Clinical Trial: Treatment of Familiar Lymphohistiocytosis

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: First Line Treatment of Familiar Lymphohistiocytosis by Alemtuzumab (CAMPATH®)

Brief Summary: The purpose of this project is to study the number of surviving patients until hematopoietic stem cell transplantation (HSCT) after first line treatment of hemophagocytic lymphohistiocytosis (HLH) by Alemtuzumab

Detailed Summary:

The hemophagocytic lymphohistiocytosis (HLH) or lymphohistiocytic activation syndrome is an inflammatory condition caused by a uncontrolled proliferation of activated lymphocytes and macrophages secreting an excess of inflammatory cytokines. Familial hemophagocytic lymphohistiocytosis (FHL) is a rare disorder of the immune system, which is invariably fatal when untreated. Treatment requires the achievement of remission of HLH prior to allogeneic hematopoietic stem cell transplantation, the only curative therapy to date.

Despite significant progress in the treatment, mortality remains high and an important number of patients will die before being eligible for HSCT.

A better understanding of the pathophysiology of FHL has opened new avenues for immunotherapy. Based on previous observations concerning the utilization of Anti-thymoglobulins (ATG) for the treatment of patients with FHL, the protocol propose a new therapeutic strategy using Alemtuzumab in association with steroids as first line treatment in FHL. This proposition is based on the hypothesis that Alemtuzumab, capable of killing T lymphocytes efficiently in vivo, should be better tolerated than ATG. In fact, in contrast to the mechanism of action of ATG, Alemtuzumab does not activate T lymphocytes.

A better tolerance and efficacy of Alemtuzumab is expected in the treatment of the hemophagocytic lymphohistiocytic syndrome. This may have a positive impact not only on survival until HSCT, but also on overall survival and quality of life with regard to long-term neurological sequelae.

This is a multicenter, open, phase I/II, non-comparative, non randomized study. Patients are recruited by the investigators during hospitalization for a first episode of lymphohistiocytic ac
Sponsor: Assistance Publique - Hôpitaux de Paris

Current Primary Outcome: Number of surviving patients until HSCT [ Time Frame: Day 1 until transplantation, up to 4 months ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Number of complete remissions following treatment [ Time Frame: Day 14, Day 21, Day 28 ]
    To assess the efficacy of the Alemtuzumab
  • Time of delay between the first administration of Alemtuzumab and complete remission [ Time Frame: Day 14, Day 21, Day 28 ]
    To assess the efficacy of the Alemtuzumab
  • Dosage of Alemtuzumab [ Time Frame: Day1-3, Day7, Day15-16, Day22-23, Day28 ]
    Pharmacokinetic
  • Number of side effects [ Time Frame: Day 1 until transplantation, up to 4 months ]
    To assess the tolerance of the Alemtuzumab


Original Secondary Outcome: Same as current

Information By: Assistance Publique - Hôpitaux de Paris

Dates:
Date Received: June 8, 2015
Date Started: June 2015
Date Completion: April 2019
Last Updated: December 2, 2016
Last Verified: December 2016