Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Angelman Syndrome: early-phase trial registry
6 of 12 indexed Angelman Syndrome trials are currently recruiting, with more weight in Phase 1-2.
Corpus placement: #1,429 of 28,707 conditions by registered trial count.
12 US clinical trials · 6 currently recruiting · #1,429 of 28,707 conditions by registered trial count
Angelman Syndrome: 12 US clinical trials tracked, 6 recruiting.
Angelman Syndrome is the subject of 12 registered US clinical trials on ClinicalTrials.gov, 6 of them currently open to new participants. 2 are in Phase 3-4 (later-stage) and 5 in Phase 1-2 (earlier-stage). The most active sponsor is Ultragenyx Pharmaceutical, running 2 of these trials.
Enrollment posture brief
Registry condition angelman-syndrome
OPEN 6 | RECR 50% | LATE 2 | EARLY 5 | RANK #1429 | SPON 2
Volume-matched condition peers
Nearest open-share peer: Abdominal Aortic Aneurysms (50% recruiting · 6 trials)
Angelman Syndrome lists 6 open slots (50% of 12 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →
Key findings
What ClinicalTrials.gov does not surface for Angelman Syndrome on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.
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Angelman Syndrome is recruiting above its size-band peers.
6 of 12 indexed trials (50%) are open to enrollment, against a 46.5% average across 27,806 conditions with fewer than 20 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.
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One sponsor concentrates Angelman Syndrome research.
Ultragenyx Pharmaceutical accounts for 2 of 12 trials (16.7%). 9 distinct sponsors appear on at least one study for this condition label.
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Most Angelman Syndrome trials plan to enrol fewer than 100 people.
Median target enrolment is 90 among the 12 trials that report a genuine target (sentinel values excluded). 8 of those (66.7%) plan for fewer than 100 participants.
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Angelman Syndrome research is mostly interventional.
10 interventional and 2 observational studies make up this index (83.3% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and recent trials
12 total, page 1 of 1
Angelman Syndrome Natural History Study
Boston Children's Hospital
NCT04507997
REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome
Ionis Pharmaceuticals
NCT06914609
Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)
Rush University Medical Center
NCT06139172
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
Ionis Pharmaceuticals
NCT05127226
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
Ultragenyx Pharmaceutical
NCT07157254
A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome
MavriX Bio
NCT07181837
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
Ultragenyx Pharmaceutical
NCT06617429
Characterization of Angelman Syndrome
Boston Children's Hospital
NCT00296764
Dietary Supplements for the Treatment of Angelman Syndrome
University of California, San Diego
NCT00348933
A Study in Adults and Adolescents With Angelman Syndrome (STARS)
Healx AI
NCT02996305
A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS)
Hoffmann-La Roche
NCT04428281
Nutritional Formulation for Angelman Syndrome
University of Colorado, Denver
NCT03644693
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 3 |
| Phase 2 | 2 |
| Phase 3 | 2 |
Top Sponsors
Named interventions
Most-linked intervention names on Angelman Syndrome records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Angelman Syndrome trials. A trial with sites in several states counts in each.
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Related
What to do with this Angelman Syndrome page
12 registered trials, 6 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 6 Angelman Syndrome trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- Ultragenyx Pharmaceutical sponsors the most Angelman Syndrome trials on record, review their full trial history before evaluating a specific study. See Ultragenyx Pharmaceutical's trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Angelman Syndrome condition card totals 12 registered trials; most-linked intervention GTX-102; top US site state California. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.