ClinicalTrials.gov record NCT07187193

Efficacy and Safety of Low-Dose Cytarabine Combined With Thalidomide in Adult Patients With Untreated LCH

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

Efficacy and Safety of Low-Dose Cytarabine Combined With Thalidomide in the Treatment of Adult Patients With Untreated Langerhans Cell Histiocytosis: A Prospective, Multicenter, Single-Arm Study.

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 to 70 years old
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.

A phase 2, single‑arm study testing a combined regimen of low‑dose cytarabine plus thalidomide in adults with newly diagnosed Langerhans Cell Histiocytosis (LCH). The planned treatment is 12 cycles of combination therapy (Ara‑C days 1–5 plus thalidomide days 1–35, every 35 days) followed by 12 cycles of thalidomide maintenance (100 mg daily, 28‑day cycles), for 24 cycles total. The study will record responses, survival outcomes, and adverse events over up to 3 years. This is a single‑group, open‑label interventional study.

What it is studying. Whether the combination of low‑dose cytarabine and thalidomide is safe and how patients with untreated adult LCH respond to this regimen.

What the researchers hope to learn. To measure event‑free survival (EFS), overall survival (OS), overall response rate (ORR), and to record adverse events for this treatment regimen in adults with untreated LCH over a follow‑up of up to three years.

What this study is about

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

Treatment of Adult Patients with Newly Diagnosed Langerhans Cell Histiocytosis (LCH) Using a Low-Dose Cytarabine Combined with Thalidomide Regimen.

Interventions being studied

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

Drug
  • Cytarabine (Ara-C)
  • Thalidomide (100mg)

Eligibility

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

Inclusion criteria

  • Clearly diagnose and treat adult patients with multisystem or single-system multifocal LCH;
  • Aged between 18 and 70 years;
  • Multisystem involvement or single-system multifocal disease;
  • No prior systemic treatment (patients who have only received local radiotherapy or surgery may be enrolled);
  • ECOG performance status score ≤ 2;
  • Judged by clinicians as suitable for treatment with this protocol;
  • Patients or their families able to understand the study protocol and willing to participate in the study, providing written informed consent.

Exclusion criteria

  • Patients with LCH involving the central nervous system;
  • Single-system single-lesion LCH;
  • Subjects who have undergone major surgery within 4 weeks prior to the first dose in the study;
  • Subjects who have received radiotherapy within 4 weeks prior to the first dose in the study; subjects with a history of myocardial infarction within the past year;
  • Patients with New York Heart Association (NYHA) class 3 or 4 congestive heart failure, or a history of NYHA class 3 or 4 congestive heart failure;
  • Pregnant or lactating women;
  • Abnormal liver and kidney function: creatinine level ≥176.8μmol/l (2mg/dl), transaminase and bilirubin levels more than 2 times the upper limit of normal (for LCH patients with liver involvement, bilirubin levels more than 10 times the upper limit of normal);
  • Abnormal blood counts: absolute neutrophil count less than 1×10\^9/L, platelet count less than 50×10\^9/L;
  • Patients or their families unable to understand the conditions and objectives of this study;
  • Any other circumstances in which the investigator deems the patient unsuitable to participate in this trial.

Study description and what participation involves

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

Detailed description from the registry

AraC 100 mg/m² d1-5+ thalidomide 100mg d1-35, q35 days ,for a total of 12 cycles;followed by thalidomide 100mg, qd1-28 q28d,for a total of 12 cycles of monotherapy maintenance reatment;

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 receive combination treatment: cytarabine (Ara‑C) 100 mg/m² on days 1–5 plus thalidomide 100 mg on days 1–35 every 35 days for 12 cycles, followed by thalidomide 100 mg on days 1–28 every 28 days as monotherapy for 12 maintenance cycles (24 cycles total). They will have regular clinic visits during treatment for safety monitoring, blood tests and other assessments, and side‑effect reporting (to be recorded and graded). Outcomes including event‑free survival and overall survival are followed until about 3 years from enrollment. Investigators will perform any additional tests or imaging needed to evaluate response and safety during the study.

How the study is structured. Interventional phase 2, single‑group (no comparison or placebo), open‑label trial. Participants receive repeated cycles of combination cytarabine plus thalidomide, then thalidomide alone for maintenance, with outcomes tracked for up to three years. The study measures event‑free survival, overall survival, response rate, and records adverse events.

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 Langerhans cell histiocytosis

Clinical trials20 trials listed for Langerhans cell histiocytosisEvery trial the registry lists for Langerhans cell histiocytosis, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Langerhans cell histiocytosis researchLatest monthly update: September 2026. Includes: Adult routine care. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Apr 12, 2025
Primary completion
Apr 12, 2027 (estimated)
Study completion
Apr 13, 2027 (estimated)
Target enrollment
50 participants (estimated)
Last updated on registry
Sep 22, 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
Cancer Institute and Hospital, Chinese Academy of Medical Sciences

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)

  • Department of Medical Oncology, National Cancer Center/National Clinical Research Center for Cancer/Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical CollegeRecruiting

    Beijing · China

Terms used on this page · explanations by MedicalUpdates.ai

ECOG performance status
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.
written informed consent
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.
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.
absolute neutrophil count
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.
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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