GeneTx & Ultragenyx Resume Angelman Syndrome Trial in U.S., U.K., & Canada
GeneTx Biotherapeutics and Ultragenyx Pharmaceutical have resumed a Phase 1/2 study of GTX-102 in the U.K., Canada, and now the U.S., after a temporary halt due to reversible muscle weakness in patients. The study, known as KIK-AS, aims to treat Angelman syndrome in children and adolescents.
The study was paused late last year when five participants experienced reversible muscle weakness following the highest dose of GTX-102. The FDA has since lifted the clinical hold, allowing the trial to resume with a modified protocol. In the U.S., the study will begin with an eight-patient dose-loading phase, aged 4 to 7 and treatment-naïve, followed by a maintenance phase. The U.K. and Canada studies will also proceed with about 12 patients split into two age groups, with a maintenance phase if safety is maintained.
GTX-102 is an antisense oligonucleotide designed to block the UBE3A antisense transcript, potentially reactivating the silent paternal copy of UBE3A in the brain. This could provide a therapeutic benefit for individuals with Angelman syndrome.
The resumption of the KIK-AS study in the U.S., following its restart in the U.K. and Canada, brings renewed hope for a potential treatment of Angelman syndrome. The modified protocol aims to ensure the safety of participants while advancing the research. Further details about the study's progress are expected in the coming months.
