Hybrid Immunotherapy for Hemophagocytic LymphoHistiocytosis

Recruiting in Palo Alto (17 mi)

+14 other locations

Overseen byMichael Jordan, MD

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Children's Hospital Medical Center, Cincinnati

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?

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.

Eligibility Criteria

Inclusion Criteria

diagnosis of hemophagocytic lymphohistiocytosis

Patients <18 years of age

The patient must have active disease at the time of enrollment

See 2 more

Treatment Details

Interventions

ATG, rabbit (Immunosuppressant)
Dexamethasone (Corticosteroid)
Etoposide (Topoisomerase Inhibitor)
Hydrocortisone (Corticosteroid)
Methotrexate (Antimetabolite)

Participant Groups

1Treatment groups

Experimental Treatment

Group I: Induction TherapyExperimental Treatment5 Interventions

ATG, rabbit: intravenous, 5 mg/kg/dose, 5 consecutive days Dexamethasone: intravenous, 20mg/m2/day x7days, 10mg/m2/day x7days, 5mg/m2/day x14days, 2.5mg/m2/day x14days, 1.25mg/m2/day x14days Etoposide: intravenous, 150 mg/m2 weekly, starting 7 days after first dose of Thymoglobulin Methotrexate and hydrocortisone: intrathecal to patients with central nervous system involvement, age\< 1 yr: 6/8mg (MTX/HC), 1-2 yrs: 8/10mg, 2-3 yrs: 10/12mg, \>3 yrs: 12/15 mg, on day 7, 14, 21 and 42