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

Phase 4

Completed

• Conditions: Iron Overload
• Interventions: Drug: deferasirox

• Registration Number: NCT01335035
• Lead Sponsor: Novartis Pharmaceuticals

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2008-12
• Study First Submitted: 2011-03-30
• Study First Submitted QC: 2011-04-12
• Study First Posted: 2011-04-13
• Primary Completion: 2011-05
• Status Verified: 2012-04
• Last Update Submitted: 2012-04-27
• Last Update Posted: 2012-05-01

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 30

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)

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
ICL670	deferasirox	-

Primary Outcome Measures

Name	Time	Method
Mean change in serum ferritin	after 52 weeks of treatment with deferasirox

Secondary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (1)

Novartis Investigative Site; 🇪🇸 Murcia, Spain