LuspaterceptPhase 2/3NCT07539779
Luspatercept in Preventing Poor Erythroid Engraftment for Hematological Malignancies With Moderate to Severe Myelofibrosis
A Phase 2/3 study testing Luspatercept in people with Luspatercept and Poor Erythroid Engraftment, run by Nanfang Hospital, Southern Medical University at 1 site in China.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Luspatercept, Poor Erythroid Engraftment, Hematological Malignancies
- What is being tested?
- Luspatercept
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 196 participants
- Where
- 1 siteChina
- Study dates
- 1 May 2026to 31 Dec 2030
- Phase
- Phase 2/3
- Are there results?
- Not yet: the study is under way
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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
Nanfang Hospital, Southern Medical University
Guangzhou, Guangdong, 510515
Qifa Liu, +86-020-62787883, liuqifa628@163.com
About this study
Official title: The Efficacy and Safety of Luspatercept in Preventing Poor Erythroid Engraftment After Allo-HSCT for Hematological Malignancies With Moderate to Severe Myelofibrosis: A Prospective, Multicenter, Randomized Controlled Study
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.
Read the detailed description
Exploring appropriate prevention strategies for poor erythroid engraftment during transplantation is not only a significant scientific issue but also a major clinical problem. Luspatercept is a recombinant fusion protein that can target and regulate the signaling pathway of the transforming growth factor-β (TGF-β) superfamily. By binding to multiple TGF-β superfamily ligands, it weakens the Smad2/3 signaling pathway, thereby promoting the maturation and generation of red blood cells. In recent years, some small-sample studies have explored the efficacy and safety of luspatercept for preventive treatment in patients with hematological malignancies who received allo-HSCT, showing certain efficacy. However, these studies were retrospective and requires large-sample, prospective, randomized controlled studies for further verification. Currently, there are no prospective studies on preventive treatment for poor erythroid engraftment after allo-HSCT in patients with hematological malignancies.
The presence of fibrosis in the bone marrow before transplantation (MF), especially moderate/severe MF, is one of the main causes of post-transplant PGF. A small-sample clinical trial in our center previously showed that in patients with MDS/MPN and acute leukemia with MF grade 2/3, applying luspatercept at +7 and +21 days after transplantation could reduce the risk of early red cell transfusion after transplantation, and showed good safety. Based on the current research status and our previous studies, 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 patients with hematological malignancies accompanied by moderate/severe MF.
- Being tested
- Luspatercept, Control
- Medical subject headings
- Hematologic Neoplasms, Primary Myelofibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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.
ControlOther
The patient will receive the best supportive treatment including blood transfusion.
| Study group | Role | What they receive |
|---|---|---|
| Luspatercept group | Experimental | Luspatercept Receives: Drug: Luspatercept |
| Control group | Other | Control Receives: Other: Control |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 28 days
Secondary outcomes (6)
Red blood cell units
The number of red blood cell units administered
Measured: 28 days
Overall survival
Will calculate time from random assignment until death from any cause.
Measured: 1 year
Disease-free survival
Will calculate time from random assignment until relapse or death from any cause
Measured: 1 year
Cumulative rate of poor graft engraftment
The definition of PGF is as follows: More than 28 days after transplantation, there is at least a continuous period of 3 days of two-line or three-line blood cell reduction (absolute neutrophil count < 0.5 × 109/L, platelet (PLT) < 20 × 109/L, hemoglobin (HGB) < 70 g/L), and blood transfusion support treatment is required; bone marrow examination indicates a low degree of bone marrow hyperplasia; complete donor engraftment; no active severe GVHD or hematological recurrence.
Measured: 1 year
Relapse
Will calculate time from random assignment until relapse
Measured: 1 year
Non-relapse mortality
Defined as death from any cause not subsequent to relapse
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Apr 2026
- Study start1 May 2026 (estimated)
- Primary completion1 Jan 2030 (estimated)
- Study completion31 Dec 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for LuspaterceptStudies of the same condition, open and under way.
- More trials run by Nanfang Hospital, Southern Medical UniversityThe sponsor's other registered studies.
