Eltrombopag in treatment of poor graft function.

Phase 1

• Conditions: Poor Graft Function (PGF) after allogeneic hematopoietic cell transplantation (allo-HCT). PGF is commonly defined as follows: one or several significant cytopenias after allo-HCT persisting or developing after allo-HCT despite full donor chimerism and in the absence of relapse or other causes (in particular graft-versus-host disease [GVHD]).; Therapeutic area: Diseases [C] - Cancer [C04]

• Registration Number: EUCTR2018-001157-27-FR
• Lead Sponsor: CHRU of Lille

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-10-09
• Last Update Posted: 28 February 2019

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 25

Inclusion Criteria

- Patient age = 6 years old
- Diagnosis of poor graft function defined as:
• Patient = day+60 after allo-HCT,
• Persisting thrombocytopenia on two different samples over at least two weeks (platelet < 20G/L with transfusion requirement) +/- neutropenia (ANC <1G/L) +/- anemia (Hb <8g/dL or transfusion requirement),
• Full donor chimerism on whole blood (= 95%),
• Biopsy proven hypocellular marrow without evidence of myelodysplasia
• No evidence for relapse,
• No evidence for active acute or chronic graft versus host disease,
• Absence of active viral infection (EBV, CMV, ADENOVIRUS, PARVOVIRUS B19),
• Absence of B9/B12 deficiency,
• Absence of hypothyroidism,
• Absence of hypogonadism,
• Absence of dialysis,
• Absence of thrombotic microangiopathy,
• Absence of macrophage activation syndrome,
• No other known causes of poor graft function.

Are the trial subjects under 18? yes
Number of subjects for this age range: 5
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 15
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5

Exclusion Criteria

• Criteria for poor graft function not fulfilled (see above),
• Patients aged less than 6 years old (or unable to swallow),
• Hepatic impairment (Child-Pugh = 5),
• Hypersensitivity to eltrombopag or to any of the excipients,
• Patients with any contra-indication to eltrombopag, filgrastim,
• Unable to understand the investigational nature of the study or give informed consent,
• History of congestive heart failure, arrhythmia requiring chronic treatment, arterial or venous,
• Thrombosis (not excluding line thrombosis) within the last 1 year, or myocardial infarction within 3 months before enrollment,
• ECOG Performance Status of 3 or greater,
• Pregnant and/or lactating women,
• Freedom privacy.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified