Study of Efficacy and Safety of Eltrombopag in Patients With Poor Graft Function

The ELTION Study - A Multicenter Open-label Interventional Study of Eltrombopag in Patients With Poor Graft Function After Allogeneic Hematopoietic Stem Cell Transplantation

ClinicalTrials.gov Identifier: NCT03718533

Novartis Reference Number: CETB115EES03

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All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. 

Study Description

The purpose of this phase II open-label, single-arm 36-week clinical trial is to evaluate the effect of eltrombopag in patients with poor graft function (PGF) after allogeneic-hematopoietic stem cells transplantation (allo-HSCT).

Condition 
Poor Graft Function
Phase 
Phase 2
Overall status 
Active, not recruiting
Enrollment count 
17 participants
Start date 
Dec 17, 2018
Completion date 
Nov 27, 2020
Gender 
All
Age(s)
18 Years - 100 Years (Adult, Older Adult)

Interventions

Drug
Eltrombopag
50mg Film-coated tablet and 25mg Film-coated tablet for oral use administration

Eligibility Criteria

Inclusion Criteria:

Patient must be able to understand and communicate with the investigator and comply with the requirements of the study and must provide written, signed and dated informed consent form before any study assessment is performed
Male of female patients ≥ 18 years of age

Patients diagnosed with primary or secondary poor graft function (PGF) defined as two or more cytopenias after day +30 post-transplant (re-tested in a peripheral blood analysis at screening):

Platelet count <20,000/ µL (mandatory)
Absolute neutrophil count (ANC) <1,000/µL
Hemoglobin <100 g/L
Presence of donor chimerism >90% in screening visit
Karnofsky status ≥90% (Karnofsky assessment must be performed within 7 days prior to Day 1)

Exclusion Criteria:

Pregnant or nursing (lactating women).
Evidence of active acute or chronic graft versus host disease (GVHD).
Evidence of any active malignancy.
Subjects who are human immune deficiency virus (HIV), hepatitis C virus (HCV), hepatitis B surface antigen (HBsAg) positive in screening visit.
Cytogenetic abnormality in chromosome 7 present before the allo-HSC.

Evidence of any clonal abnormality on cytogenetics (in bone marrow analysis).

A local post-transplant conventional cytogenetic assessment should be available within 8 weeks before Day 1.
If the cytogenetics is not valuable, i.e, it does not show metaphases, a FISH for MDS-related most frequent abnormalities including chromosome 7 is accepted.

As a consequence, patients with dry tap bone marrow aspiration are NOT eligible.

Evidence of bone marrow involvement or progression of the underlying disease assessed by the applicable methods in each case.
Evidence of thrombotic microangiopathy.
Evidence of possible causes of cytopenia other than PGF (active infections, myelotoxic drugs, hypersplenism…).
Prior use of any thrombopoietin receptor (TPO-R) agonists for PGF.
AST or ALT levels >3 x ULN.
Creatinine level ≥1.5 x ULN.
Total bilirubin level ≥1.5 x ULN.
Previous thromboembolic event (other than line-related upper extremity thrombosis)
Hypersensitivity to eltrombopag or its components.

Clinically significant ECG abnormality history or current diagnosis of cardiac disease indicating significant risk of safety for subjects participating in the study such as uncontrolled or significant cardiac disease or impaired cardiac function including any of the following:

. Corrected QTc > 450 msec (male subjects), > 460 msec (female subjects) using Fredericia correction (QTcF) on the screening ECG
. Myocardial infarction
. Uncontrolled congestive heart failure
. Unstable angina
. Congenital long QT syndrome.
Administration of an investigational drug within 30 days or 5 half-lives, whichever is longer, preceding the first dose of study treatment.
Patient with liver cirrhosis.
Risk factors for Torsade de Pointes including uncorrected hypokalemia or hypomagnesemia.
Subjects with any serious and/ or unstable pre-existing medical, psychiatric disorder or other conditions that could interfere with patient´s safety, obtaining informed consent or compliance with the study procedures as per investigator discretion.

Study Locations

Spain
Novartis Investigative Site
-
Malaga, 29010
Andalucia
Spain
Novartis Investigative Site
-
Salamanca, 37007
Castilla Y Leon
Spain
Novartis Investigative Site
-
Valencia, 46010
Comunidad Valenciana
Spain
Novartis Investigative Site
-
Palma De Mallorca, 07120
Islas Baleares
Spain
Novartis Investigative Site
-
San Sebastian, 20080
Pais Vasco
Spain
Novartis Investigative Site
-
Vigo, 36212
Pontevedra
Spain
Novartis Investigative Site
-
Barcelona, 08041
-
Spain
Novartis Investigative Site
-
Madrid, 28034
-
Spain

Have a question?

Call 1-999-669-6682 or email [email protected]