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FDA Lifts Hold on Angelman Syndrome Drug Trial: GTX-102 Resumes with New Protocols

After pausing due to muscle weakness, the GTX-102 trial resumes with new protocols. Families of Angelman syndrome patients hope for safer, promising results.

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FDA Lifts Hold on Angelman Syndrome Drug Trial: GTX-102 Resumes with New Protocols

The FDA has lifted the clinical hold on a Phase 1/2 study (NCT04259281) testing GTX-102 in children and adolescents with Angelman syndrome. The study, known as KIK-AS, is now resuming in the U.S., U.K., and Canada with modified protocols. Ultragenyx Pharmaceutical Inc., in collaboration with GeneTx Biotherapeutics, is leading the development of this promising therapy.

Last year, the study was paused due to five participants experiencing reversible muscle weakness after receiving the highest dose of GTX-102. The new protocols address this issue by narrowing the dose range and changing the administration method to intrathecal. In the U.S., eight treatment-naïve patients aged 4 to 7 will be enrolled, with an active treatment group and an age-matched control group. The U.K. and Canada will involve two age groups (4 to 7 and 8 to 17) receiving different doses of GTX-102, followed by a maintenance phase.

GTX-102 is an antisense oligonucleotide designed to block the UBE3A antisense transcript, reactivating the silent paternal copy of UBE3A in the brain. The study aims to assess the safety, tolerability, and preliminary efficacy of multiple doses of GTX-102 in up to 20 participants. GeneTx and Ultragenyx aim to begin treating participants in all three regions over the next several months.

The resumption of the GTX-102 study brings hope to families affected by Angelman syndrome. With the modified protocols, researchers aim to ensure the safety of participants while gathering valuable data on the potential benefits of this novel therapy. The study's results could significantly impact the lives of children and adolescents with Angelman syndrome.

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