Clinical trial NCT06811935: PEDI Pediatric Autoimmune Neuropsychiatric Disorder Associated With Streptococcus Study
Official title: Feasibility of a Study to Determine the Incidence and Natural History of Children With Pediatric Acute-Onset Neuropsychiatric Syndrome (PANS)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: PANS
PANDAS
Lead sponsor: University of Wisconsin, Madison
Sponsor class: OTHER
Collaborators: name: National Institute of Mental Health (NIMH)
First posted: 2025-02-06
Enrollment: 30
Sex: ALL
Minimum age (years): 3
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Children between the ages of 3 and 17 years with PANS or PANDAS.

Brief summary: The objective of the Pediatric Epidemiological Data and Incidence (PEDI) PANDAS study is to demonstrate the feasibility of enrollment and retention of subjects in a study to determine the incidence and natural history of children with Pediatric Acute-Onset Neuropsychiatric Syndrome (PANS) with regard to spectrum, course and outcome. The investigators aim to demonstrate they can recruit and retain 85% of children who are eligible for this study. Eligible children are those who fit criteria for PANS and Pediatric Autoimmune Neuropsychiatric Disorder Associated with Streptococcus (PANDAS). Each child will be followed for one year.

Detailed description: Specific Aim 1: To establish proof-of-concept of prospectively determining the incidence of PANDAS/PANS and the role of Group A streptococcus (GAS) in this group of disorders.

Specific Aim 1a: To prospectively identify and enroll children with PANDAS and PANS in geographically distinct populations of children in the US in order to determine the incidence and spectrum of these disorders.

Specific Aim 1b: To determine the role of GAS in new onset PANDAS/PANS.

Specific Aim 2: To establish proof-of-concept for studying the clinical course and outcome over the first year of treatment prospectively.

Eligibility criteria: Inclusion Criteria:

* Child age 3-17 years old
* Child and their parent/guardian are able to complete all study activities in English
* Abrupt onset of symptoms (within 7 days) occurring in the past 3 months. Willing to come in-person for at least the baseline visit.
* Pediatric patient who is a current member of the primary care population at either UW Health or Dartmouth Health
* Potential participant's family plans to be followed within UW Health or Dartmouth Health for at least one year
* Suspected or confirmed diagnosis of PANS or PANDAS based on published criteria:

  * PANS: (A) Abrupt, dramatic, overnight onset of obsessive compulsive disorder (OCD) or severely restricted food intake; (B) concurrent abrupt onset of additional severe neuropsychiatric symptoms from at least 2 of the following 7 categories:

    1. Anxiety
    2. Emotional lability and/or depression
    3. Irritability, aggression and/or severe oppositional behaviors
    4. Developmental regression
    5. Deterioration in school performance
    6. Sensory or motor abnormalities, including heightened sensitivity to sensory stimuli, hallucinations, dysgraphia, complex motor, and/or vocal tics
    7. Somatic signs and symptoms, including sleep disturbances, enuresis or urinary frequency consistent with a known neurologic or medical disorder.

AND (C) symptoms not better explained by another medical or neurologic disorder

* PANDAS

  * Presence of OCD and/or tic disorder
  * Pre-pubertal onset of symptoms (i.e., 3-12 years old)
  * Episodic course characterized by very acute and severe onset and dramatic symptom exacerbations
  * Neurologic abnormalities present during symptom exacerbations-motoric hyperactivity and adventitious movements
  * Temporal relationships between group A streptococcus (GAS) infections and symptom exacerbations

Exclusion Criteria:

* History of tics, OCD or food restriction/avoidance that contradicts a history of sudden onset or was present before the onset of this investigation
* Current symptom(s) of Pica
* Previous participation in the study
* Not suitable for study participation at the discretion of the site investigator (e.g., the child does not have verbal or cognitive ability adequate for self-report assessment)
* Patient or family is unable to complete the study questionnaires or procedures in English as the primary language

Primary outcomes:
measure: Percentage of Eligible Participants Enrolled
timeFrame: up to 12 months
Secondary outcomes:
measure: Percentage of Participants Retained at 12 months
timeFrame: up to 12 months
Other outcomes:
Locations:
city: Lebanon
country: United States
facility: Dartmouth Hitchcock Medical Center
state: New Hampshire
city: Madison
country: United States
facility: UW School of Medicine and Public Health
state: Wisconsin
Study officials:
affiliation: UW School of Medicine and Public Health
name: Ellen R Wald, MD
role: PRINCIPAL_INVESTIGATOR
References: