Study Comparing Human Normal Immunoglobulin and Placebo for Children with Pediatric Acute-Onset Neuropsychiatric Syndrome (PANS)

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What is this study about?

This clinical trial is focused on studying a condition known as Pediatric Acute-Onset Neuropsychiatric Syndrome (PANS). PANS is a disorder that can cause sudden and severe changes in behavior and mood in children, often including symptoms like obsessive-compulsive disorder (OCD), anxiety, and mood swings. The study will test a treatment called Panzyga, which is a solution containing human normal immunoglobulin, a type of protein derived from blood that is used to help the immune system. The trial will compare the effects of Panzyga to a placebo, which is a solution of 0.9% sodium chloride, to see if Panzyga is more effective in reducing the symptoms of PANS.

The purpose of the study is to determine if Panzyga can better alleviate the symptoms associated with PANS compared to the placebo. Participants in the study will receive either Panzyga or the placebo through an intravenous infusion, which means the solution is given directly into a vein. The study will last for several weeks, during which the participants’ symptoms will be monitored and assessed by healthcare professionals. The main goal is to see if there is an improvement in the severity of the symptoms from the beginning of the study to the end of the treatment period.

Throughout the study, the participants’ progress will be evaluated using various scales and questionnaires designed to measure changes in their symptoms and overall behavior. These assessments will help determine the effectiveness of Panzyga in providing a lasting benefit for children with PANS. The study aims to provide valuable information on whether Panzyga can be a superior treatment option for managing the symptoms of this challenging condition.

1 joining the study

Upon joining the study, eligibility is confirmed based on specific criteria, including age and diagnosis of pediatric acute-onset neuropsychiatric syndrome (PANS).

Legal representatives or guardians must provide informed consent, and if applicable, the patient may also provide assent.

2 initial assessment

An initial assessment is conducted to establish a baseline for symptoms using the Clinician-rated CY-BOCS score, which measures the severity of obsessive-compulsive symptoms.

3 treatment phase

The treatment phase involves the administration of Panzyga or a placebo. Panzyga is a solution for infusion containing human normal immunoglobulin, administered intravenously.

The primary objective is to determine if Panzyga is more effective than the placebo in reducing the severity of symptoms associated with PANS.

4 monitoring and follow-up

Throughout the study, symptoms are monitored using various scales, including the Clinical Global Impression (CGI) and the Parent & Child OC Impact Scale.

The primary endpoint is the improvement in neuropsychiatric symptoms, measured by changes in the CY-BOCS score from the baseline to Week 9.

5 end of treatment assessment

At the end of the treatment phase, an assessment is conducted to evaluate the changes in symptoms and the effectiveness of the treatment.

6 follow-up period

A follow-up period extends to Week 18 to assess the durability of the clinical benefits associated with Panzyga treatment.

The CY-BOCS score at the end of the follow-up period is compared to the Week 9 scores to evaluate the lasting effects of the treatment.

Who Can Join the Study?

Patients must be between the ages of 6 and 17 years old.
Patients need to have a confirmed diagnosis of moderate to severe Pediatric acute-onset neuropsychiatric syndrome (PANS). This includes having noticeable and stable obsessive-compulsive disorder (OCD) symptoms. OCD symptoms should be rated as 4 or higher on a specific scale, with little change between measurements.
The diagnosis should be based on a sudden and dramatic start of OCD that meets specific diagnostic criteria. This should be confirmed by a tool called MINI-KID-7.
Patients should also have other sudden and severe neuropsychiatric symptoms from at least two of the following categories, which cannot be explained by other known disorders:

Anxiety, especially separation anxiety.
Emotional lability, which means extreme mood swings, and/or depression.
Irritability, aggression, and/or very oppositional behaviors.
Behavioral regression, such as acting younger than their age, like talking baby talk or having temper tantrums.
Deterioration in school performance.
Sensory or motor abnormalities.
Somatic signs and symptoms, including sleep problems, bed wetting, or frequent urination.

The patient’s legal representative(s) or guardian(s) must sign an informed consent form. If the patient is old enough to understand the study, they should also provide written agreement.
The legal representative(s) or guardian(s) must be able to understand and follow the study’s requirements.
For patients with Pediatric autoimmune neuropsychiatric disorders associated with streptococcal infection (PANDAS), additional criteria include:

An episodic pattern of symptom severity, meaning symptoms come and go.
A link between the start or worsening of symptoms and infections with group A streptococcal infection, shown by a positive throat culture or specific antibody tests.

Who Cannot Join the Study?

Patients who have a known allergy or severe reaction to the study medication or its ingredients cannot participate.
Patients with a history of certain medical conditions that might interfere with the study results are excluded.
Patients who are currently participating in another clinical trial are not eligible.
Patients who have received certain treatments or medications recently that could affect the study outcomes are excluded.
Patients with a history of substance abuse or dependency that could interfere with the study are not eligible.
Patients who are unable to comply with the study procedures or follow-up visits are excluded.
Patients with any other condition that the study doctors believe would make participation unsafe or affect the study results are not eligible.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy

Other Sites

Site Name	City	Country
IRCCS Istituto Giannina Gaslini	Genoa	Italy
Queen Silvia Childrens Hospital – Sahlgrenska University Hospital – Vaestra Goetalandsregionen	Gothenburg	Sweden
Azienda Socio Sanitaria Locale N. 8 Di Cagliari	Cagliari	Italy
Karolinska University Hospital	Solna	Sweden
A.O.U. Policlinico G. Martino Di Messina	Messina	Italy
Uatghdchac Dmlsl Sqgvm Da Remz Le Sbavmkkf	Rome	Italy

Trial status

Country	Status	Recruitment Start
Italy	Not recruiting	07.06.2023
Sweden	Not recruiting	07.06.2023

Trial locations

Investigated drugs:

Human Normal Immunoglobulin

Panzyga is a medication used in this clinical trial to treat pediatric acute-onset neuropsychiatric syndrome (PANS). It is an intravenous immunoglobulin (IVIG) therapy, which means it is made from antibodies that help the immune system fight infections and other diseases. The goal of using Panzyga in this study is to see if it can reduce the severity of symptoms in children with PANS.

Pediatric acute-onset neuropsychiatric syndrome (PANS) – This condition is characterized by a sudden onset of obsessive-compulsive symptoms or severely restricted food intake, along with at least two additional neuropsychiatric symptoms. These can include anxiety, emotional lability, depression, irritability, aggression, behavioral regression, and sensory or motor abnormalities. The symptoms appear abruptly and can be severe, impacting the child’s daily functioning. PANS is thought to be triggered by infections, environmental factors, or other stressors, leading to an abnormal immune response. The progression of the disease can vary, with symptoms fluctuating in intensity and duration. It is important to monitor and manage the symptoms to improve the quality of life for affected children.

Trial ID:

2024-511673-30-00

Protocol code:

NGAM-13

NCT ID:

NCT04508530

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study Comparing Human Normal Immunoglobulin and Placebo for Children with Pediatric Acute-Onset Neuropsychiatric Syndrome (PANS)

What is this study about?

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