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

Sponsor

Novartis Pharmaceuticals (Industry)

Overall Status

Completed

CT.gov ID

NCT01335035

Collaborator

(none)

Enrollment

Locations

Arm

3.3

Patients Per Site

Study Details

Study Description

Brief Summary

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.

Condition or Disease	Intervention/Treatment	Phase
Iron Overload	Drug: deferasirox	Phase 4

Study Design

Study Type:

Interventional

Actual Enrollment :

30 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Study Start Date :

Dec 1, 2008

Actual Primary Completion Date :

May 1, 2011

Arms and Interventions

Arm	Intervention/Treatment
Experimental: ICL670	Drug: deferasirox Other Names: ICL670

Arm

Intervention/Treatment

Experimental: ICL670

Drug: deferasirox

Other Names:

ICL670

Outcome Measures

Primary Outcome Measures

Mean change in serum ferritin [after 52 weeks of treatment with deferasirox]

Secondary Outcome Measures

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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	Country
1	Novartis Investigative Site	Andalucia	Spain
2	Novartis Investigative Site	Asturias	Spain
3	Novartis Investigative Site	Canarias	Spain
4	Novartis Investigative Site	Castilla y Leon	Spain
5	Novartis Investigative Site	Cataluna	Spain
6	Novartis Investigative Site	Comunidad Valenciana	Spain
7	Novartis Investigative Site	Islas baleares	Spain
8	Novartis Investigative Site	Madrid	Spain
9	Novartis Investigative Site	Murcia	Spain