Angelman Syndrome clinical trials at University of California Health

4 in progress, 2 open to eligible people

Showing trials for

  • GTX-102 in Pediatric Subjects with AS

    open to eligible people ages 4-17

    The primary objective of this study is to evaluate the effect of GTX-102 in cognitive function in participants with Angelman Syndrome (AS).

    at UCSD UCSF

  • Angelman Syndrome Natural History Study

    open to all eligible people

    The goal of this study is to conduct a prospective, longitudinal natural history study of children and adults with Angelman Syndrome using investigator-observed and parent-reported outcome measures to obtain data that will be useful for future…

    at UCSD

  • (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS)

    Sorry, in progress, not accepting new patients

    This is a phase I, multicenter, non-randomized, adaptive, open-label, multiple ascending, intra-participant, dose-escalation study with a long-term extension (LTE) part and an optional open-label extension (OOE) part. The objective of the study is…

    at UCLA

  • Long-term Extension of GTX-102 in Angelman Syndrome

    Sorry, accepting new patients by invitation only

    The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)

    at UCLA UCSD

Our lead scientists for Angelman Syndrome research studies include .

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