Rare Disease Research, LLC | Atlanta, GA
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About
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).
Full description
This is a Phase 1/2, open-label, multiple-dose, study to evaluate the safety, tolerability, and plasma and CSF concentrations of GTX-102 in pediatric participants with AS.
The study includes a Loading phase followed by a Maintenance phase. Participants may continue on GTX-102 during the Maintenance phase of the study until GTX-102 is commercially available, intolerable toxicity occurs, the parent/legal guardian withdraws consent, the participant enrolls in another experimental study, or this study is terminated.
This study was previously posted by GeneTX Biotherapeutics, LLC and was transferred to Ultragenyx in July 2022.
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74 participants in 13 patient groups
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Central trial contact
HCPs Contact: Medical Information; Patients Contact: Trial Recruitment
Data sourced from clinicaltrials.gov
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