Ignite Creation Date:
2025-12-24 @ 12:08 PM
Ignite Modification Date:
2025-12-28 @ 1:41 PM
Study NCT ID:
NCT04609761
Status:
RECRUITING
Last Update Posted:
2024-11-08
First Post:
2020-10-05
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Open-label Trial of IVIG in Children With PANS
Sponsor:
Göteborg University
Organization:
Study Overview
Official Title:
Intravenous Immunoglobulin (IVIG) Treatment in Children With Pediatric Acute-onset Neuropsychiatric Syndrome (PANS): an Open-label Trial in South-western Sweden
Status:
RECRUITING
Status Verified Date:
2024-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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.
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).
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).
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?: