Histiocytosis,Langerhans-CellPhase 1/2NCT07890701
A Single-Arm,Multicenter Phase, Phase II Clinical Trial of Adebrelimab Combined With Trametinib in the Treatment of Refractory Recurrent Langerhans Cell Histiocytosis in Children and Adolescents
A Phase 1/2 study testing treated group in people with Histiocytosis,Langerhans-Cell, run by Second Affiliated Hospital of Wenzhou Medical University at 1 site in China.
Trial summary
Based on clinical observation data from two previously enrolled cases, the adezbelimab combined with trametinib regimen showed good safety in pediatric refractory relapsed LCH, with good drug tolerance. Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Histiocytosis,Langerhans-Cell
- What is being tested?
- treated group
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteChina
- Study dates
- 20 Oct 2026to 1 Nov 2027
- Phase
- Phase 1/2
- 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 Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- LCH diagnostic criteria: lesion biopsy pathology for LCH, immunohistochemical CD1a and/or CD207 (Langerin) positive [refer to the Chinese Children's Histiocytic Group (CCHG) CCHG-LCH-2019 research protocol];
- Cases must meet one of the following criteria for refractory or relapsed LCH:
- 1) Refractory LCH: SS-LCH patients have responded to at least first-line therapy and have not met improvement criteria with second-line treatments based on klatropine or clofarabine; or MS-LCH patients do not achieve improvement after one course of first-line treatment; 2) Recurrent LCH: After complete response in SS-LCH or MS-LCH patients, new lesions reappear or the original lesion enlarges again.
- 3. Age 1-18 years; 4. All patients must sign informed consent forms. 5. ECOG PS score: 0-1; (Or according to the Karnofsky/Lansky Performance State Scale, performance score >=50%) 6. Expected survival time≥ 6 months; 7. Essential organ functions are basically normal:
- Good cardiac function: echocardiography shows cardiac ejection fraction ≥50%, ECG shows no obvious abnormalities;
- Good renal function: serum creatinine ≤2.0×ULN;
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- Good liver function: ALT and AST ≤ 5.0×ULN; Total bilirubin ≤ 2.5× ULN (Gilbert syndrome requires total bilirubin ≤ 3.0× ULN); For patients with liver disease caused by histiocytosis: abnormal levels of bilirubin, aspartate aminotransferase, alanine aminotransferase, and albumin may be observed.
- Good bone marrow proliferation: absolute neutrophil count (ANC) ≥1.0×109/L, hemoglobin ≥80 g/L (may receive red blood cell transfusion), platelet count ≥ 75×109/L (transfusion-independent, defined as no platelet transfusion within 7 days prior to enrollment).
- Blood oxygen saturation >91% when not receiving oxygen;
Who cannot join
- Those who have used similar anti-PD-1 or anti-PD-L1 monoclonal antibodies or related pathway targeted drugs;
- Participating in other clinical investigators simultaneously;
- Previous treatment toxicity has not recovered and remains above grade 1 (CTCAE V5.0 criteria) (excluding alopecia and neurotoxicity);
- Those allergic to the active ingredients and excipients of the investigational drugs trimetinib and/or adezbelizumab;
- History of autoimmune diseases, including but not limited to systemic lupus erythematosus, psoriasis, rheumatoid arthritis, inflammatory bowel disease, Hashimoto's thyroiditis, etc.;
- Individuals with a past or current active tuberculosis infection;
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- Active infections requiring systemic treatment;
- Complicated by serious diseases, including but not limited to: uncontrolled diabetes, active peptic ulcers, etc.;
- Positive antibodies against HIV, TP, HCV, HBV Ag, and HBV DNA copy count above the upper limit of normal detection units;
- Thyroid dysfunction (FT3, FT4, T3, T4);
- Administering live or attenuated vaccines within 4 weeks before dosing, during treatment, or within 5 months after the last dose;
- Candidates deemed unsuitable for the trial due to other reasons determined by the investigator;
- Those who, due to personal reasons, are unable to follow the established plan during treatment;
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
Second Affiliated Hospital of Wenzhou Medical University
Wenzhou, Zhejiang
About this study
Based on clinical observation data from two previously enrolled cases, the adezbelimab combined with trametinib regimen showed good safety in pediatric refractory relapsed LCH, with good drug tolerance. Both children benefited clinically from the combination regimen, preliminarily validating the feasibility and effectiveness of this regimen in treating refractory relapsed LCH in children. To systematically validate the clinical efficacy and safety of this combination regimen in treating refractory relapsed LCH, expand the sample size, and improve evidence-based medical evidence, this study plans to conduct a prospective single-arm, multicenter Phase II clinical trial of adebrelimab combined with trametinib for refractory Langerhans cell histiocytosis, aiming to optimize treatment regimens for children with refractory relapsed LCH and further improve clinical survival rates and long-term quality of life.
