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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03348618
Recruitment Status : Active, not recruiting
First Posted : November 21, 2017
Last Update Posted : November 19, 2019
Sponsor:
Collaborator:
Octapharma
Information provided by (Responsible Party):
IMMUNOe Research Centers

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.

Condition or disease Intervention/treatment Phase
Pediatric Acute-Onset Neuropsychiatric Syndrome Pediatric Autoimmune Neuropsychiatric Disorders Associated With Streptococcal Infections Biological: IVIG Phase 4

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 21 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Actual Study Start Date : November 24, 2017
Estimated Primary Completion Date : November 2019
Estimated Study Completion Date : March 2020


Arm Intervention/treatment
Experimental: IVIG
IVIG dose at 1 g/Kg/body weight
Biological: IVIG
Intravenous immunoglobulin
Other Name: Octagam 5%




Primary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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

  5. 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



Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03348618


Locations
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United States, Colorado
IMMUNOe Research Centers
Centennial, Colorado, United States, 80112
United States, Nebraska
Midwest Pediatrics
Papillion, Nebraska, United States, 68046
United States, North Carolina
Allergy, Asthma & Immunology Relief Research Institute
Charlotte, North Carolina, United States, 28204
Sponsors and Collaborators
IMMUNOe Research Centers
Octapharma
Investigators
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Principal Investigator: Isaac Melamed, MD IMMUNOe Research Centers

Publications:
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Responsible Party: IMMUNOe Research Centers
ClinicalTrials.gov Identifier: NCT03348618    
Other Study ID Numbers: IIS201701-PANS
First Posted: November 21, 2017    Key Record Dates
Last Update Posted: November 19, 2019
Last Verified: March 2019

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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
Keywords provided by IMMUNOe Research Centers:
Obsessive-Compulsive Disorder
Anxiety
PANS
PANDAS
Children
Additional relevant MeSH terms:
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Streptococcal Infections
Syndrome
Disease
Pathologic Processes
Gram-Positive Bacterial Infections
Bacterial Infections
gamma-Globulins
Immunoglobulins, Intravenous
Rho(D) Immune Globulin
Immunologic Factors
Physiological Effects of Drugs