Langerhans Cell HistiocytosisPhase 2NCT07187193
Efficacy and Safety of Low-Dose Cytarabine Combined With Thalidomide in Adult Patients With Untreated LCH
A Phase 2 study testing Cytarabine (Ara-C) and Thalidomide (100mg) in people with Langerhans Cell Histiocytosis, run by Cancer Institute and Hospital, Chinese Academy of Medical Sciences at 1 site in China.
Trial summary
Treatment of Adult Patients with Newly Diagnosed Langerhans Cell Histiocytosis (LCH) Using a Low-Dose Cytarabine Combined with Thalidomide Regimen. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Langerhans Cell Histiocytosis
- What is being tested?
- Cytarabine (Ara-C) and Thalidomide (100mg)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteChina
- Study dates
- 12 Apr 2025to 13 Apr 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 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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;
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
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
Xinxin XX Cao, doctor, +86-18618315968, caoxinxin@126.com
About this study
Official title: 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.
Treatment of Adult Patients with Newly Diagnosed Langerhans Cell Histiocytosis (LCH) Using a Low-Dose Cytarabine Combined with Thalidomide Regimen.
Read the detailed description
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;
- Conditions
- Langerhans Cell Histiocytosis
- Being tested
- Cytarabine (Ara-C), Thalidomide (100mg)
- 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.
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.
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.
| Study group | Role | What they receive |
|---|---|---|
| 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); Receives: Drug: Cytarabine (Ara-C); Drug: Thalidomide (100mg) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From enrollment to the end of treatment until 3 years
Secondary outcomes (3)
Overall survival (OS)
OS was defined as the time from cytarabine and thalidomide initiation to death or last follow-up.
Measured: From enrollment to the end of treatment until 3 years
Overall response rate (ORR)
The overall response rate (ORR) was defined as the cumulative proportion of patients achieving either a complete response (CR) or partial response (PR) following therapy.
Measured: From enrollment to the end of treatment until 3 years
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 until 3 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Sep 2025
- Study start12 Apr 2025
- Primary completion12 Apr 2027 (estimated)
- Study completion13 Apr 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 HistiocytosisStudies 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 prospective, multicenter, single-arm study.Planned enrollment of 50 patients.
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Xinxin XX Cao, doctor, Study chair, NCC, CICAMS
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07187193
- Sponsor's protocol ID
- NCCH006
- Status last verified
- Sep 2025
- First submitted
- 25 Aug 2025
- Last updated
- 22 Sep 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 22 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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.
- ClinicalTrials.gov ID
- NCT07187193
- Acronym / sponsor ID
- NCCH006
- Conditions
- Langerhans Cell Histiocytosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Cancer Institute and Hospital, Chinese Academy of Medical SciencesUniversity, hospital or other
- Study start
- 12 Apr 2025 (actual)
- Primary completion
- 12 Apr 2027 (estimated)
- Study completion
- 13 Apr 2027 (estimated)
- First posted
- 22 Sep 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.
Treatment of Adult Patients with Newly Diagnosed Langerhans Cell Histiocytosis (LCH) Using a Low-Dose Cytarabine Combined with Thalidomide Regimen.
Detailed description
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;
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- A prospective, multicenter, single-arm study.Planned enrollment of 50 patients.
| Arm | Type | Receives |
|---|---|---|
| Newly diagnosed LCH patients | Experimental | Drug: Cytarabine (Ara-C), Drug: Thalidomide (100mg)(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); |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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;
Key exclusion criteria (10 listed)
- 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;
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 |
|---|---|---|
| Cytarabine (Ara-C)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. | Drug | Newly diagnosed LCH patients |
| Thalidomide (100mg)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. | Drug | Newly diagnosed LCH patients |
Primary outcome
Event-Free Survival (EFS)
Time frame: From enrollment to the end of treatment until 3 years
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.
3 secondary outcomes
Overall survival (OS)
Time frame: From enrollment to the end of treatment until 3 years
Overall response rate (ORR)
Time frame: From enrollment to the end of treatment until 3 years
Adverse events
Time frame: From enrollment to the end of treatment until 3 year
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 22 Sep 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.
Xinxin XX Cao, doctor study chair
NCC, CICAMS
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 Cytarabine (Ara-C) and Thalidomide (100mg) in people with Langerhans Cell Histiocytosis, 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 10 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: looking for participants now, as of the sponsor's last update on 22 Sep 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?
Contact Xinxin XX Cao, doctor (+86-18618315968, caoxinxin@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Event-Free Survival (EFS) (time frame: From enrollment to the end of treatment until 3 years). 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 22 Sep 2025.