Octapharma/PACE Foundation IVIG Trial
Octapharma has recently announced that the IVIG Trial had passed the standard safety and administrative checks required by the FDA. The pause in candidate recruiting to perform this pre-scheduled safety check has now ended, and they can resume screening for new candidates.
For recruitment requirements and trial locations, please see the Clinical Trial website. Additional Trial Sites will be opening soon. Currently, Trial Sites are located in Arizona, Arkansas, California, Colorado, Massachusetts, and New Hampshire. All potential trial candidates should contact Octapharma directly (see contact information below) in order to be referred to a designated trial site. Please see attached updated recruitment flyer and read ASPIRE’s Q & A with PACE Team led by Paul Ryan below.
Clinical operations at Octapharma USA, Inc.
In cooperation with Octapharma, the PACE Foundation is pleased to announce that they are now actively recruiting candidates for the National PANS IVIG Trial. Attached below, please find an Octapharma Advocacy Group letter that describes the eligibility criteria for entry into the study, including help with travel expenses related to trial participation. Also attached is an Octapharma Trial Flyer. Because this FDA-sanctioned IVIG clinical study will include a large number of participants, the necessity to recruit trial candidates is urgent, so please share it with other families and providers. For more information, please contact Octapharma directly at firstname.lastname@example.org or 201-604-1124
Read ASPIRE’s Q & A with PACE Team, led by Paul Ryan, providing further information on this critical IVIG trial. Please see below.
Eligibility & Information from The ProPANS study team
Children with Pediatric Acute-Onset Neuropsychiatric Syndrome (PANS) may qualify to participate in the ProPANS IVIG clinical research study sponsored by Octapharma. This clinical research study will be conducted at a select number of medical universities and institutions. The study’s goal is to find out if the investigational study drug, Panzyga, can help children manage inflammation associated with PANS.
All study participants receive access to the IVIG study drug. Study participants visit the study clinic for health assessments about every 3 weeks and the study lasts approximately 6 months.
Children may be eligible to participate if they meet the following requirements:
- 6-17 years in age
- Moderate to severe PANS with prominent and stable obsessive-compulsive disorder symptoms
- Onset of initial PANS symptoms was not more than 6 months prior to planned Screening. Or, for children with relapsing symptoms, the initial symptoms were less than 12 months prior to Screening and recurrence is within 6 months of Screening
- Do not have symptoms consistent with autism, schizophrenia, bipolar disorder, or any other psychiatric disorder Study participants receive at no cost:
Study participants receive at no cost:
- Access to the IVIG study drug (Panzyga) All participants will receive the study drug and placebo (no active ingredients).
- Participants will be randomly assigned to receive the study drug in either the first or second half of the study
- Study support and study-related monitoring by a healthcare team
- Help with study-related travel expenses, including reimbursements for lodging, transportation, and meals may be provided
- The opportunity to help advance PANS research.
For clinical trial information – Please visit ClinicalTrials.Gov (Identifier: NCT04508530). For more information about enrollment in the trial or to find the site nearest you, contact: Snehal Udavat. Clinical operations at Octapharma USA, Inc. 201-604-1124 or email@example.com.
For information about the study: Clinical Trials Website
ASPIRE’s Q & A with PACE Team led by Paul Ryan
How many years did PACE and Octapharma spend getting the study to the recruitment stage?
From the initial conversations about developing a national IVIG Trial, through attaining a Phase 3 FDA trial approval, the launch took roughly three years.
What were some of PACE’s and Octapharma’s most significant milestones along the way?
The main steps in creating and approving the trial included multiple and extensive trial protocol discussions, safety meetings with the FDA, complex and substantial conversations with clinical partner universities about budgetary, contract, IRB, etc., and finally planning for physical site preparation and trial recruitment awareness programs. We are pleased that the trial is underway.
How was Octapharma’s Panzyga chosen as the IVIG product for this study?
Following discussions with a number of pharmaceutical companies, Octapharma expressed a strong commitment to helping children with PANS. Octapharma has been a dedicated and collaborative partner to PACE and has taken the lead in getting FDA approval for this Phase 3 IVIG Efficacy Trial.
According to the eligibility requirements, patients must have prominent and stable obsessive-compulsive disorder symptoms. Several families have asked us what you mean by stable? Do you mean consistent symptoms of OCD that occupy at least 50% of the patient’s waking day or something else? Or do you mean they have to be stable enough not to warrant possible hospitalization in an OCD clinic or similar?
There are really two questions here. The term “prominent and stable OCD symptoms” are medical definitions, which are laid out in Clinical Trials NCT04508530.
The second question, including inquiries about consistent “OCD” and the stability of the patient, should be directed to the Trial Clinicians by parents when the family is assigned a Trial Clinic. The central number to enter the trial and be assigned to a trial clinic is 1-201-604-1124.
Where is the study clinic located? Is it just at your Arizona CPAE clinic or at additional CPAE and PANS Clinics in your network? (https://pacefoundation4kids.org/clinics)
Access to the IVIG Trial will be available through an Octapharma based central recruitment number at 1-201-604-1124. The clinical trial sites that are activated to date are the University of Arizona CPAE Center and the University of Arkansas. Additionally, a number of PANS clinics throughout the country will also be activated for full trial participation over the next few months. Interested parties should contact the central Octapharma trial registration number (1-201-604-1124) immediately for further information and expedited assignment to a trial clinic.
Why does the study exclude those with PANS with comorbid psychiatric disorders like autism?
Admissions requirements are very stringent in order to achieve accurate results. Given PANS can present with Autism-like symptoms, there could be questions around whether or not the treatment is working if the child has been diagnosed with additional co-morbidities.
Roughly how many participants would you like to see enrolled?
When does enrollment close?
Enrollment will remain open until all required slots have been filled.
Is it a double-blind placebo study? Why was it designed like that?
Per the published clinical trial guidelines NCT04508530 regarding quality assurance, the trial is placebo-controlled with quadruple masking/blinding (e.g., Participant, Care Provider, Investigator, Outcomes Assessor).