- Conditions
- Histiocytosis,Langerhans-Cell
- Being tested
- treated group
- 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.
treated groupDrug
Preliminary treatment: Trametinib is administered for 1-2 weeks (no more than 2 weeks) depending on tumor burden; For patients with poor treatment response (disease activity score with at least one other than blood count markers above 1), start on day 11 with cytacitine 100mg/m2 once daily for 12 hours for 2-4 days.
Combination therapy: each cycle lasts 28 days, with atezolimab on day 1 and trametinib on days 22-28, totaling 6 cycles.
| Study group | Role | What they receive |
|---|---|---|
| Adebrelimab | Experimental | Not described Receives: Drug: treated group |
What is being measured
The primary outcome is the main question the study is designed to answer.
the quantitative Disease Activity Score, DAS
Measured: Monthly assessments during the first three months of treatment, and every three months thereafter; Assessments are conducted at 3 months, half a year, 1 year, 2 years, and 3 years after discontinuation.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Oct 2026
- Study start20 Oct 2026 (estimated)
- Primary completion1 Oct 2027 (estimated)
- Study completion1 Nov 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Histiocytosis,Langerhans-CellStudies of the same condition, open and under way.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Second Affiliated Hospital of Wenzhou Medical University
- Sponsor type
- University, hospital or other
- Collaborators
- Children's Hospital of Fudan University, Anhui Provincial Children's Hospital, Xiangya Hospital of Central South University, First Affiliated Hospital of Guangxi Medical University, Xian Children's Hospital, Children's Hospital of Soochow University, The Affiliated Hospital Of Guizhou Medical University, The Second Hospital of Anhui Medical University, The First Affiliated Hospital of University of Science and Technology of China, Children's Hospital of Chongqing Medical University, Tongji Hospital, Fujian Medical University Union Hospital, Zhangzhou Municipal Hospital of Fujian Province, Jiangxi Provincial Children Hospital, Sichuan Cancer Hospital and Research Institute, Xuzhou Children Hospital, Shanghai Children's Medical Center, Zhengzhou Children's Hospital, China
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07890701
- Sponsor's protocol ID
- SAHoWMU-CR2026-06-123
- Status last verified
- Oct 2026
- First submitted
- 16 Oct 2026
- Last updated
- 22 Oct 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 22 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-Arm,Multicenter Phase, Phase II Clinical Trial of Adebrelimab Combined With Trametinib in the Treatment of Refractory Recurrent Langerhans Cell Histiocytosis in Children and Adolescents
- ClinicalTrials.gov ID
- NCT07890701
- Acronym / sponsor ID
- SAHoWMU-CR2026-06-123
- Conditions
- Histiocytosis,Langerhans-Cell
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 2 participants (estimated)
- Lead sponsor
- Second Affiliated Hospital of Wenzhou Medical UniversityUniversity, hospital or other
- Collaborators
- Children's Hospital of Fudan University, Anhui Provincial Children's Hospital, Xiangya Hospital of Central South University, First Affiliated Hospital of Guangxi Medical University, Xian Children's Hospital, Children's Hospital of Soochow University, The Affiliated Hospital Of Guizhou Medical University, The Second Hospital of Anhui Medical University, The First Affiliated Hospital of University of Science and Technology of China, Children's Hospital of Chongqing Medical University, Tongji Hospital, Fujian Medical University Union Hospital, Zhangzhou Municipal Hospital of Fujian Province, Jiangxi Provincial Children Hospital, Sichuan Cancer Hospital and Research Institute, Xuzhou Children Hospital, Shanghai Children's Medical Center, Zhengzhou Children's Hospital, China
- Study start
- 20 Oct 2026 (estimated)
- Primary completion
- 1 Oct 2027 (estimated)
- Study completion
- 1 Nov 2027 (estimated)
- First posted
- 22 Oct 2026 (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.
