Open-Label Single-Arm Pilot Study in Adult Allogeneic Hematopoietic Stem Cell Transplant Recipients With Transfusional Iron Overload

Novartis Pharmaceuticals30 enrolled

Overview

This study has been designed to establish the efficacy and safety of deferasirox in patients with iron overload from 6 to 18 months after allogeneic hematopoietic stem cell transplant (HSCT). The purpose of this study is to assess the mean change in serum ferritin after 52 weeks of treatment with deferasirox, in patients with iron overload (defined with serum ferritin levels ≥ 1000 ng/ml and receiving \> 20 RBC concentrates) after allogeneic HSCT.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Iron Overload

Interventions

deferasiroxDRUG

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Male or female patients 18 years of age and older * Patients to undergo allogeneic HSCT between 6 and 18 months prior to inclusion. * Patients with screening ANC \> 1000/mm3 and ferritin values ≥ 1000 ng/mL (ferritin values must be measured in two measurements at one-week intervals). * Patients receiving at least 20 RBC concentrate units or 100mL/kg RBC during their lives. * Patients giving their informed consent (prior to performing any study procedure) Exclusion Criteria: * Haemosiderosis not related to transfusion. * Patients with concomitant active malignancy. * Active known viral hepatitis or known HIV-positive. * Mean levels of alanine aminotransferase (ALT) \> 5x ULN * Treatment with any iron chelating agent after allogeneic HSCT. * Uncontrolled hypertension. Other protocol-defined inclusion/exclusion criteria may app

Locations (9)

Novartis Investigative Site

Andalucia, Spain

Novartis Investigative Site

Asturias, Spain

Novartis Investigative Site

Canarias, Spain

Novartis Investigative Site

Castilla Y Leon, Spain

Outcomes

Primary Outcomes

Mean change in serum ferritin

Time frame: after 52 weeks of treatment with deferasirox

Secondary Outcomes

Mean change in the no. of sideroblasts, assessed by Perls staining

Time frame: after 52 weeks of treatment with deferasirox

Mean change in liver iron concentration (LIC), assessed by liver MRI.

Time frame: after 52 weeks of treatment with deferasirox

Incidence of chronic graft-versus-host disease ("limited" or "extensive", according to Shulman criteria)

Time frame: up to 52 weeks of study

Incidence of infections (bacterial, viral, or fungal)

Time frame: up to 52 weeks of study

Incidence of venous occlusive disease during the study

Time frame: up to 52 weeks of study

Data from ClinicalTrials.gov

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