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
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Leukemia Graft Failure | Drug: eltrombopag | Phase 2 |
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 25 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | RevErsing Poor GrAft Function With eLtrombopag After allogeneIc Hematopoietic Cell trAnsplantation: a Prospective, Phase II Study by the SFGM-TC |
| Actual Study Start Date : | September 5, 2019 |
| Estimated Primary Completion Date : | December 2021 |
| Estimated Study Completion Date : | December 2021 |
| Arm | Intervention/treatment |
|---|---|
|
Experimental: eltrombopag
Eligible patients will receive the investigational drug eltrombopag
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Drug: eltrombopag
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).
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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 :
The QLQ-C30 consists of thirty items:
| Ages Eligible for Study: | 6 Years and older (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Diagnosis of poor graft function defined as:
Exclusion Criteria:
| Contact: Ibrahim Yakoub-Agha, MD,PhD | (0)3.20.44.55.51 ext +33 | ibrahim.yakoubagha@chru-lille.fr |
| France | |
| Hôpital Claude Huriez, CHU | Recruiting |
| Lille, France | |
| Principal Investigator: | Ibrahim Yakoub-Agha, MD,PhD | University Hospital, Lille |
| Tracking Information | |||||
|---|---|---|---|---|---|
| First Submitted Date ICMJE | February 28, 2019 | ||||
| First Posted Date ICMJE | May 14, 2019 | ||||
| Last Update Posted Date | November 19, 2020 | ||||
| Actual Study Start Date ICMJE | September 5, 2019 | ||||
| Estimated Primary Completion Date | December 2021 (Final data collection date for primary outcome measure) | ||||
| Current Primary Outcome Measures ICMJE |
Platelet response [ Time Frame: at 12 weeks ] Platelet response defined as a platelet count ≥ 20G/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.
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| Original Primary Outcome Measures ICMJE | Same as current | ||||
| Change History | |||||
| Current Secondary Outcome Measures ICMJE |
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| Original Secondary Outcome Measures ICMJE | Same as current | ||||
| Current Other Pre-specified Outcome Measures | Not Provided | ||||
| Original Other Pre-specified Outcome Measures | Not Provided | ||||
| Descriptive Information | |||||
| Brief Title ICMJE | RevErsing Poor GrAft Function With eLtrombopag After allogeneIc Hematopoietic Cell trAnsplantation | ||||
| Official Title ICMJE | RevErsing Poor GrAft Function With eLtrombopag After allogeneIc Hematopoietic Cell trAnsplantation: a Prospective, Phase II Study by the SFGM-TC | ||||
| Brief 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 |
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| Detailed Description | Not Provided | ||||
| Study Type ICMJE | Interventional | ||||
| Study Phase ICMJE | Phase 2 | ||||
| Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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| Condition ICMJE |
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| Intervention ICMJE | Drug: eltrombopag
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).
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| Study Arms ICMJE | Experimental: eltrombopag
Eligible patients will receive the investigational drug eltrombopag
Intervention: Drug: eltrombopag
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| Publications * | Not Provided | ||||
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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| Recruitment Information | |||||
| Recruitment Status ICMJE | Recruiting | ||||
| Estimated Enrollment ICMJE |
25 | ||||
| Original Estimated Enrollment ICMJE | Same as current | ||||
| Estimated Study Completion Date ICMJE | December 2021 | ||||
| Estimated Primary Completion Date | December 2021 (Final data collection date for primary outcome measure) | ||||
| Eligibility Criteria ICMJE |
Inclusion Criteria:
Exclusion Criteria:
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| Sex/Gender ICMJE |
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| Ages ICMJE | 6 Years and older (Child, Adult, Older Adult) | ||||
| Accepts Healthy Volunteers ICMJE | No | ||||
| Contacts ICMJE |
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| Listed Location Countries ICMJE | France | ||||
| Removed Location Countries | |||||
| Administrative Information | |||||
| NCT Number ICMJE | NCT03948529 | ||||
| Other Study ID Numbers ICMJE | 2017_52 2018-001157-27 ( EudraCT Number ) |
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| Has Data Monitoring Committee | Yes | ||||
| U.S. FDA-regulated Product |
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| IPD Sharing Statement ICMJE | Not Provided | ||||
| Responsible Party | University Hospital, Lille | ||||
| Study Sponsor ICMJE | University Hospital, Lille | ||||
| Collaborators ICMJE | Not Provided | ||||
| Investigators ICMJE |
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| PRS Account | University Hospital, Lille | ||||
| Verification Date | November 2020 | ||||
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ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |
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