Thalidomide treatment for symptomatic large granular lymphocytic leukemia

The Efficacy of Thalidomide for the Symptomatic Large Granular Lymphocytic Leukemia - a Prospective Phase Ⅱ Multicenter Clinical Trial From China

This study is testing if thalidomide can help people with symptomatic large granular lymphocytic leukemia feel better by seeing how well it works on its own.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 48 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China (other)
• Drugs / interventions: methotrexate
• Locations: 1 site (Tianjin, Tianjin Municipality)
• Trial ID: NCT06530576 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the efficacy of thalidomide monotherapy in patients with symptomatic large granular lymphocytic leukemia (LGLL). Patients will receive thalidomide at doses of 50 to 100 mg daily for up to three treatment cycles, with evaluations for response after each cycle. If partial remission is not achieved, methotrexate will be added to the treatment regimen. The primary endpoint is the overall response rate to thalidomide monotherapy.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18 and older with symptomatic LGLL who have not responded to previous treatments or are untreated.

Not a fit: Patients with asymptomatic LGLL or those who have already achieved remission may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new effective option for patients suffering from symptomatic LGLL.

How similar studies have performed: Previous studies using thalidomide in combination therapies for LGLL have shown promising results, indicating potential for success with this monotherapy approach.

Eligibility criteria

Inclusion Criteria:

1. The patient fully understands the study, voluntarily participates, and signs the informed consent form (ICF);
2. The patient must meet the diagnostic criteria for LGLL;
3. The patient can be of any gender, aged 18 years or older;
4. 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;
5. The patient has indications for LGLL treatment, meeting at least one of the following criteria:

   1. ANC \< 0.5 × 10\^9/L, or neutropenia with recurrent infections
   2. HGB \< 100 g/L or requiring red blood cell transfusions for maintenance
   3. PLT \< 50 × 10\^9/L
   4. Concurrent autoimmune disease requiring treatment
   5. Symptomatic splenomegaly
   6. Severe B symptoms (unexplained fever, temperature over 38°C; night sweats; weight loss of 10% or more within six months)
   7. Pulmonary hypertension;
6. ECOG score of 0-2;
7. The patient's expected survival period is 6 months or more.

Exclusion Criteria:

1. Unable to understand or follow the study procedures;
2. Diagnosed or treated for malignancies other than LGLL within the past five years;
3. 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;
4. 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;
5. Caprini thrombosis analysis score indicating high risk (Appendix 2);
6. Known history of HIV infection or active HBV infection, or any uncontrolled active systemic infection requiring intravenous antibiotics; Note: Active HBV infection is defined as: a. HBV DNA ≥ 2000 IU/ml; b. ALT ≥ 2 times the ULN; c. Exclusion of hepatitis due to the disease itself, drugs, or other causes. All three conditions must be met. If a patient initially has active HBV infection and turns into inactive HBV infection after anti-HBV treatment, they can be included in the study provided they receive adequate anti-HBV treatment.
7. 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;
8. Pregnant or breastfeeding women, and women of childbearing age who are not using contraception;
9. Hypersensitivity to the drugs or their components used in the study.

Where this trial is running

Tianjin, Tianjin Municipality

• Institute of Hematology & Blood Diseases Hospital — Tianjin, Tianjin Municipality, China (RECRUITING)

Study contacts

• Principal investigator: Shuhua Yi, Doctor — Institute of Hematology & Blood Diseases Hospital, China
• Study coordinator: Shuhua Yi, Doctor

Conditions: T-LGL Leukemia, NK-LGL Leukemia, Large granular lymphocyte leukemia, Thalidomide, Overall response rate