Low-dose cytarabine combined with thalidomide for adults with newly diagnosed LCH

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.

This trial tests whether combining low-dose cytarabine with thalidomide is safe and effective for adults newly diagnosed with multisystem or multifocal Langerhans cell histiocytosis.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Ages: 18 Years to 70 Years
• Sex: All
• Sponsor: Cancer Institute and Hospital, Chinese Academy of Medical Sciences (other)
• Locations: 1 site (Beijing)
• Trial ID: NCT07187193 on ClinicalTrials.gov

What this trial studies

Adults aged 18–70 with newly diagnosed multisystem or single-system multifocal LCH who have not received prior systemic therapy will receive induction cycles of cytarabine 100 mg/m² daily on days 1–5 plus thalidomide 100 mg daily on days 1–35 every 35 days for up to 12 cycles. Patients completing induction move to maintenance thalidomide 100 mg daily given on a 28-day cycle for up to 12 cycles. The phase 2, single-arm protocol monitors safety, response, and tolerability with regular clinical and laboratory assessments. Patients with CNS involvement, single-lesion disease, recent major surgery or radiotherapy, or significant cardiac issues are excluded.

Who should consider this trial

Good fit: Adults 18–70 years old with newly diagnosed multisystem or single-system multifocal LCH, ECOG performance status ≤2, no prior systemic therapy, and able to attend the Beijing treatment center are ideal candidates.

Not a fit: Patients with isolated single-lesion LCH, central nervous system involvement, recent major surgery or radiotherapy, significant recent cardiac events, or prior systemic treatment are excluded and unlikely to benefit from this protocol.

Why it matters

Potential benefit: If successful, this regimen could provide an effective, outpatient-friendly systemic option that controls disease with potentially manageable toxicity in adults with newly diagnosed multisystem or multifocal LCH.

How similar studies have performed: Cytarabine has shown activity in adult LCH and thalidomide has immunomodulatory effects, but the specific low-dose cytarabine plus thalidomide combination has limited large-trial data and is relatively untested in this exact regimen.

Eligibility criteria

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.

Where this trial is running

Beijing

• Department of Medical Oncology, National Cancer Center/National Clinical Research Center for Cancer/Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College — Beijing, China (RECRUITING)

Study contacts

• Study coordinator: Xinxin XX Cao, doctor

Conditions: Langerhans Cell Histiocytosis