Post Hematopoietic Stem Cell Transplantation

Novartis

Status and phase

Completed

Phase 4

Conditions

Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major

Treatments

Drug: ICL670

Study type

Interventional

Funder types

Industry

Identifiers

NCT01610297

CICL670ATR04

Details and patient eligibility

About

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.

Enrollment

27 patients

Sex

All

Ages

2+ years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion 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