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Stem Cell Transplant ComplicationsPhase 2/3NCT05466201

The Use of Eltrombopag Post HSCT in BMFD

A Phase 2/3 study testing Eltrombopag 25 MG Oral Tablet and Supportive care in people with Stem Cell Transplant Complications, run by The First Affiliated Hospital of Soochow University at 1 site in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Bone marrow failure disease(BMFD) is a kind of bone marrow due to congenital or acquired hematopoietic stem cells (hemopoietic stem cell, HSC) function damage. Allogenic hemopoietic stem cell transplantation (Allo-HSCT) might be the most possible treatment to cure the disease.However, 5-26% of patients have been… Read more about this study

Who is it for?
Ages 12 to 65 years
Conditions the study is for
Stem Cell Transplant Complications
What is being tested?
Eltrombopag 25 MG Oral Tablet and Supportive care
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
118 participants
Where
1 siteChina
Study dates
22 Oct 2022to 31 Oct 2025
Phase
Phase 2/3
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 12 to 65 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Patients diagnosed as bone marrow failure disease who received allo-HSCT; Physical strength score 0-3 according to WHO standard

Who cannot join

  • single or double umbilical cord blood transplantation;
  • allergic to any of the research drugs involved in the protocol;
  • simultaneously suffering from another malignant tumor;
  • pregnant or lactating women;
  • participating in other clinical researchers at the same time;
  • patients with at least one following high risk factors of thrombosis: past medical history of thromboembolism, concurrent grade 2 to 3 hypertension (systolic BP>=160mmHg or diastolic BP>=100mmHg) , diabetes, obesity(BMI>30), family history of stroke, smoke for more than 10 years , or history of catheter thrombosis;
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  • severe cataract;
  • Severe infectious diseases (uncured tuberculosis, pulmonary aspergillosis, viral infection, active hepatitis B/C; for positive HBsAg and HBcAg, patient is excluded if hepatitis B DNA nucleic acid test is positive, DNA negative patients can enter this clinical trial; patients with hepatitis C who have a positive hepatitis C RNA nucleic acid test are excluded).;
  • Abnormal liver and kidney function: creatinine level ≥177 μmol/l (1.5mg/dl), transaminase and bilirubin levels increased significantly (3 times or more than the upper limit of normal), and who cannot be enrolled at the discretion of clinician.
  • In moribund condition or concurrent severe liver, kidney, heart, nerve, lung, infectious or metabolic diseases, the severity of which will cause the patient to be unable to tolerate the treatment regimen, or may die within 7-10 days

Trial locations and contacts

1 site in 1 country.

China1 site

  • The First Affiliated Hospital of Soochow University

    Suzhou, Jiangsu, 215006

Other trials in China

About this study

Official title: A Multicenter Clinical Study of a TPO Receptor Agonist (Eltrombopag) in the Acceleration of Engraftment Post Hematopoietic Stem Cell Transplantation of Bone Marrow Failure Diseases

Bone marrow failure disease(BMFD) is a kind of bone marrow due to congenital or acquired hematopoietic stem cells (hemopoietic stem cell, HSC) function damage. Allogenic hemopoietic stem cell transplantation (Allo-HSCT) might be the most possible treatment to cure the disease.However, 5-26% of patients have been reported to have delayed platelet engraftment (DPE), which is defined as persistent severe thrombocytopenia (<20 × 109/L) for >35 days after transplantation . To date, no standard treatment and prevention has been recommended for DPE. In patients with DPE, the amount of transfusion, the increased risk of infection, and the prolonged average hospital stay were independent risk factors affecting the prognosis of allo-HSCT patients. Due to continuous and progressive failure in the bone marrow hematopoiesis, thrombocytopenia post HSCT is more common in BMFD patients and often achieves low response to conventional therapy, such as platelet transfusion. Therefore, it is of great significance to effectively promote hematopoietic reconstruction to improve the prognosis of transplant patients.

Read the detailed description

Bone marrow failure disease(BMFD) is a kind of bone marrow due to congenital or acquired hematopoietic stem cells (hemopoietic stem cell, HSC) function damage. Generally, BMFD can be divided into two categories based on pathogenesis, which is primary and secondary BMFD. The latter is commonly seen secondary to infection, cancer, drugs, while aplastic anemia(AA), hypoplastic myelodysplastic syndromes(hMDS), paroxysmal nocturnal haemoglobinuria(PNH) and Fanconi anaemia(FA) are included in primary BMFD. Although immunotherapy or allogenic hemopoietic stem cell transplantation (Allo-HSCT) can be selected based on different individuals, allo-HSCT is still the most effective treatment for diseases that pose a greater threat to life or have a higher degree of malignancy, such as sereve aplastic anemia(SAA), MDS and PNH. Patients undergoing allo-HSCT typically achieve neutrophil and megakaryocyte reconstruction within 2 weeks and 3 weeks after transplantation respectively. However, 5-26% of patients have been reported to have delayed platelet engraftment (DPE), which is defined as persistent severe thrombocytopenia (<20 × 109/L) for >35 days after transplantation . To date, no standard treatment and prevention has been recommended for DPE. In patients with DPE, the amount of transfusion, the increased risk of infection, and the prolonged average hospital stay were independent risk factors affecting the prognosis of allo-HSCT patients. Due to continuous and progressive failure in the bone marrow hematopoiesis, thrombocytopenia post HSCT is more common in BMFD patients and often achieves low response to conventional therapy, such as platelet transfusion. Therefore, it is of great significance to effectively promote hematopoietic reconstruction to improve the prognosis of transplant patients.

