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

NCT03348618 | Unknown Phase 4 DMC FDA Drug

• 1 other identifier: IIS201701-PANS
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 3

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.

Conditions

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

Keywords

Obsessive-Compulsive Disorder; Anxiety; PANS; PANDAS; Children

Outcome Measures

Primary Outcomes (5)

• 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)

Study Arms (1)

IVIG

EXPERIMENTAL

IVIG dose at 1 g/Kg/body weight

Biological: IVIG

Interventions

IVIG BIOLOGICAL

Intravenous immunoglobulin

Also known as: Octagam 5%

IVIG

Eligibility Criteria

• Age: 4 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• IMMUNOe Research Centers: lead
• Octapharma: collaborator

Study Sites (3)

IMMUNOe Research Centers

Centennial, Colorado, 80112, United States

Location

Midwest Pediatrics

Papillion, Nebraska, 68046, United States

Location

Allergy, Asthma & Immunology Relief Research Institute

Charlotte, North Carolina, 28204, United States

Location

Related Publications (4)

• Swedo SE, Leckman JF Rose NR. From research subgroup to clinical syndrome: Modifying the PANDAS criteria to describe PANS (Pediatric Acute-onset Neuropsychiatric Syndrome). Pediatr Therapeut 2012, 2:2.

  BACKGROUND

• Chang K, Frankovich J, Cooperstock M, Cunningham MW, Latimer ME, Murphy TK, Pasternack M, Thienemann M, Williams K, Walter J, Swedo SE; PANS Collaborative Consortium. Clinical evaluation of youth with pediatric acute-onset neuropsychiatric syndrome (PANS): recommendations from the 2013 PANS Consensus Conference. J Child Adolesc Psychopharmacol. 2015 Feb;25(1):3-13. doi: 10.1089/cap.2014.0084. Epub 2014 Oct 17.

  PMID: 25325534 BACKGROUND

• Swedo SE, Leonard HL, Garvey M, Mittleman B, Allen AJ, Perlmutter S, Lougee L, Dow S, Zamkoff J, Dubbert BK. Pediatric autoimmune neuropsychiatric disorders associated with streptococcal infections: clinical description of the first 50 cases. Am J Psychiatry. 1998 Feb;155(2):264-71. doi: 10.1176/ajp.155.2.264.

  PMID: 9464208 BACKGROUND

• Melamed I, Kobayashi RH, O'Connor M, Kobayashi AL, Schechterman A, Heffron M, Canterberry S, Miranda H, Rashid N. Evaluation of Intravenous Immunoglobulin in Pediatric Acute-Onset Neuropsychiatric Syndrome. J Child Adolesc Psychopharmacol. 2021 Mar;31(2):118-128. doi: 10.1089/cap.2020.0100. Epub 2021 Feb 18.

  PMID: 33601937 DERIVED

MeSH Terms

Conditions

Pediatric acute-onset neuropsychiatric syndrome; Pediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcal infections; Obsessive-Compulsive Disorder; Anxiety Disorders

Interventions

Immunoglobulins, Intravenous

Condition Hierarchy (Ancestors)

Mental Disorders

Intervention Hierarchy (Ancestors)

Immunoglobulin G; Immunoglobulin Isotypes; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Officials

• Isaac Melamed, MD

  IMMUNOe Research Centers

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 14, 2017
• First Posted: November 21, 2017
• Study Start: November 24, 2017
• Primary Completion: November 1, 2019
• Study Completion: March 1, 2020
• Last Updated: November 19, 2019

Record last verified: 2019-03

Locations

US (3)