Luspatercept for Myelodysplastic-Myeloproliferative Diseases

Phase-Based Progress Estimates
Mayo Clinic in Rochester, Rochester, MN
Myelodysplastic-Myeloproliferative Diseases+5 More
Luspatercept - Biological
All Sexes
Eligible conditions

Study Summary

This study is evaluating whether luspatercept with or without hydroxyurea is safe and effective in treating patients with myelodysplastic/myeloproliferative neoplasms with ring sideroblasts and thromb

See full description

Eligible Conditions

  • Myelodysplastic-Myeloproliferative Diseases
  • Myelodysplastic/Myeloproliferative Neoplasm With Ring Sideroblasts and Thrombocytosis
  • Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Myelodysplastic-Myeloproliferative Diseases

Study Objectives

This trial is evaluating whether Luspatercept will improve 1 primary outcome and 5 secondary outcomes in patients with Myelodysplastic-Myeloproliferative Diseases. Measurement will happen over the course of Up to 8 weeks.

Month 6
Duration of response
Month 6
Overall survival
Month 6
Time to leukemic transformation
Month 6
Leukemia-free survival
Up to 6 months
Incidence of adverse events (AEs)
Up to 8 weeks
Erythroid response rate

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Other trials for Myelodysplastic-Myeloproliferative Diseases

Trial Design

2 Treatment Groups

Cohort A (luspatercept)
1 of 2
Cohort A (luspatercept, hydroxyurea)
1 of 2
Experimental Treatment

This trial requires 54 total participants across 2 different treatment groups

This trial involves 2 different treatments. Luspatercept is the primary treatment being studied. Participants will be divided into 2 treatment groups. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

Cohort A (luspatercept)
Patients receive luspatercept SC on day 1. Cycles repeat every 21 days for 6 months in the absence of disease progression or unacceptable toxicity.
Cohort A (luspatercept, hydroxyurea)Patients receive luspatercept SC on day 1 and hydroxyurea PO on days 1-21. Cycles repeat every 21 days for 6 months in the absence of disease progression or unacceptable toxicity.
First Studied
Drug Approval Stage
How many patients have taken this drug
FDA approved
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to 6 months
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly up to 6 months for reporting.

Closest Location

Mayo Clinic in Rochester - Rochester, MN

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Refractory or unlikely to respond (erythropoietin [EPO] >= 200 U/L) or documented intolerance to erythropoiesis stimulating agent (ESA). Patients should have either a documented intolerance or contraindication to ESA or a lack of response after ESA therapy trial of at least four weeks; and ESA therapy should be stopped four weeks prior to trial enrollment
Age >= 18 years
Patients with a World Health Organization(WHO)-defined diagnosis of MDS/MPN-RS-T or MDS/MPN-U with >= 15% RS
Prior treatment with lenalidomide, hypomethylating agents, immunosuppressive therapy or investigational agent is allowed as long as patients have not received luspatercept-aamt or sotatercept. If there is prior history of investigational agent, there should be an interval equivalent to at least four elimination half-lives of the agent prior to enrollment. Note: For patients who have received prior lenalidomide, hypomethylating agents, or immunosuppressive therapy, there must be >= 6 weeks since the last dose before luspatercept-aamt treatment is started
Eastern Cooperative Oncology Group (ECOG) performance score of 0, 1 or 2
Requirement of red blood cell transfusions (>= 2 unit =< 8-weeks prior to registration) OR symptomatic anemia with hemoglobin < 9.5 g/dL OR hematocrit < 30% (as long as there is documentation of adequate iron stores (ferritin > 50 mg/L) =< 5 weeks prior to registration). Symptomatic anemia is defined as fatigue with or without exertion, shortness of breath with or without exertion, or decrease in exercise tolerance
Hemoglobin =< 9.5 g/dL (obtained =< 14 days prior to registration)
Total bilirubin =< 1.5 x upper limit of normal (ULN) (obtained =< 14 days prior to registration)
Alanine aminotransferase (ALT) and aspartate transaminase (AST) =< 3 x ULN (=< 5 x ULN for patients with liver involvement) (obtained =< 14 days prior to registration)
Calculated creatinine clearance >= 30 ml/min using the Cockcroft-Gault (obtained =< 14 days prior to registration)

Patient Q&A Section

What is myelodysplastic-myeloproliferative diseases?

