Luvometinib in Pediatric SS-LCH With Special-site Single/Multifocal Bone Lesions

January 18, 2026 updated by: Ju Gao, West China Second University Hospital

A Multi-center, Open-label, Single-arm Study of Luvometinib Monotherapy in Pediatric Langerhans Cell Histiocytosis With Single-system Special-site Single and Multifocal Bone Involvement

Langerhans cell histiocytosis (LCH) is the most common type of histiocytic disorder in children, affecting about 2.6 to 8.9 out of every million kids each year. It can look very different from one child to another-some cases get better on their own-but when it affects special bones (like the base of the skull, temporal bone, eye socket, or spine) or when there are multiple bone lesions in one system, children often face a higher risk of long-term complications and the disease coming back.Current guidelines in China and around the world recommend treating these children with whole-body therapy, usually a chemotherapy combination of vinblastine and prednisone. However, even with longer treatment courses, about 27.6% of children with multiple bone lesions still have the disease return, and less than 70% stay free of events after 5 years. Some even develop lasting nerve system problems.

In recent years, researchers discovered that nearly all children with LCH have overactive MAPK signaling pathways in their cells. This discovery opened the door to using MAPK inhibitors as a new treatment. Studies have shown that these drugs work well and are safe for children with relapsed or hard-to-treat LCH. Even better, in some kids with single-system bone disease, the disease did not come back after stopping the drug-suggesting it might even cure certain cases.

Luvometinib (also called FCN-159), a new MAPK inhibitor developed by Fosun Pharma in Shanghai, was approved in 2025 for treating adult LCH. A Phase II clinical study showed very encouraging results: 82.8% of patients saw their disease improve or disappear, and 74.4% stayed free of progression after 12 months. The drug was well tolerated, with side effects that were mild and manageable-no serious problems forced anyone to stop treatment.Compared to traditional chemotherapy, luvometinib has fewer and milder side effects, does not weaken the immune system, and lets children continue normal daily life and school. It is a simple oral pill taken once a day, so there's no need for intravenous lines or hospital stays, making treatment much easier and improving quality of life for both the child and the family.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

62

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • China
      • Hefei, China
        • Recruiting
        • Anhui Provincial Children's Hospital
        • Contact:
    • Anhui
      • Hefei, Anhui, China
        • Recruiting
        • The Second Affiliated Hospital of Anhui Medical University
        • Contact:
    • Guangxi
      • Nanning, Guangxi, China
        • Recruiting
        • The Second Affiliated Hospital of Guangxi Medical University
        • Contact:
          • Hong Ying Wei
          • Phone Number: 86+15977767103
          • Email: whylhr@qq.com
    • Guizhou
      • Guiyang, Guizhou, China
        • Recruiting
        • Affiliated Hospital of Guizhou Medical University
        • Contact:
      • Zunyi, Guizhou, China
        • Recruiting
        • Zunyi Medical University Affiliated Hospital, Guizhou Provincial Children's Hospital
        • Contact:
    • Jiangxi
      • Nanchang, Jiangxi, China
        • Recruiting
        • Jiangxi Provincial Children's Hospital
        • Contact:
    • Shaanxi
      • Xi'an, Shaanxi, China
        • Recruiting
        • Xi'an Children's Hospital
        • Contact:
      • Xi'an, Shaanxi, China
        • Recruiting
        • Xi'an Northwest Women's and Children's Hospital
        • Contact:
    • Sichuan
      • Chengdu, Sichuan, China
        • Recruiting
        • West China Second Hospital, Sichuan University
        • Contact:
    • Xinjiang
      • Ürümqi, Xinjiang, China
        • Recruiting
        • The First Affiliated Hospital of Xinjiang Medical University
        • Contact:
    • Yunan
      • Kunming, Yunan, China
        • Recruiting
        • Kunming Children's Hospital
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Children aged 0-18 years, both sexes.
  2. Pathologically confirmed diagnosis of LCH (CD1a+ and/or CD207+), with no prior treatment specific to LCH.
  3. Patients assessed as having single-system multifocal bone involvement, single-site bone involvement at central nervous system risk sites (central nervous system risk sites include craniofacial region [excluding parietal, occipital, and frontal bones], orbital, ear, and oral regions), or single-site vertebral bone involvement with intraspinal space-occupying lesion compressing the spinal cord.
  4. Signed informed consent, willing to receive treatment according to this protocol and undergo follow-up.

Exclusion Criteria:

  1. Patients with other underlying diseases, such as primary immunodeficiency, heart failure, renal insufficiency, hepatitis virus infection, HIV infection, post-organ transplantation, etc.
  2. Secondary malignancy.
  3. QTcF > 0.47 seconds on electrocardiogram prior to enrollment.
  4. Ophthalmologic screening prior to enrollment reveals retinal vein occlusion, retinal pigment epithelial detachment, or other ocular diseases.
  5. Patients with LCH carrying category 3 MEK mutations, specifically the following mutation sites: L98_I103del, L98_K104del, P105_A106del, P105_I107delinsL, L101_I103delinsF, E102_I103delinsF, E102_I103del, E102_I103delinsV, E102_I103delinsVN, E102_K104delinsQ, I103_A106del.
  6. Refusal to sign the informed consent form.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Luvometinib
Drug: Luvometinib
5 mg/m² orally once daily for 1 year

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Event-free survival rate
Time Frame: 2-year
2-year

Secondary Outcome Measures

Outcome Measure
Time Frame
Objective response rate
Time Frame: 1 month and 3 month
1 month and 3 month
Overall survival rate
Time Frame: 2-year
2-year
Safety of luvometinib
Time Frame: 1-year
1-year
Quality of life assessment in children
Time Frame: Before therapy，1month，3months，6months， 12months
Before therapy，1month，3months，6months， 12months
Survey on satisfaction with drug treatment in children
Time Frame: Before therapy，1month，3months，6months， 12months
Before therapy，1month，3months，6months， 12months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

West China Second University Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

June 19, 2026

Primary Completion (Estimated)

December 31, 2030

Study Completion (Estimated)

December 31, 2030

Study Registration Dates

First Submitted

January 18, 2026

First Submitted That Met QC Criteria

January 18, 2026

First Posted (Actual)

January 27, 2026

Study Record Updates

Last Update Posted (Actual)

January 27, 2026

Last Update Submitted That Met QC Criteria

January 18, 2026

Last Verified

January 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • WCSH-NCP-SS-LCH-2026

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

IPD might be shared in the future upon approval by the ethics committee and under a data use agreement.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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