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

Pediatric Acute-Onset Neuropsychiatric Syndrome Clinical Trial

— 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)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03348618
• Other study ID #: IIS201701-PANS
• Status: Active, not recruiting
• Phase: Phase 4
• Start date: November 24, 2017
• Est. completion date: March 2020

Study information

• Verified date: March 2019
• Source: IMMUNOe Research Centers
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Active, not recruiting
• Enrollment: 21
• Est. completion date: March 2020
• Est. primary completion date: November 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years to 16 Years

Eligibility

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

Related Conditions & MeSH terms

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

Intervention

Biological:
• IVIG
Intravenous immunoglobulin

Locations

Country	Name	City	State
United States	IMMUNOe Research Centers	Centennial	Colorado
United States	Allergy, Asthma & Immunology Relief Research Institute	Charlotte	North Carolina
United States	Midwest Pediatrics	Papillion	Nebraska

Sponsors (2)

Lead Sponsor	Collaborator
IMMUNOe Research Centers	Octapharma

Country where clinical trial is conducted

United States, 

References & Publications (3)

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. — View 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.

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. Erratum in: Am J Psychiatry 1998 Apr;155(4):578. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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)
Primary	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)
Primary	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)
Primary	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)
Primary	Change in biomarkers levels	Neuro-immune biomarkers	Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)