Luvometinib for children with single-system multifocal or special-site bone LCH

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

This project tests whether the drug luvometinib can help children with Langerhans cell histiocytosis that affects certain bones or has multiple bone lesions.

Quick facts

• Phase: Not applicable
• Study type: Interventional
• Enrollment: 62 (estimated)
• Ages: 0 Years to 18 Years
• Sex: All
• Sponsor: West China Second University Hospital (Academic / other)
• Drugs / interventions: Luvometinib, chemotherapy, prednisone
• Locations: 11 sites (Hefei, Anhui and 10 other locations)
• Trial ID: NCT07371182 on ClinicalTrials.gov

What this trial studies

This interventional protocol enrolls children aged 0–18 with pathologically confirmed LCH limited to single-system multifocal bone disease or single-site bone lesions at central nervous system risk locations who have not received LCH-specific treatment. Enrolled patients will receive luvometinib and be followed for clinical response, recurrence rates, and safety outcomes including neurologic and ophthalmologic monitoring. The trial targets lesions in craniofacial, orbital, temporal, ear, oral regions, and vertebral lesions with intraspinal compression. Sites for enrollment are multiple tertiary hospitals in China and participants will undergo regular imaging and clinical assessments during and after therapy.

Who should consider this trial

Good fit: Children aged 0–18 with pathologically confirmed LCH who have single-system multifocal bone involvement or single-site bone disease at CNS-risk locations and have not received prior LCH-specific therapy are the intended candidates.

Not a fit: Children with multisystem LCH, prior LCH-specific treatment, significant organ dysfunction or excluded comorbidities (for example primary immunodeficiency, active hepatitis/HIV, secondary malignancy) are unlikely to be eligible or to benefit from this protocol.

Why it matters

Potential benefit: If successful, luvometinib could lower relapse risk and reduce long-term neurologic complications for children with these bone-involved forms of LCH.

How similar studies have performed: Other therapies that target the MAPK pathway, including BRAF and MEK inhibitors, have shown benefit in LCH, so this trial builds on that therapeutic approach.

Eligibility criteria

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.

Where this trial is running

Hefei, Anhui and 10 other locations

• The Second Affiliated Hospital of Anhui Medical University — Hefei, Anhui, China (Recruiting)
• The Second Affiliated Hospital of Guangxi Medical University — Nanning, Guangxi, China (Recruiting)
• Affiliated Hospital of Guizhou Medical University — Guiyang, Guizhou, China (Recruiting)
• Zunyi Medical University Affiliated Hospital, Guizhou Provincial Children's Hospital — Zunyi, Guizhou, China (Recruiting)
• Jiangxi Provincial Children's Hospital — Nanchang, Jiangxi, China (Recruiting)
• Xi'an Children's Hospital — Xi'an, Shaanxi, China (Recruiting)
• Xi'an Northwest Women's and Children's Hospital — Xi'an, Shaanxi, China (Recruiting)
• West China Second Hospital, Sichuan University — Chengdu, Sichuan, China (Recruiting)
• The First Affiliated Hospital of Xinjiang Medical University — Ürümqi, Xinjiang, China (Recruiting)
• Kunming Children's Hospital — Kunming, Yunan, China (Recruiting)
• Anhui Provincial Children's Hospital — Hefei, China (Recruiting)

Study contacts

• Study coordinator: Xue Tang

Conditions Langerhans Cell Histiocytosis