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

NCT07187193 | Recruiting Phase 2

• 1 other identifier: NCCH006
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Langerhans Cell Histiocytosis

Outcome Measures

Primary Outcomes (1)

• Event-Free Survival (EFS)

  The primary endpoint was event-free survival (EFS), with events defined as poor response (stable disease or progressive disease) to cytarabine and thalidomide therapy, or death from any cause. EFS was measured from initiation of cytarabine and thalidomide therapy until the date of the first documented event, with censoring of patients without events at the last follow-up.

  From enrollment to the end of treatment until 3 years

Secondary Outcomes (3)

• Overall survival (OS)

  From enrollment to the end of treatment until 3 years

• Overall response rate (ORR)

  From enrollment to the end of treatment until 3 years

• Adverse events

  From enrollment to the end of treatment until 3 year

Other Outcomes (2)

• FACT-G (Functional Assessment of Cancer Therapy-General)

  From enrollment to the end of treatment until 3 years

• Correlation between next-generation sequence (NGS) / circulating free DNA (cfDNA) and efficacy/EFS

  From enrollment to the end of treatment until 3 years

Study Arms (1)

Newly diagnosed LCH patients

EXPERIMENTAL

(1) The histopathological diagnosis of LCH is confirmed; (2) Multi-system involvement or single-system with multiple lesions; (3) No prior systemic treatment (patients who have only received local radiotherapy or surgery are eligible);

Drug: Cytarabine (Ara-C); Drug: Thalidomide (100mg)

Interventions

Cytarabine (Ara-C) DRUG

The treatment regimen consists of Ara-C administered at a dose of 100 mg/m² from days 1 to 5, combined with thalidomide at a dose of 100 mg from days 1 to 35, repeated every 35 days for a total of 12 cycles. Following this, thalidomide will be given as monotherapy at a dose of 100 mg from days 1 to 28, administered every 28 days for an additional 12 cycles of maintenance treatment. In total, participants will undergo 24 cycles of treatment.

Newly diagnosed LCH patients

Thalidomide (100mg) DRUG

The treatment regimen consists of AraC administered at a dose of 100 mg/m² from days 1 to 5, combined with thalidomide at a dose of 100 mg from days 1 to 35, repeated every 35 days for a total of 12 cycles. Following this, thalidomide will be given as monotherapy at a dose of 100 mg from days 1 to 28, administered every 28 days for an additional 12 cycles of maintenance treatment. In total, participants will undergo 24 cycles of treatment.

Newly diagnosed LCH patients

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Cancer Institute and Hospital, Chinese Academy of Medical Sciences: lead

Study 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 College

Beijing, China

RECRUITING

MeSH Terms

Conditions

Histiocytosis, Langerhans-Cell

Interventions

Cytarabine; Thalidomide

Condition Hierarchy (Ancestors)

Lung Diseases, Interstitial; Lung Diseases; Respiratory Tract Diseases; Histiocytosis; Lymphatic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Arabinonucleosides; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Phthalimides; Phthalic Acids; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Piperidones; Piperidines; Isoindoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• Xinxin XX Cao, doctor

  NCC, CICAMS

  STUDY CHAIR

Central Study Contacts

Xinxin XX Cao, doctor

CONTACT

+86-18618315968; caoxinxin@126.com

Huilei HL Miao, doctor

CONTACT

+86-18801317695; miaohl13@163.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: A prospective, multicenter, single-arm study.Planned enrollment of 50 patients.

Study Record Dates

• First Submitted: August 25, 2025
• First Posted: September 22, 2025
• Study Start: April 12, 2025
• Primary Completion (Estimated): April 12, 2027
• Study Completion (Estimated): April 13, 2027
• Last Updated: September 22, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)