ClinicalTrials.gov record NCT07371182

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

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

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
AgeUnder 1 month old to 18 years old
SexAll
LocationChina
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This open-label study gives daily oral luvometinib to children (0–18 years) with single-system LCH that affects special skull/face/ear/orbit sites or involves multiple bone lesions. Treatment is 5 mg/m² taken once daily for one year, and participants will be followed for outcomes including event-free survival and other measures.

What it is studying. The study tests luvometinib given as a daily oral pill for one year in children with single-system special-site or multifocal bone LCH. It measures 2-year event-free survival as the primary outcome and also looks at early objective response, overall survival, safety during one year of treatment, and quality of life and treatment satisfaction over 12 months.

What the researchers hope to learn. To determine 2-year event-free survival after luvometinib treatment; to measure objective response at 1 and 3 months; to record 2-year overall survival; to assess safety over one year; and to evaluate quality of life and treatment satisfaction during 12 months.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Luvometinib

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • Children aged 0-18 years, both sexes.
  • Pathologically confirmed diagnosis of LCH (CD1a+ and/or CD207+), with no prior treatment specific to LCH.
  • 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.
  • Signed informed consent, willing to receive treatment according to this protocol and undergo follow-up.

Exclusion criteria

  • Patients with other underlying diseases, such as primary immunodeficiency, heart failure, renal insufficiency, hepatitis virus infection, HIV infection, post-organ transplantation, etc.
  • Secondary malignancy.
  • QTcF > 0.47 seconds on electrocardiogram prior to enrollment.
  • Ophthalmologic screening prior to enrollment reveals retinal vein occlusion, retinal pigment epithelial detachment, or other ocular diseases.
  • 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.
  • Refusal to sign the informed consent form.

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants take oral luvometinib at 5 mg/m² once daily for one year. Before enrollment they will have pathology confirmation and screening tests including an ECG and an eye exam (specific ECG or eye findings can exclude participation). During treatment they attend clinic visits for safety monitoring and assessments of disease response and complete quality-of-life and treatment-satisfaction questionnaires before treatment and at about 1, 3, 6 and 12 months. Event-free and overall survival are followed for up to about 2 years.

How the study is structured. An open-label, single-group interventional drug study: all participants receive luvometinib (5 mg/m² orally once daily) for one year. There is no placebo or comparison arm. Participants have regular assessments for response, safety monitoring, and quality-of-life questionnaires, with outcomes tracked up to two years.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Langerhans cell histiocytosis

Clinical trials20 trials listed for Langerhans cell histiocytosisEvery trial the registry lists for Langerhans cell histiocytosis, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Langerhans cell histiocytosis researchLatest monthly update: September 2026. Includes: Adult routine care. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Jun 19, 2026 (estimated)
Primary completion
Dec 31, 2030 (estimated)
Study completion
Dec 31, 2030 (estimated)
Target enrollment
62 participants (estimated)
Last updated on registry
Jan 27, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
West China Second University Hospital
Principal investigator
Ju Gao, Professor of Pediatrics
West China Second University Hospital

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (11)

Terms used on this page · explanations by MedicalUpdates.ai

prednisone
Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
relapsed
A condition that improved with treatment and has since returned or worsened.
Phase II
A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.
prior treatment
Treatment a person has already had for the condition. A 'line of therapy' is one course of treatment; studies often require, or limit, the number of prior lines.
informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
follow-up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
QTcF
A recording of the heart's electrical activity. QTc is one measurement from it; some drugs are avoided if it is prolonged.
enrollment
The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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