Paul Harmatz, MD, on Challenges With Assessing Neurocognitive Outcomes


The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed challenges with clinical trials in neurocognitive diseases including MPS2.

“You have to make [a trial] short enough that that person comes in healthy, stays healthy for 6 months, and leaves. That's why having a 2-year trial for neurocognitive functional outcome is a very big challenge. People in the muscular dystrophy field face that same challenge. You want to move in, show a benefit, and quickly move out before some treatment-unrelated but disease related component really makes any effect hard to interpret.”

RGX-121 gene therapy (REGENXBIO), delivered via image-guided injection to the cisterna magna, yielded dose-dependent reductions in D2S6 and other cerebrospinal fluid glycosaminoglycans in patients with neuronopathic mucopolysaccharidosis type 2 (MPS2). The updated data, from the phase 1/2 CAMPSIITE trial (NCT03566043), were presented by Paul Harmatz, MD, pediatric gastroenterologist and professor in residence, University of California at San Francisco, at the American Society of Gene and Cell Therapy (ASGCT) 2023 Annual Meeting, held May 16-20, in Los Angeles, California.

CGTLive spoke with Harmatz to learn more about the trial and the challenges with conducting such trials in the MPS2 population. He outlined some of these challenges, including the heterogeneity of disease courses in patients with MPS2. Specifically, he talked about challenges with assessing neurocognitive functional outcomes and how disease factors may affect these evaluations, in patients with MPS2 as well as neurological diseases including muscular dystrophies. The phase 3 portion of CAMPSIITE is now enrolling patients to evaluate efficacy of RGX-121.

Harmatz P, Ficicioglu C, Giugliani R, et al. RGX-121 gene therapy for the treatment of neuronopathic mucopolysaccharidosis type II (MPS II): Interim analysis of data from the first in human study. Presented at: ASGCT 2023 Annual Meeting; May 16-20; Los Angeles, California. Poster #807
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