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New hope for rare cancer: drug cocktail targets stubborn LCH

NCT ID NCT07204041

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 2 trial tests a combination of three drugs—selinexor, thalidomide, and dexamethasone—in 40 adults with relapsed or refractory Langerhans cell histiocytosis (LCH), a rare immune cell disorder. The goal is to see if the XTD regimen can shrink tumors and delay disease progression. Participants take the drugs orally in 28-day cycles for up to 12 cycles.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Selinexor, thalidomide, and dexamethasone (XTD regimen)
What this could lead to
If successful, this could offer a new treatment option for adults with hard-to-treat Langerhans cell histiocytosis, potentially controlling the disease and improving survival.
What could go wrong
This is a small, early-phase trial with no comparison group, so results may not apply broadly. The drugs have known side effects like fatigue, blood clots, and nerve damage.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 40 people

The number the study aims to enrol. It can still change while the study runs.

Started

Aug 2025

Expected to finish

Aug 2027

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 80 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Organ pathology confirmed diagnosis of LCH; * Age 18 years or older; * Multi-system involvement, or single system with multiple lesions; * Disease not relieved after receiving at least one systemic treatment, or disease relapsed after improvement; * ECOG performance status score ≤2; * Clinical physician determines suitability for this treatment protocol; * Subjects can understand the study protocol and are willing to participate in this study, providing written informed consent. Exclusion Criteria: * Single system single lesion LCH * Underwent major surgery within 4 weeks prior to the first administration of the study drug; * Underwent radiotherapy within 4 weeks prior to the first administration of the study drug; * History of myocardial infarction within the past year; suffers from New York Heart Association (NYHA) class 3 or 4 congestive heart failure, or has a history of NYHA class 3 or 4 congestive heart failure, unless left ventricular ejection fraction (LVEF) ≥ 50% in the echocardiogram (ECHO) screening performed within 1 month before entering the study; * Pregnant or breastfeeding women (women of childbearing age with positive pregnancy test at baseline or who have not undergone pregnancy testing. Postmenopausal women must have been menopausal for at least 12 months); * Abnormal liver and kidney function: creatinine level ≥176.8μmol/l (2mg/dl), transaminase and bilirubin levels more than 2 times the upper limit of normal (for LCH patients with liver involvement, transaminase levels more than 10 times and bilirubin levels more than 3 times the upper limit of normal); * Severe hematological abnormalities: absolute neutrophil count less than 1 × 10\^9/L, platelet less than 50×10\^9/L; * Presence of uncontrolled infections; * Any other circumstances that the investigator believes to be inappropriate for the patient to participate in this trial;

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Conditions

The condition(s) this trial relates to.

Histiocytosis, Langerhans-Cell Langerhans cell histiocytosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Department of Medical Oncology, National Cancer Center/National Clinical Research Center for Cancer/Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, beijing,

    Beijing, China

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