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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01335035
First received: March 30, 2011
Last updated: April 27, 2012
Last verified: April 2012
  Purpose

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 Intervention Phase
Iron Overload
Drug: deferasirox
Phase 4

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • Mean change in serum ferritin [ Time Frame: after 52 weeks of treatment with deferasirox ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Mean change in the no. of sideroblasts, assessed by Perls staining [ Time Frame: after 52 weeks of treatment with deferasirox ] [ Designated as safety issue: No ]
  • Mean change in liver iron concentration (LIC), assessed by liver MRI. [ Time Frame: after 52 weeks of treatment with deferasirox ] [ Designated as safety issue: No ]
  • Incidence of chronic graft-versus-host disease ("limited" or "extensive", according to Shulman criteria) [ Time Frame: up to 52 weeks of study ] [ Designated as safety issue: Yes ]
  • Incidence of infections (bacterial, viral, or fungal) [ Time Frame: up to 52 weeks of study ] [ Designated as safety issue: Yes ]
  • Incidence of venous occlusive disease during the study [ Time Frame: up to 52 weeks of study ] [ Designated as safety issue: Yes ]

Enrollment: 30
Study Start Date: December 2008
Primary Completion Date: May 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ICL670 Drug: deferasirox
Other Name: ICL670

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01335035

Locations
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, Spain
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01335035     History of Changes
Other Study ID Numbers: CICL670AES04, EudraCT: 2008-003207-30
Study First Received: March 30, 2011
Last Updated: April 27, 2012
Health Authority: Spain: Agencia Española del Medicamento y Productos Sanitarios

Keywords provided by Novartis:
Iron metabolism,
Allogenetic Hematopoietic Stem Cell Transplant,
haematological diseases

Additional relevant MeSH terms:
Iron Overload
Iron Metabolism Disorders
Metabolic Diseases
Deferasirox
Iron Chelating Agents
Chelating Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on July 20, 2014