: baseline, 24 weeks ] [ Designated as safety issue: No ]
    Absolute change in liver iron concentration (MRI) from baseline after 24 weeks of treatment
  • serum ferritin vs liver iron concentration [ Time Frame: baseline, week 24 and week 52 ] [ Designated as safety issue: No ]
    baseline, week 24, week 52 serum ferritin versus baseline, week 24, week 52 liver iron concentration
  • change in serum ferritin [ Time Frame: baseline, 52 weeks ] [ Designated as safety issue: No ]
    Absolute change in serum ferritin from baseline after 52 weeks of treatment
  • endocrine laboratory parameters [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
    Absolute change from baseline in Total and free testosterone (males), LH and FSH (females), TSH, total and free T4, total and free T3, fasting plasma slucoes, insulin, insulin reisitance and cortisol.
  • pk parameters [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
    PAS will be used in all pharmacikinetic data analysis and PK summary statistics. Th Pk parameters, AUCtau, Cmas and tmax may be determined using non-comparmental methos for deferasirox and its iron complex. Biofluid concentrations will be expressed in mass per volume units.
  • adverse events [ Time Frame: 52 weeks ] [ Designated as safety issue: Yes ]
    number of participants with adverse events as a measure of safety and tolerability
Not Provided
Not Provided
 
An Open Label, Multi-center, Efficacy and Safety Study of Deferasirox in Iron Overloaded Patients With Non-transfusion Dependent Thalassemia.
An Open Label, Multi-center, Efficacy and Safety Study of Deferasirox in Iron Overloaded Patients With Non-transfusion Dependent Thalassemia

To assess the efficacy of deferasirox in patients with non-transfusion dependent thalassemia based on change in liver iron concentration from baseline after 52 weeks of treatment. To provide further assessment of the long-term efficacy and safety of deferasirox in NTDT patients with iron overload (LIC ≥ 5 mg Fe/g liver dw and SF ≥ 300 ng/mL) over 260 weeks.

To assess the efficacy of deferasirox in patients with non-transfusion dependent thalassemia based on change in liver iron concentration from baseline after 24 and 52 . Also, to evaluate the impact of deferasirox on the Quality of Life for adults and pediatric patients; correlate changes in serum ferritin and LIC, evaluate efficacy changes according to genotype, evaluate higher doses of deferasirox, assess endocrine function and examine pk parameters.To also assess the efficacy of treatment in patients with very high LIC (>15 mg Fe/g dw) at baseline and those who need re-treatment after having reached the target LIC < 3 mg Fe/g dw during the study

Interventional
Phase 4
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Non-transfusion Dependent Thalassemia
Drug: ICL670 deferasirox
deferasirox
Deferasirox
one arm, deferasirox, LIC based dose titration
Intervention: Drug: ICL670 deferasirox
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
134
January 2020
January 2020   (final data collection date for primary outcome measure)

Inclusion Criteria:

Non-transfusion dependent congenital or chronic anemia inclusive of beta-thalassemia intermedia, HbE beta-thalassemia or alpha-thalassemia intermedia (HbH disease)/ Liver iron concentration >/= 5 mg Fe/g dw Serum Ferritin >/= 300 ng/mL -

Exclusion Criteria:

HbS-beta Thalassemia anticipated regular transfusion program during the study Blood transfusion 6 months prior to study start significant proteinuria Creatinine clearance >/= 40 ml/min ALT >5 x ULN active hepatitis B or C cirrhosis

-

Both
10 Years and older
No
Contact information is only displayed when the study is recruiting subjects
China,   Greece,   Italy,   Lebanon,   Thailand,   Tunisia,   Turkey,   United Kingdom
 
NCT01709838
CICL670E2419, 2012-000650-64
No
Novartis ( Novartis Pharmaceuticals )
Novartis Pharmaceuticals
Not Provided
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
Novartis
May 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP