A Study to Evaluate the Benefit of Octagam 5%® in Subjects With Pediatric Acute-onset Neuropsychiatric Syndrome (PANS)

Study Overview
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Brief Summary
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This study will evaluate the use of intravenous immunoglobulins (IVIG) at a dose of 1g/Kg/body weight given every three weeks for 6 infusions in pediatric subjects ages 4 - 16 years with moderate to severe PANS. The study will compare biomarkers and behavioral scales before treatment, after the last infusion, 2 months, and at a minimum 6 months post-treatment.

Detailed Description
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IVIG at an immunomodulatory dose of 1 g/Kg body weight has been known to induce suppression of systemic inflammation and has been used in the treatment of autoimmune diseases. It has been proven beneficial in inflammatory conditions affecting the nervous system.

Official Title
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A Multi-site, Open-Label, Pilot Study to Evaluate the Benefit of Octagam 5%® in Subjects With Pediatric Acute-onset Neuropsychiatric Syndrome (PANS)

Conditions
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Pediatric Acute-Onset Neuropsychiatric Syndrome, Pediatric Autoimmune Neuropsychiatric Disorders Associated With Streptococcal Infections

Intervention / Treatment
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* Biological: IVIG


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male and female children, 4-16 years of age Diagnosis of moderate to severe PANS based on accepted criteria Must be willing to follow study procedures and comply with wash-out period If using prophylactic antibiotics, must be on stable dose for 3 months Exclusion Criteria: History of rheumatic fever, including Sydenham chorea (the neurologic manifestation) Previous IVIG therapy within the last 6 months Allergic reactions to blood products Patients who, in the investigator's opinion, might not be suitable for the trial. Steroid use

Ages Eligible for Study
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Minimum Age: 4 Years
Maximum Age: 16 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: IVIG<br>IVIG dose at 1 g/Kg/body weight | Biological: IVIG<br>* Intravenous immunoglobulin<br>* Other names: Octagam 5%;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Anxiety Disorders Interview Schedule for DSM-IV, Child, Parent Versions (ADIS) | ADIS Child and Parent Interviews are designed to diagnose children with emotional disorder, where anxiety is a prominent component. Problem behaviors and diagnoses include school refusal behavior, separation anxiety, social phobia, specific phobia, panic disorder, agoraphobia, OCD and PTSD. Assessment of ADHD allows for differentiation of inattentive type, hyperactive-type, and combined type. | Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks) | 
| Change in Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS) | The scale is a clinician-rated, 10-item scale, each item rated from 0 (no symptoms) to 4 (extreme symptoms) (total range, 0 to 40), with separate subtotals for severity of obsessions and compulsions. | Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks) | 
| Change in Yale Global Tic Severity Scale (YGTSS) | The CY-BOCS rating scale is designed to rate the severity and type of symptoms in patients with obsessive compulsive disorder (OCD). Ratings of the characteristics of obsessive or compulsive ideation and actions on a scale of 0 - 4, with 0 as none and 4 as extreme. | Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks) | 
| Change in Clinical Global Impressions (CGI) | CGI is a 3-item observer-rated scale used to measure symptom severity, global improvement, and therapeutic response. Items 1 and 2 are rated on a 7-point scale; item 3 is rated from 0 to 4 | Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks) | 
| Change in biomarkers levels | Neuro-immune biomarkers | Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Anxiety, Obsessive-Compulsive Disorder, PANS, PANDAS, Children