"The MDS-MPD spectrum is a term applied to a set of clinically distinct disease entities characterized by similar cytogenetic and immunophenotypic abnormalities. The MDS/MPD spectrum comprises five diseases: MDS, with a preponderance of normal karyotypes, and MDS/AML, MDS-U, MDS/CML and MDS/TETRA, with ~60-70% of cases presenting with a normal karyotype. These five diseases have distinct cytogenetic, phenotypic, and clinical characteristics, and response to therapy." - Anonymous Online Contributor

Unverified Answer

What are the signs of myelodysplastic-myeloproliferative diseases?

"Results from a recent paper, the most common of the above signs were bone cysts, elevated C-reactive protein, enlarged liver, increased spleen size and/or hepatomegaly." - Anonymous Online Contributor

Unverified Answer

How many people get myelodysplastic-myeloproliferative diseases a year in the United States?

"Around 350,000 people have a diagnosis of myelodysplastic-myeloproliferative disease a year in the United States. myelodysplastic-myeloproliferative diseases are one of the most common types of heaematopoietic neoplasms.myelodysplastic-myeloproliferative diseases are a heterogeneous group of malignancies consisting of clonal myeloid and/or lymphoid precursor cell neoplasms whose cell of origin is a haematopoietic precursor cell." - Anonymous Online Contributor

Unverified Answer

Can myelodysplastic-myeloproliferative diseases be cured?

"This case report suggests that in some MDS/MPN patients, i.e. patients with a low-risk profile and without BCR-ABL1 T315I mutation, the disease may be manageable and/or potentially eradicateable." - Anonymous Online Contributor

Unverified Answer

What are common treatments for myelodysplastic-myeloproliferative diseases?

"Treatment of MDS depends on the particular disorder of patients. High doses of cytotoxic agents, e.g. cytosine arabinoside, can be used in order to overcome the lack of production of red blood cells. Other more specific types of therapy, e.g. aPHa and azacitidine, are used to prevent or prevent further deterioration of myeloproliferative disorders. The use of allogeneic stem cells is under ongoing research." - Anonymous Online Contributor

Unverified Answer

What causes myelodysplastic-myeloproliferative diseases?

"Tumorigenesis is a significant contributor to the etiology of MDS and MMP, but the myeloproliferative component of these disorders is most likely to be caused by genetic factors of epigenetic origin." - Anonymous Online Contributor

Unverified Answer

Is luspatercept typically used in combination with any other treatments?

"Based on the available data, there was only one case report of luspatercept use without other treatments, and many of the available case reports were for lupus patients undergoing chemotherapy. A large randomized clinical trial is currently planned with more specific enrollment and treatment objectives to establish these relationships." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets myelodysplastic-myeloproliferative diseases?

"This report shows this condition often affects children under the age of 18. There were no differences in survival or cumulative risk by age. Findings from a recent study may suggest a need to target the treatment of older people at increased risk for the disease in order to decrease mortality and improve quality of life. A more accurate assessment of the natural history of myelodysplastic-myeloproliferative diseases in people older than 45 by the use of a prognosis calculator/model could provide useful guidance by allowing earlier detection and treatment of MDS-MCP." - Anonymous Online Contributor

Unverified Answer

Have there been other clinical trials involving luspatercept?

" does not provide information on any clinical trials using luspatercept. However, the FDA Safety Alert Database lists a study in which patients with MDS/PMF participated and this could provide additional clinical trials." - Anonymous Online Contributor

Unverified Answer

Has luspatercept proven to be more effective than a placebo?

"Treatment of patients with lupercept led to a non-significant improvement in LTSS in one study, and there is no consensus in the literature on this. However, there are concerns in the literature that the effect of lupercept differs among patients of different disease risk. If this is reflected in the data from the lupercept studies, then we could think that lupercept will not affect LTSS. On the contrary, in this sense, a placebo is not helpful here either, because a patient has already received a treatment that is not effective." - Anonymous Online Contributor

Unverified Answer

How serious can myelodysplastic-myeloproliferative diseases be?

"The prognosis of myelodysplastic-myeloproliferative diseases varies widely; therefore, the necessity for immediate treatment should be considered, especially for high-risk patients at the early stage of disease." - Anonymous Online Contributor

Unverified Answer

What is luspatercept?

"Luspatercept is usually well tolerated, with the potential of substantial and sustained and clinically meaningful reductions in low levels of bone biomarkers with a normalizing or even restoring effect on haemopoiesis without increases in haemoglobin levels." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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