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Post Hematopoietic Stem Cell Transplantation

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ClinicalTrials.gov Identifier: NCT01610297
Recruitment Status : Completed
First Posted : June 4, 2012
Results First Posted : July 21, 2016
Last Update Posted : October 24, 2016
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Tracking Information
First Submitted Date  ICMJE May 30, 2012
First Posted Date  ICMJE June 4, 2012
Results First Submitted Date  ICMJE April 21, 2016
Results First Posted Date  ICMJE July 21, 2016
Last Update Posted Date October 24, 2016
Study Start Date  ICMJE September 2013
Actual Primary Completion Date October 2015   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 9, 2016)
Number of Participants With Adverse Events, Serious Adverse Events and Deaths as a Measure of Safety and Tolerability [ Time Frame: 12 months ]
To determine the safety; incidence, type and severity of adverse events including renal, hepatic, biochemistry and hematologic parameters of deferasirox in the treatment of iron overload after hematopoietic stem cell transplantation (HSCT) in patients with beta-thalassemia major in 12 months period
Original Primary Outcome Measures  ICMJE
 (submitted: June 1, 2012)
•Safety of deferasirox in the treatment of iron overload after HSCT in patients with beta-thalassemia major [ Time Frame: 12 months ]
• To determine the safety; incidence, type and severity of adverse events including renal, hepatic, biochemistry and hematologic parameters of deferasirox in the treatment of iron overload after HSCT in patients with beta-thalassemia major in 12 months period
Change History Complete list of historical versions of study NCT01610297 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: September 12, 2016)
  • Change in Serum Ferritin Level. [ Time Frame: Baseline, 12 Months ]
    Blood samples were collected and serum levels were assessed at study baseline (BL) and at 12 months.
  • Change in the Further Parameters of Iron Overload (Liver Iron Concentration by Magnetic Resonance Imaging (MRI Examination) [ Time Frame: Baseline, 12 month ]
    Liver Iron Concentration (LIC) values between 3 and 7 mg Fe / g dry weight are indicative of mild iron deposition, while values between 7 and 15 mg Fe / g dry weight are indicative of moderate iron deposition which have been associated with liver disease. Values >15 mg Fe/g dry weight are indicative of severe iron deposition which is associated with progressive liver fibrosis, increased morbidity and mortality
  • The Percentage of Patients Reaching Serum Ferritin Levels Lower Than 500 μg/L [ Time Frame: Week 28 and Week 52 ]
    Serum Ferritin values between 1000-2500 μg/L are indicative of mild to moderate iron overload while values >2500 μg/L are indicative of severe iron overload and levels constantly higher than 2500 μg/L has been shown to to increase the risk of cardiac complications and endocrine disease. Maintaining levels <1000 μg/L is associated with increased survival and less morbidity.
  • Change in the Further Parameters of Iron Overload (Cardiac Iron Concentration by Magnetic Resonance Imaging (MRI Examination) [ Time Frame: Baseline, 12 month ]
    Cardiac MRI values between 10 to 20 milliseconds (ms) are indicative of moderate cardiac iron deposition associated with declining left ventricular ejection fraction and arrhythmias while values <10 ms are indicative of deposition sufficient to risk cardiac decompensation and associated with overt heart failure and mortality.
Original Secondary Outcome Measures  ICMJE
 (submitted: June 1, 2012)
• Efficacy of deferasirox in the treatment of iron overload after HSCT in patients with beta-thalassemia major by 12th month. [ Time Frame: 12 Months ]
• To evaluate change the serum ferritin level and change from baseline to 12th month • To evaluate change in the further parameters of iron overload (cardiac iron and liver iron concentration by MR examination) from baseline to 12th month • To determine the percentage of patients reaching serum ferritin levels lower than 500 µg/L at week 28 and at week 52.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Post Hematopoietic Stem Cell Transplantation
Official Title  ICMJE A Phase II, Multi-center, Single-arm, Prospective Study to Evaluate the Safety and Efficacy of Deferasirox in Beta-thalassemia Major Patients After Hematopoietic Stem Cell Transplantation.
Brief Summary This was a prospective, single-arm, multicenter, national, phase II clinical study. The purpose of this Phase II study was to examine the safety and efficacy of deferasirox to decrease iron overload (IOL) in the posttransplant period in patients with beta-thalassemia major.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major
Intervention  ICMJE Drug: ICL670
Oral dose of ICL670 at 10 mg/kg daily
Study Arms  ICMJE Experimental: ICL670
Oral dose of ICL670 at 10 mg/kg daily
Intervention: Drug: ICL670
Publications * Yesilipek MA, Karasu G, Kaya Z, Kuskonmaz BB, Uygun V, Dag I, Ozudogru O, Ertem M. A Phase II, Multicenter, Single-Arm Study to Evaluate the Safety and Efficacy of Deferasirox after Hematopoietic Stem Cell Transplantation in Children with β-Thalassemia Major. Biol Blood Marrow Transplant. 2018 Mar;24(3):613-618. doi: 10.1016/j.bbmt.2017.11.006. Epub 2017 Nov 16.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: June 29, 2015)
27
Original Estimated Enrollment  ICMJE
 (submitted: June 1, 2012)
30
Actual Study Completion Date  ICMJE October 2015
Actual Primary Completion Date October 2015   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria

  • Patients who had hematopoietic stem cell transplantation (HSCT) for beta-thalassemia major
  • hematopoietic stem cell transplantation (HSCT) was performed minimum 6 months and maximum 2 years ago
  • The washout period after the immunosuppressive therapy should be at least 3 months.
  • Signifacant IOL should be present including:

A. Serum ferritin >1000 μg/L or B. cardiac MRI <20 ms or C. liver iron concentration ≥ 5 mg/g dry weight measured by R2* MRI

Exclusion Criteria

  • Patients who had any contraindication for treatment with deferasirox according to the prescribing information

    •Patients who depended on transfusion

  • Patients with clinical symptoms of cardiac dysfunction (shortness of breath at rest or exertion, orthopnea, exercise intolerance, lower extremity edema, arrhythmias)
  • Patients who were experiencing severe complication of HSCT e.g. acute Graft-versus host disease (GVHD)
  • Significant proteinuria / Increase in serum creatinine
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Turkey
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01610297
Other Study ID Numbers  ICMJE CICL670ATR04
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Novartis ( Novartis Pharmaceuticals )
Study Sponsor  ICMJE Novartis Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
PRS Account Novartis
Verification Date September 2016

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