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

Study Overview
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Brief Summary
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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.

Official Title
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Open-Label Single-Arm Pilot Study of Deferasirox (Exjade®) in Adult Allogeneic Hematopoietic Stem Cell Transplant Recipients With Transfusional Iron Overload

Conditions
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Iron Overload

Intervention / Treatment
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* Drug: deferasirox


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ICL670<br> | Drug: deferasirox<br> <br> * Other names: ICL670;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mean change in serum ferritin |  | after 52 weeks of treatment with deferasirox | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mean change in the no. of sideroblasts, assessed by Perls staining |  | after 52 weeks of treatment with deferasirox | 
| Mean change in liver iron concentration (LIC), assessed by liver MRI. |  | after 52 weeks of treatment with deferasirox | 
| Incidence of chronic graft-versus-host disease (limited or extensive, according to Shulman criteria) |  | up to 52 weeks of study | 
| Incidence of infections (bacterial, viral, or fungal) |  | up to 52 weeks of study | 
| Incidence of venous occlusive disease during the study |  | up to 52 weeks of study | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Iron metabolism,, Allogenetic Hematopoietic Stem Cell Transplant,, haematological diseases