NCT07539779

Brief Summary

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an important treatment for hematological malignancies. Poor erythroid engraftment after transplantation is a serious complication, especially in patients with moderate to severe myelofibrosis (MF). Currently, there is a lack of effective prevention strategies for poor erythroid engraftment after transplantation. Luspatercept, a novel TGF-β superfamily signaling pathway modulator, has shown potential in small-sample studies for the treatment and prevention of post-transplant anemia. Given the high proportion and poor prognosis of poor engraftment function in hematological malignancies with moderate to severe myelofibrosis after transplantation, we plan to conduct a prospective, multicenter, randomized controlled study to explore the efficacy and safety of luspatercept in preventing poor erythroid engraftment after allo-HSCT in hematological malignancies with moderate to severe myelofibrosis.

Trial Health

63
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
196

participants targeted

Target at P75+ for phase_2

Timeline
57mo left

Started May 2026

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
not yet recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

April 13, 2026

Completed
7 days until next milestone

First Posted

Study publicly available on registry

April 20, 2026

Completed
11 days until next milestone

Study Start

First participant enrolled

May 1, 2026

Completed
3.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2030

Expected
12 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2030

Last Updated

April 20, 2026

Status Verified

April 1, 2026

Enrollment Period

3.7 years

First QC Date

April 13, 2026

Last Update Submit

April 13, 2026

Conditions

LuspaterceptPoor Erythroid EngraftmentHematological MalignanciesMyelofibrosis (MF)

Outcome Measures

Primary Outcomes (1)

  • Cumulative incidence of poor erythroid engraftment

    Poor erythroid engraftment after allo-HSCT is defined as HGB \< 70g/L at 28 days post-transplantation and the inability to discontinue red blood cell transfusion in the case of complete donor engraftment.

    28 days

Secondary Outcomes (6)

  • Red blood cell units

    28 days

  • Overall survival

    1 year

  • Disease-free survival

    1 year

  • Cumulative rate of poor graft engraftment

    1 year

  • Relapse

    1 year

  • +1 more secondary outcomes

Study Arms (2)

Luspatercept group

EXPERIMENTAL

Luspatercept

Drug: Luspatercept

Control group

OTHER

Control

Other: Control

Interventions

LuspaterceptDRUG

On the 7th day after allo-HSCT, the first dose of Luspatercept 1.0mg/kg was administered subcutaneously. If the peripheral blood HGB was \< 70g/L on the 21st day after allo-HSCT, the second dose of Luspatercept 1.0mg/kg was given subcutaneously; if the peripheral blood HGB was ≥ 70g/L on the 21st day after allo-HSCT, no second dose of Luspatercept subcutaneous injection was given.

Luspatercept group
ControlOTHER

The patient will receive the best supportive treatment including blood transfusion.

Control group

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Age 18-65 years old, gender not restricted;
  • ECOG score 0-2 points;
  • Hematological malignancies with moderate to severe myelofibrosis
  • Willing to undergo the first allo-HSCT with a suitable donor
  • In a CR state before transplantation.

You may not qualify if:

  • Has previously undergone allo-HSCT;
  • ECOG score is 3-5;
  • Expected lifespan after transplantation is less than 30 days;
  • Has severe cardiac dysfunction, severe arrhythmia or severe pulmonary dysfunction (obstructive and/or restrictive ventilation disorder);
  • Has severe liver dysfunction, with liver function indicators (aspartate aminotransferase (AST), alanine aminotransferase (ALT), total bilirubin (TBIL)) more than twice the upper limit of normal;
  • Has severe renal dysfunction, with creatinine (Cr) more than twice the upper limit of normal or 24-hour creatinine clearance rate (Ccr) lower than 30 ml/min;
  • Has severe active bleeding;
  • Patients judged by the investigator to be unsuitable for participating in this trial.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Nanfang Hospital, Southern Medical University

Guangzhou, Guangdong, 510515, China

Location

Related Publications (5)

  • Wobus M, Mies A, Asokan N, Oelschlagel U, Mobus K, Winter S, Cross M, Weidner H, Rauner M, Hofbauer LC, Bornhauser M, Platzbecker U. Luspatercept restores SDF-1-mediated hematopoietic support by MDS-derived mesenchymal stromal cells. Leukemia. 2021 Oct;35(10):2936-2947. doi: 10.1038/s41375-021-01275-5. Epub 2021 May 17.

