Treatment of Resistant Langerhans Cell Histiocytosis With ENBREL
Information source: Baylor College of Medicine
ClinicalTrials.gov processed this data on August 20, 2015
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Histiocytosis, Langerhans-Cell
Intervention: Etanercept (Drug)
Phase: Phase 2
Sponsored by: Baylor College of Medicine
Official(s) and/or principal investigator(s):
Kenneth McClain, MD, Principal Investigator, Affiliation: Baylor College of Medicine
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
Official title: Treatment of Resistant Langerhans Cell Histiocytosis With Etanercept (ENBREL, IMMUNEX, SEATTLE)
Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: Response of Langerhans cell histiocytosis
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.
Minimum age: N/A.
Maximum age: 65 Years.
- 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
Locations and Contacts
Texas Children's Hospital, Houston, Texas 77030, United States
Starting date: October 2001
Last updated: August 22, 2006