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Treatment of Resistant Langerhans Cell Histiocytosis With ENBREL

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00048373
Recruitment Status : Completed
First Posted : November 1, 2002
Last Update Posted : January 9, 2020
The Methodist Hospital Research Institute
Information provided by (Responsible Party):
Kenneth McClain, Baylor College of Medicine

Brief Summary:
The purpose of this study is to learn about the response of Langerhans cell histiocytosis (LCH) to Enbrel in patients who have failed to respond to standard therapies. We are also looking specifically at what side effects Enbrel has on patients. We expect to enroll 20 patients on this study and anticipate the subjects active participation to last up to one year.

Condition or disease Intervention/treatment Phase
Histiocytosis, Langerhans-Cell Drug: Etanercept Phase 2

Detailed Description:

After subjects have completed the pretreatment evaluations, they will receive an injection of Enbrel - the study drug -(given under the skin) twice weekly. If the subjects disease stabilizes or regresses, they may continue to receive treatment for up to 1 year.

Subjects with disease involvement of risk organs (this means patients have disease in their liver, lung, spleen, or bone marrow): will be admitted to the hospital for observation during the first week of administration of Enbrel. If after the first two doses there appears to be no problems, subjects may be followed as an outpatient with twice weekly evaluation visits (similar to those performed before treatment began) until abnormal blood tests have become normal. Then the frequency of clinic visits will decrease and be similar to those described below for subjects without disease involvement of risk organs.

Subjects without disease involvement of risk organs will be treated as an outpatient. While receiving the treatment, subjects may not receive any other chemotherapy agents. Doctors will be monitoring subjects closely for side effects. Most side effects usually disappear after the treatment is stopped. In the meantime, however, the doctor may prescribe medication to keep these side effects under control.

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Study Type : Interventional  (Clinical Trial)
Enrollment : 20 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Treatment of Resistant Langerhans Cell Histiocytosis With Etanercept (ENBREL, IMMUNEX, SEATTLE)
Study Start Date : October 2001
Study Completion Date : April 2004

Primary Outcome Measures :
  1. Response of Langerhans cell histiocytosis

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Age: Patients of any age greater than 1 year and up to 65 years of age. An internal medicine board-certified physician will also evaluate adult patients.
  • Histologic diagnosis: Patients must have a histologically confirmed LCH that is refractory to standard therapy.
  • Recovery from prior therapy: Patients must have recovered from the toxic effects of all prior therapy but may have abnormal hematologic, hepatic, or other lab values secondary to the disease.
  • Life expectancy: Patients must have a life expectancy of at least 8 weeks.
  • Performance status: Patients must have a Lansky performance status greater than 40 or Karnofsky status greater than 40.
  • Informed consent: All patients or their legal guardians (if the patient is < 18 years of age) must sign a document of informed consent indicating their awareness of the investigational nature and the risks of this study. When appropriate the patient will be included in all discussions in order to obtain verbal consent.
  • Hematologic status: Patients of any hematologic status may be enrolled since resistant LCH may require considerable transfusion support.
  • Durable Power of Attorney (DPA): A DPA must be offered to all patients 18 years of age or older.


  • Women of childbearing potential who are pregnant or lactating are excluded.
  • Patients with active infections must be treated prior to entry.
  • Significant other diseases that the investigator feels will complicate review/evaluation of the study data (example: uncontrolled diabetes, multiple sclerosis).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00048373

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United States, Texas
Texas Children's Hospital
Houston, Texas, United States, 77030
Sponsors and Collaborators
Baylor College of Medicine
The Methodist Hospital Research Institute
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Principal Investigator: Kenneth McClain, MD Baylor College of Medicine
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Responsible Party: Kenneth McClain, Professor, Pediatrics-Oncology, Baylor College of Medicine Identifier: NCT00048373    
Other Study ID Numbers: H10339
First Posted: November 1, 2002    Key Record Dates
Last Update Posted: January 9, 2020
Last Verified: January 2020
Keywords provided by Kenneth McClain, Baylor College of Medicine:
Additional relevant MeSH terms:
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Histiocytosis, Langerhans-Cell
Lymphatic Diseases
Lung Diseases, Interstitial
Lung Diseases
Respiratory Tract Diseases
Anti-Inflammatory Agents, Non-Steroidal
Analgesics, Non-Narcotic
Sensory System Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anti-Inflammatory Agents
Antirheumatic Agents
Gastrointestinal Agents
Immunosuppressive Agents
Immunologic Factors