    PMID: 34002031RESULT

  • Fenaux P, Santini V, Spiriti MAA, Giagounidis A, Schlag R, Radinoff A, Gercheva-Kyuchukova L, Anagnostopoulos A, Oliva EN, Symeonidis A, Berger MH, Gotze KS, Potamianou A, Haralampiev H, Wapenaar R, Milionis I, Platzbecker U. A phase 3 randomized, placebo-controlled study assessing the efficacy and safety of epoetin-alpha in anemic patients with low-risk MDS. Leukemia. 2018 Dec;32(12):2648-2658. doi: 10.1038/s41375-018-0118-9. Epub 2018 Mar 30.

    PMID: 29895954RESULT

  • Platzbecker U, Della Porta MG, Santini V, Zeidan AM, Komrokji RS, Shortt J, Valcarcel D, Jonasova A, Dimicoli-Salazar S, Tiong IS, Lin CC, Li J, Zhang J, Giuseppi AC, Kreitz S, Pozharskaya V, Keeperman KL, Rose S, Shetty JK, Hayati S, Vodala S, Prebet T, Degulys A, Paolini S, Cluzeau T, Fenaux P, Garcia-Manero G. Efficacy and safety of luspatercept versus epoetin alfa in erythropoiesis-stimulating agent-naive, transfusion-dependent, lower-risk myelodysplastic syndromes (COMMANDS): interim analysis of a phase 3, open-label, randomised controlled trial. Lancet. 2023 Jul 29;402(10399):373-385. doi: 10.1016/S0140-6736(23)00874-7. Epub 2023 Jun 10.

    PMID: 37311468RESULT

  • de Witte T, Bowen D, Robin M, Malcovati L, Niederwieser D, Yakoub-Agha I, Mufti GJ, Fenaux P, Sanz G, Martino R, Alessandrino EP, Onida F, Symeonidis A, Passweg J, Kobbe G, Ganser A, Platzbecker U, Finke J, van Gelder M, van de Loosdrecht AA, Ljungman P, Stauder R, Volin L, Deeg HJ, Cutler C, Saber W, Champlin R, Giralt S, Anasetti C, Kroger N. Allogeneic hematopoietic stem cell transplantation for MDS and CMML: recommendations from an international expert panel. Blood. 2017 Mar 30;129(13):1753-1762. doi: 10.1182/blood-2016-06-724500. Epub 2017 Jan 17.

    PMID: 28096091RESULT

  • Gratwohl A, Baldomero H, Aljurf M, Pasquini MC, Bouzas LF, Yoshimi A, Szer J, Lipton J, Schwendener A, Gratwohl M, Frauendorfer K, Niederwieser D, Horowitz M, Kodera Y; Worldwide Network of Blood and Marrow Transplantation. Hematopoietic stem cell transplantation: a global perspective. JAMA. 2010 Apr 28;303(16):1617-24. doi: 10.1001/jama.2010.491.

    PMID: 20424252RESULT

MeSH Terms

Conditions

Hematologic NeoplasmsPrimary Myelofibrosis

Interventions

luspatercept

Condition Hierarchy (Ancestors)

Neoplasms by SiteNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesMyeloproliferative DisordersBone Marrow Diseases

Study Officials

  • Qifa Liu

    Nanfang Hospital, Southern Medical University

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Li Xuan

CONTACT

+86-020-6164161318956985366@163.com

Qifa Liu

CONTACT

+86-020-62787883liuqifa628@163.com

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
NONE
Purpose
PREVENTION
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 13, 2026

First Posted

April 20, 2026

Study Start

May 1, 2026

Primary Completion (Estimated)

January 1, 2030

Study Completion (Estimated)

December 31, 2030

Last Updated

April 20, 2026

Record last verified: 2026-04

Data Sharing

IPD Sharing
Will not share

Locations

CN(1)