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

November 15, 2019 updated by: IMMUNOe Research Centers

A Multi-site, Open-Label, Pilot Study to Evaluate the Benefit of Octagam 5%® in Subjects With Pediatric Acute-onset Neuropsychiatric Syndrome (PANS)

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.

Study Overview

Status

Unknown

Conditions

Intervention / 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.

Study Type

Interventional

Enrollment (Anticipated)

21

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Colorado
      • Centennial, Colorado, United States, 80112
        • IMMUNOe Research Centers
    • Nebraska
      • Papillion, Nebraska, United States, 68046
        • Midwest Pediatrics
    • North Carolina
      • Charlotte, North Carolina, United States, 28204
        • Allergy, Asthma & Immunology Relief Research Institute

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

4 years to 16 years (CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: IVIG
IVIG dose at 1 g/Kg/body weight
Biological: IVIG
Intravenous immunoglobulin
Other Names:
  • Octagam 5%

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in Anxiety Disorders Interview Schedule for DSM-IV, Child, Parent Versions (ADIS)
Time Frame: Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
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)
Change in Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS)
Time Frame: Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
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)
Change in Yale Global Tic Severity Scale (YGTSS)
Time Frame: Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
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)
Change in Clinical Global Impressions (CGI)
Time Frame: Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
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)
Change in biomarkers levels
Time Frame: Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
Neuro-immune biomarkers
Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

IMMUNOe Research Centers

Collaborators

Octapharma

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

November 24, 2017

Primary Completion (ANTICIPATED)

November 1, 2019

Study Completion (ANTICIPATED)

March 1, 2020

Study Registration Dates

First Submitted

November 14, 2017

First Submitted That Met QC Criteria

November 17, 2017

First Posted (ACTUAL)

November 21, 2017

Study Record Updates

Last Update Posted (ACTUAL)

November 19, 2019

Last Update Submitted That Met QC Criteria

November 15, 2019

Last Verified

March 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • IIS201701-PANS

Drug and device information, study documents

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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