Based on clinical observation data from two previously enrolled cases, the adezbelimab combined with trametinib regimen showed good safety in pediatric refractory relapsed LCH, with good drug tolerance. Both children benefited clinically from the combination regimen, preliminarily validating the feasibility and effectiveness of this regimen in treating refractory relapsed LCH in children. To systematically validate the clinical efficacy and safety of this combination regimen in treating refractory relapsed LCH, expand the sample size, and improve evidence-based medical evidence, this study plans to conduct a prospective single-arm, multicenter Phase II clinical trial of adebrelimab combined with trametinib for refractory Langerhans cell histiocytosis, aiming to optimize treatment regimens for children with refractory relapsed LCH and further improve clinical survival rates and long-term quality of life.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Adebrelimab | Experimental | Drug: treated group |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- LCH diagnostic criteria: lesion biopsy pathology for LCH, immunohistochemical CD1a and/or CD207 (Langerin) positive [refer to the Chinese Children's Histiocytic Group (CCHG) CCHG-LCH-2019 research protocol];
- Cases must meet one of the following criteria for refractory or relapsed LCH:
- 1) Refractory LCH: SS-LCH patients have responded to at least first-line therapy and have not met improvement criteria with second-line treatments based on klatropine or clofarabine; or MS-LCH patients do not achieve improvement after one course of first-line treatment; 2) Recurrent LCH: After complete response in SS-LCH or MS-LCH patients, new lesions reappear or the original lesion enlarges again.
- 3. Age 1-18 years; 4. All patients must sign informed consent forms. 5. ECOG PS score: 0-1; (Or according to the Karnofsky/Lansky Performance State Scale, performance score >=50%) 6. Expected survival time≥ 6 months; 7. Essential organ functions are basically normal:
- Good cardiac function: echocardiography shows cardiac ejection fraction ≥50%, ECG shows no obvious abnormalities;
Key exclusion criteria (13 listed)
- Those who have used similar anti-PD-1 or anti-PD-L1 monoclonal antibodies or related pathway targeted drugs;
- Participating in other clinical investigators simultaneously;
- Previous treatment toxicity has not recovered and remains above grade 1 (CTCAE V5.0 criteria) (excluding alopecia and neurotoxicity);
- Those allergic to the active ingredients and excipients of the investigational drugs trimetinib and/or adezbelizumab;
- History of autoimmune diseases, including but not limited to systemic lupus erythematosus, psoriasis, rheumatoid arthritis, inflammatory bowel disease, Hashimoto's thyroiditis, etc.;
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 |
|---|---|---|
| treated groupPreliminary treatment: Trametinib is administered for 1-2 weeks (no more than 2 weeks) depending on tumor burden; For patients with poor treatment response (disease activity score with at least one other than blood count markers above 1), start on day 11 with cytacitine 100mg/m2 once daily for 12 hours for 2-4 days. Combination therapy: each cycle lasts 28 days, with atezolimab on day 1 and trametinib on days 22-28, totaling 6 cycles. | Drug | Adebrelimab |
Primary outcome
the quantitative Disease Activity Score, DAS
Time frame: Monthly assessments during the first three months of treatment, and every three months thereafter; Assessments are conducted at 3 months, half a year, 1 year, 2 years, and 3 years after discontinuation.
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 Oct 2026.
- Individual participant dataSharing planned: Undecided
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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.
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 1/2 study testing treated group in people with Histiocytosis,Langerhans-Cell, run by Second Affiliated Hospital of Wenzhou Medical University 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 9 inclusion and 13 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: enrolling by invitation, as of the sponsor's last update on 22 Oct 2026. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
the quantitative Disease Activity Score, DAS (time frame: Monthly assessments during the first three months of treatment, and every three months thereafter; Assessments are conducted at 3 months, half a year, 1 year, 2 years, and 3 years after discontinuation.). 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 Oct 2026.