ClinicalTrials.gov record NCT02670707

Vinblastine/Prednisone Versus Single Therapy With Cytarabine for Langerhans Cell Histiocytosis (LCH)

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

Randomization of Cytarabine Monotherapy Versus Standard-of-Care Vinblastine/Prednisone for Frontline Treatment of Langerhans Cell Histiocytosis (TXCH LCH0115)

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.
PhasePhase 3A later-stage study, usually with more participants, designed to give stronger evidence about how well a treatment works and how safe it is. It often compares the treatment with standard care or a placebo, and results may support an application for approval.
AgeUp to 21 years old
SexAll
LocationUnited States
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 randomized Phase 3 trial compares single-agent cytarabine with standard vinblastine plus prednisone as first-line chemotherapy for Langerhans cell histiocytosis (LCH). The study uses imaging (including PET/CT) and collects tumor and blood samples to study genetic and other biomarkers and to assess response. Participants receive assigned chemotherapy in repeated cycles and are followed for up to 5 years after therapy.

What it is studying. The study tests cytarabine monotherapy compared with standard vinblastine/prednisone when used as frontline treatment for LCH. It also evaluates PET/CT imaging to detect disease and follow response, and looks for links between genetic or blood biomarkers (including BRAF-V600E) and treatment response.

What the researchers hope to learn. Compare 1-year event-free survival between cytarabine monotherapy and vinblastine/prednisone; describe durable responses at 2 and 5 years and overall survival; compare numbers and types of treatment toxicities; measure rate of achieving non-active disease; determine time to eradication of BRAF-V600E or other LCH-defining mutations; assess PET/CT for identifying high-risk organ involvement and correlate imaging with disease activity and circulating mutation levels; analyze response using RECIST and metabolic imaging; study risk factors and timing for diabetes insipidus and neurodegenerative disease; and collect and store serial biological samples for future research.

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 a type of cancer that can damage tissue or cause lesions to form in one or more places in the body. Langerhans cell histiocytosis (LCH) is a cancer that begins in LCH cells (a type of dendritic cell which fights infection). Sometimes there are mutations (changes) in LCH cells as they form. These include mutations of the BRAF gene. These changes may make the LCH cells grow and multiply quickly. This causes LCH cells to build up in certain parts of the body, where they can damage tissue or form lesions. For most patients with LCH, standard-of-care vinblastine/prednisone are used as front-line therapy while cytarabine therapy has been used as therapy for patients who develop recurrence. No alternate treatment strategy has been developed for frontline therapy in LCH. The purpose of this research study is to compare previously used vinblastine/prednisone to single therapy with cytarabine for LCH. We will evaluate the utility of an imaging study called a positron emission tomography (PET) scan to more accurately assess areas of LCH involvement not otherwise seen in other imaging studies as well as response to therapy. We also want to identify if genetic and other biomarkers (special proteins in patient's blood and in patient's cancer) relate to the response of patients LCH to study treatment.

Interventions being studied

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

Drug
  • Cytarabine
  • Vinblastine/prednisone

Eligibility

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

Inclusion criteria

  • Patient must have biopsy-confirmed diagnosis of Langerhans cell histiocytosis.
  • Patient must be between 0-21 years of age.
  • Patient must have a Karnofsky performance score ≥ 50% or Lansky performance score ≥ 50%.

Exclusion criteria

  • Patient may not have received any prior systemic cytotoxic or other chemotherapies for LCH or any other malignant disorder prior to the initiation of protocol therapy on TXCH LCH0115 with the exception of:
  • Steroid pretreatment: Systemic glucocorticosteroids (prednisone, methylprednisone, dexamethasone, etc.) for less than or equal to 120 hours (5 days) in the 7 days prior to initiating protocol therapy or for less than or equal to 336 hours (14 days) in the 28 days before the initiation of protocol therapy does not affect eligibility. The dose of steroid previously given does not affect eligibility. Patients who have only received surgical or radiation therapy, intralesional injection of steroids, inhalational steroids, systemic mineralocorticoids (hydrocortisone), or topical steroids may also be enrolled.
  • Patient may not have disease limited to a single skin or bone site, with the following exceptions:
  • Central Nervous System (CNS) risk lesions/special site disease: patients with single bone sites that are CNS-risk (sphenoid, mastoid, orbital, zygomatic, ethmoid, maxillary, or temporal bones, the cranial fossa, pituitary gland or neurodegenerative disease) or are "special sites" (odontoid peg, vertebral lesion with intraspinal soft tissue extension) require systemic therapy as standard of care and thus are eligible for the study.
  • Functionally critical lesions: A single lesion not described above which may cause "functionally critical anatomic abnormality" wherein attempts at local therapy (such as surgical curettage or radiation) would cause unacceptable morbidity. These patients may be enrolled with written approval of the Coordinating Center PI or Vice-Chair and documentation of the rationale justifying systemic therapy.
  • Asynchronous multisite LCH presentation: A patient may also have any single site of disease involvement at the time of enrollment if they previously had at least one other site of LCH disease in the past (which may have been treated with local therapy/surgery as described), as long as no systemic therapy was previously given per protocol guidelines.
  • Patient may not have severe renal disease (creatinine greater than 3 times normal for age OR creatinine clearance < 50 ml/m2/1.73m\^2).
  • Patient may not have severe hepatic disease (direct bilirubin greater than 3 mg/dl OR aspartate aminotransferase (AST) greater than 500 IU/L), unless hepatic injury is due to LCH.
  • Female patients may not be pregnant or breastfeeding.
  • Patients of reproductive potential not willing to use an adequate method of birth control for the duration of the study.
  • Patients who are HIV positive may not be enrolled.
  • NOTE: Patients excluded for laboratory abnormalities or performance score only may be enrolled on the study with written approval from the Coordinating Center PI or Vice-Chair.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Show the detailed description from the registry

