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Priya Life Science Clinical trial register
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Trackers› Clinical trials› Angelman Syndrome
Clinical trial register · United States · industry-sponsored

Angelman Syndrome Trials in the USA

Every industry-sponsored study on the ClinicalTrials.gov registry that lists a US site and is open, about to open, active-but-closed, or enrolling by invitation. This deliberately excludes academic and other non-industry sponsors — the full US registry across all sponsors runs to over 22,000 recruiting studies alone.

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Source: ClinicalTrials.gov. Complete coverage of the industry-sponsored slice — all 9094 matching records are in this view (non-industry sponsors are out of scope by design, not truncated). Synced 17 Sep 2026, 08:23 UTC
9094
Studies in scope
industry-sponsored, all four open statuses
4935
Recruiting now
open to new participants
2188
Lead sponsors
across recruiting studies
Top states
California 7 Illinois 7 Massachusetts 5 Missouri 3 North Carolina 3
Narrow the registerReset
Recruitment status
Phase
Lead sponsor
Ultragenyx Pharmaceutical Inc3 Ionis Pharmaceuticals, Inc.2 MavriX Bio, LLC1 OHB Pediatrics Ltd.1
State
California7 Illinois7 Massachusetts5 Missouri3 North Carolina3
All 13 states
Target enrolment
to
7 of 9094 studies
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Status
Study
Lead sponsor
Phase
Enrol
US states
Start
Recruiting
BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome.
NCT07605429Angelman Syndrome
OHB Pediatrics Ltd.
Phase 3
165
California, Illinois, North Carolina
2026-06-23
Recruiting
A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome
NCT07181837Angelman Syndrome
MavriX Bio, LLC
Phase 1/2
12
California, Illinois, Massachusetts
2025-10-29
Active, closed
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
NCT07157254Angelman Syndrome
Ultragenyx Pharmaceutical Inc
Phase 2
60
California, Illinois, Maryland, Missouri, North Carolina, Ohio, Texas
2025-10-13
Recruiting
REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome
NCT06914609Angelman Syndrome
Ionis Pharmaceuticals, Inc.
Phase 3
158
California, Colorado, District of Columbia, Florida, Georgia, Illinois, Massachusetts, Missouri
2025-06-10
Active, closed
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
NCT06617429Angelman Syndrome
Ultragenyx Pharmaceutical Inc
Phase 3
129
California, Colorado, Florida, Georgia, Illinois, Massachusetts, Missouri, New York
2024-12-03
Active, closed
Long-term Extension of GTX-102 in Angelman Syndrome
NCT06415344Angelman Syndrome
Ultragenyx Pharmaceutical Inc
Phase 3
255
California, Georgia, Illinois, Massachusetts, New York
2024-07-31
Active, closed
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
NCT05127226Angelman Syndrome
Ionis Pharmaceuticals, Inc.
Phase 1/2
71
California, Colorado, Illinois, Massachusetts, North Carolina, Texas
2021-12-22
Rows per page Showing 1–7 of 7
1
Cite this view

Priya Life Science (2026). Clinical trial register: industry-sponsored studies with a US investigative site, filtered. Derived from ClinicalTrials.gov. Retrieved 18 September 2026. https://priyalifescience.com/clinical-trials-usa/condition/angelman-syndrome

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Listing here is informational and not medical advice; eligibility is determined by each study team. Compare pay for the teams running these studies in the US Pharma Salary Guide, see who's behind them in the US Company Directory, or track approvals on the FDA Approvals Tracker. For Irish, UK and EU sites, see the Ireland, UK and EU registers.