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Angelman Syndrome clinical trials at University of California Health

3 in progress, 1 open to eligible people

Showing trials for
  • 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 clinical trials.

    at UCSD

  • GTX-102 in Children With Angelman Syndrome

    Sorry, in progress, not accepting new patients

    The primary objective of the study is to evaluate the safety and tolerability of multiple-ascending doses of GTX-102 administered by intrathecal (IT) injection to participants with Angelman Syndrome (AS).

    at UCLA

  • Pharmacodynamics Of RO7248824 In Participants With Angelman Syndrome

    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 an LTE part. The objective of the study is to investigate the safety, tolerability, PK and PD of RO7248824 in participants administered IT with AS. Two linked sets of dose escalation cohorts are planned based on two different age groups, namely participants with AS aged ≥ 5 to ≤ 12 years in cohorts A1 to A4 (with at least 2 participants ≤ 8 years old in each cohort) and AS participants aged ≥ 1 to ≤ 4 years in cohorts B1 to B5. The two sets of cohorts will be run in parallel, with each cohort A1-A4 preceding and gating the linked cohort B1-B5 (e.g., A1 precedes B1).

    at UCLA

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