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ClinicalTrials.gov 6 recruiting now official registry

Angelman Syndrome clinical trials

6 of 12 indexed Angelman Syndrome trials are currently recruiting.

12 US clinical trials · 6 currently 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.

The research picture

Angelman Syndrome has 12 registered US clinical trials, 6 of them open to new participants right now, about 50% of the total.

6
recruiting participants now
50%
of trials open to enrollment
2
in Phase 3–4 (later-stage)
2
top sponsor: Ultragenyx Pharmaceutical
Phase 13Phase 22Phase 32

Active & Recent Trials

12 total, page 1 of 1

RECRUITING 300 participants

Angelman Syndrome Natural History Study

Boston Children's Hospital

NCT04507997

RECRUITING Phase 3 158 participants

REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome

Ionis Pharmaceuticals

NCT06914609

RECRUITING NA 92 participants

Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)

Rush University Medical Center

NCT06139172

RECRUITING Phase 1 70 participants

HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome

Ionis Pharmaceuticals

NCT05127226

RECRUITING Phase 2 60 participants

A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)

Ultragenyx Pharmaceutical

NCT07157254

RECRUITING Phase 1 12 participants

A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome

MavriX Bio

NCT07181837

ACTIVE NOT RECRUITING Phase 3 129 participants

Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)

Ultragenyx Pharmaceutical

NCT06617429

COMPLETED 302 participants

Characterization of Angelman Syndrome

Boston Children's Hospital

NCT00296764

COMPLETED NA 90 participants

Dietary Supplements for the Treatment of Angelman Syndrome

University of California, San Diego

NCT00348933

COMPLETED Phase 2 88 participants

A Study in Adults and Adolescents With Angelman Syndrome (STARS)

Healx AI

NCT02996305

COMPLETED Phase 1 74 participants

A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS)

Hoffmann-La Roche

NCT04428281

COMPLETED NA 15 participants

Nutritional Formulation for Angelman Syndrome

University of Colorado, Denver

NCT03644693

Phase Distribution

PhaseTrial count
Phase 1 3
Phase 2 2
Phase 3 2

Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.

Reading the Angelman Syndrome Trial Landscape

12 indexed Angelman Syndrome studies · 6 recruiting (50%).

Phase mix: 2 late (III/IV) vs 5 early (I/II).

Top sponsor: Ultragenyx Pharmaceutical (2 trials), 8 others listed. 12 shown below, recruiting first.

How to read this condition's trial landscape

That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.

Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.

All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation, eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.

Frequently Asked Questions

How many clinical trials are there for Angelman Syndrome?

PlainTrial tracks 12 US clinical trials for Angelman Syndrome, of which 6 are currently recruiting participants. Data sourced from ClinicalTrials.gov.

How do I find a recruiting trial for Angelman Syndrome?

Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.

Is this data current?

Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.

Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.

Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2026 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.

Verify with NIH →

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page aggregates every registered trial for this condition, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.