This study is a single-arm, prospective, exploratory clinical trial aimed at exploring the efficacy and safety of zeprumetostat in patients with relapsed/refractory large granular T-cell leukemia. The study is expected to enroll 10 patients with relapsed/refractory large granular T-cell leukemia for treatment with zeprumetostat . The patients will undergo a maximum 4-week screening period and then enter the treatment phase, where they will receive zeprumetostat 350mg twice daily orally. The treatment lasts for 28 consecutive days as one cycle. If adverse events occur during the treatment, they will be handled according to the suspension and resumption standards. The efficacy and safety will be evaluated after the patients complete two cycles of administration.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
The patients will undergo a maximum 4-week screening period and then enter the treatment phase, where they will receive zeprumetostat 350mg twice daily orally. The treatment lasts for 28 consecutive days as one cycle. If adverse events occur during the treatment, they will be handled according to the suspension and resumption standards.
The proportion of achieving hematological remission after two cycles of treatment
Hematological response is evaluated by hemoglobin (Hb), absolute neutrophil count (ANC), platelet count (PLT), absolute lymphocyte count (ALC), absolute large granular lymphocyte count, and blood transfusion.
Time frame: 8 weeks
The incidence of adverse reactions during treatment
Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event
Time frame: 8 weeks
The time to achieve partial hematological remission
HPR is assessed by Hb, ANC, PLT, ALC, absolute large granular lymphocyte count and blood transfusion
Time frame: 8 weeks
Time to achieve complete hematologic response
HCR is assessed by Hb, ANC, PLT, ALC, and absolute large granular lymphocyte count.
Time frame: 8 weeks
Time to achieve complete molecular response
CMR is assessed by Hb, ANC, PLT, ALC, and absolute large granular lymphocyte count
Time frame: 8 weeks
Change of the health-related quality of life after two cycles of treatment
Medical Outcomes Study Questionnaire Short Form 36 Health Survey (SF-36) is used to assess the health-related quality of life of patients. The SF-36 has eight scaled scores; the scores are weighted sums of the questions in each section. Scores range from 0 - 100. Lower scores = more disability, higher scores = less disability
Time frame: Baseline and 2 cycles of therapy (Each cycle is 28 days)
Time from treatment benefit to disease recurrence
Duration time was calculated from response to relapse.
Time frame: A minimum of 2 years of planned follow-up
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