A Study to Investigate the Safety and Efficacy of a New Drug in Children With a Disease That is Called "Reactivated Primary Haemophagocytic Lymphohistiocytosis"
The purpose of this study is to assess the safety, tolerability and preliminary efficacy of a new drug aimed at controlling disease reactivation in patients diagnosed with primary haemophagocytic lymphohistiocytosis and having previously shown partial response to current recommended treatment. The new drug will be administered on top of a glucocorticosteroid, which is usually part of the current recommended treatment.
Primary Hemophagocytic Lymphohistiocytosis
Drug: Cyclosporine A
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Pilot, Open-label, Single Arm, Multicentre Study to Explore Safety, Tolerability, Pharmacokinetics and Efficacy of Intravenous Multiple Administrations of NI-0501, an Anti-interferon Gamma (Anti-IFNγ) Monoclonal Antibody, in Paediatric Patients With Primary Haemophagocytic Lymphohistiocytosis Which Has Reactivated.|
- Safety and tolerability of NI-0501 in haemophagocytic lymphohistiocytosis reactivating patients [ Time Frame: 12 weeks ] [ Designated as safety issue: Yes ]
- Vital signs
- Physical examination
- Recording of adverse Events
- Routine laboratory assessments
- Preliminary efficacy [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]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.
|Study Start Date:||January 2013|
|Estimated Primary Completion Date:||February 2014 (Final data collection date for primary outcome measure)|
|Experimental: NI-0501||Biological: NI-0501 Drug: Dexamethasone Drug: Cyclosporine A|
|Contact: Cristina de Min, MD||+41-22-593- ext firstname.lastname@example.org|
|Azienda Ospedaliero Universitaria Meyer||Recruiting|
|Florence, Italy, 50139|
|Contact: Maurizio Arico, MD +39 055 566- ext 2739 email@example.com|