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

Официальное название

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

Обзор

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).

Вмешательства

• Препарат Luspatercept
  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.

Первичные конечные точки

• Proportion of subjects achieving hematologic improvement - erythroids (HI-E) according to IWG 2006 criteria [Срок оценки: within 12 weeks]

Вторичные конечные точки (4)

• Proportion of subjects achieving RBC-TI according to IWG 2006 criteria [Срок оценки: within 12 weeks]
• Median time to HI-E or RBC-TI [Срок оценки: within 12 weeks]
• Mean change in serum ferritin [Срок оценки: within 12 weeks]
• Incidence of the adverse event [Срок оценки: within 12 weeks]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 1 центр

• Regenerative Medicine Center — Тяньцзинь

Идентификаторы

NCT: NCT05925504 · Lusp-MDS

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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