ClinicalTrials.gov record NCT06530576

Thalidomide for the Symptomatic Large Granular Lymphocytic Leukemia

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

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

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 2A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.
Age18 years old and older
SexAll
LocationChina
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is a phase 2, single-group study testing oral thalidomide in adults with symptomatic T-LGL or NK-LGL leukemia. Treatment is given in 4-month cycles (thalidomide 50–100 mg nightly) for up to three cycles; methotrexate may be added if a sufficient response is not reached. The main outcome is overall response rate; safety and remission outcomes are also measured.

What it is studying. The study tests whether thalidomide given alone produces hematologic responses in symptomatic large granular lymphocytic leukemia and measures overall response rate. It also records safety (adverse events) and tracks outcomes such as complete remission rate, the effect of adding methotrexate when needed, progression-free survival, overall survival, and duration of remission.

What the researchers hope to learn. Primary scientific goal: measure overall response rate (complete plus partial responses) to thalidomide monotherapy. Secondary goals: assess safety (adverse events), complete remission rate, how much adding methotrexate changes response, progression-free survival, overall survival, and duration of remission.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Thalidomide and methotrexate

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.
  • 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.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

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.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants take oral thalidomide nightly (50–100 mg) in 4‑month treatment cycles with response checks after each cycle. If response is inadequate, oral methotrexate (10 mg/m2 once weekly) may be added and the combined regimen continued; treatment is allowed for up to three 4‑month cycles. If complete remission is achieved, one additional consolidation cycle may be given before stopping; patients who still lack partial remission after two cycles of the combination are withdrawn. The study includes regular safety monitoring and recording of adverse events and other assessments during treatment and follow‑up.

How the study is structured. Interventional, open-label, single-group phase 2 trial. Participants receive oral thalidomide nightly in 4-month cycles; effectiveness is assessed after each cycle. If response criteria are not met per protocol, methotrexate is added orally once weekly. Treatment continues up to three 4-month cycles or until protocol-defined stopping rules.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on large granular lymphocytic leukemia

Clinical trials16 trials listed for large granular lymphocytic leukemiaEvery trial the registry lists for large granular lymphocytic leukemia, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in large granular lymphocytic leukemia researchLatest monthly update: September 2026. Includes: Treatment response reports. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Aug 10, 2024
Primary completion
Jun 10, 2026 (estimated)
Study completion
Jul 1, 2027 (estimated)
Target enrollment
48 participants (estimated)
Last updated on registry
Nov 18, 2025
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Institute of Hematology & Blood Diseases Hospital, China

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

Terms used on this page · explanations by MedicalUpdates.ai

complete response
No sign of the condition can be found with the tests used in the study. It does not always mean the condition is gone permanently, and the definition depends on the condition and the study.
endpoint
Something the study measures to judge its results, such as a symptom score, a blood test or survival. Outcomes are grouped as primary, secondary and sometimes exploratory.
informed consent form
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
relapsed
A condition that improved with treatment and has since returned or worsened.
ANC
The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
ECOG score
A scale from 0 to 5 that describes how a person's condition affects daily activity. 0 means fully active; 1 means restricted in strenuous activity; 2 means able to self-care but unable to work; higher numbers mean more limitation.
upper limit of normal
The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
creatinine clearance
A measure of kidney function, calculated from a blood creatinine level together with age, weight and sex.
biopsy
Removal of a small piece of tissue so it can be examined under a microscope. 'Biopsy-proven' or 'histologically confirmed' means the diagnosis was confirmed by examining tissue under a microscope.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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