A Study to Evaluate the Benefit of Octagam 5%® in Subjects With Pediatric Acute-onset Neuropsychiatric Syndrome (PANS)
- Conditions
- Pediatric Autoimmune Neuropsychiatric Disorders Associated With Streptococcal InfectionsPediatric Acute-Onset Neuropsychiatric Syndrome
- Interventions
- Biological: IVIG
- Registration Number
- NCT03348618
- Lead Sponsor
- IMMUNOe Research Centers
- Brief Summary
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
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.
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- All
- Target Recruitment
- 21
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description IVIG IVIG IVIG dose at 1 g/Kg/body weight
- Primary Outcome Measures
Name Time Method Change in Anxiety Disorders Interview Schedule for DSM-IV, Child, Parent Versions (ADIS) Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks) 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.
Change in Yale Global Tic Severity Scale (YGTSS) Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks) 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.
Change in Clinical Global Impressions (CGI) Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks) 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
Change in Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS) Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks) 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.
Change in biomarkers levels Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks) Neuro-immune biomarkers
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (3)
IMMUNOe Research Centers
🇺🇸Centennial, Colorado, United States
Midwest Pediatrics
🇺🇸Papillion, Nebraska, United States
Allergy, Asthma & Immunology Relief Research Institute
🇺🇸Charlotte, North Carolina, United States