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    Summary
    EudraCT Number:2020-000867-21
    Sponsor's Protocol Code Number:NGAM-13
    National Competent Authority:Sweden - MPA
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2022-08-05
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedSweden - MPA
    A.2EudraCT number2020-000867-21
    A.3Full title of the trial
    A superiority Phase III study to compare the effect of Panzyga versus placebo in patients with pediatric acute-onset neuropsychiatric syndrome
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase III Study to Compare the Effect of Panzyga and Placebo in Patients with PANS (Pediatric Acute-Onset Neuropsychiatric Syndrome)
    A.3.2Name or abbreviated title of the trial where available
    N/A
    A.4.1Sponsor's protocol code numberNGAM-13
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorOctapharma Pharmazeutika Produktionsges.m.b.H.
    B.1.3.4CountryAustria
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportOctapharma Pharmazeutika Produktionsges.m.b.H.
    B.4.2CountryAustria
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationOctapharma Pharmazeutika Produktionsges.m.b.H.
    B.5.2Functional name of contact pointGlobal Clinical Project Manager
    B.5.3 Address:
    B.5.3.1Street AddressOberlaaer Str. 235
    B.5.3.2Town/ cityVienna
    B.5.3.3Post code1100
    B.5.3.4CountryAustria
    B.5.4Telephone number+431610321698
    B.5.5Fax number+431610329249
    B.5.6E-mailAT1ClinicalDepartment@octapharma.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Panzyga
    D.2.1.1.2Name of the Marketing Authorisation holderOctapharma
    D.2.1.2Country which granted the Marketing AuthorisationAustria
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namePanzyga
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNImmunoglobulin G
    D.3.9.1CAS number 308067-58-5
    D.3.9.2Current sponsor codeN/A
    D.3.9.3Other descriptive nameHUMAN NORMAL IMMUNOGLOBULIN (IV)
    D.3.9.4EV Substance CodeSUB12041MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) Yes
    D.3.11.7Plasma derived medicinal product Yes
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Pediatric acute-onset neuropsychiatric syndrome (PANS)
    E.1.1.1Medical condition in easily understood language
    PANS is a condition which can cause to suddenly change the way a patient feels and acts. Possible causes for PANS are infections, changes in the body's chemical reactions, and inflammations.
    E.1.1.2Therapeutic area Psychiatry and Psychology [F] - Behavioral Disciplines and Activities [F04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 25.0
    E.1.2Level LLT
    E.1.2Classification code 10086608
    E.1.2Term PANS
    E.1.2System Organ Class 10037175 - Psychiatric disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    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.
    E.2.2Secondary objectives of the trial
    The secondary objectives of this study are to:
    • verify the sustainability of the reduction of the severity of symptoms in pediatric patients treated with Panzyga
    • assess the efficacy of Panzyga treatment in reducing functional impairment associated with PANS.
    E.2.3Trial contains a sub-study No
    E.3Principal 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)
    E.4Principal 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 four weeks before randomization (Section 4.2).
    19. Severe liver disease (alanine aminotransferase [ALT] three times above normal value).
    20. Known hepatitis B, hepatitis C or HIV infection as per patient medical history.
    21. Cardiac insufficiency (New York Heart Association [NYHA] classification III-IV), cardiomyopathy, significant cardiac dysrhythmia requiring treatment.
    22. Medical conditions with symptoms and effects that could alter protein catabolism and/or IgG utilization (e.g. protein-losing enteropathies, nephrotic syndrome).
    23. Pregnant and/or lactating women.
    E.5 End points
    E.5.1Primary 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.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 6 (Cycle 3)
    E.5.2Secondary end point(s)
    To assess behavioral changes by means of the following standardized assessments:
    • 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
    • Clinical Global Impression (CGI)
    • Parent & Child OC Impact Scale – Revised (COIS-RP and COIS-RC)
    • The Swanson, Nolan, and Pelham Rating Scale (SNAP-IV, 90 item)
    • Parent Tic Questionnaire (PTQ)
    E.5.2.1Timepoint(s) of evaluation of this end point
    every three weeks until Week 18
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over Yes
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days15
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months6
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 92
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 65
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 27
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 92
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2022-09-27
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial Status
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