LeukemiaPhase 2REGALIANCT03948529
RevErsing Poor GrAft Function With eLtrombopag After allogeneIc Hematopoietic Cell trAnsplantation
A Phase 2 study testing eltrombopag in people with Leukemia and Graft Failure, run by University Hospital, Lille at 1 site in France.
Trial summary
Poor graft function (PGF) after allogeneic hematopoietic cell transplantation (allo-HCT) is a misunderstood complication associated with poor outcome and limited therapeutic options. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Leukemia, Graft Failure
- What is being tested?
- eltrombopag
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteFrance
- Study dates
- 5 Sep 2019to 21 Aug 2023
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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,
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- 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.
Who cannot join
- 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,
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- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: RevErsing Poor GrAft Function With eLtrombopag After allogeneIc Hematopoietic Cell trAnsplantation: a Prospective, Phase II Study by the SFGM-TC
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
- Conditions
- Leukemia, Graft Failure
- Being tested
- eltrombopag
- Keywords
- Poor Graft Function (PGF),, Allogeneic hematopoietic cell transplantation (Allo-HCT), Eltrombopag.
- Medical subject headings
- Leukemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
eltrombopagDrug
eltrombopag at the starting dose of 50mg/day. After 2 weeks of eltrombopag initiation and in the absence of platelet response, eltrombopag will be increased every two weeks (50mg increase) up to a maximum dose of 150mg/day (2 maximum escalation from D1, with maximum dose escalation phase of 4 weeks).
| Study group | Role | What they receive |
|---|---|---|
| eltrombopag | Experimental | Eligible patients will receive the investigational drug eltrombopag Receives: Drug: eltrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
Platelet response
Platelet response defined as a platelet count ≥ 30G/L at 12 weeks measured on at least two serial measurements performed 1 week apart and sustained for 1 month or more without support of platelet transfusions.
Measured: at 12 weeks
Secondary outcomes (8)
Time to erythroid response
Time to erythroid response defined as an increase of at least 1.5g/dL without transfusion, that is sustained for at least 2 weeks and transfusion requirements at 12 and 24 weeks for BRC as compared with transfusion requirements during the eight weeks preceding study entry
Measured: at 12 and 24 weeks
Time to neutrophil response
Time to neutrophil response defined as an increase of ANC above 1G/L, which is sustained for at least 7 days,
Measured: at least 7 days
Percentage of patients presenting best bone marrow response at 12 and 24 weeks of treatment assessed by bone marrow aspirate and bone marrow biopsy with fibrosis staining.
Measured: at 12 and 24 weeks
Transfusion requirements
Transfusion requirements for BRC and platelets as compared with transfusions requirements during the eight weeks preceding study entry
Measured: at 12 and 24 weeks
Proportion of patients presenting grade 3 or 4 adverse events from the first to the last administration of eltombopag.
All adverse events will be reported on the adverse events reporting form of the case report file. Each adverse event will be recorded individually.
The severity of the adverse event will be determined as follows :
- Severe (grade 3): significant interference with the patient's daily activity and unacceptable,
- Life-threatening (grade 4).
Measured: from 1st administration of eltrombopag to 1 month after the last administration of eltrombopag,
Quality of life evaluation using the European Organisation for Research Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30 questionnaire).
The QLQ-C30 consists of thirty items:
- 05 functional scales to explore the physical (1 to 5), executive (6 to 7), social (26 and 27), cognitive (20 and 25), and emotional (21 to 24) dimensions of the quality of life.
- 09 symptomatic scales to explore fatigue (10, 12 and 18), nausea symptoms (14 and 15), pain (9 and 19), dyspnea (8), insomnia (11), anorexia (13), diarrhea (17), constipation (16) and financial difficulties (28).
- 01 scale measuring the overall quality of life of each patient (29 and 30). The results of these different scales allow the calculation of a score that varies from 0 (worst) to 100 (better). A high overall health score reflects good health and a good quality of life. A high score for a symptom scale reflects a high level of symptoms. An average difference of 5 to 10 scores between two visits indicates a minor change, from 10 to 20 a moderate change and a difference of more than 20 points a significant change.
