A Clinical Study on the Efficacy and Safety of Zeprumetostat in Relapsed/Refractory Large Granular Lymphocyte Leukemia

NCT07692529 | Not Yet Recruiting Phase 1

• 1 other identifier: EZH2-LGLL
• Study Type: interventional
• Target: 10
• Locations: 0 countries
• Sites: N/A

Brief Summary

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.

Conditions

Evaluation of the Efficacy and Safety of Zeprumetostat in Relapsed/Refractory Large Granular Lymphocyte Leukemia (T-LGLL)

Outcome Measures

Primary Outcomes (1)

• 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.

  8 weeks

Secondary Outcomes (6)

• The incidence of adverse reactions during treatment

  8 weeks

• The time to achieve partial hematological remission

  8 weeks

• Time to achieve complete hematologic response

  8 weeks

• Time to achieve complete molecular response

  8 weeks

• Change of the health-related quality of life after two cycles of treatment

  Baseline and 2 cycles of therapy (Each cycle is 28 days）

Study Arms (1)

zeprumetostat

EXPERIMENTAL

Drug: zeprumetostat

Interventions

zeprumetostat DRUG

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.

zeprumetostat

Eligibility Criteria

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

You may qualify if:

• Hemoglobin \< 100g/L or RBC transfusion dependence
• Neutrophil count \<0.5×10\^9/L or neutrophil count decreased with recurrent infection
• Progressive splenomegaly and/or Massive Splenomegaly
• Combined with autoimmune diseases requiring treatment, such as rheumatoid arthritis, autoimmune thyroiditis, etc.
• Severe B symptoms Failure or intolerance to a first-line therapy ECOG performance status ≤2 Expected survival ≥ 6 months Willing and able to comply with the requirements for this study and written informed consent.

You may not qualify if:

• 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 Having a history of mental illness or suffering from severe cerebrovascular diseases or cognitive sequelae History of pulmonary fibrosis, interstitial pneumonia, pneumoconiosis, radiation pneumonitis, drug-related pneumonia, severe impairment of lung function, etc.
• Difficulty in swallowing, chronic diarrhea and intestinal obstruction, there are multiple factors that affect the administration and absorption of medications.
• Received attenuated vaccine 4 in weeks before enrollment Participation in another clinical trial within 4 weeks before the start of this trial Have an allergy to zeprumetostat or any other part of this medicine. Previously treated with other EZH2 inhibitor. Pregnant or breast-feeding patients Patients considered to be ineligible for the study by the investigator for reasons other than the above

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

MeSH Terms

Conditions

Leukemia, Large Granular Lymphocytic

Condition Hierarchy (Ancestors)

Leukemia, T-Cell; Leukemia, Lymphoid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director of the Red Blood Cell Diseases Center & Director of the Regenerative Medicine Clinic

Study Record Dates

• First Submitted: June 30, 2026
• First Posted: July 9, 2026
• Study Start (Estimated): August 30, 2026
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): February 26, 2028
• Last Updated: July 9, 2026

Record last verified: 2026-07