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Trial record 1 of 1 for:    NCT04508530
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Study To Compare The Effect of Panzyga Versus Placebo in Patients With Pediatric Acute-onset Neuropsychiatric Syndrome

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ClinicalTrials.gov Identifier: NCT04508530
Recruitment Status : Not yet recruiting
First Posted : August 11, 2020
Last Update Posted : August 11, 2020
Sponsor:
Information provided by (Responsible Party):
Octapharma

Brief Summary:
A Superiority Study To Compare The Effect of Panzyga Versus Placebo in Patients with Pediatric Acute-onset Neuropsychiatric Syndrome

Condition or disease Intervention/treatment Phase
Pediatric Acute-Onset Neuropsychiatric Syndrome Biological: Panzyga Other: Placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 92 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Superiority Study To Compare The Effect of Panzyga Versus Placebo in Patients With Pediatric Acute-onset Neuropsychiatric Syndrome
Estimated Study Start Date : January 1, 2021
Estimated Primary Completion Date : March 30, 2023
Estimated Study Completion Date : March 30, 2023

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Panzyga
Panzyga 10% IVIG
Biological: Panzyga
Panzyga 10% IVIG

Placebo Comparator: Placebo
Placebo
Other: Placebo
Placebo




Primary Outcome Measures :
  1. CY-BOCS score - Primary [ Time Frame: 9 weeks ]
    Improvement of neuropsychiatric symptomatology and behavior in PANS patients determined by clinician-rated Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS) score. The mean changes in the total CY-BOCS score from Baseline to Week 9 will be compared between Panzyga and placebo treatment to demonstrate superiority. The CY-BOCS scale score is a clinician-rated, semi-structured interview for rating the presence or absence, as well as the severity of obsessive-compulsive symptoms.The CY-BOCS yields a total obsession score, a total compulsion score and combined total score (maximum total CY-BOCS score=40).


Secondary Outcome Measures :
  1. CY-BOCS score - Secondary [ Time Frame: 18 weeks ]
    CY-BOCS score at the end of the follow-up period at Week 18 will be compared to the Week 9 scores within the (Panzyga - Placebo) treatment sequence group

  2. Clinical Global Impression [ Time Frame: 18 weeks ]
    To assess behavioral changes via the Clinical Global Impression (CGI) assessment. The Clinical Global Impression - Severity scale (CGI-S) is a 7-point scale that requires the clinician to rate the severity of the patient's illness at the time of assessment, relative to the clinician's past experience with patients who have the same diagnosis. Considering total clinical experience, a patient is assessed on severity of mental illness at the time of rating 1 = normal; not at all ill; 2, borderline mentally ill; 3, mildly ill; 4, moderately ill; 5, markedly ill; 6, severely ill; or 7, extremely ill. The Clinical Global Impression - Improvement scale (CGI-I) is a 7-point scale that requires the clinician to assess how much the patient's illness has improved or worsened relative to a baseline state at the beginning of the intervention and is rated as: 1, very much improved; 2, much improved; 3, minimally improved; 4, no change; 5, minimally worse; 6, much worse; or 7, very much worse.

  3. Parent OC Impact Scale [ Time Frame: 18 weeks ]
    To assess behavioral changes via the Parent OC Impact Scale - COIS-RP assessment. The Child Obsessive-Compulsive Impact Scale-Revised consists of parallel 33-item parent and child-report versions assessing impairment due to OCD across multiple functional domains. Items are rated on a 4-point scale from 0 (not at all) to 3 (very much).

  4. Child OC Impact Scale [ Time Frame: 18 weeks ]
    To assess behavioral changes via the Child OC Impact Scale COIS-RC assessment. The Child Obsessive-Compulsive Impact Scale-Revised consists of parallel 33-item parent and child-report versions assessing impairment due to OCD across multiple functional domains. Items are rated on a 4-point scale from 0 (not at all) to 3 (very much).

  5. SNAP-IV 90 item Scale [ Time Frame: 18 weeks ]
    To assess behavioral changes via the Swanson, Nolan, And Pelham Scale - Version IV (SNAP-IV) 90 item Scale. It is a 90-item, parent- or teacher-reported scale which elicits reporting on each of the DSM cardinal symptoms of Attention-Deficit Hyperactivity Disorder (ADHD), Oppositional Defiant Disorder (ODD), and Conduct Disorder (CD) in children and adolescents. The 90-item scale also includes items on aggression, mood, and school performance and behavior. It is used extensively in ADHD and disruptive behavior disorder intervention trials and is sensitive to change

  6. Parent Tic Questionnaire (PTQ) [ Time Frame: 18 weeks ]
    To assess behavioral changes via the Parent Tic Questionnaire (PTQ). The PTQ assesses tic severity in the past week, allowing for individual parent ratings of tic presence or absence.



Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Patients ≥6 to ≤17 years of age.
  2. Confirmed diagnosis of moderate to severe PANS (Children's Yale-Brown Obsessive Compulsive Scale [CY-BOCS] score ≥16) based on the following criteria:

    1. Abrupt dramatic onset of obsessive-compulsive disorder (OCD).
    2. Concurrent presence of additional neuropsychiatric symptoms, with similarly severe and acute onset, from at least two of the following seven categories, that are not better explained by a known neurologic or medical disorder, such as Sydenham chorea (SC), systemic lupus erythematosus, Tourette disorder, or other:

      • Anxiety (particularly, separation anxiety)
      • Emotional lability (extreme mood swings) and/or depression
      • Irritability, aggression and/or severely oppositional behaviors
      • Behavioral (developmental) regression (examples, talking baby talk,throwing temper tantrums, etc.)
      • Deterioration in school performance
      • Sensory or motor abnormalities
      • Somatic signs and symptoms, including sleep disturbances, bed wetting or urinary frequency
  3. Signed informed consent of patient's legal representative(s)/guardians(s). If patients are old enough to understand the risks and benefits of the study (as determined by each institution), they should provide written assent/consent.
  4. Legal representative(s)/guardians(s) must be capable of understanding and complying with the relevant aspects of the study protocol.

Patients who will additionally meet the following optional inclusion criteria will be identified as patients with Pediatric autoimmune neuropsychiatric disorders associated with streptococcal infection (PANDAS):

  1. An episodic (relapsing-remitting) course of symptom severity
  2. Temporal association between symptoms onset or exacerbation and infections with group A streptococcal infection (GAS, positive throat culture and/or anti-GAS antibody titers)

Exclusion Criteria:

  1. Onset of initial PANS symptoms more than six months prior to Screening. In patients with relapsing symptoms, initial symptoms may not have been more than 12 months prior to Screening, must have fully resolved, and their recurrence must be less than 6 months prior to Screening.
  2. Contraindication to receiving intravenous immunoglobulin (IVIG), including:

    1. History of severe hypersensitivity, e.g. anaphylaxis or severe systemic response, to immunoglobulin, blood or plasma derived products, or any component of Panzyga.
    2. Known immunoglobulin (Ig) A deficiency with antibodies to IgA.
    3. Hyperviscosity syndromes or known or suspected hypercoagulable conditions as inferred from clinical history, which can increase risks of thrombosis associated with IVIG administration.
    4. History of arterial or venous thrombotic or thromboembolic events (TEEs) within the last year prior Baseline. History of acquired or inherited thrombophilia any time prior to Baseline.
    5. Need for live virus vaccine within three months after receiving study drug.
    6. Renal dysfunction (creatinine 120 µmol/L or <1.35 mg/dL), history of renal dysfunction, or known risk factor for renal dysfunction (chronic renal insufficiency, diabetes mellitus, taking known nephrotoxic medication).
  3. Severely restricted food intake likely to require parenteral nutrition, or <5th percentile BMI-for-age.
  4. Presence of symptoms consistent with autism or schizophrenia, bipolar disorder, or other psychotic disorder.
  5. Presence of serious or unstable medical illness, psychiatric (e.g. high suicide risk) or behavioral symptoms that would make participation unsafe or study procedures too difficult to tolerate.
  6. Treatment with systemic corticosteroids within eight weeks before randomization.
  7. History of rheumatic fever, including SC (neurological manifestation).
  8. Past treatment of neuropsychiatric symptoms with immunomodulatory therapy (such as systemic corticosteroids, IVIG or plasmapheresis).
  9. Initiation of cognitive behavioral therapy (CBT) within eight weeks before randomization.
  10. Start of treatment or change in dosing with selective serotonin reuptake inhibitors [SSRIs] within eight weeks before randomization. Treatment with alpha-2 agonists or antipsychotics within eight weeks before randomization.
  11. Completion of antibiotics or antiviral drugs course for acute infection within one week before randomization. Use of antibiotics at a prophylactic dose is allowed if started at least eight weeks before randomization.
  12. Severe liver disease (alanine aminotransferase [ALT] three times above normal value).
  13. Known hepatitis B, hepatitis C or HIV infection.
  14. Cardiac insufficiency (New York Heart Association [NYHA] classification III-IV), cardiomyopathy, significant cardiac dysrhythmia requiring treatment.
  15. Medical conditions with symptoms and effects that could alter protein catabolism and/or IgG utilization (e.g. protein-losing enteropathies, nephrotic syndrome).
  16. Pregnant and lactating women.
  17. Female patients of childbearing potential unwilling to use a protocol-required method of contraception (as per protocol Section 7.3.9 b) from Screening throughout the study treatment period and for 30 days following the last dose of study drug. A woman of childbearing potential is defined as a fertile woman or adolescent, from the beginning of menstruation, unless permanently sterile.
  18. Participation in another interventional clinical trial that is either blinded or involves an investigational (not approved) product within three months before Baseline or during the course of the clinical study. Participation in observational clinical trials or openlabel trials involving an approved product may be permitted after consultation with the medical monitor.

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): NCT04508530


Contacts
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Contact: Mikaela Raymond 866-337-1868 ctgov@clinicalresearchmgt.com

Sponsors and Collaborators
Octapharma
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Responsible Party: Octapharma
ClinicalTrials.gov Identifier: NCT04508530    
Other Study ID Numbers: NGAM-13
First Posted: August 11, 2020    Key Record Dates
Last Update Posted: August 11, 2020
Last Verified: August 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Syndrome
Disease
Pathologic Processes