The Tapering Dose of Luspatercept in Patients With Lower-risk Myelodysplastic Syndromes

NCT05925504 | Recruiting Phase 2

• 1 other identifier: Lusp-MDS
• Study Type: interventional
• Target: 36
• Locations: 1 country
• Sites: 1

Brief Summary

This is a prospective, single center, single-arm, phase 2 study. The aim of this study is to evaluate the efficacy and safety of Luspatercept for Patients with Lower-risk Myelodysplastic Syndromes (MDS).

Conditions

Lower Risk MDS Per IPSS-R

Outcome Measures

Primary Outcomes (1)

• Proportion of subjects achieving hematologic improvement - erythroids (HI-E) according to IWG 2006 criteria

  within 12 weeks

Secondary Outcomes (4)

• Proportion of subjects achieving RBC-TI according to IWG 2006 criteria

  within 12 weeks

• Median time to HI-E or RBC-TI

  within 12 weeks

• Mean change in serum ferritin

  within 12 weeks

• Incidence of the adverse event

  within 12 weeks

Study Arms (1)

Luspatercept

EXPERIMENTAL

open-label, single-arm

Drug: Luspatercept

Interventions

Luspatercept DRUG

The starting dose is 1.75mg/kg once every 3 weeks by subcutaneous injection. For rapid hemoglobin rise after 2 consecutive doses at the 1.75mg/kg starting dose, decrease the dose of Luspatercept or interrupt treatment. Otherwise, continue treatment with the dose of 1.75mg/kg once every 3 weeks.

Luspatercept

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead
• Beijing Health Alliance Charitable Foundation: collaborator

Study Sites (1)

Regenerative Medicine Center

Tianjin, Tianjin Municipality, 300131, China

RECRUITING

MeSH Terms

Interventions

luspatercept

Central Study Contacts

Zhen Gao

CONTACT

‭15522360862‬; gaozhen@ihcams.ac.cn

Jingyu Zhao

CONTACT

13752253515; zhaojingyu@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Physician of Regenerative Medical Center

Study Record Dates

• First Submitted: June 21, 2023
• First Posted: June 29, 2023
• Study Start: July 1, 2023
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): September 30, 2026
• Last Updated: March 12, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)