Phase 3 study to test efficacy of new treatment for pediatric acute-onset neuropsychiatric syndrome

Octapharma has launched a phase 3, multi-center superiority study to compare the effectiveness of PANZYGA® (immune globulin intravenous, human - ifas) 10% Liquid Preparation versus placebo in patients with pediatric acute-onset neuropsychiatric syndrome (PANS).

PANS is a condition defined by sudden onset of obsessive-compulsive symptoms and/or severe eating restrictions, along with at least two other cognitive, behavioral, or neurological symptoms, according to the National Institutes of Health (NIH).

PANS is a newly defined symptom-based condition that mainly occurs in children and adolescents. Octapharma is proud to sponsor this important research because few studies have described PANS clinical characteristics. We are hopeful that PANZYGA® can make a difference in the lives of children and adolescents impacted by the syndrome."

Flemming Nielsen, Octapharma USA President

Researchers aim to enroll 92 patients from age 6 to 17 with a confirmed diagnosis of moderate to severe PANS. Approximately 30 study sites are planned for the prospec-tive, randomized, double-blind, parallel group, placebo-controlled superiority study. The primary objective of the trial is to evaluate if PANZYGA® is superior to placebo (0.9% w/v sodium chloride) for reducing the severity of symptoms associated with PANS in pediatric patients. The secondary objectives of this study are to determine the sustainability of the reduction of the severity of symptoms in pediatric patients treated with PANZYGA®; and to assess the efficacy of PANZYGA® treatment in reducing functional impairment associated with PANS.

"PANS has been a very challenging disease to treat," said Michael Daines, M.D., Division chief of allergy, immunology and rheumatology at the University of Arizona and the study's lead investigator. "It is very hard on families to see their children afflicted with sudden onset, debilitating, and difficult to treat OCD as well as other cognitive and behavioral issues. These changes impact the ability of children to function in school and at home. The search for safe and effective therapies for PANS has been difficult but there is strong evidence that immunomodulation can mitigate or cure this dis-ease. IVIG has been used in prior studies and in some has shown significant efficacy. This phase 3 trial with PANZYGA® should help define the role of IVIG as an im-munomodulatory drug for the management of children with PANS."

The study will include three infusions of PANZYGA® or placebo administered over two days every three weeks for a total of nine weeks, with an additional double-blind, crossover safety and efficacy follow-up phase of three infusions of PANZYGA® or pla-cebo administered over two days every three weeks for a total of nine weeks. The trial will be conducted in a two-stage adaptive design with one interim analysis. An un-blinded interim analysis will be performed by an independent statistician after 40 pa-tients have completed the first nine-week treatment period, to adjust sample size, if re-quired. The entire study team will remain blinded until the end of the study.

The PACE Foundation is very proud to have been involved with facilitating the launch of this pivotal study in conjunction with Octapharma, our consortium of leading aca-demic research centers including the University of Arizona, Stanford University, UCLA, Harvard University, University of Wisconsin, University of Arkansas and our corporate partner Banner Health. As a leading na-tional non-profit organization dedicated to improving the diagnosis, treatment and quality of life of persons with Pediatric Autoimmune Neurological Disorders, through advocacy, education and research, the PACE Foundation is committed to building public/private partnerships that lead to meaningful outcomes for patients in need."

Paul Ryan, PACE Foundation

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