Mallinckrodt confirmed screening of the first patient in the company’s phase 4, multicenter, multiple-dose, open label study to assess the effects of H.P. Acthar Gel as a therapy option in patients with severe noninfectious intermediate uveitis, posterior uveitis, or panuveitis (NIPPU).
H.P. Acthar Gel is approved by the FDA for the treatment of severe acute and chronic allergic and inflammatory processes involving the eye and its adnexa – such as keratitis, iritis, iridocyclitis, diffuse posterior uveitis and choroiditis, optic neuritis, chorioretinitis, anterior segment inflammation – one of 19 indications.
“I am pleased that the first patient has been screened in this important phase 4 study, the results of which may potentially provide further evidence to support Acthar as a treatment option in appropriate uveitis patients.” C. Stephen Foster, MD, Founder and President, Massachusetts Eye Research and Surgery Institution, Professor of Ophthalmology, Harvard Medical School, Founder of the Ocular Immunology and Uveitis Foundation, Waltham, Mass, said in a company news release. “In my experience, a considerable number of uveitis patients can have persistent disease that may not be resolved by first-line treatment.”
“The screening of the first patient in this study is an important milestone in enhancing our understanding of Acthar’s effectiveness and potential utility as a treatment option for certain uveitis patients,” Tunde Otulana, MD, Chief Medical Officer at Mallinckrodt, said in the news release. “We believe this exploratory evaluation of patients with severe uveitis – despite being on one or more standard treatments – can potentially help physicians better understand which individuals may benefit from the drug as a treatment alternative.”
About the Trial
The phase 4 clinical study is titled “Efficacy and Safety of H.P. Acthar Gel in Subjects With Severe Noninfectious Intermediate Uveitis, Posterior Uveitis, or Panuveitis (NIPPU).” This is a phase 4, multicenter, multiple dose, open label study to examine the effects of Acthar in adult subjects, with a target enrollment of 30. Subjects with current severe NIPPU who meet entry criteria will be treated with H.P. Acthar Gel 1 mL (80 units [U]) subcutaneously (SC) 2 times per week for 36 weeks. Initial treatment will be followed by a taper to Acthar 1 mL (80 U) SC once a week for two weeks, then 0.5 mL (40 U) SC once a week for 2 weeks. All subjects will have a follow-up contact 28 (± 14) days after their last dose of study drug.
Response will be evaluated by vitreous haze (time frame: from baseline to 36 weeks) for the proportion of subjects with a 2-step or more reduction from baseline (or achievement of Grade 0) in vitreous haze at 36 weeks. Full study details can be found on clinicaltrials.gov.
Vitreous haze is the obscuration of fundus details by vitreous cells and protein exudation. It impacts vision more profoundly than anterior inflammation and therefore is used commonly as an outcome measure for clinical trials involving intermediate, posterior, or panuveitis.