- 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 2/3
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 196 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Nanfang Hospital, Southern Medical University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Qifa Liu, Principal investigator, Nanfang Hospital, Southern Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07539779
- Sponsor's protocol ID
- NFEC-2026-016
- Status last verified
- Apr 2026
- First submitted
- 13 Apr 2026
- Last updated
- 20 Apr 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 34002031Fenaux 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.
PubMed 29895954Platzbecker 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.
PubMed 37311468de 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.
PubMed 28096091Gratwohl 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.
PubMed 20424252
Study at a glance
From the registry record, last updated by the sponsor on 20 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Efficacy and Safety of Luspatercept in Preventing Poor Erythroid Engraftment After Allo-HSCT for Hematological Malignancies With Moderate to Severe Myelofibrosis: A Prospective, Multicenter, Randomized Controlled Study
- ClinicalTrials.gov ID
- NCT07539779
- Acronym / sponsor ID
- NFEC-2026-016
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 196 participants (estimated)
- Lead sponsor
- Nanfang Hospital, Southern Medical UniversityUniversity, hospital or other
- Study start
- 1 May 2026 (estimated)
- Primary completion
- 1 Jan 2030 (estimated)
- Study completion
- 31 Dec 2030 (estimated)
- First posted
- 20 Apr 2026 (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.
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.
Detailed description
Exploring appropriate prevention strategies for poor erythroid engraftment during transplantation is not only a significant scientific issue but also a major clinical problem. Luspatercept is a recombinant fusion protein that can target and regulate the signaling pathway of the transforming growth factor-β (TGF-β) superfamily. By binding to multiple TGF-β superfamily ligands, it weakens the Smad2/3 signaling pathway, thereby promoting the maturation and generation of red blood cells. In recent years, some small-sample studies have explored the efficacy and safety of luspatercept for preventive treatment in patients with hematological malignancies who received allo-HSCT, showing certain efficacy. However, these studies were retrospective and requires large-sample, prospective, randomized controlled studies for further verification. Currently, there are no prospective studies on preventive treatment for poor erythroid engraftment after allo-HSCT in patients with hematological malignancies.
The presence of fibrosis in the bone marrow before transplantation (MF), especially moderate/severe MF, is one of the main causes of post-transplant PGF. A small-sample clinical trial in our center previously showed that in patients with MDS/MPN and acute leukemia with MF grade 2/3, applying luspatercept at +7 and +21 days after transplantation could reduce the risk of early red cell transfusion after transplantation, and showed good safety. Based on the current research status and our previous studies, 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 patients with hematological malignancies accompanied by moderate/severe MF.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Luspatercept group | Experimental | Drug: LuspaterceptLuspatercept |
| Control group | Other | Other: ControlControl |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (8 listed)
- 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;
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 |
|---|---|---|
| LuspaterceptOn 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. | Drug | Luspatercept group |
| ControlThe patient will receive the best supportive treatment including blood transfusion. | Other | Control group |
Primary outcome
Cumulative incidence of poor erythroid engraftment
Time frame: 28 days
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.
6 secondary outcomes
Red blood cell units
Time frame: 28 days
Overall survival
Time frame: 1 year
Disease-free survival
Time frame: 1 year
Cumulative rate of poor graft engraftment
Time frame: 1 year
Relapse
Time frame: 1 year
Non-relapse mortality
Time frame: 1 year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Apr 2026.
- Results publication: PubMed 34002031Wobus 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.
- Results publication: PubMed 29895954Fenaux 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.
- Results publication: PubMed 37311468Platzbecker 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.
- Results publication: PubMed 28096091de 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.
- Results publication: PubMed 20424252Gratwohl 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.
- Individual participant dataSharing planned: No
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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.
Qifa Liu principal investigator
Nanfang Hospital, Southern Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Luspatercept in people with Luspatercept and Poor Erythroid Engraftment, run by Nanfang Hospital, Southern Medical 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 5 inclusion and 8 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: opening soon, as of the sponsor's last update on 20 Apr 2026. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Li Xuan (+86-020-61641613, 18956985366@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cumulative incidence of poor erythroid engraftment (time frame: 28 days). 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 NCT07539779, 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 20 Apr 2026.