CompletedPhase 3

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

United States71 participantsStarted 2021-06-30

Plain-language summary

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

Age range6 Years – 17 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. 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:
✓. 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)

✕. 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:
✕. 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.

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

Timeframe: 9 Weeks

NCT IDNCT04508530

SponsorOctapharma

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-08-27

Results submitted2025-11-19

Who can participate

Age range6 Years – 17 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. 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:
✓. 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)

✕. 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:
✕. 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.