T-LGL LeukemiaPhase 2NCT06530576
Thalidomide for the Symptomatic Large Granular Lymphocytic Leukemia
A Phase 2 study testing Thalidomide and methotrexate in people with T-LGL Leukemia and NK-LGL Leukemia, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial 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%. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- T-LGL Leukemia, NK-LGL Leukemia
- What is being tested?
- Thalidomide and methotrexate
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 1 siteChina
- Study dates
- 10 Aug 2024to 1 Jul 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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.
Who cannot join
- 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);
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Institute of Hematology & Blood Diseases Hospital
Tianjin, Tianjin Municipality, 300020
Shuhua Yi, Dr., 86-022-23909106, yishuhua@ihcams.ac.cn
Lugui Qiu, Dr., 86-022-23909286, qiulg@ihcams.ac.cn
About this study
Official title: The Efficacy of Thalidomide for the Symptomatic Large Granular Lymphocytic Leukemia - a Prospective Phase Ⅱ Multicenter Clinical Trial From China
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.
Read the detailed description
The regimen is an oral treatment with a cycle of 4 months. All patients first receive monotherapy with thalidomide at a dose of 50-100 mg/QN. After 4 months, the efficacy is evaluated. If effective, the monotherapy continues for another 4 months. If complete remission (CR) is achieved, the treatment is consolidated for another 4 months before stopping, for a total of 3 courses. If CR is achieved at any point within the first year, the treatment is consolidated for one more course and then stopped. If partial remission (PR) is not achieved within the first cycle, or CR is not achieved within the second cycle, methotrexate 10 mg/m2 orally once a weekis a is dded for consolidation, with each cycle lasting 4 months. After a maximum of 3 cycles, treatment is stopped. Patients who do not achieve PR after 2 cycles of the TM regimen will be withdrawn from the study, with a maximum of 3 cycles allowed.
- Conditions
- T-LGL Leukemia, NK-LGL Leukemia
- Being tested
- Thalidomide and methotrexate
- Keywords
- Large granular lymphocyte leukemia, Thalidomide, Overall response rate
- Medical subject headings
- Leukemia, Large Granular Lymphocytic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Thalidomide and methotrexateDrug
Thalidomide at a dose of 50-100 mg/QN, Methotrexate 10 mg/m2 orally once a week.
| Study group | Role | What they receive |
|---|---|---|
| Thalidomide | Experimental | All patients first receive thalidomide 100mg monotherapy. Receives: Drug: Thalidomide and methotrexate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall response rate
complete remission rate+ partial remission rate
Measured: up to 5 years
Secondary outcomes (6)
The safety of thalidomide
Incidence of adverse events, serious adverse events and significant adverse event
Measured: up to 5 years
Complete remession rate
Hematological PR was defined as an improvement in blood counts ANC > 0.5 × 109/L; HGB increased by >1 g/dL; PLT > 50 × 109/L
Measured: up to 5 years
The rate of improvement in efficacy after TM combination
The effectiveness of the TM regimen is measured by subtracting the response rate of single-agent thalidomide
Measured: up to 5 years
Progression-free survival
Progression-free survival
Measured: up to 5 years
Overall survival
The time from the start of treatment to the patient's death from any cause
Measured: up to 5 years
Duration of remission
the time from response to progression/death (P/D)
Measured: up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jul 2024
- Study start10 Aug 2024
- Primary completion10 Jun 2026 (estimated)
- Study completion1 Jul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for T-LGL LeukemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Thalidomide at a dose of 50-100 mg/QN
- Masking
- None (open label)
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, China
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Shuhua Yi, Doctor, Principal investigator, Institute of Hematology & Blood Diseases Hospital, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06530576
- Sponsor's protocol ID
- IIT2024041
- Status last verified
- Aug 2025
- First submitted
- 28 Jul 2024
- Last updated
- 18 Nov 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Efficacy of Thalidomide for the Symptomatic Large Granular Lymphocytic Leukemia - a Prospective Phase Ⅱ Multicenter Clinical Trial From China
- ClinicalTrials.gov ID
- NCT06530576
- Acronym / sponsor ID
- IIT2024041
- Conditions
- T-LGL Leukemia, NK-LGL Leukemia
- Keywords
- Large granular lymphocyte leukemia, Thalidomide, Overall response rate
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 10 Aug 2024 (actual)
- Primary completion
- 10 Jun 2026 (estimated)
- Study completion
- 1 Jul 2027 (estimated)
- First posted
- 31 Jul 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
The regimen is an oral treatment with a cycle of 4 months. All patients first receive monotherapy with thalidomide at a dose of 50-100 mg/QN. After 4 months, the efficacy is evaluated. If effective, the monotherapy continues for another 4 months. If complete remission (CR) is achieved, the treatment is consolidated for another 4 months before stopping, for a total of 3 courses. If CR is achieved at any point within the first year, the treatment is consolidated for one more course and then stopped. If partial remission (PR) is not achieved within the first cycle, or CR is not achieved within the second cycle, methotrexate 10 mg/m2 orally once a weekis a is dded for consolidation, with each cycle lasting 4 months. After a maximum of 3 cycles, treatment is stopped. Patients who do not achieve PR after 2 cycles of the TM regimen will be withdrawn from the study, with a maximum of 3 cycles allowed.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Thalidomide at a dose of 50-100 mg/QN
| Arm | Type | Receives |
|---|---|---|
| Thalidomide | Experimental | Drug: Thalidomide and methotrexateAll patients first receive thalidomide 100mg monotherapy. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- 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:
Key exclusion criteria (9 listed)
- 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);
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Thalidomide and methotrexateThalidomide at a dose of 50-100 mg/QN, Methotrexate 10 mg/m2 orally once a week. | Drug | Thalidomide |
Primary outcome
Overall response rate
Time frame: up to 5 years
complete remission rate+ partial remission rate
6 secondary outcomes
The safety of thalidomide
Time frame: up to 5 years
Complete remession rate
Time frame: up to 5 years
The rate of improvement in efficacy after TM combination
Time frame: up to 5 years
Progression-free survival
Time frame: up to 5 years
Overall survival
Time frame: up to 5 years
Duration of remission
Time frame: up to 5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Nov 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Shuhua Yi, Doctor principal investigator
Institute of Hematology & Blood Diseases Hospital, China
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Thalidomide and methotrexate in people with T-LGL Leukemia and NK-LGL Leukemia, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 18 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Shuhua Yi, Doctor (86-22-23909106, yishuhua@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall response rate (time frame: up to 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Nov 2025.