Clinical trial NCT01610297: Post Hematopoietic Stem Cell Transplantation
Official 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.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major
Keywords: hematopoietic stem cell transplantation, beta-thalassemia major, deferasirox, iron overload
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2012-06-04
Enrollment: 27
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Number of Participants With Adverse Events, Serious Adverse Events and Deaths as a Measure of Safety and Tolerability
timeFrame: 12 months
Secondary outcomes:
measure: Change in Serum Ferritin Level.
timeFrame: Baseline, 12 Months
measure: Change in the Further Parameters of Iron Overload (Liver Iron Concentration by Magnetic Resonance Imaging (MRI Examination)
timeFrame: Baseline, 12 month
measure: The Percentage of Patients Reaching Serum Ferritin Levels Lower Than 500 μg/L
timeFrame: Week 28 and Week 52
measure: Change in the Further Parameters of Iron Overload (Cardiac Iron Concentration by Magnetic Resonance Imaging (MRI Examination)
timeFrame: Baseline, 12 month
Other outcomes:
Locations:
city: Ankara
country: Turkey
facility: Novartis Investigative Site
city: Ankara
country: Turkey
facility: Novartis Investigative Site
city: Antalya
country: Turkey
facility: Novartis Investigative Site
city: Antalya
country: Turkey
facility: Novartis Investigative Site
city: Istanbul
country: Turkey
facility: Novartis Investigative Site
city: Izmir
country: Turkey
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
citation: 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 beta-Thalassemia Major. Biol Blood Marrow Transplant. 2018 Mar;24(3):613-618. doi: 10.1016/j.bbmt.2017.11.006. Epub 2017 Nov 16.