Being tested
Eltrombopag 25 MG Oral Tablet, Supportive care
Keywords
Hematopoietic stem cell transplantation, Bone marrow failure syndrome, Eltrombopag, Thrombocytopenia
Medical subject headings
Bone Marrow Failure Disorders, Thrombocytopenia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Eltrombopag 25 MG Oral TabletDrug

    Also called TPO-RA

    Since thrombopoietin receptor agonists (TPO-RAs) have never been regularly used for promotion of cell engraftment post hematopoietic stem cell transplantation HSCT), we design this eltrombopag intervention to evaluate the safety and efficacy of TPO-RAs post HSCT.

  • Supportive careDrug

    Also called support

    Patients receive transfusion of blood products, including platelets and red blood cells.

Study groups in NCT05466201
Study groupRoleWhat they receive
supportive careOtherpatients in this group will receive supportive care, including blood products transfusion, anti-infective therapy rather than rhTPO or TPO-RAs.
Receives: Drug: Supportive care
Eltrombopag groupExperimentalEltrombopag treatment will be started at the dose of 50mg/d from the 1st day post hematopoietic stem cell transplantation, and the dose will be titrated by 25mg each every 7 days up to 100mg/d according to the tolerability. If not tolerable, reduce the dose to the previous tolerable level (if not tolerable at 50mg/d, reduce to 25mg/d) and maintain this dose for the following 7 days, with the attempt to restart dose escalation after this 7-day period.
Receives: Drug: Eltrombopag 25 MG Oral Tablet

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number and proportion of platelet engraftment on day 35 post HSCT.

    Platelet engraftment is defined as platelet count >20×109/L in consecutive 7 days (namely day 28 to day 35 after HSCT) without platelet transfusion in the prior 7 days (in other words, no platelet transfusion after day 21).

    Measured: From day 28 to 35 post HSCT

Secondary outcomes (3)

  1. Number and proportion of subjects achieving neutrophil engraftment at day 35 post HSCT.

    Neutrophil engraftment is defined as ANC>0.5×109/L for consecutive 3 days without administration of G-csf

    Measured: From day 28 to 35 post HSCT

  2. Hematopoietic reconstruction time.

    Reconstruction time is defined as the time from day 1 after allo-HSCT to platelet or neutrophil engraftment

    Measured: From day 1 to day 180 post HSCT

  3. Overall survival (OS)

    OS is defined as the time from the date of day 1 post HSCT to the date of death due to any cause.

    Measured: From day 1 to day 720 post HSCT or the time of the patients enrolled dead or quitting

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted20 Jul 2022
  2. Study start22 Oct 2022
  3. Primary completion31 Jul 2025
  4. Study completion31 Oct 2025

Resources

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 2/3
Primary purpose
Prevention
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
118 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05466201
Sponsor's protocol ID
SOOCHOW-HY-2022-08
Status last verified
Nov 2025
First submitted
12 Jul 2022
Last updated
3 Dec 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Nash RA, Kurzrock R, DiPersio J, Vose J, Linker C, Maharaj D, Nademanee AP, Negrin R, Nimer S, Shulman H, Ashby M, Jones D, Appelbaum FR, Champlin R. A phase I trial of recombinant human thrombopoietin in patients with delayed platelet recovery after hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2000;6(1):25-34. doi: 10.1016/s1083-8791(00)70049-8.

    PubMed 10707996

  • Marotta S, Marano L, Ricci P, Cacace F, Frieri C, Simeone L, Trastulli F, Vitiello S, Cardano F, Pane F, Risitano AM. Eltrombopag for post-transplant cytopenias due to poor graft function. Bone Marrow Transplant. 2019 Aug;54(8):1346-1353. doi: 10.1038/s41409-019-0442-3. Epub 2019 Jan 24.

    PubMed 30679824

  • Mahat U, Rotz SJ, Hanna R. Use of Thrombopoietin Receptor Agonists in Prolonged Thrombocytopenia after Hematopoietic Stem Cell Transplantation. Biol Blood Marrow Transplant. 2020 Mar;26(3):e65-e73. doi: 10.1016/j.bbmt.2019.12.003. Epub 2019 Dec 9.

    PubMed 31830528

  • Zeidan AM, Battiwalla M, Berlyne D, Winkler T. Aplastic Anemia and MDS International Foundation (AAMDSIF): Bone marrow failure disease scientific symposium 2016. Leuk Res. 2017 Feb;53:8-12. doi: 10.1016/j.leukres.2016.11.011. Epub 2016 Nov 24.

    PubMed 27923195

  • Dominietto A, Raiola AM, van Lint MT, Lamparelli T, Gualandi F, Berisso G, Bregante S, Frassoni F, Casarino L, Verdiani S, Bacigalupo A. Factors influencing haematological recovery after allogeneic haemopoietic stem cell transplants: graft-versus-host disease, donor type, cytomegalovirus infections and cell dose. Br J Haematol. 2001 Jan;112(1):219-27. doi: 10.1046/j.1365-2141.2001.02468.x.

    PubMed 11167808

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Eltrombopag 25 MG Oral Tablet and Supportive care in people with Stem Cell Transplant Complications, run by The First Affiliated Hospital of Soochow University at 1 site in China.

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 1 inclusion and 10 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: finished, as of the sponsor's last update on 3 Dec 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 China. 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 combined 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.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

Number and proportion of platelet engraftment on day 35 post HSCT. (time frame: From day 28 to 35 post HSCT). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05466201, which also lists 5 linked publications.

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 3 Dec 2025.

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