New hope for rare cancer: drug cocktail targets stubborn LCH
NCT ID NCT07204041
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 Hide 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;
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Langerhans cell histiocytosis (LCH) are added.
Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Could existing cancer drugs tackle rare tumors? new trial aims to find out
- New targeted drug combo aims to curb relapse in kids with rare immune disorder
- Scientists hunt for genetic clues in rare blood disorders
- New pill could spare kids with rare bone disease from chemo
- Old drug, new hope: lenalidomide tackles rare histiocyte cancers
- New pill shows promise for rare childhood diseases