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Hybrid Immunotherapy for Hemophagocytic LymphoHistiocytosis

Information source: Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Hemophagocytic Lymphohistiocytosis

Intervention: ATG, rabbit (Drug); Etoposide (Drug); Methotrexate (Drug); hydrocortisone (Drug); Dexamethasone (Drug)

Phase: Phase 2

Status: Recruiting

Sponsored by: Children's Hospital Medical Center, Cincinnati

Official(s) and/or principal investigator(s):
Michael Jordan, MD, Principal Investigator, Affiliation: Children's Hospital Medical Center, Cincinnati

Overall contact:
Sharon Mitchell, Phone: 513-636-4174, Email: Sharon.Mitchell@cchmc.org

Summary

Despite good progress during the last decade, hemophagocytic lymphohistiocytosis (HLH) remains difficult to treat. Two different treatment regimens have been used successfully. The first one, a treatment regimen based on two drugs called etoposide and dexamethasone, has been used worldwide. The second regimen, based on two drugs called Anti-thymocyte globulin (ATG) and prednisone, has been used mostly at one hospital in Paris, for over 15 years. With either regimen, about three quarters of treated children survive the most difficult time, the first two months after diagnosis. These two different regimens appear to work somewhat differently, and we suspect that combining them may give better results than either regimen alone. We are conducting this clinical trial to test the combination of ATG, dexamethasone, and etoposide for the treatment of HLH. The purpose of this research study is to find out what effects (good and bad) this drug combination has on you and your HLH.

Clinical Details

Official title: An Open Label Phase II Pilot Study of Hybrid ImmunoTherapy(ATG/Dexamethasone/Etoposide) for Hemophagocytic LymphoHistiocytosis:HIT-HLH

Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome: Complete Response Rate

Secondary outcome:

Time to Response

Overall Survival

Incidence of Infection

Incidence and Time to Relapse

Overall Survival to day +100

Gather Biologic Samples

Detailed description: Hemophagocytic lymphohistiocytosis (HLH) is a rare immunological disorder first recognized almost 70 years ago.(1) Genetic and animal studies have indicated that the familial form of HLH is clearly due to a deficiency of cytotoxic killing. Patients with HLH present with a potentially fatal syndrome of 'hyperimmunity.' These patients have severe inflammation, associated with cytopenias and variably severe bone marrow, liver, or CNS damage. Tissue damage and mortality appear to be due to hypercytokinemia related to persistent immune hyperactivation. An animal model of HLH and correlative human studies all suggest that excessive and abnormal activation of T cells drives the pathophysiology of this disorder, and that suppressing this excessive activation is critical for successful therapy of HLH. It is believed a combination of the two proven induction regimens for hemophagocytic lymphohistiocytosis (HLH) (anti-thymocyte globulin (ATG)- and etoposide-based) will result in response rates and overall survival rates at eight weeks which are comparable or better than the current standard of care (induction therapy per the HLH-94 protocol).

Eligibility

Minimum age: N/A. Maximum age: 18 Years. Gender(s): Both.

Criteria:

Inclusion Criteria:

- diagnosis of hemophagocytic lymphohistiocytosis

- Patients <18 years of age

- The patient must have active disease at the time of enrollment

- Patient's legal guardians must sign an Institutional Review Board approved consent

form indicating their awareness of the investigational nature and the risks of this study.

- Eligible subjects must be enrolled with the protocol coordinating center

Exclusion Criteria:

- Recent treatment, within 3 months, with another therapeutic regimen for HLH

- Known active malignancy

- Known rheumatologic diagnosis which may be the underlying cause of HLH

- Pregnancy (as determined by serum or urine test) or active breast feeding

- Failure to provide signed informed consent

Locations and Contacts

Sharon Mitchell, Phone: 513-636-4174, Email: Sharon.Mitchell@cchmc.org

Phoenix Children's Hospital, Phoenix, Arizona 85254, United States; Recruiting

University of California, San Francisco Department of Pediatrics, San Francisco, California 94143, United States; Recruiting

Stanford University, Stanford, California 94305, United States; Recruiting
Michael Jeng

University of Colorado, Aurora, Colorado 80045, United States; Recruiting
Timothy Garrington

Nemours, Wilmington, Delaware 19803, United States; Recruiting
Jonathan Powell

Children's National Medical Center, Washington, District of Columbia 20010, United States; Recruiting
Stephan Ladisch

Nemours, Jacksonville, Florida 32827, United States; Recruiting
Michael Joyce

Florida All Children's Hospital, St. Petersburg, Florida 33701, United States; Recruiting

Tulane University Medical Center, New Orleans, Louisiana 70118, United States; Completed

Children's Hospital Boston, Boston, Massachusetts 02115, United States; Recruiting

Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio 45229, United States; Recruiting

The Hospital for Sick Children, Toronto, Ontario, Canada; Recruiting
Ahmed Naqvi

Oregon Health and Science University, Portland, Oregon 21703, United States; Recruiting
Evan Shereck

Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19145, United States; Recruiting

Texas Children's Cancer Center/Baylor College of Medicine, Houston, Texas 77030, United States; Recruiting

Additional Information

Starting date: April 2010
Last updated: August 3, 2015

Page last updated: August 23, 2015

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