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

Iron Overload Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01335035
• Other study ID #: CICL670AES04
• Secondary ID: EudraCT: 2008-00
• Status: Completed
• Phase: Phase 4
• First received: March 30, 2011
• Last updated: April 27, 2012
• Start date: December 2008

Study information

• Verified date: April 2012
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Health authority: Spain: Agencia Española del Medicamento y Productos Sanitarios
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 30
• Est. primary completion date: May 2011
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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

Study Design

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Iron Overload

Intervention

Drug:
• deferasirox

Locations

Country	Name	City	State
Spain	Novartis Investigative Site	Andalucia
Spain	Novartis Investigative Site	Asturias
Spain	Novartis Investigative Site	Canarias
Spain	Novartis Investigative Site	Castilla y Leon
Spain	Novartis Investigative Site	Cataluna
Spain	Novartis Investigative Site	Comunidad Valenciana
Spain	Novartis Investigative Site	Islas baleares
Spain	Novartis Investigative Site	Madrid
Spain	Novartis Investigative Site	Murcia

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Country where clinical trial is conducted

Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Mean change in serum ferritin		after 52 weeks of treatment with deferasirox	No
Secondary	Mean change in the no. of sideroblasts, assessed by Perls staining		after 52 weeks of treatment with deferasirox	No
Secondary	Mean change in liver iron concentration (LIC), assessed by liver MRI.		after 52 weeks of treatment with deferasirox	No
Secondary	Incidence of chronic graft-versus-host disease ("limited" or "extensive", according to Shulman criteria)		up to 52 weeks of study	Yes
Secondary	Incidence of infections (bacterial, viral, or fungal)		up to 52 weeks of study	Yes
Secondary	Incidence of venous occlusive disease during the study		up to 52 weeks of study	Yes