Thalidomide for the Symptomatic Large Granular Lymphocytic Leukemia

NCT06530576 | Recruiting Phase 2 DMC

• 1 other identifier: IIT2024041
• Study Type: interventional
• Target: 48
• Locations: 1 country
• Sites: 1

Brief Summary

There is currently no standard first-line treatment for LGLL. The investigators used the TPM regimen (thalidomide + prednison + methotrexate ) to treat LGLL since 2020, enrolling a total of 54 patients and achieving an overall response rate (ORR) of 88.9% and a complete response (CR) rate of 75.9%. To further explore this hypothesis, the investigators designed this study to observe the efficacy of thalidomide monotherapy in patients with symptomatic LGLL. The investigators speculate that thalidomide plays a major role in the significant improvement of the TPM regimen compared to the MTX regimen. Patients with LGLL are treated with thalidomide at 50 to 100 mg. If the desired response is not achieved at specific time points, methotrexate is added. Thalidomide monotherapy is administered for up to 3 courses, and the TM regimen can also be used for up to 3 courses. The overall response rate with thalidomide monotherapy serves as the primary study endpoint.

Conditions

T-LGL Leukemia; NK-LGL Leukemia

Keywords

Large granular lymphocyte leukemia; Thalidomide; Overall response rate

Outcome Measures

Primary Outcomes (1)

• Overall response rate

  complete remission rate+ partial remission rate

  up to 5 years

Secondary Outcomes (6)

• The safety of thalidomide

  up to 5 years

• Complete remession rate

  up to 5 years

• The rate of improvement in efficacy after TM combination

  up to 5 years

• Progression-free survival

  up to 5 years

• Overall survival

  up to 5 years

Study Arms (1)

Thalidomide

EXPERIMENTAL

All patients first receive thalidomide 100mg monotherapy.

Drug: Thalidomide and methotrexate

Interventions

Thalidomide and methotrexate DRUG

Thalidomide at a dose of 50-100 mg/QN, Methotrexate 10 mg/m2 orally once a week.

Thalidomide

Eligibility Criteria

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

You may qualify if:

• The patient fully understands the study, voluntarily participates, and signs the informed consent form (ICF);
• The patient must meet the diagnostic criteria for LGLL;
• The patient can be of any gender, aged 18 years or older;
• The patient is either untreated or not properly treated previously, or has poor efficacy (not reaching PR) or relapsed after being treated with regimens not based on methotrexate/thalidomide;
• The patient has indications for LGLL treatment, meeting at least one of the following criteria:
• ANC \< 0.5 × 10\^9/L, or neutropenia with recurrent infections
• HGB \< 100 g/L or requiring red blood cell transfusions for maintenance
• PLT \< 50 × 10\^9/L
• Concurrent autoimmune disease requiring treatment
• Symptomatic splenomegaly
• Severe B symptoms (unexplained fever, temperature over 38°C; night sweats; weight loss of 10% or more within six months)
• Pulmonary hypertension;
• ECOG score of 0-2;
• The patient's expected survival period is 6 months or more.

You may not qualify if:

• Unable to understand or follow the study procedures;
• Diagnosed or treated for malignancies other than LGLL within the past five years;
• Non-lymphoma-related liver or kidney function impairment: ALT \> 3 times the upper limit of normal (ULN), AST \> 3 times the ULN, total bilirubin (TBIL) \> 2 times the ULN, serum creatinine clearance \< 30 ml/min;
• Other serious medical conditions that could affect the study (e.g., uncontrolled diabetes, gastric ulcer, other serious heart or lung diseases), with the judgment resting with the investigator;
• Caprini thrombosis analysis score indicating high risk (Appendix 2);
• Patients who have undergone major surgery (excluding lymph node biopsy) within the past 14 days or are expected to undergo major surgery during the treatment;
• Pregnant or breastfeeding women, and women of childbearing age who are not using contraception;
• Hypersensitivity to the drugs or their components used in the study.

Sponsors & Collaborators

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

Study Sites (1)

Institute of Hematology & Blood Diseases Hospital

Tianjin, Tianjin Municipality, 300020, China

RECRUITING

MeSH Terms

Conditions

Leukemia, Large Granular Lymphocytic

Interventions

Thalidomide; Methotrexate

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

Intervention Hierarchy (Ancestors)

Phthalimides; Phthalic Acids; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Piperidones; Piperidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Isoindoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Aminopterin; Pterins; Pteridines

Study Officials

• Shuhua Yi, Doctor

  Institute of Hematology & Blood Diseases Hospital, China

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Shuhua Yi, Doctor

CONTACT

86-22-23909106; yishuhua@ihcams.ac.cn

Lugui Qiu, Doctor

CONTACT

86-22-23909172; qiulg@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: SPONSOR

Model Details: Thalidomide at a dose of 50-100 mg/QN

Study Record Dates

• First Submitted: July 28, 2024
• First Posted: July 31, 2024
• Study Start: August 10, 2024
• Primary Completion (Estimated): June 10, 2026
• Study Completion (Estimated): July 1, 2027
• Last Updated: November 18, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)