Pediatric Acute-Onset Neuropsychiatric SyndromePhase 4PANSNCT03348618
A Study to Evaluate the Benefit of Octagam 5%® in Subjects With Pediatric Acute-onset Neuropsychiatric Syndrome (PANS)
A Phase 4 study testing IVIG in people with Pediatric Acute-Onset Neuropsychiatric Syndrome and Pediatric Autoimmune Neuropsychiatric Disorders Associated With Streptococcal Infections, run by IMMUNOe Research Centers at 3 sites in United States.
Trial summary
This study will evaluate the use of intravenous immunoglobulins (IVIG) at a dose of 1g/Kg/body weight given every three weeks for 6 infusions in pediatric subjects ages 4 - 16 years with moderate to severe PANS. Read more about this study
- Who is it for?
- Ages 4 to 16 years
- Conditions the study is for
- Pediatric Acute-Onset Neuropsychiatric Syndrome, Pediatric Autoimmune Neuropsychiatric Disorders Associated With Streptococcal Infections
- What is being tested?
- IVIG
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 3 sitesUnited States
- Study dates
- 24 Nov 2017to Mar 2020
- Phase
- Phase 4
- Are there results?
- None posted in the registry
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 4 to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male and female children, 4-16 years of age
- Diagnosis of moderate to severe PANS based on accepted criteria
- Must be willing to follow study procedures and comply with wash-out period
- If using prophylactic antibiotics, must be on stable dose for 3 months
Who cannot join
- History of rheumatic fever, including Sydenham chorea (the neurologic manifestation)
- Previous IVIG therapy within the last 6 months
- Allergic reactions to blood products
- Patients who, in the investigator's opinion, might not be suitable for the trial.
- Steroid use
Trial locations and contacts
3 sites in 1 country.
United States3 sites
IMMUNOe Research Centers
Centennial, Colorado, 80112
Allergy, Asthma & Immunology Relief Research Institute
Charlotte, North Carolina, 28204
Midwest Pediatrics
Papillion, Nebraska, 68046
About this study
Official title: A Multi-site, Open-Label, Pilot Study to Evaluate the Benefit of Octagam 5%® in Subjects With Pediatric Acute-onset Neuropsychiatric Syndrome (PANS)
This study will evaluate the use of intravenous immunoglobulins (IVIG) at a dose of 1g/Kg/body weight given every three weeks for 6 infusions in pediatric subjects ages 4 - 16 years with moderate to severe PANS. The study will compare biomarkers and behavioral scales before treatment, after the last infusion, 2 months, and at a minimum 6 months post-treatment.
Read the detailed description
IVIG at an immunomodulatory dose of 1 g/Kg body weight has been known to induce suppression of systemic inflammation and has been used in the treatment of autoimmune diseases. It has been proven beneficial in inflammatory conditions affecting the nervous system.
- Conditions
- Pediatric Acute-Onset Neuropsychiatric Syndrome, Pediatric Autoimmune Neuropsychiatric Disorders Associated With Streptococcal Infections
- Being tested
- IVIG
- Keywords
- Obsessive-Compulsive Disorder, Anxiety, PANS, PANDAS, Children
- Medical subject headings
- Pediatric acute-onset neuropsychiatric syndrome, Pediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcal infections, Obsessive-Compulsive Disorder, Anxiety Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IVIGBiological
Also called Octagam 5%
Intravenous immunoglobulin
| Study group | Role | What they receive |
|---|---|---|
| IVIG | Experimental | IVIG dose at 1 g/Kg/body weight Receives: Biological: IVIG |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Anxiety Disorders Interview Schedule for DSM-IV, Child, Parent Versions (ADIS)
ADIS Child and Parent Interviews are designed to diagnose children with emotional disorder, where anxiety is a prominent component. Problem behaviors and diagnoses include school refusal behavior, separation anxiety, social phobia, specific phobia, panic disorder, agoraphobia, OCD and PTSD. Assessment of ADHD allows for differentiation of inattentive type, hyperactive-type, and combined type.
Measured: Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
Change in Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS)
The scale is a clinician-rated, 10-item scale, each item rated from 0 (no symptoms) to 4 (extreme symptoms) (total range, 0 to 40), with separate subtotals for severity of obsessions and compulsions.
Measured: Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
Change in Yale Global Tic Severity Scale (YGTSS)
The CY-BOCS rating scale is designed to rate the severity and type of symptoms in patients with obsessive compulsive disorder (OCD). Ratings of the characteristics of obsessive or compulsive ideation and actions on a scale of 0 - 4, with 0 as none and 4 as extreme.
