PANS Pediatric Acute-Onset Neuropsychiatric Syndrome Clinical Trial
Official title:
Intravenous Immunoglobulin (IVIG) Treatment in Children With Pediatric Acute-onset Neuropsychiatric Syndrome (PANS): an Open-label Trial in South-western Sweden
Open-label prospective trial to study efficacy, safety and tolerability of intravenous immunoglobulin (IVIG) once monthly for 6 months in children and adolescents with PANS. Number of subjects: 10. Age range: 4-17 years. Pediatric Acute-onset Neuropsychiatric Syndrome (PANS) is a recently defined research diagnosis describing an abrupt, dramatic onset of neuropsychiatric symptoms including obsessions/compulsions and/or food restriction in children. Immunologic mechanisms are suspected, but treatment trials are few.
Open-label prospective trial to study efficacy, safety and tolerability of intravenous immunoglobulin (IVIG) once monthly for 6 months in children and adolescents with PANS (including the subgroup PANDAS). Number of subjects: 10. Age range: 4-17 years. Pediatric Acute-onset Neuropsychiatric Syndrome (PANS) is a recently defined research diagnosis describing an abrupt, dramatic onset of neuropsychiatric symptoms including obsessions/compulsions and/or food restriction in children. Immunologic mechanisms are suspected, but treatment trials are few. The primary objective of this study is to evaluate the efficacy of intravenous immunoglobulin (IVIG), 2 g/kg given every 4 weeks for 6 months, to patients with post-infectious PANS/PANDAS, in improving neuropsychiatric symptoms and impairment. The secondary objectives of this study are to evaluate changes from baseline to follow-up at 3 months, 6 months and 12 months in: - Obsessive Compulsive Disorder (OCD) symptoms - adaptive functioning - quality of life - cognitive functioning - for patients with baseline inflammation signs on cerebral Magnetic Resonance Imaging (MRI) to evaluate changes in these measures after IVIG therapy after 6 months. - number of days of work/school/daily activities missed per subject year due to PANS/PANDAS before and after IVIG therapy - parental care load, e.g. need for sick leave, before and after IVIG therapy - Immunoglobulin (IgG, IgM and IgA) levels at baseline, 3 months, 6 months and 12 months - sustainability of any improvement at 12 months after initiation of IVIG measured with the PANS scale, CGI-S and CGI-I To assess the safety and tolerability of high dose IVIG therapy - Clinical signs and symptoms (nausea, headache, local reactions) - ALAT - Hemoglobin, complete blood count including leucocyte differential Investigational product: Intravenous immunoglobulin IVIG (Privigen), 2 g/kg BW every 4th week for 6 months (= 6 infusions). ;