Langerhans Cell Histiocytosis (LCH)Phase 2NCT07204041
Efficacy and Safety of XTD Regimen (Selinexor, Thalidomide and Dexamethasone) in Adult Patients With Relapsed/Refractory LCH
A Phase 2 study testing Selinexor, Thalidomide (100mg) and Dexamethasone in people with Langerhans Cell Histiocytosis (LCH), run by Cancer Institute and Hospital, Chinese Academy of Medical Sciences at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Langerhans Cell Histiocytosis (LCH)
- What is being tested?
- Selinexor, Thalidomide (100mg) and Dexamethasone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteChina
- Study dates
- 1 Aug 2025to 2 Aug 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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);
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- 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;
Trial locations and contacts
1 site in 1 country.
China1 site
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
About this study
Official title: Efficacy and Safety of XTD Regimen (Selinexor, Thalidomide and Dexamethasone) in Adult Patients With Relapsed/Refractory Langerhans Cell Histiocytosis: A Prospective, Multicenter, Single-Arm Study.
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)
- Being tested
- Selinexor, Thalidomide (100mg), Dexamethasone
- Medical subject headings
- Histiocytosis, Langerhans-Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SelinexorDrug
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.
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.
DexamethasoneDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Drug: Selinexor; Drug: Thalidomide (100mg); Drug: Dexamethasone |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From enrollment to the end of treatment at 8 weeks
Secondary outcomes (3)
ORR
The overall response rate (ORR) was defined as the cumulative proportion of patients attaining either a complete response (CR) or partial response (PR) .
Measured: From enrollment to the end of treatment at 8 weeks
OS
OS was measured from XTD initiation to death or last follow-up
Measured: From enrollment to the end of treatment at 8 weeks
Adverse Events
Toxicities were recorded and graded per National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 5.0.
Measured: From enrollment to the end of treatment at 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Oct 2025
- Study start1 Aug 2025
- Primary completion1 Aug 2027 (estimated)
- Study completion2 Aug 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Langerhans Cell Histiocytosis (LCH)Studies of the same condition, open and under way.
- More trials run by Cancer Institute and Hospital, Chinese Academy of Medical SciencesThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- A multicenter, prospective, interventional study, with a planned number of subjects: approximately 40 cases.
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07204041
- Sponsor's protocol ID
- NCCH0027
- Status last verified
- Sep 2025
- First submitted
- 24 Sep 2025
- Last updated
- 2 Oct 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of XTD Regimen (Selinexor, Thalidomide and Dexamethasone) in Adult Patients With Relapsed/Refractory Langerhans Cell Histiocytosis: A Prospective, Multicenter, Single-Arm Study.
- ClinicalTrials.gov ID
- NCT07204041
- Acronym / sponsor ID
- NCCH0027
- Conditions
- Langerhans Cell Histiocytosis (LCH)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Cancer Institute and Hospital, Chinese Academy of Medical SciencesUniversity, hospital or other
- Study start
- 1 Aug 2025 (actual)
- Primary completion
- 1 Aug 2027 (estimated)
- Study completion
- 2 Aug 2027 (estimated)
- First posted
- 2 Oct 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- A multicenter, prospective, interventional study, with a planned number of subjects: approximately 40 cases.
| Arm | Type | Receives |
|---|---|---|
| Adult patients with relapsed or refractory LCH | Experimental | Drug: Selinexor, Drug: Thalidomide (100mg), Drug: Dexamethasone(1) Relapsed or refractory LCH; (2) Age ≥ 18 years; (3) Multi-focal single system or multi-system involvement; (4) ECOG PS 0-2 score |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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;
Key exclusion criteria (9 listed)
- 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);
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SelinexorThe 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. | Drug | Adult patients with relapsed or refractory LCH |
| Thalidomide (100mg)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. | Drug | Adult patients with relapsed or refractory LCH |
| DexamethasoneThe 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. | Drug | Adult patients with relapsed or refractory LCH |
Primary outcome
PFS
Time frame: From enrollment to the end of treatment at 8 weeks
PFS defined as the time from XTD initiation to first documented disease progression, relapse after XTD, death from any cause, or last follow-up.
3 secondary outcomes
ORR
Time frame: From enrollment to the end of treatment at 8 weeks
OS
Time frame: From enrollment to the end of treatment at 8 weeks
Adverse Events
Time frame: From enrollment to the end of treatment at 8 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Oct 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Selinexor, Thalidomide (100mg) and Dexamethasone in people with Langerhans Cell Histiocytosis (LCH), run by Cancer Institute and Hospital, Chinese Academy of Medical Sciences at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 2 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
PFS (time frame: From enrollment to the end of treatment at 8 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Oct 2025.