Measured: Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
Change in Clinical Global Impressions (CGI)
CGI is a 3-item observer-rated scale used to measure symptom severity, global improvement, and therapeutic response. Items 1 and 2 are rated on a 7-point scale; item 3 is rated from 0 to 4
Measured: Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
Change in biomarkers levels
Neuro-immune biomarkers
Measured: Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Nov 2017
- Study start24 Nov 2017
- Primary completionNov 2019 (estimated)
- Study completionMar 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Pediatric Acute-Onset Neuropsychiatric SyndromeStudies of the same condition, open and under way.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 21 participants (estimated)
Sponsor and investigators
- Lead sponsor
- IMMUNOe Research Centers
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Octapharma
- Responsible party
- Sponsor
- Investigators
- Isaac Melamed, MD, Principal investigator, IMMUNOe Research Centers
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03348618
- Sponsor's protocol ID
- IIS201701-PANS
- Acronym
- PANS
- Status last verified
- Mar 2019
- First submitted
- 14 Nov 2017
- Last updated
- 19 Nov 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Swedo SE, Leckman JF Rose NR. From research subgroup to clinical syndrome: Modifying the PANDAS criteria to describe PANS (Pediatric Acute-onset Neuropsychiatric Syndrome). Pediatr Therapeut 2012, 2:2.
Chang K, Frankovich J, Cooperstock M, Cunningham MW, Latimer ME, Murphy TK, Pasternack M, Thienemann M, Williams K, Walter J, Swedo SE; PANS Collaborative Consortium. Clinical evaluation of youth with pediatric acute-onset neuropsychiatric syndrome (PANS): recommendations from the 2013 PANS Consensus Conference. J Child Adolesc Psychopharmacol. 2015 Feb;25(1):3-13. doi: 10.1089/cap.2014.0084. Epub 2014 Oct 17.
PubMed 25325534Swedo SE, Leonard HL, Garvey M, Mittleman B, Allen AJ, Perlmutter S, Lougee L, Dow S, Zamkoff J, Dubbert BK. Pediatric autoimmune neuropsychiatric disorders associated with streptococcal infections: clinical description of the first 50 cases. Am J Psychiatry. 1998 Feb;155(2):264-71. doi: 10.1176/ajp.155.2.264.
PubMed 9464208Melamed I, Kobayashi RH, O'Connor M, Kobayashi AL, Schechterman A, Heffron M, Canterberry S, Miranda H, Rashid N. Evaluation of Intravenous Immunoglobulin in Pediatric Acute-Onset Neuropsychiatric Syndrome. J Child Adolesc Psychopharmacol. 2021 Mar;31(2):118-128. doi: 10.1089/cap.2020.0100. Epub 2021 Feb 18.
PubMed 33601937
Study at a glance
From the registry record, last updated by the sponsor on 19 Nov 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-site, Open-Label, Pilot Study to Evaluate the Benefit of Octagam 5%® in Subjects With Pediatric Acute-onset Neuropsychiatric Syndrome (PANS)
- ClinicalTrials.gov ID
- NCT03348618
- Acronym / sponsor ID
- PANS / IIS201701-PANS
- Conditions
- Pediatric Acute-Onset Neuropsychiatric Syndrome, Pediatric Autoimmune Neuropsychiatric Disorders Associated With Streptococcal Infections
- Keywords
- Obsessive-Compulsive Disorder, Anxiety, PANS, PANDAS, Children
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 21 participants (estimated)
- Lead sponsor
- IMMUNOe Research CentersIndustry (pharma, biotech, devices)
- Collaborators
- Octapharma
- Study start
- 24 Nov 2017 (actual)
- Primary completion
- Nov 2019 (estimated)
- Study completion
- Mar 2020 (estimated)
- First posted
- 21 Nov 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will evaluate the use of intravenous immunoglobulins (IVIG) at a dose of 1g/Kg/body weight given every three weeks for 6 infusions in pediatric subjects ages 4 - 16 years with moderate to severe PANS. The study will compare biomarkers and behavioral scales before treatment, after the last infusion, 2 months, and at a minimum 6 months post-treatment.
