Efficacy and Safety of XTD Regimen (Selinexor, Thalidomide and Dexamethasone) in Adult Patients With Relapsed/Refractory LCH

NCT07204041 | Active Not Recruiting Phase 2

• 1 other identifier: NCCH0027
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

In adult patients with relapsed/refractory Langerhans cell histiocytosis (LCH), a treatment regimen of XTD regimen (Selinexor, Thalidomide and Dexamethasone) is planned to be used.

Conditions

Langerhans Cell Histiocytosis (LCH)

Outcome Measures

Primary Outcomes (1)

• PFS

  PFS defined as the time from XTD initiation to first documented disease progression, relapse after XTD, death from any cause, or last follow-up.

  From enrollment to the end of treatment at 8 weeks

Secondary Outcomes (3)

• ORR

  From enrollment to the end of treatment at 8 weeks

• OS

  From enrollment to the end of treatment at 8 weeks

• Adverse Events

  From enrollment to the end of treatment at 8 weeks

Other Outcomes (2)

• Correlation between the positivity of NGS in MAPK pathway and therapeutic efficacy/PFS

  From enrollment to the end of treatment at 8 weeks

• Fact-G

  From enrollment to the end of treatment at 8 weeks

Study Arms (1)

Adult patients with relapsed or refractory LCH

EXPERIMENTAL

(1) Relapsed or refractory LCH; (2) Age ≥ 18 years; (3) Multi-focal single system or multi-system involvement; (4) ECOG PS 0-2 score

Drug: Selinexor; Drug: Thalidomide (100mg); Drug: Dexamethasone

Interventions

Selinexor DRUG

The combined treatment period includes 12 cycles: receiving Selinexor (60mg, D1, 8, 15, 22), Thalidomide (100mg, D1-28), and Dexamethasone (40mg, D1, 8, 15, 22) as oral treatment, with each cycle lasting 28 days, for a total of 12 cycles of combined treatment, or until disease progression, death, or occurrence of intolerable toxicity. Alternatively, until disease progression, death, or occurrence of intolerable toxicity.

Adult patients with relapsed or refractory LCH

Thalidomide (100mg) DRUG

The combined treatment period includes 12 cycles: receiving Selinexor (60mg, D1, 8, 15, 22), Thalidomide (100mg, D1-28), and Dexamethasone (40mg, D1, 8, 15, 22) as oral treatment, with each cycle lasting 28 days, for a total of 12 cycles of combined treatment, or until disease progression, death, or occurrence of intolerable toxicity. Alternatively, until disease progression, death, or occurrence of intolerable toxicity.

Adult patients with relapsed or refractory LCH

Dexamethasone DRUG

The combined treatment period includes 12 cycles: receiving Selinexor (60mg, D1, 8, 15, 22), Thalidomide (100mg, D1-28), and Dexamethasone (40mg, D1, 8, 15, 22) as oral treatment, with each cycle lasting 28 days, for a total of 12 cycles of combined treatment, or until disease progression, death, or occurrence of intolerable toxicity. Alternatively, until disease progression, death, or occurrence of intolerable toxicity.

Adult patients with relapsed or refractory LCH

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Organ pathology confirmed diagnosis of LCH;
• Age 18 years or older;
• Multi-system involvement, or single system with multiple lesions;
• Disease not relieved after receiving at least one systemic treatment, or disease relapsed after improvement;
• ECOG performance status score ≤2;
• Clinical physician determines suitability for this treatment protocol;
• Subjects can understand the study protocol and are willing to participate in this study, providing written informed consent.

You may not qualify if:

• Single system single lesion LCH
• Underwent major surgery within 4 weeks prior to the first administration of the study drug;
• Underwent radiotherapy within 4 weeks prior to the first administration of the study drug;
• History of myocardial infarction within the past year; suffers from New York Heart Association (NYHA) class 3 or 4 congestive heart failure, or has a history of NYHA class 3 or 4 congestive heart failure, unless left ventricular ejection fraction (LVEF) ≥ 50% in the echocardiogram (ECHO) screening performed within 1 month before entering the study;
• Pregnant or breastfeeding women (women of childbearing age with positive pregnancy test at baseline or who have not undergone pregnancy testing. Postmenopausal women must have been menopausal for at least 12 months);
• 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, transaminase levels more than 10 times and bilirubin levels more than 3 times the upper limit of normal);
• Severe hematological abnormalities: absolute neutrophil count less than 1 × 10\^9/L, platelet less than 50×10\^9/L;
• Presence of uncontrolled infections;
• Any other circumstances that the investigator believes to be inappropriate for the patient 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,

Beijing, China

Location

MeSH Terms

Conditions

Histiocytosis, Langerhans-Cell

Interventions

selinexor; Thalidomide; Dexamethasone

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Phthalimides; Phthalic Acids; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Piperidones; Piperidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Isoindoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Steroids, Fluorinated

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 multicenter, prospective, interventional study, with a planned number of subjects: approximately 40 cases.

Study Record Dates

• First Submitted: September 24, 2025
• First Posted: October 2, 2025
• Study Start: August 1, 2025
• Primary Completion (Estimated): August 1, 2027
• Study Completion (Estimated): August 2, 2027
• Last Updated: October 2, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)