ClinicalTrials.gov record NCT07728201

Response-adapted Luvometinib With or Without Cytarabine in Langerhans Cell Histiocytosis

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

Luvometinib Monotherapy or Combined With Cytarabine for Langerhans Cell Histiocytosis: A Response-adapted, Interventional, Prospective Study

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.
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.
PhasePhase 2A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.
Age10 years old and older
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 Phase 2 interventional study evaluates a response-adapted treatment plan for people aged 10 years and older with Langerhans cell histiocytosis who need systemic therapy. All participants receive six 35-day cycles of oral luvometinib for induction; a PET/CT at the end of induction determines the next steps. People with a complete metabolic response continue luvometinib maintenance alone, while those without a complete metabolic response who are suitable receive luvometinib plus cytarabine followed by luvometinib maintenance. The primary measure is objective response rate after six cycles by blinded independent central review.

What it is studying. Tests a response-adapted treatment approach using daily oral luvometinib for induction, adding subcutaneous cytarabine for participants who do not achieve a complete metabolic response on PET/CT, and then continuing luvometinib maintenance; the main outcome is metabolic response on PET after six cycles.

What the researchers hope to learn. Scientific goals are to measure objective metabolic response rate after six cycles of luvometinib induction (primary endpoint) and to collect safety data. Secondary goals include estimating progression-free survival at 24 months, overall survival at 12 and 24 months, later objective response rates and disease control at cycles 12/18/24, time to response, and clinical benefit measures.

What this study is about

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

This single-center, prospective, interventional phase 2 study evaluates a response-adapted treatment strategy for patients aged 10 years and older with histologically confirmed Langerhans cell histiocytosis requiring systemic therapy. All participants receive six 35-day cycles of luvometinib induction. Post-induction treatment follows the cycle 6 PET/CT response: participants with complete metabolic response continue luvometinib maintenance without cytarabine, whereas participants without complete metabolic response who are judged suitable to continue protocol treatment receive luvometinib plus cytarabine followed by luvometinib maintenance; participants with progression or otherwise unsuitable to continue protocol treatment may receive other standard therapy or discontinue study treatment per protocol. The primary endpoint is objective response rate after six cycles by blinded independent central review.

Interventions being studied

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

Drug
  • Luvometinib
  • Luvometinib, Cytarabine.

Eligibility

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

Inclusion criteria

  • Histologically confirmed Langerhans cell histiocytosis (LCH).
  • Age 10 years or older.
  • Systemic treatment indication and at least one PET response criteria-evaluable lesion.
  • Expected survival of at least 12 weeks, as judged by the investigator, and able to undergo protocol-specified treatment, assessments, and follow-up.
  • ECOG performance status 0-2 or Lansky score >=60.
  • Adequate organ function as defined in the protocol.
  • Written informed consent from adult participants or legal guardians, with participant assent when applicable.

Exclusion criteria

  • Hypersensitivity to luvometinib or any excipient.
  • Concurrent other malignant tumor.
  • Pregnancy or breastfeeding.
  • Failure to meet protocol contraception requirements.
  • Active bacterial, fungal, or viral infection.
  • Significant retinal disease or glaucoma.
  • NYHA class >=3 heart failure or LVEF <50%.
  • Psychiatric disease or other condition preventing protocol compliance.
  • Investigator judgment that participation is unsuitable.

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 once daily for an initial induction period of six 35‑day cycles, then have a PET/CT scan after cycle 6 to assess response. If the scan shows a complete metabolic response, participants continue luvometinib maintenance for 18 additional 35‑day cycles; if not and they are judged suitable, participants receive luvometinib plus subcutaneous cytarabine (100 mg/m² on days 1–5 of each 35‑day cycle) for 12 cycles, followed by luvometinib maintenance for 6 cycles. Study staff will record side effects and serious adverse events during treatment and for a period afterward; participants who progress or are otherwise unsuitable may stop study treatment and receive other standard therapy. Follow-up for safety and outcomes continues unless the participant withdraws consent.

How the study is structured. Interventional, non-randomized, single-masked Phase 2 study. All participants receive six 35‑day cycles of oral luvometinib (induction). After the cycle 6 PET/CT assessment, participants with complete metabolic response follow the CMR path and continue luvometinib maintenance for 18 additional 35‑day cycles (unless progression, unacceptable toxicity, death, or withdrawal occurs). Participants without complete metabolic response who are judged suitable follow the non-CMR path: luvometinib plus subcutaneous cytarabine (100 mg/m² on days 1–5 of each 35‑day cycle) for 12 cycles, then luvometinib maintenance for six additional 35‑day cycles. Safety and responses are followed over time, with the primary response assessed by a blinded independent central review.

Questions you could ask

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

  • The registry says recruitment has not started. When is it expected to open, and can I register interest now?
  • 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 30, 2026 (estimated)
Primary completion
Jul 31, 2028 (estimated)
Study completion
Jun 30, 2030 (estimated)
Target enrollment
30 participants (estimated)
Last updated on registry
Jul 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
Shanghai Ninth People's Hospital Affiliated to Shanghai Jiao Tong University
Principal investigator
Qi Zhu, Principal Investigator
Shanghai Ninth People's Hospital Affiliated to Shanghai Jiao Tong University
Collaborators
  • Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.

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 (1)

Terms used on this page · explanations by MedicalUpdates.ai

histologically confirmed
Removal of a small piece of tissue so it can be examined under a microscope. 'Biopsy-proven' or 'histologically confirmed' means the diagnosis was confirmed by examining tissue under a microscope.
PET/CT
A scan in which a small amount of a radioactive tracer is injected so that activity in tissues can be seen. Often combined with a CT or MRI scan.
standard therapy
Treatment generally considered appropriate for a condition in a particular clinical setting. It can vary by patient, place and over time. In many studies the new treatment is added to, or compared with, standard of care.
primary endpoint
The main result the study is designed to measure and report on. Other results, called secondary outcomes, are also collected.
blinded
Who does not know which intervention a participant is receiving. It can apply to participants, the people giving the treatment, those assessing results, or those analysing the data.
follow-up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
ECOG performance status
A scale from 0 to 5 that describes how a person's condition affects daily activity. 0 means fully active; 1 means restricted in strenuous activity; 2 means able to self-care but unable to work; higher numbers mean more limitation.
Written 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.
assent
A child's or dependent adult's agreement to take part, given alongside the formal consent of a parent or legal representative.
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 website; 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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