Clinical trial NCT02389400: Methotrexate and Cytosine in Adult Langerhans Cell Histiocytosis
Official title: Efficacy and Tolerance of Combination Chemotherapy With Methotrexate and Cytosine Arabinoside in Newly Diagnosed Adult With Langerhans Cell Histiocytosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Langerhans Cell Histiocytosis
Keywords: adult with Langerhans cell histiocytosis
newly diagnosed
methotrexate
cytosine arabinoside
Lead sponsor: Peking Union Medical College Hospital
Sponsor class: OTHER
First posted: 2015-03-17
Enrollment: 43
Sex: ALL
Minimum age (years): 18
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Study Type: Interventional

Study Design:

Endpoint Classification: Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment purpose: This single arm clinical trial is studying efficacy and tolerance of combination chemotherapy with methotrexate and cytosine arabinoside in newly diagnosed adult with Langerhans cell histiocytosis.

Detailed description: This single arm clinical trial is studying efficacy and tolerance of combination chemotherapy with methotrexate and cytosine arabinoside in newly diagnosed adult with Langerhans cell histiocytosis.

Eligibility criteria: Inclusion Criteria:

1.DISEASE CHARACTERISTICS:

* Histopathologically confirmed diagnosis of Langerhans cell histiocytosis according to the criteria defined by the Histiocyte Society
* Demonstration of CD1a antigenic determinants on the surface of lesional cells (by immunocytology or immunohistology) or Birbeck granules in lesional cells by electron microscopy
* Considered at risk or low risk according to the following criteria:

  * Multi-system at risk disease, defined as involvement of one or more risk organs (i.e., hematopoietic system, liver, spleen, or lungs)

    1.No single-system lung involvement
  * Multi-system low-risk disease

    1.Multiple organs involved but without involvement of risk organs
  * Single-system disease 1.Multifocal bone disease (i.e., lesions in 2 or more different bones) 2.Localized special site involvement, such as CNS-risk lesions with intracranial soft tissue extension or vertebral lesions with intraspinal soft tissue extension 3.Vault lesions are not regarded as CNS-risk lesions 2.PATIENT CHARACTERISTICS:

    1. Not pregnant or nursing
    2. Negative pregnancy test
    3. Fertile patients must use effective contraception 3.PRIOR CONCURRENT THERAPY:

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    1. No prior treatment for Langerhans cell histiocytosis

Exclusion Criteria:

Primary outcomes:
measure: disease free survival
timeFrame: 5 years
Secondary outcomes:
measure: overall free survival
timeFrame: 5 years
Other outcomes:
measure: Response
timeFrame: 2 years
Locations:
city: Beijing
country: China
facility: Peking union medical college hospital
Study officials:
affiliation: Peking Union Medical College Hospital
name: minghui Duan, Dr.
role: PRINCIPAL_INVESTIGATOR
References:
citation: Han X, Ouyang M, Duan M, Zhang W, Zhu T, Li J, Wang S, Zhou D. The combination of methotrexate and cytosine arabinoside in newly diagnosed adult Langerhans cell histiocytosis: a prospective phase II interventional clinical trial. BMC Cancer. 2020 May 18;20(1):433. doi: 10.1186/s12885-020-06872-8.