Octapharma has initiated a Phase III superiority study of Panzyga (immune globulin intravenous, human – ifas) in pediatric acute-onset neuropsychiatric syndrome (PANS) patients.
The multi-centre study is designed to compare the effectiveness of 10% Liquid Preparation of the drug candidate to placebo (0.9% w/v sodium chloride).
As per the National Institutes of Health (NIH), PANS is characterised by a sudden onset of obsessive-compulsive symptoms and/or severe eating restrictions. These conditions are accompanied by at least two other cognitive, behavioural or neurological symptoms.
The new prospective, randomised, double-blind, parallel group, placebo-controlled study is designed to enrol 92 moderate to severe PANS patients aged six to 17 at about 30 sites.
The primary objective is the superiority of Panzyga over placebo in decreasing the severity of symptoms caused by PANS in paediatric patients, said Octapharma.
The secondary objectives include the sustainability of the reduction of the symptoms severity and the drug’s efficacy in mitigating functional impairment related to PANS.
During the study, participants will receive three infusions of the drug or placebo over two days every three weeks for a total of nine weeks.
A double-blind, crossover safety and efficacy follow-up phase will also involve three infusions given over two days every three weeks for nine weeks.
With a two-stage adaptive design, the trial will have one interim analysis, which will be conducted after 40 patients have completed the first nine weeks of treatment.
The sample size will be adjusted based on the interim findings if required.
Study’s lead investigator Michael Daines said: “PANS has been a very challenging disease to treat. The search for safe and effective therapies for PANS has been difficult, but there is strong evidence that immunomodulation can mitigate or cure this disease.
“IVIG has been used in prior studies and in some has shown significant efficacy. This Phase III trial with Panzyga should help define the role of IVIG as an immunomodulatory drug for the management of children with PANS.”
In May this year, Octapharma USA secured regulatory approval to conduct a Phase III trial of Octagam 10% to treat Covid-19 patients with severe disease progression.