Clinical trial NCT02889016: Neurobiologic, Immunologic, and Rheumatologic Markers in Youth With PANS
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Pediatric Acute-Onset Neuropsychiatric Syndrome
Pediatric Autoimmune Neuropsychiatric Disorders Associated With Streptococcal Infections
PANS
PANDAS
Keywords: PANS
PANDAS
Lead sponsor: Stanford University
Sponsor class: OTHER
First posted: 2016-09-05
Enrollment: 500
Sex: ALL
Minimum age (years): 4
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The investigators to enroll 500 children with PANS/PANDAS and 100 healthy controls. Children's parents will also be enrolled to report information about their children as well as their own caregiver burden. The 500 patients with PANS will meet diagnostic criteria. These patients are targeted because this study is seeking to specifically assess the immunologic and neurological markers of PANS.

Brief summary: This study is an investigation of the neurologic, immunologic, and rheumatologic markers of Pediatric Acute-Onset Neuropsychiatric Syndrome (PANS). PANS is a condition characterized by the abrupt, dramatic onset of obsessive compulsive disorder (OCD) and/or eating restriction accompanied by equally abrupt and severe co-morbid neuropsychiatric symptoms, which include anxiety, emotional lability, depression, irritability, aggression, oppositionality, deterioration in school performance, behavioral (developmental) regression, sensory amplification, movement abnormalities, sleep disturbance, and urinary frequency. PANS is thought to be caused by infection, inflammation, or alternate triggers that is associated with a brain response that leads to these symptoms. The purpose of this study is to examine specific neurologic, immunologic, rheumatologic, and genomic, components in children with the acute-onset of psychiatric symptoms. This research may begin to uncover a much larger story of autoimmune processes that are involved in psychiatric disorders of childhood. By better understanding the etiologic components of psychiatric phenomenon, future treatments may be better targeted to underlying causes.

Detailed description: The investigators will recruit 500 children, 1-18 years old at onset with PANS/PANDAS. They will be treatment naive and within one month of onset/exacerbation. The 500 children with PANS will be gender- and age-matched to 100 healthy children, to allow examination of immunologic, neurologic, genomic, and behavioral differences between these two groups of children.

Eligibility criteria: Inclusion Criteria:

* Children with PANS

  1. Age 1-18 at onset of PANS
  2. Diagnosis of PANS: abrupt onset of OCD or food restriction, and at least two of the following associated symptoms: frequent urination, worsening handwriting/cognition, inattention, anorexia, separation anxiety, oppositionality, irritability/rage outbursts, and emotional lability.
  3. Patients must live within 90 miles of Stanford University and have a new onset of PANS illness
  4. Patients must have an established pediatrician within 90 miles of Stanford University for 3 years.
* Healthy Controls

  1. Age 4-18
  2. No psychiatric diagnosis

Exclusion Criteria:

* Any neuropsychiatric illness that may obscure the clear diagnosis of PANS

Primary outcomes:
measure: Cerebral blood flow
timeFrame: Through study completion, up to 12 years
measure: EEG patterns
timeFrame: Through study completion, up to 12 years
measure: Rapid Eye Movement (REM) motor disinhibition
timeFrame: Through study completion, up to 12 years
Secondary outcomes:
measure: Global Impairment Scores
timeFrame: Every 2-4 weeks for up to 12 years
measure: Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS)
timeFrame: Every 2-4 weeks for up to 12 years
measure: Columbia Impairment Scale
timeFrame: Every 2-4 weeks for up to 12 years
measure: Caregiver Burden Inventory
timeFrame: Every 2-4 weeks for up to 12 years
measure: Neurological findings
timeFrame: Every 2-4 weeks for up to 12 years
Other outcomes:
Locations:
city: Palo Alto
country: United States
facility: Stanford University
state: California
Study officials:
References:
citation: Swedo SE, Leckman JF, Rose NR (2012) From Research Subgroup to Clinical Syndrome: Modifying the PANDAS Criteria to Describe PANS (Pediatric Acute-onset Neuropsychiatric Syndrome). Pediatr Therapeut 2:113. doi: 10.4172/2161-0665.1000113
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: Murphy TK, Gerardi DM, Leckman JF. Pediatric acute-onset neuropsychiatric syndrome. Psychiatr Clin North Am. 2014 Sep;37(3):353-74. doi: 10.1016/j.psc.2014.06.001.