To be eligible to participate in this study, patients physician must have determined that they have LCH which is not treatable by surgical intervention or observation alone. If patient chooses to participate in this study, they will be assigned randomly (like flipping a coin) to one of two LCH chemotherapy treatment groups. A computer will randomly determine if they will begin to receive vinblastine/prednisone treatment or cytarabine treatment. Patient will have an equal chance (50%) to receive vinblastine/prednisone or to receive cytarabine. Neither the patient nor their doctor will be able to choose the group assignment, but the patient and their study doctor will know which treatment they are receiving. The patient will need to have the following tests, exams, or procedures. Most of these are part of regular cancer care and may be done even if the patient does not want to join this study. Some of them may not need to be repeated if they have had them done recently. The patient's doctor will tell them which ones they need to repeat.

  • History and Physical
  • Blood tests
  • Urine tests
  • Biopsy to confirm diagnosis
  • Bone Marrow biopsy/aspirate (if patient is less than 2 years old or for any patient clinical concern for bone marrow disease)*
  • Lumbar puncture for spinal fluid tests (if patient has disease in pituitary gland or brain)
  • The bone marrow sample procedure is as follows: The skin above the hipbone will be made numb. This area will then be sterilized and a small incision will be made. A bone marrow needle will be inserted into the hipbone and bone marrow will be withdrawn. Patient will sign a separate consent that explains this procedure in more detail.

We will also do x-rays and scans of the inside of the body. These scans may include chest and bone x-rays, ultrasound, computerized tomography (CT), magnetic resonance imaging (MRI) and/or positron emission tomography (PET). The specific tests depend on age, sites of LCH and other clinical factors. An ultrasound is a sound wave machine that uses a computer to make pictures of the tissues of the body. A CT is an x-ray machine that uses a computer to make pictures of the organs of the body. An MRI is a scan which uses a magnetic machine to make pictures of the inside of the body. A PET scan is an x-ray technique that uses a sugar solution that you drink to see activity inside the body. The PET and CT scans will look at where the LCH is in the body. If tests show that the patient can participate in this study and they choose to participate, treatment will begin based on randomization to either the cytarabine ("experimental") arm or the vinblastine/prednisone ("standard") arm as described below. Patient will have a history and physical and blood tests before each cycle. The cancer drugs will be given to the patient intravenously (IV). An IV is a tube placed inside a vein to give medicine to the patient. If at anytime during the study the cancer gets worse, the patient will be taken off the study treatment.

  • Cytarabine ("experimental") arm

Initial therapy I (Weeks 1-6)

  • Cytarabine for five consecutive days. This five-day cycle will be repeated every 21 days for a total of two cycles.

Patient will have scans to see how the LCH responded to the treatment during week 6. Patients who have Complete Response (CR), Partial Response (PR), or Stable Disease (SD) at the end of Initial Therapy I, will proceed to Initial Therapy II (Weeks 7-12). Patients who have PR or SD at the end of Initial Therapy I will require reassessment again at the end of Initial Therapy II. Patients who have CR at the end of Initial Therapy I do not require disease reassessment again until Continuation Week 24. Initial therapy II (Weeks 7-12)

  • Cytarabine for five consecutive days. This five-day cycle will be repeated every 21 days for a total of two cycles.

Patient will have scans to see how the LCH responded to the treatment during week 12 (unless they had no active disease at week 6). If the patient had LCH in their bone marrow (BM) at the beginning of the study, they will have another BM biopsy. If the cancer is stable and does not respond to therapy by Week 12, the patient will be taken off the study treatment. If the patient has no evidence of active disease at this time they will proceed to Continuation therapy. Continuation therapy (Weeks 13-52)

  • Cytarabine for five consecutive days. This five-day cycle will be repeated every 28 days for 10 additional cycles to complete one year of therapy

Patient will have scans to see how the LCH responded to the treatment at Week 24 and at the end of therapy. If the patient had LCH in their bone marrow (BM) at the beginning of the study, they will have another BM biopsy.

  • Vinblastine/prednisone ("standard") arm

Initial therapy I (Weeks 1-6)

  • Prednisone given by mouth two times a day daily on days 1-28 AND
  • Vinblastine will be given IV (into a vein) one day a week for 6 weeks

Patient will have scans to see how the LCH responded to the treatment after week 6. If the patient had LCH in their bone marrow (BM) at the beginning of the study, they will have another BM biopsy. If the disease is completely gone by Week 6, patient will proceed to Continuation therapy. If the disease is only partly gone or unchanged patient will receive additional therapy with Initial therapy II. Patient will have scans to see how the LCH responded to the treatment during Week 12 and if they had LCH in their bone marrow (BM) at the beginning of the study, they will have another BM biopsy. Initial therapy II (Weeks 7-12, given only if the patient does not have evidence of active disease by Week 6)

  • Prednisone by mouth on the first three days of each week during weeks 7-12 AND
  • Vinblastine IV weekly during weeks 7-12

If the disease is gone or better after this additional therapy Continuation therapy will begin. Continuation therapy

  • Prednisone by mouth twice daily day on days 1-5 every 3 weeks AND
  • Vinblastine IV once every 3 weeks
  • 6-Mercaptopurine by mouth daily NOTE: Only high risk patients will receive 6-Mercaptopurine

Patient will have scans to see how the LCH responded to the treatment at Week 24 and at the end of therapy. If the patient had LCH in their bone marrow (BM) at the beginning of the study, they will have another BM biopsy. STUDY FOLLOW-UP: Patient will be in this study for 5 years after completion of their therapy. The doctors will exam the patient at 1 month, 3 months, 6 months, 9 months, 12 months, then at 18 and 24 months, then yearly until 5 years after the patient is taken off treatment. At these visits the patient may have routine blood tests, scans, and the biology tests.

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.

If they join, participants are randomly assigned to receive either single‑agent cytarabine or vinblastine with prednisone; drugs are given intravenously (and prednisone by mouth in the vinblastine arm) on scheduled cycles. Cytarabine is given as five consecutive daily IV doses per cycle, with initial cycles repeated every 21 days and later continuation cycles every 28 days to complete about one year of therapy; vinblastine/prednisone is given on its scheduled weekly and then every‑3‑week cycles as described in the protocol. In the vinblastine/prednisone arm, patients with high‑risk organ involvement (liver, spleen, or hematopoietic system) may also receive 6‑mercaptopurine during continuation therapy as part of standard care. Before and during treatment participants will have physical exams, blood and urine tests, imaging (which may include PET/CT), and confirmation biopsy; some patients will need bone‑marrow biopsy (required if under 2 years old or if marrow disease is suspected) and lumbar puncture if pituitary or brain disease is present. Serial samples (viable white blood cells, plasma, tumor tissue, bone marrow and cerebrospinal fluid when collected) may be stored for future research. After finishing therapy participants are followed with clinic visits, tests and scans for up to 5 years (visits at 1, 3, 6, 9, 12, 18, 24 months and then yearly).

How the study is structured. A randomized, open-label Phase 3 trial assigning participants 1:1 to two treatment arms (cytarabine alone or vinblastine plus prednisone). Treatments are given in repeated cycles (IV drugs and oral steroids where specified). Scans, blood tests, biopsies, and possible spinal fluid testing are used to assess disease and response. Participants are followed with visits and tests during treatment and for up to five years after finishing therapy.

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 United States. 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
Mar 7, 2016
Primary completion
January 2027 (estimated)
Study completion
January 2029 (estimated)
Target enrollment
124 participants (estimated)
Last updated on registry
Sep 11, 2025
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
Baylor College of Medicine
Principal investigator
Olive Eckstein, Assistant Professor
Baylor College of Medicine

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.
positron emission tomography
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.
biomarkers
Something measurable in the body, such as a blood test result or an imaging finding, that indicates how a condition or treatment is behaving.
biopsy
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.
Karnofsky
A scale from 100 (no complaints) down to 0 that describes how well a person can carry out daily activities. Studies often require a minimum score.
standard of care
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.
enrollment
The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
creatinine clearance
A measure of kidney function, calculated from a blood creatinine level together with age, weight and sex.
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.
Lumbar puncture
A procedure in which a needle is placed between the bones of the lower back to collect spinal fluid or to give a treatment.
CT
An X-ray scan that builds detailed cross-section images of the body. 'High-resolution' CT (HRCT) is used to look closely at the lungs.
magnetic resonance imaging
A scan that uses magnets and radio waves, not X-rays, to make detailed pictures of the inside of the body. Some implants and metal objects mean a person cannot have one.
randomization
Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
Complete Response
No sign of the condition can be found with the tests used in the study. It does not always mean the condition is gone permanently, and the definition depends on the condition and the study.
FOLLOW-UP
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.

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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