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A Phase III Study to Compare the Effect of Panzyga and Placebo in Patients with PANS (Pediatric Acute-Onset Neuropsychiatric Syndrome)

Phase 1
Conditions
Pediatric acute-onset neuropsychiatric syndrome (PANS)
MedDRA version: 25.0Level: LLTClassification code 10086608Term: PANSSystem Organ Class: 10037175 - Psychiatric disorders
Therapeutic area: Psychiatry and Psychology [F] - Behavioral Disciplines and Activities [F04]
Registration Number
EUCTR2020-000867-21-SE
Lead Sponsor
Octapharma Pharmazeutika Produktionsges.m.b.H.
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
ot Recruiting
Sex
All
Target Recruitment
92
Inclusion Criteria

1. Patients =6 to =17 years of age.
2. Confirmed diagnosis of moderate to severe PANS with prominent and stable obsessive-compulsive disorder (OCD) symptoms (i.e. Clinical Global Impression (CGI)—Severity-OCD rating of = 4 or higher on 2 ratings without a change of more than 1 unit between measurements) based on the following criteria:
a. Abrupt dramatic onset of OCD meeting DSM-5 diagnostic criteria for OCD as confirmed by the MINI-KID-7
b. 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)
Are the trial subjects under 18? yes
Number of subjects for this age range: 92
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1.Onset of current PANS episode more than 12 months prior to first investigational medicinal product (IMP) treatment.
2.a. In patients with relapsing episodes: Onset of initial PANS episode more than 24 months prior to first IMP treatment
b. In patients with relapsing episodes: Absence of significant improvement and stabilization between the episodes according to investigator's judgment.
3.Contraindications to receiving intravenous immunoglobulin (IVIG), including:
a.History of severe hypersensitivity, e.g. anaphylaxis or severe systemic response, to immunoglobulin, blood or plasma derived products, or any component of Panzyga.
b.Immunoglobulin (Ig) A deficiency with antibodies to IgA (<7 mg/dL).
c.Hyperviscosity syndromes or known or suspected hypercoagulable conditions as inferred from clinical history, which can increase risks of thrombosis associated with IVIG administration.
d.History of arterial or venous thrombotic or thromboembolic events (TEEs) within the last year prior to Baseline. History of acquired or inherited thrombophilia any time prior to Baseline.
e.Need for live virus vaccine within three months after receiving study drug.
f.Renal dysfunction (creatinine >120 µmol/L or 1.36 mg/dL), history of renal dysfunction, or known risk factor for renal dysfunction (chronic renal insufficiency, diabetes mellitus, taking known nephrotoxic medication).
4.Severely restricted food intake likely to require parenteral nutrition, and <5th percentile BMI-for-age (BMI Percentile Calculator for Child and Teen based on Centers for Disease Control and Prevention growth charts for children and teens ages 2 through 19 years)
5.Body mass index = 40 kg/m2
6.Presence of symptoms consistent with autism or schizophrenia, bipolar disorder, or other psychotic disorder (unless psychotic symptoms have onset coincident with PANS).
7.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.
8.Treatment with systemic corticosteroids within eight weeks before randomization.
9. Treatment with NSAIDs within five days before randomization.
10. Treatment with melatonin within one week before randomization.
11.History of rheumatic fever, including SC (neurological manifestation).
12.Past treatment of neuropsychiatric symptoms with immunomodulatory therapy (such as IVIG, rituximab or mycophenolate mofetil) or plasmapheresis.
13.Initiation of cognitive behavioral therapy (CBT) within eight weeks before randomization.
14.Start of treatment or change in dosing with selective serotonin reuptake inhibitors [SSRIs] within eight weeks before randomization.
15.Treatment with alpha-2 agonists or antipsychotics within eight weeks before randomization.
16.Start of treatment or change in dosing with stimulants (Methylphenidate, Amphetamine and similar products) for Attention-Deficit Hyperactivity Disorder (ADHD) within four weeks prior to randomization.
17.Active use of tetrahydrocannabinol (THC) containing agents within four weeks prior to enrollment or during the trial. Use of cannabidiol- (CBD) / cannabimovone- (CBM) containing agents without THC is allowed if started more than eight weeks before enrollment in a stable dose/frequency.
18.Use of antibiotics or antiviral drugs at therapeutic dose within one week before randomization. Use of antibiotics at a prophylactic dose is allowed if started at least fou

Study & Design

Study Type
Interventional clinical trial of medicinal product
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Main Objective: The primary objective of this study is to confirm that Panzyga is superior to placebo (0.9% w/v sodium chloride) for reducing the severity of symptoms associated with PANS in pediatric patients.;Secondary Objective: The secondary objectives of this study are to:<br>• verify the sustainability of the reduction of the severity of symptoms in pediatric patients treated with Panzyga<br>• assess the efficacy of Panzyga treatment in reducing functional impairment associated with PANS.;Primary end point(s): Improvement of neuropsychiatric symptomatology and behavior in PANS patients determined by clinician-rated 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.;Timepoint(s) of evaluation of this end point: Week 6 (Cycle 3)
Secondary Outcome Measures
NameTimeMethod
Secondary end point(s): To assess behavioral changes by means of the following standardized assessments:<br>• To assess the durability of the clinical benefit associated with Panzyga treatment, the 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 to assess the durability of the clinical benefit associated with Panzyga treatment<br>• Clinical Global Impression (CGI)<br>• Parent & Child OC Impact Scale – Revised (COIS-RP and COIS-RC)<br>• The Swanson, Nolan, and Pelham Rating Scale (SNAP-IV, 90 item)<br>• Parent Tic Questionnaire (PTQ)<br>;Timepoint(s) of evaluation of this end point: every three weeks until Week 18
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