Measured: at 12 and 24 weeks
Immune function (T/B/NK cells counts)
Measured: at 12 and 24 weeks
Overall survival, relapse-free survival and non-relapse mortality
No relapse disease will be investigated using the Fine-Gray Test. No relapse mortality represent all death without relapse of the underlind disease.
Measured: at 24 weeks of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 May 2019
- Study start5 Sep 2019
- Primary completion24 Feb 2023
- Study completion21 Aug 2023
Why it stopped: inclusions difficulties.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for LeukemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Lille
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Ibrahim Yakoub-Agha, MD,PhD, Principal investigator, University Hospital, Lille
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03948529
- Sponsor's protocol ID
- 2017_52
- Other IDs
- 2018-001157-27, 2018
- Acronym
- REGALIA
- Status last verified
- Sep 2025
- First submitted
- 28 Feb 2019
- Last updated
- 30 Sep 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- RevErsing Poor GrAft Function With eLtrombopag After allogeneIc Hematopoietic Cell trAnsplantation: a Prospective, Phase II Study by the SFGM-TC
- ClinicalTrials.gov ID
- NCT03948529
- Acronym / sponsor ID
- REGALIA / 2017_52
- Conditions
- Leukemia, Graft Failure
- Keywords
- Poor Graft Function (PGF),, Allogeneic hematopoietic cell transplantation (Allo-HCT), Eltrombopag.
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: inclusions difficulties.
- Enrolment
- 9 participants (actual)
- Lead sponsor
- University Hospital, LilleUniversity, hospital or other
- Study start
- 5 Sep 2019 (actual)
- Primary completion
- 24 Feb 2023 (actual)
- Study completion
- 21 Aug 2023 (actual)
- First posted
- 14 May 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| eltrombopag | Experimental | Drug: eltrombopagEligible patients will receive the investigational drug eltrombopag |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (17 listed)
- Diagnosis of poor graft function defined as:
- Written informed consent must be obtained before any study-trial specific procedure are performed,
- Affiliation to a social security system.
Key exclusion criteria (13 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| eltrombopageltrombopag at the starting dose of 50mg/day. After 2 weeks of eltrombopag initiation and in the absence of platelet response, eltrombopag will be increased every two weeks (50mg increase) up to a maximum dose of 150mg/day (2 maximum escalation from D1, with maximum dose escalation phase of 4 weeks). | Drug | eltrombopag |
Primary outcome
Platelet response
Time frame: at 12 weeks
Platelet response defined as a platelet count ≥ 30G/L at 12 weeks measured on at least two serial measurements performed 1 week apart and sustained for 1 month or more without support of platelet transfusions.
8 secondary outcomes
Time to erythroid response
Time frame: at 12 and 24 weeks
Time to neutrophil response
Time frame: at least 7 days
Percentage of patients presenting best bone marrow response at 12 and 24 weeks of treatment assessed by bone marrow aspirate and bone marrow biopsy with fibrosis staining.
Time frame: at 12 and 24 weeks
Transfusion requirements
Time frame: at 12 and 24 weeks
Proportion of patients presenting grade 3 or 4 adverse events from the first to the last administration of eltombopag.
Time frame: from 1st administration of eltrombopag to 1 month after the last administration of eltrombopag,
Quality of life evaluation using the European Organisation for Research Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30 questionnaire).
Time frame: at 12 and 24 weeks
Immune function (T/B/NK cells counts)
Time frame: at 12 and 24 weeks
Overall survival, relapse-free survival and non-relapse mortality
Time frame: at 24 weeks of treatment
Results and safety
The study was stopped early (reason given: inclusions difficulties.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Sep 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ibrahim Yakoub-Agha, MD,PhD principal investigator
University Hospital, Lille
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing eltrombopag in people with Leukemia and Graft Failure, run by University Hospital, Lille at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 30 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: inclusions difficulties.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Platelet response (time frame: at 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Sep 2025.