Clinical trial NCT03348618: A Study to Evaluate the Benefit of Octagam 5%® in Subjects With Pediatric Acute-onset Neuropsychiatric Syndrome (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
Overall status: UNKNOWN
Phase: PHASE4
Conditions: Pediatric Acute-Onset Neuropsychiatric Syndrome
Pediatric Autoimmune Neuropsychiatric Disorders Associated With Streptococcal Infections
Keywords: Obsessive-Compulsive Disorder
Anxiety
PANS
PANDAS
Children
Lead sponsor: IMMUNOe Research Centers
Sponsor class: INDUSTRY
Collaborators: name: Octapharma
First posted: 2017-11-21
Enrollment: 21
Sex: ALL
Minimum age (years): 4
Maximum age (years): 16
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Change in Anxiety Disorders Interview Schedule for DSM-IV, Child, Parent Versions (ADIS)
timeFrame: Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
measure: Change in Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS)
timeFrame: Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
measure: Change in Yale Global Tic Severity Scale (YGTSS)
timeFrame: Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
measure: Change in Clinical Global Impressions (CGI)
timeFrame: Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
measure: Change in biomarkers levels
timeFrame: Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
Secondary outcomes:
Other outcomes:
Locations:
city: Centennial
country: United States
facility: IMMUNOe Research Centers
state: Colorado
city: Papillion
country: United States
facility: Midwest Pediatrics
state: Nebraska
city: Charlotte
country: United States
facility: Allergy, Asthma & Immunology Relief Research Institute
state: North Carolina
Study officials:
affiliation: IMMUNOe Research Centers
name: Isaac Melamed, MD
role: PRINCIPAL_INVESTIGATOR
References:
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.
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.
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.
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.