NCT04609761

Open-label Trial of IVIG in Children With PANS

Intravenous Immunoglobulin (IVIG) Treatment in Children With Pediatric Acute-onset Neuropsychiatric Syndrome (PANS): an Open-label Trial in South-western Sweden

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 17 (actual)
• Sponsor: Göteborg University · Academic / Other
• Sex: All
• Age: 4 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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).

Conditions

• PANS Pediatric Acute-Onset Neuropsychiatric Syndrome

Interventions

Type	Name	Description
DRUG	Privigen Injectable Product (Intravenous Immunoglobulin)	Privigen, 2 g/kg BW every 4th week for 6 months (= 6 infusions).

Timeline

• Start date: 2021-01-12
• Primary completion: 2025-10-15
• Completion: 2025-10-15
• First posted: 2020-10-30
• Last updated: 2026-03-06

Locations

1 site across 1 country: Sweden