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

Status

Indicates the current recruitment status or the expanded access status.
Unknown

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Anticipated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
21

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
IMMUNOe Research Centers

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
November 24, 2017

Primary Completion (ANTICIPATED)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
November 1, 2019

Study Completion (ANTICIPATED)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
March 1, 2020

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 14, 2017

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 17, 2017

First Posted (ACTUAL)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 21, 2017

Study Record Updates

Last Update Posted (ACTUAL)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
November 19, 2019

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 15, 2019

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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