Angelman Syndrome Clinical Trials

14 recruiting

Angelman Syndrome Trials at a Glance

14 actively recruiting trials for angelman syndrome are listed on ClinicalTrialsFinder across 6 cities in 17 countries. The largest study group is Phase 2 with 3 trials, with the heaviest enrollment activity in Chicago, Los Angeles, and Paris. Lead sponsors running angelman syndrome studies include IRCCS Eugenio Medea, Ionis Pharmaceuticals, Inc., and Centre Hospitalier Universitaire de Liege.

Browse angelman syndrome trials by phase

Treatments under study

About Angelman Syndrome Clinical Trials

Looking for clinical trials for Angelman Syndrome? There are currently 14 studies actively recruiting participants. Clinical trials offer access to new treatments before they are widely available, and every approved therapy in use today was first tested through a clinical trial.

Below you can browse trials, sign up for alerts when new Angelman Syndrome trials open, and view eligibility criteria for each study. Each listing includes the study phase, locations, and enrollment details.

Frequently Asked Questions

Common questions about Angelman Syndrome clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 114 of 14 trials

Recruiting
Phase 3

REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome

Angelman Syndrome
Ionis Pharmaceuticals, Inc.158 enrolled38 locationsNCT06914609
Recruiting
Phase 2

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

Angelman Syndrome
Ultragenyx Pharmaceutical Inc60 enrolled22 locationsNCT07157254
Recruiting

A Natural History Study of Angelman Syndrome

Angelman Syndrome
Massachusetts General Hospital40 enrolled1 locationNCT07417137
Recruiting

Use of Eye Tracking to Study Social Perception Abnormalities in Children With Angelman Syndrome

Angelman Syndrome
Assistance Publique - Hôpitaux de Paris60 enrolled1 locationNCT06737718
Recruiting
Phase 1Phase 2

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

Angelman Syndrome
MavriX Bio, LLC12 enrolled3 locationsNCT07181837
Recruiting

IDMet (RaDiCo Cohort) (RaDiCo-IDMet)

Beckwith-Wiedemann SyndromePrader-Willi SyndromePseudohypoparathyroidism+6 more
Institut National de la Santé Et de la Recherche Médicale, France2,000 enrolled20 locationsNCT05945576
Recruiting
Not Applicable

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

Tuberous SclerosisFragile X SyndromeRett Syndrome+5 more
Rush University Medical Center92 enrolled1 locationNCT06139172
Recruiting
Phase 1Phase 2

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

Angelman Syndrome
Ionis Pharmaceuticals, Inc.70 enrolled11 locationsNCT05127226
Recruiting

Natural History Study for Patients With Angelman Syndrome

Angelman Syndrome
Centre Hospitalier Universitaire de Liege30 enrolled1 locationNCT06229769
Recruiting

Angelman Syndrome Natural History Study

Angelman Syndrome
Boston Children's Hospital300 enrolled11 locationsNCT04507997
Recruiting

Study of the Prevalence of Autistic Traits in Angelman Syndrome

Angelman Syndrome
IRCCS Eugenio Medea46 enrolled1 locationNCT06337383
Recruiting

Structural-functional Connectome in Drug-resistant Epilepsies and Neurodevelopmental Syndromes With Epilepsy

EpilepsyAngelman SyndromeDup15q Syndrome
IRCCS Eugenio Medea120 enrolled1 locationNCT06353620
Recruiting

Angelman Natural History Study - FAST Spain

Angelman Syndrome
Puerta de Hierro University Hospital40 enrolled2 locationsNCT06115109
Recruiting

The Global Angelman Syndrome Registry

Angelman Syndrome
Foundation for Angelman Syndrome Therapeutics, Australia5,000 enrolled1 locationNCT05293184