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Completed

NCT Number: NCT01610297

Post Hematopoietic Stem Cell Transplantation

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.

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Key information

Conditions

Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Iron Metabolism Disorders Iron Overload Metabolic Diseases Nutritional and Metabolic Diseases Thalassemia beta-Thalassemia

Age range

2 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Novartis Investigative Site, Ankara, Turkey, Turkey (Türkiye)

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

ICL670

Drug

Oral dose of ICL670 at 10 mg/kg daily

Primary outcomes

  1. 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

Secondary outcomes

  1. 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.

  2. 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

  3. 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.

  4. 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.

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

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.

Important dates

Study start
2013
Primary completion
2015
Study completion
2015
First posted
Jun 4, 2012
Registry last updated
Oct 24, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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