Evaluating Luspatercept for Patients with Lower-risk Myelodysplastic Syndromes

Safety and Efficacy Study of the Tapering Dose of Luspatercept in Patients With Lower-risk Myelodysplastic Syndromes

This study is testing if a new drug called Luspatercept can help people with lower-risk Myelodysplastic Syndromes who haven't responded to other treatments feel better and improve their blood levels.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 36 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China (Academic / other)
• Drugs / interventions: chemotherapy, radiation
• Locations: 1 site (Tianjin, Tianjin Municipality)
• Trial ID: NCT05925504 on ClinicalTrials.gov

What this trial studies

This phase 2 clinical trial is a prospective, single-center, single-arm study aimed at assessing the efficacy and safety of Luspatercept in patients diagnosed with lower-risk Myelodysplastic Syndromes (MDS). Participants must have an IPSS-R score of 3.5 or lower and a baseline hemoglobin level below 100g/L. The study will involve administering Luspatercept to eligible patients who have not responded to or are intolerant of prior erythropoiesis-stimulating agents. The trial will monitor patient outcomes to determine the treatment's effectiveness and safety profile.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18 and older with a diagnosis of lower-risk MDS and specific eligibility criteria related to prior treatments and health status.

Not a fit: Patients with platelet counts below 50 x 10^9/L or those with certain types of MDS, such as secondary MDS or MDS associated with specific cytogenetic abnormalities, may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with lower-risk MDS, potentially improving their hemoglobin levels and overall quality of life.

How similar studies have performed: While this approach is being evaluated in this specific context, similar studies using Luspatercept have shown promise in treating other forms of anemia, indicating potential for success.

Eligibility criteria

Inclusion Criteria:

* Male or female age ≥ 18 years
* Subject has diagnosis of MDS according to WHO classification that meets IPSS-R score ≤3.5
* Hemoglobin \< 100g/L at baseline
* Refractory or intolerant to prior ESA treatment or EPO≥500U/L
* ECOG performance status ≤2
* Willing and able to comply with the requirements for this study and written informed consent.

Exclusion Criteria:

* Platelet counts \< 50 x 10\^9/L
* Previously treated with either luspatercept or sotatercept
* Use any of the following prior to this study

  * Immunomodulatory drugs such as lenalidomide \[IMiD\] for ≥4 weeks
  * Immunosuppressive therapy \[IST\] for ≥4 weeks
  * Demethylating agents \[HMA\] ≥ 1 cycle of treatment
* MDS associated with del 5q cytogenetic abnormality
* Secondary MDS, i.e. MDS that is known to have arisen as the result of chemical injury or treatment with chemotherapy and/or radiation for other diseases.
* Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding.
* Prior allogeneic or autologous stem cell transplant.
* Prior history of malignancies, other than MDS, unless the subject is free of the disease (including completion of any active or adjuvant treatment for prior malignancy) for ≥ 5 years. However, subjects with the following history/concurrent conditions are allowed: basal or squamous cell carcinoma of the skin, superficial bladder cancer, prostate intraepithelial neoplasia, carcinoma in situ of the cervix or other indolent tumors.
* Uncontrolled systemic fungal, bacterial, or viral infection (defined as ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics, antiviral therapy, and/or other treatment), known human immunodeficiency virus (HIV), known evidence of active infectious hepatitis B, and/or known evidence of active hepatitis C.
* Clinically significant cardiac disease, including any of the follow: uncontrolled angina pectoris, myocardial infarction, unstable cardiac arrhythmias, congestive heart failure and New York Heart Association (NYHA) grade 2-4 cardiac failure.
* Abnormal liver function: two consecutive examinations with an interval of ≥1 week suggest that ALT and AST are 2.5 times higher than the upper limit of normal values
* Renal impairment: creatinine clearance \<60ml/min
* Any severe and/or uncontrolled medical conditions or other conditions that could affect their participation in the study, including clinically significant cardiac diseases, refractory hypertension, metabolic disorders and other diseases that seriously affect the function of the gastrointestinal tract.
* Had a history of any psychiatric diseases, cerebrovascular disease or cognitive sequelae of head injury.
* Major surgery within 8 weeks prior to this study. Subjects must be completely recovered from any previous surgery prior to this study.
* Received attenuated vaccine in 4 weeks before enrollment.
* Participation in another clinical trial within 4 weeks before the start of this trial.
* History of severe allergic or anaphylactic reactions or hypersensitivity to recombinant proteins or excipients in the luspatercept.
* Pregnant or breast-feeding patients
* Patients considered to be ineligible for the study by the investigator for reasons other than the above.

Where this trial is running

Tianjin, Tianjin Municipality

• Regenerative Medicine Center — Tianjin, Tianjin Municipality, China (Recruiting)

Study contacts

• Study coordinator: Zhen Gao

Conditions Lower Risk MDS Per IPSS-R