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

Primary Purpose

Iron Overload

Status
Completed
Phase
Phase 4
Locations
Spain
Study Type
Interventional
Intervention
deferasirox
Sponsored by
Novartis Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Iron Overload focused on measuring Iron metabolism,, Allogenetic Hematopoietic Stem Cell Transplant,, haematological diseases

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

ICL670

Arm Description

Outcomes

Primary Outcome Measures

Mean change in serum ferritin

Secondary Outcome Measures

Mean change in the no. of sideroblasts, assessed by Perls staining
Mean change in liver iron concentration (LIC), assessed by liver MRI.
Incidence of chronic graft-versus-host disease ("limited" or "extensive", according to Shulman criteria)
Incidence of infections (bacterial, viral, or fungal)
Incidence of venous occlusive disease during the study

Full Information

First Posted
March 30, 2011
Last Updated
April 27, 2012
Sponsor
Novartis Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT01335035
Brief Title
Open-Label Single-Arm Pilot Study in Adult Allogeneic Hematopoietic Stem Cell Transplant Recipients With Transfusional Iron Overload
Official Title
Open-Label Single-Arm Pilot Study of Deferasirox (Exjade®) in Adult Allogeneic Hematopoietic Stem Cell Transplant Recipients With Transfusional Iron Overload
Study Type
Interventional

2. Study Status

Record Verification Date
April 2012
Overall Recruitment Status
Completed
Study Start Date
December 2008 (undefined)
Primary Completion Date
May 2011 (Actual)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals

4. Oversight

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Iron Overload
Keywords
Iron metabolism,, Allogenetic Hematopoietic Stem Cell Transplant,, haematological diseases

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
30 (Actual)

8. Arms, Groups, and Interventions

Arm Title
ICL670
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
deferasirox
Other Intervention Name(s)
ICL670
Primary Outcome Measure Information:
Title
Mean change in serum ferritin
Time Frame
after 52 weeks of treatment with deferasirox
Secondary Outcome Measure Information:
Title
Mean change in the no. of sideroblasts, assessed by Perls staining
Time Frame
after 52 weeks of treatment with deferasirox
Title
Mean change in liver iron concentration (LIC), assessed by liver MRI.
Time Frame
after 52 weeks of treatment with deferasirox
Title
Incidence of chronic graft-versus-host disease ("limited" or "extensive", according to Shulman criteria)
Time Frame
up to 52 weeks of study
Title
Incidence of infections (bacterial, viral, or fungal)
Time Frame
up to 52 weeks of study
Title
Incidence of venous occlusive disease during the study
Time Frame
up to 52 weeks of study

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis Pharmaceuticals
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Novartis Investigative Site
City
Andalucia
Country
Spain
Facility Name
Novartis Investigative Site
City
Asturias
Country
Spain
Facility Name
Novartis Investigative Site
City
Canarias
Country
Spain
Facility Name
Novartis Investigative Site
City
Castilla y Leon
Country
Spain
Facility Name
Novartis Investigative Site
City
Cataluna
Country
Spain
Facility Name
Novartis Investigative Site
City
Comunidad Valenciana
Country
Spain
Facility Name
Novartis Investigative Site
City
Islas baleares
Country
Spain
Facility Name
Novartis Investigative Site
City
Madrid
Country
Spain
Facility Name
Novartis Investigative Site
City
Murcia
Country
Spain

12. IPD Sharing Statement

Citations:
PubMed Identifier
24997153
Citation
Vallejo C, Batlle M, Vazquez L, Solano C, Sampol A, Duarte R, Hernandez D, Lopez J, Rovira M, Jimenez S, Valcarcel D, Belloch V, Jimenez M, Jarque I; Subcommittee of Non-Infectious Complications of the Grupo Espanol de Trasplante Hematopoyetico (GETH). Phase IV open-label study of the efficacy and safety of deferasirox after allogeneic stem cell transplantation. Haematologica. 2014 Oct;99(10):1632-7. doi: 10.3324/haematol.2014.105908. Epub 2014 Jul 4.
Results Reference
derived

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

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