Clinical Trial: A Study to Investigate the Safety and Efficacy of an Anti-IFNγ mAb in Children Affected by Primary Haemophagocytic Lymphohistiocytosis

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

Official Title: A Phase 2/3, Open-label, Single Arm, Multicentre Study to Assess Safety, Tolerability, Pharmacokinetics and Efficacy of Intravenous Multiple Administrations of NI-0501, an Anti-interferon Gamma (Anti-

Brief Summary: The purpose of this study is to assess the safety, tolerability and efficacy of a new drug aimed at controlling disease activity in patients diagnosed with primary haemophagocytic lymphohistiocytosis. The new drug can be administered as the first-line therapy, to patients not previously treated with the current standard of care, or can be given to patients who have either failed or were unable to tolerate the current standard of care. Administration will be on top of a glucocorticosteroid, which is usually part of the current recommended treatment.

Detailed Summary:
Sponsor: NovImmune SA

Current Primary Outcome: Overall response rate [ Time Frame: end of treatment ]

Original Primary Outcome: Safety and tolerability of NI-0501 in haemophagocytic lymphohistiocytosis reactivating patients [ Time Frame: 12 weeks ]

  • Vital signs
  • Physical examination
  • Recording of adverse Events
  • Routine laboratory assessments


Current Secondary Outcome:

Original Secondary Outcome: Preliminary efficacy [ Time Frame: 12 weeks ]

Evolution of clinical signs (such as fever, splenomegaly, central nervous system symptoms) and laboratory parameters (such as complete blood cell count, fibrinogen, serum triglycerides, ferritin, soluble Interleukin-2 receptor levels), which characterize the disease, to assess response and time to response.


Information By: NovImmune SA

Dates:
Date Received: March 21, 2013
Date Started: January 2013
Date Completion:
Last Updated: February 7, 2017
Last Verified: February 2017