INTERVENTIONALPROTOCOL REGISTERED2021-003009-23
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION582 in Patients with Angelman Syndrome
Angelman syndrome is a neurogenetic disorder caused by a loss of function of the maternally inherited UBE3A gene (chromosome 15) which codes for UBE3A ubiquitin protein ligase. AS presents early in...
Locally preserved from EU Clinical Trials Register (EudraCT)Open EU Clinical Trials Register (EudraCT)↗last source update not reported
What this record can show
Protocol only - no registry results posted
This locally preserved record separates the registered protocol from source-reported registry results. Neither is a treatment recommendation.
Registry facts
Study at a glance
- Phase
- Not reported
- Enrollment
- Not reported
- Start date
- 2022-07-19
- Sponsor
- Ionis Pharmaceuticals, Inc.
- Design
- Not reported
- Locations
- 0
- Results record
- Not present in registry snapshot
Angelman syndrome is a neurogenetic disorder caused by a loss of function of the maternally inherited UBE3A gene (chromosome 15) which codes for UBE3A ubiquitin protein ligase. AS presents early in...Angelman syndrome is a neurogenetic disorder caused by a loss of function of the maternally inherited UBE3A gene (chromosome 15) which codes for UBE3A ubiquitin protein ligase. AS presents early in...
Linked local records
Related local records
Local links use governed condition terms or exact source-reported intervention names. They are navigation candidates, not efficacy claims.
Conditions
No published condition report matched.
Cannabinoids and active compounds
No governed compound link was found.
Medicines
No exact medicine-name link was found.
Interventions
What was registered
No structured intervention was reported.
Eligibility
Population and criteria
- Sex
- Not reported
- Minimum age
- Not reported
- Maximum age
- Not reported
- Healthy volunteers
- Not reported
No structured eligibility criteria were reported.
Publications
Locally readable linked articles
No exact PMID-linked public article is currently readable locally.
Snapshot provenance
- Snapshot
- 250af192-de5d-484c-92e4-b4221986d1e4
- Retrieved
- 14/09/2026, 12:10:31
- SHA-256
- 8b24648e8192c87e9791b227cc353ad373d29ba49c2169d052d75e40c2f75205