Detailed description
IVIG at an immunomodulatory dose of 1 g/Kg body weight has been known to induce suppression of systemic inflammation and has been used in the treatment of autoimmune diseases. It has been proven beneficial in inflammatory conditions affecting the nervous system.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| IVIG | Experimental | Biological: IVIGIVIG dose at 1 g/Kg/body weight |
Who may qualify
- Ages
- 4 to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Male and female children, 4-16 years of age
- Diagnosis of moderate to severe PANS based on accepted criteria
- Must be willing to follow study procedures and comply with wash-out period
- If using prophylactic antibiotics, must be on stable dose for 3 months
Key exclusion criteria (5 listed)
- History of rheumatic fever, including Sydenham chorea (the neurologic manifestation)
- Previous IVIG therapy within the last 6 months
- Allergic reactions to blood products
- Patients who, in the investigator's opinion, might not be suitable for the trial.
- Steroid use
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 |
|---|---|---|
| IVIGAlso: Octagam 5%Intravenous immunoglobulin | Biological | IVIG |
Primary outcome
Change in Anxiety Disorders Interview Schedule for DSM-IV, Child, Parent Versions (ADIS)
Time frame: Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
ADIS Child and Parent Interviews are designed to diagnose children with emotional disorder, where anxiety is a prominent component. Problem behaviors and diagnoses include school refusal behavior, separation anxiety, social phobia, specific phobia, panic disorder, agoraphobia, OCD and PTSD. Assessment of ADHD allows for differentiation of inattentive type, hyperactive-type, and combined type.
Change in Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS)
Time frame: Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
The scale is a clinician-rated, 10-item scale, each item rated from 0 (no symptoms) to 4 (extreme symptoms) (total range, 0 to 40), with separate subtotals for severity of obsessions and compulsions.
Change in Yale Global Tic Severity Scale (YGTSS)
Time frame: Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
The CY-BOCS rating scale is designed to rate the severity and type of symptoms in patients with obsessive compulsive disorder (OCD). Ratings of the characteristics of obsessive or compulsive ideation and actions on a scale of 0 - 4, with 0 as none and 4 as extreme.
Change in Clinical Global Impressions (CGI)
Time frame: Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
CGI is a 3-item observer-rated scale used to measure symptom severity, global improvement, and therapeutic response. Items 1 and 2 are rated on a 7-point scale; item 3 is rated from 0 to 4
Change in biomarkers levels
Time frame: Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)
Neuro-immune biomarkers
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Nov 2019.
- BackgroundSwedo SE, Leckman JF Rose NR. From research subgroup to clinical syndrome: Modifying the PANDAS criteria to describe PANS (Pediatric Acute-onset Neuropsychiatric Syndrome). Pediatr Therapeut 2012, 2:2.
- Background: PubMed 25325534Chang K, Frankovich J, Cooperstock M, Cunningham MW, Latimer ME, Murphy TK, Pasternack M, Thienemann M, Williams K, Walter J, Swedo SE; PANS Collaborative Consortium. Clinical evaluation of youth with pediatric acute-onset neuropsychiatric syndrome (PANS): recommendations from the 2013 PANS Consensus Conference. J Child Adolesc Psychopharmacol. 2015 Feb;25(1):3-13. doi: 10.1089/cap.2014.0084. Epub 2014 Oct 17.
- Background: PubMed 9464208Swedo SE, Leonard HL, Garvey M, Mittleman B, Allen AJ, Perlmutter S, Lougee L, Dow S, Zamkoff J, Dubbert BK. Pediatric autoimmune neuropsychiatric disorders associated with streptococcal infections: clinical description of the first 50 cases. Am J Psychiatry. 1998 Feb;155(2):264-71. doi: 10.1176/ajp.155.2.264.
- Derived from this study: PubMed 33601937Melamed I, Kobayashi RH, O'Connor M, Kobayashi AL, Schechterman A, Heffron M, Canterberry S, Miranda H, Rashid N. Evaluation of Intravenous Immunoglobulin in Pediatric Acute-Onset Neuropsychiatric Syndrome. J Child Adolesc Psychopharmacol. 2021 Mar;31(2):118-128. doi: 10.1089/cap.2020.0100. Epub 2021 Feb 18.
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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.
Isaac Melamed, MD principal investigator
IMMUNOe Research Centers
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing IVIG in people with Pediatric Acute-Onset Neuropsychiatric Syndrome and Pediatric Autoimmune Neuropsychiatric Disorders Associated With Streptococcal Infections, run by IMMUNOe Research Centers at 3 sites in United States.
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 4 inclusion and 5 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: status not confirmed, as of the sponsor's last update on 19 Nov 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. 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. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
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?
Change in Anxiety Disorders Interview Schedule for DSM-IV, Child, Parent Versions (ADIS) (time frame: Measurements at baseline, one week post treatment (19 weeks), and 6 + months post-treatment (43 + weeks)). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03348618, which also lists 4 linked publications.
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 19 Nov 2019.