Pediatric Acute-Onset Neuropsychiatric SyndromePhase 3NCT04508530
Phase III Study To Compare The Effect of Panzyga Versus Placebo in Patients With Pediatric Acute-onset Neuropsychiatric Syndrome (PANS/PANDAS)
A Phase 3 study testing Panzyga in people with Pediatric Acute-Onset Neuropsychiatric Syndrome, run by Octapharma at 12 sites in 3 countries.
Trial summary
A Superiority Study To Compare The Effect of Panzyga Versus Placebo in Patients with Pediatric Acute-onset Neuropsychiatric Syndrome Read more about this study
- Who is it for?
- Ages 6 to 17 years
- Conditions the study is for
- Pediatric Acute-Onset Neuropsychiatric Syndrome
- What is being tested?
- Panzyga
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 71 participants
- Where
- 12 sitesin 3 countries
- Study dates
- 30 Jun 2021to 30 Oct 2024
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
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- 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.
- 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):
- 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)
Who cannot join
- 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.
- 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).
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- 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.
- Treatment with NSAIDs within five days before randomization.
- Treatment with melatonin within one week before randomization
- History of rheumatic fever, including SC (neurological manifestation).
- Past treatment of neuropsychiatric symptoms with immunomodulatory therapy (such as IVIG, rituximab or mycophenolate mofetil) or plasmapheresis.
- Initiation of cognitive behavioral therapy (CBT) within eight weeks before randomization.
- 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.
- 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.
- 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.
- 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 four weeks before randomization (Section 4.2).
- Severe liver disease (alanine aminotransferase [ALT] three times above normal value).
- Known hepatitis B, hepatitis C or HIV infection as per patient medical history.
- Cardiac insufficiency (New York Heart Association [NYHA] classification III-IV), cardiomyopathy, significant cardiac dysrhythmia requiring treatment.
- Medical conditions with symptoms and effects that could alter protein catabolism and/or IgG utilization (e.g. protein-losing enteropathies, nephrotic syndrome).
- Pregnant and/or lactating women.
- 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 four weeks 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.
- 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 open-label trials involving an approved product may be permitted after consultation with the medical monitor.
Trial locations and contacts
12 sites in 3 countries.
12 sites in 3 countries
United States7 sites
Octapharma Research Site
Boston, Massachusetts, 02114
Octapharma Research Site
Centennial, Colorado, 80112
Octapharma Research Site
Lebanon, New Hampshire, 03756
Octapharma Research Site
Little Rock, Arkansas, 72202
Octapharma Research Site
Los Angeles, California, 90024
Octapharma Research Site
Palo Alto, California, 94305
Octapharma Research Site
Tucson, Arizona, 85712
Italy3 sites
Octapharma Research Site
Genova, 16147
Octapharma Research Site
Roma, 00161
Octapharma Research Site
Roma, 00186
Sweden2 sites
Octapharma Research Site
Gothenburg, 41685
Octapharma Research Site
Stockholm, 17164
About this study
Official title: A Superiority Phase III Study To Compare The Effect of Panzyga Versus Placebo in Patients With Pediatric Acute-onset Neuropsychiatric Syndrome (PANS/PANDAS)
A Superiority Study To Compare The Effect of Panzyga Versus Placebo in Patients with Pediatric Acute-onset Neuropsychiatric Syndrome
- Being tested
- Panzyga, Placebo
- Medical subject headings
- Pediatric acute-onset neuropsychiatric syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PanzygaBiological
Panzyga 10% IVIG
PlaceboOther
Placebo
| Study group | Role | What they receive |
|---|---|---|
| Panzyga | Experimental | Panzyga 10% IVIG Receives: Biological: Panzyga |
| Placebo | Placebo comparator | Placebo Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
The CY-BOCS scores at the end of the follow up period were compared with the Week 9 scores to examine the durability of any clinical benefit following Panzyga treatment in the first 9-week treatment period and whether there was any worsening after crossing over to placebo treatment.
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 of the total CY-BOCS score at Week 18 to the Week 9 score was calculated within the (Panzyga - Placebo) treatment sequence group. A value of 0 means there was no change from Week 9 to Week 18. Negative values indicate a worse outcome. Positive values mean a better outcome.
Measured: 9 Weeks (Week 9 to Week 18)
Clinical Global Impression - Improvement (CGI-I) Score at Week 9
The CGI-I rating scale was used to assess behavioral changes in PANS patients. 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. The mean CGI-I scores at Week 9 were compared between Panzyga and placebo treatment.
Measured: 9 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Aug 2020
- Study start30 Jun 2021
- Primary completion27 Aug 2024
- Study completion30 Oct 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Pediatric Acute-Onset Neuropsychiatric SyndromeStudies of the same condition, open and under way.
- More trials run by OctapharmaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 71 participants (actual)
Sponsor and investigators
- Lead sponsor
- Octapharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04508530
- Sponsor's protocol ID
- NGAM-13
- Status last verified
- Dec 2025
- First submitted
- 7 Aug 2020
- Last updated
- 7 Jan 2026
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Superiority Phase III Study To Compare The Effect of Panzyga Versus Placebo in Patients With Pediatric Acute-onset Neuropsychiatric Syndrome (PANS/PANDAS)
- ClinicalTrials.gov ID
- NCT04508530
- Acronym / sponsor ID
- NGAM-13
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 71 participants (actual)
- Lead sponsor
- OctapharmaIndustry (pharma, biotech, devices)
- Study start
- 30 Jun 2021 (actual)
- Primary completion
- 27 Aug 2024 (actual)
- Study completion
- 30 Oct 2024 (actual)
- First posted
- 11 Aug 2020 (actual)
- Results
- Posted 7 Jan 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A Superiority Study To Compare The Effect of Panzyga Versus Placebo in Patients with Pediatric Acute-onset Neuropsychiatric Syndrome
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Panzyga | Experimental | Biological: PanzygaPanzyga 10% IVIG |
| Placebo | Placebo comparator | Other: PlaceboPlacebo |
Who may qualify
- Ages
- 6 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (16 listed)
- 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:
- 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.
- 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):
Key exclusion criteria (32 listed)
- 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.
- Contraindication to receiving intravenous immunoglobulin (IVIG), including:
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PanzygaPanzyga 10% IVIG | Biological | Panzyga |
| PlaceboPlacebo | Other | Placebo |
Primary outcome
Percentage Change in CY-BOCS Score From Baseline to Week 9
Time frame: 9 Weeks
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.
2 secondary outcomes
Percentage Change in Total CY-BOCS Score From Week 9 to Week 18 Within the (Panzyga - Placebo) Treatment Sequence Group
Time frame: 9 Weeks (Week 9 to Week 18)
Clinical Global Impression - Improvement (CGI-I) Score at Week 9
Time frame: 9 Weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jan 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
12 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Panzyga in people with Pediatric Acute-Onset Neuropsychiatric Syndrome, run by Octapharma at 12 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 32 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States, Italy, Sweden. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 Jan 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage Change in CY-BOCS Score From Baseline to Week 9 (time frame: 9 Weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Jan 2026.