Phase III Study To Compare The Effect of Panzyga Versus Placebo in Patients With Pediatric Acute-onset Neuropsychiatric Syndrome (PANS/PANDAS)

NCT04508530 | Completed Phase 3 Results FDA Drug

• 1 other identifier: NGAM-13
• Study Type: interventional
• Target: 71
• Locations: 3 countries
• Sites: 12

Brief Summary

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

Conditions

Pediatric Acute-Onset Neuropsychiatric Syndrome

Outcome Measures

Primary Outcomes (1)

• Percentage Change in CY-BOCS Score From Baseline to Week 9

  The primary endpoint of this study was the percentage change of neuropsychiatric symptomatology and behavior in PANS patients determined by clinician-rated Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS) score. 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 (minimum total CY-BOCS score=0, maximum total CY-BOCS score=40). The mean percentage change in the total CY-BOCS score from Baseline to Week 9 was calculated and compared between Panzyga and Placebo treatment to demonstrate superiority. A value of 0 means there was no change from baseline to week 9. Negative values indicate a worse outcome. Positive values mean a better outcome.

  9 Weeks

Secondary Outcomes (2)

• Percentage Change in Total CY-BOCS Score From Week 9 to Week 18 Within the (Panzyga - Placebo) Treatment Sequence Group

  9 Weeks (Week 9 to Week 18)

• Clinical Global Impression - Improvement (CGI-I) Score at Week 9

  9 Weeks

Other Outcomes (1)

• Patients With PANS Improvement From Baseline to Week 9

  9 Weeks

Study Arms (2)

Panzyga

EXPERIMENTAL

Panzyga 10% IVIG

Biological: Panzyga

Placebo

PLACEBO COMPARATOR

Placebo

Other: Placebo

Interventions

Panzyga BIOLOGICAL

Panzyga 10% IVIG

Panzyga

Placebo OTHER

Placebo

Placebo

Eligibility Criteria

• Age: 6 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Patients ≥6 to ≤17 years of age.
• 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:
• Abrupt dramatic onset of OCD meeting DSM-5 diagnostic criteria for OCD as confirmed by the MINI-KID-7
• 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
• 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.
• Legal representative(s)/guardians(s) must be capable of understanding and complying with the relevant aspects of the study protocol.
• An episodic (relapsing-remitting) course of symptom severity
• Temporal association between symptoms onset or exacerbation and infections with group A streptococcal infection (GAS, positive throat culture and/or anti-GAS antibody titers)

You may not qualify if:

• Onset of current PANS episode more than 12 months prior to first investigational medicinal product (IMP) treatment.
• 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.
• Contraindication to receiving intravenous immunoglobulin (IVIG), including:
• History of severe hypersensitivity, e.g. anaphylaxis or severe systemic response, to immunoglobulin, blood or plasma derived products, or any component of Panzyga.
• Immunoglobulin (Ig) A deficiency with antibodies to IgA (\<7 mg/dL).
• Hyperviscosity syndromes or known or suspected hypercoagulable conditions as inferred from clinical history, which can increase risks of thrombosis associated with IVIG administration.
• 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.
• Need for live virus vaccine within three months after receiving study drug.
• 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). For Italy, estimated glomerular filtration rate (eGFR) needs to be calculated with the 2009 Schwartz equation (eGFR = k \* height/Serum creatinine (Scr), where k is 0.413) \[2\]. eGFR must not be below 30.
• 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)
• Body mass index ≥ 40 kg/m2
• Presence of symptoms consistent with autism or schizophrenia, bipolar disorder, or other psychotic disorder (unless psychotic symptoms have onset coincident with PANS).
• 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.
• Treatment with systemic corticosteroids within eight weeks before randomization.

Sponsors & Collaborators

• Octapharma: lead

Study Sites (12)

Octapharma Research Site

Tucson, Arizona, 85712, United States

Location

Octapharma Research Site

Little Rock, Arkansas, 72202, United States

Location

Octapharma Research Site

Los Angeles, California, 90024, United States

Location

Octapharma Research Site

Palo Alto, California, 94305, United States

Location

Octapharma Research Site

Centennial, Colorado, 80112, United States

Location

Octapharma Research Site

Boston, Massachusetts, 02114, United States

Location

Octapharma Research Site

Lebanon, New Hampshire, 03756, United States

Location

Octapharma Research Site

Genova, 16147, Italy

Location

Octapharma Research Site

Roma, 00161, Italy

Location

Octapharma Research Site

Roma, 00186, Italy

Location

Octapharma Research Site

Gothenburg, 41685, Sweden

Location

Octapharma Research Site

Stockholm, 17164, Sweden

Location

MeSH Terms

Conditions

Pediatric acute-onset neuropsychiatric syndrome

Interventions

Panzyga

Results Point of Contact

• Title: Patrick Murphy
• Organization: Clinical Research Management Group

p.murphy@crmg-usa.com

4138210022

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 7, 2020
• First Posted: August 11, 2020
• Study Start: June 30, 2021
• Primary Completion: August 27, 2024
• Study Completion: October 30, 2024
• Last Updated: January 7, 2026
• Results First Posted: January 7, 2026

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

SE (2); IT (3); US (7)