TerminatedPhase 2

RevErsing Poor GrAft Function With eLtrombopag After allogeneIc Hematopoietic Cell trAnsplantation

Stopped: inclusions difficulties.

France9 participantsStarted 2019-09-05

Plain-language summary

Poor graft function (PGF) after allogeneic hematopoietic cell transplantation (allo-HCT) is a misunderstood complication associated with poor outcome and limited therapeutic options. Despite the lack of standardized diagnostic criteria, 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. Not only PGF can alter patients' quality of life by leading to recurrent transfusions, bleeding events and infections, but it is also associated with poor survival after allo-HCT. Although PGF is relatively frequent, there is no well-codified behavior in the literature or in the recommendations issued by the various learned societies of transplantation. The aim objective of the investigator's study is to demonstrate that eltrombopag improve PGF after allo-HCT

Who can participate

Age range

6 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * 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 \< 30G/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. * Written informed consent must be obtained before any study-trial specific procedure are performed, * Affiliation to a social security system. 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), * Patients with bone morrow fibrosis, * Patients with a cytogenetic abnormality of chromosome 7 * 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…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Platelet response

Timeframe: at 12 weeks

Trial details

NCT IDNCT03948529

SponsorUniversity Hospital, Lille

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2023-02-24

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