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

Primary Purpose

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

Status
Unknown status
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
IVIG
Sponsored by
IMMUNOe Research Centers
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Pediatric Acute-Onset Neuropsychiatric Syndrome focused on measuring Obsessive-Compulsive Disorder, Anxiety, PANS, PANDAS, Children

Eligibility Criteria

4 Years - 16 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • IMMUNOe Research Centers
  • Midwest Pediatrics
  • Allergy, Asthma & Immunology Relief Research Institute

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

IVIG

Arm Description

IVIG dose at 1 g/Kg/body weight

Outcomes

Primary Outcome Measures

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.
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.
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.
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
Change in biomarkers levels
Neuro-immune biomarkers

Secondary Outcome Measures

Full Information

First Posted
November 14, 2017
Last Updated
November 15, 2019
Sponsor
IMMUNOe Research Centers
Collaborators
Octapharma
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1. Study Identification

Unique Protocol Identification Number
NCT03348618
Brief Title
A Study to Evaluate the Benefit of Octagam 5%® in Subjects With Pediatric Acute-onset Neuropsychiatric Syndrome (PANS)
Acronym
PANS
Official Title
A Multi-site, Open-Label, Pilot Study to Evaluate the Benefit of Octagam 5%® in Subjects With Pediatric Acute-onset Neuropsychiatric Syndrome (PANS)
Study Type
Interventional

2. Study Status

Record Verification Date
March 2019
Overall Recruitment Status
Unknown status
Study Start Date
November 24, 2017 (Actual)
Primary Completion Date
November 2019 (Anticipated)
Study Completion Date
March 2020 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
IMMUNOe Research Centers
Collaborators
Octapharma

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pediatric Acute-Onset Neuropsychiatric Syndrome, Pediatric Autoimmune Neuropsychiatric Disorders Associated With Streptococcal Infections
Keywords
Obsessive-Compulsive Disorder, Anxiety, PANS, PANDAS, Children

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
21 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
IVIG
Arm Type
Experimental
Arm Description
IVIG dose at 1 g/Kg/body weight
Intervention Type
Biological
Intervention Name(s)
IVIG
Other Intervention Name(s)
Octagam 5%
Intervention Description
Intravenous immunoglobulin
Primary Outcome Measure Information:
Title
Change in Anxiety Disorders Interview Schedule for DSM-IV, Child, Parent Versions (ADIS)
Description
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.
Time Frame
Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
Title
Change in Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS)
Description
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.
Time Frame
Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
Title
Change in Yale Global Tic Severity Scale (YGTSS)
Description
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.
Time Frame
Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
Title
Change in Clinical Global Impressions (CGI)
Description
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
Time Frame
Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
Title
Change in biomarkers levels
Description
Neuro-immune biomarkers
Time Frame
Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
16 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Isaac Melamed, MD
Organizational Affiliation
IMMUNOe Research Centers
Official's Role
Principal Investigator
Facility Information:
Facility Name
IMMUNOe Research Centers
City
Centennial
State/Province
Colorado
ZIP/Postal Code
80112
Country
United States
Facility Name
Midwest Pediatrics
City
Papillion
State/Province
Nebraska
ZIP/Postal Code
68046
Country
United States
Facility Name
Allergy, Asthma & Immunology Relief Research Institute
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28204
Country
United States

12. IPD Sharing Statement

Citations:
Citation
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.
Results Reference
background
PubMed Identifier
25325534
Citation
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.
Results Reference
background
PubMed Identifier
9464208
Citation
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. Erratum In: Am J Psychiatry 1998 Apr;155(4):578.
Results Reference
background
PubMed Identifier
33601937
Citation
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.
Results Reference
derived

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

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