Clinical trial NCT01335035: 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
Overall status: COMPLETED
Phase: PHASE4
Conditions: Iron Overload
Keywords: Iron metabolism,
Allogenetic Hematopoietic Stem Cell Transplant,
haematological diseases
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2011-04-13
Enrollment: 30
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Mean change in serum ferritin
timeFrame: after 52 weeks of treatment with deferasirox
Secondary outcomes:
measure: Mean change in the no. of sideroblasts, assessed by Perls staining
timeFrame: after 52 weeks of treatment with deferasirox
measure: Mean change in liver iron concentration (LIC), assessed by liver MRI.
timeFrame: after 52 weeks of treatment with deferasirox
measure: Incidence of chronic graft-versus-host disease ("limited" or "extensive", according to Shulman criteria)
timeFrame: up to 52 weeks of study
measure: Incidence of infections (bacterial, viral, or fungal)
timeFrame: up to 52 weeks of study
measure: Incidence of venous occlusive disease during the study
timeFrame: up to 52 weeks of study
Other outcomes:
Locations:
city: Andalucia
country: Spain
facility: Novartis Investigative Site
city: Asturias
country: Spain
facility: Novartis Investigative Site
city: Canarias
country: Spain
facility: Novartis Investigative Site
city: Castilla y Leon
country: Spain
facility: Novartis Investigative Site
city: Cataluna
country: Spain
facility: Novartis Investigative Site
city: Comunidad Valenciana
country: Spain
facility: Novartis Investigative Site
city: Islas baleares
country: Spain
facility: Novartis Investigative Site
city: Madrid
country: Spain
facility: Novartis Investigative Site
city: Murcia
country: Spain
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
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.