Angelman Syndrome clinical trials
The registry lists 52 studies for Angelman Syndrome across more than 10 countries. 16 are recruiting or about to start. 42 of them are mainly about Angelman Syndrome (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-06.
Get an email when these change. Save the Angelman Syndrome studies recruiting or about to start — once a week, free: studies that open, close, add sites or post results.
See an example email
Example · made-up studies
Breast cancer · Recruiting: 2 came in, 3 changed
What changed in your saved searches, Oct 31 – Nov 6:
A study of drug A after surgery for early breast cancer
Came in · now Recruiting
Drug B with hormone therapy in advanced breast cancer
Changed
· Sites: 4 added, including Seoul and Busan
· Enrollment: 300 → 450 participants
Exercise during chemotherapy
Now Active, not recruiting
One email a week for all your saved searches. Stop any time.
Who runs them
Ultragenyx Pharmaceutical Inc (4) · Ionis Pharmaceuticals, Inc. (3) · Hoffmann-La Roche (3) · Boston Children's Hospital (3) · Neurim Pharmaceuticals Ltd. (2) · IRCCS Eugenio Medea (2) · Assistance Publique - Hôpitaux de Paris (2) · Puerta de Hierro University Hospital (2)
What is being tested here
Drugs under test in Angelman Syndrome studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| Obudanersen | 1 | 2 | 3 |
| Apazunersen sodium | 0 | 2 | 4 |
| Gaboxadol | 0 | 1 | 3 |
| Levodopa | 0 | 1 | 3 |
| Minocycline | 0 | 0 | 2 |
| Alogabat | 0 | 0 | 1 |
| Ercanetide | 0 | 0 | 1 |
| Carbidopa | 0 | 0 | 1 |
| Creatine | 0 | 0 | 1 |
| Levomefolate calcium | 0 | 0 | 1 |
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 4, phase 3 first
- Aug 2027REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome · Phase 3 · Ionis Pharmaceuticals, Inc.
- Nov 2026Early Check: Expanded Screening in Newborns · RTI International
- Dec 2026Web Intervention for Parents of Youth With Genetic Syndromes (WINGS) · Rush University Medical Center
- Feb 2027Use of Eye Tracking to Study Social Perception Abnormalities in Children With Angelman Syndrome · Assistance Publique - Hôpitaux de Paris
Who these studies take
Of the 14 Angelman Syndrome studies recruiting now, 6 (43%) set no upper age limit and 7 (50%) stop at 75 or younger. 14 (100%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 0 (0%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 14 (100%) | 14 (100%) |
| 18 | 12 (86%) | 12 (86%) |
| 30 | 12 (86%) | 12 (86%) |
| 40 | 12 (86%) | 12 (86%) |
| 50 | 12 (86%) | 12 (86%) |
| 65 | 8 (57%) | 8 (57%) |
| 75 | 8 (57%) | 8 (57%) |
| 85 | 7 (50%) | 7 (50%) |
Across all 52 studies, recruiting or not: 73% stop at 75 or younger and 71% at 65; 94% take children; 4% are for women only and 2% for men only; 15% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
What changed recently
From the registry's own updates since Oct 14, 2026.
Stopped3
- Long-term Extension of GTX-102 in Angelman SyndromeTerminatedNov 3, 2026NCT06415344
- Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)TerminatedNov 3, 2026NCT06617429
- A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)TerminatedNov 3, 2026NCT07157254
Studies
Recruiting first, then most recently updated. Showing 50 of 52. Browse all 52 → or search them.
- BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome.RecruitingNCT07605429Phase 33 countries
- CHAMPION: A Study to Evaluate the Efficacy and Safety of Obudanersen (ION582) in Children and Adults With Angelman Syndrome (AS)RecruitingNCT07782827Phase 31 country
- A Natural History Study of Angelman SyndromeRecruitingNCT074171371 country
- The Global Angelman Syndrome RegistryRecruitingNCT052931841 country
- A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman SyndromeRecruitingNCT07181837Phase 1/21 country
- Parent and Infant Inter(X)Action Intervention (PIXI)Enrolling by invitationNCT03836300Not applicable1 country
- Use of Eye Tracking to Study Social Perception Abnormalities in Children With Angelman SyndromeRecruitingNCT067377181 country
- IDMet (RaDiCo Cohort) (RaDiCo-IDMet)RecruitingNCT059455761 country
- Expanded Access/Compassionate Use of Rugonersen in Patients With Angelman SyndromeAvailableNCT07136454
- Natural History Study for Patients With Angelman SyndromeRecruitingNCT062297691 country
- Angelman Syndrome Natural History StudyRecruitingNCT045079972 countries
- Study of the Prevalence of Autistic Traits in Angelman SyndromeRecruitingNCT063373831 country
- Structural-functional Connectome in Drug-resistant Epilepsies and Neurodevelopmental Syndromes With EpilepsyRecruitingNCT063536201 country
- Angelman Natural History Study - FAST SpainRecruitingNCT061151091 country
- Angelman Syndrome Natural History Study-FAST UKRecruitingNCT051008101 country
- Ghrelin in Patients With a Rare Disease Associated With Intellectual Disability, and Hyperphagia, and/or Overweight, and/or ObesityRecruitingNCT047688031 country
- Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)TerminatedNCT06617429Phase 36 countries
- Long-term Extension of GTX-102 in Angelman SyndromeTerminatedNCT06415344Phase 38 countries
- A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)TerminatedNCT07157254Phase 28 countries
- REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman SyndromeActive, not recruitingNCT06914609Phase 312 countries
- Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)Active, not recruitingNCT06139172Not applicable1 country
- HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman SyndromeActive, not recruitingNCT05127226Phase 1/26 countries
- Early Check: Expanded Screening in NewbornsActive, not recruitingNCT036552231 country
- Italian Angelman Syndrome RegistryCompletedNCT036505691 country
- A Study of the Safety and Tolerability of GTX-102 in Children With Angelman SyndromeCompletedNCT04259281Phase 1/28 countries
- A Study to Investigate the Pharmacokinetics (PK) and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype.CompletedNCT05630066Phase 26 countries
- A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS)CompletedNCT04428281Phase 14 countries
- A Study in Adults and Adolescents With Angelman Syndrome (STARS)CompletedNCT02996305Phase 22 countries
- An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman SyndromeTerminatedNCT03882918Phase 32 countries
- An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman SyndromeCompletedNCT05011851Phase 21 country
- Efficacy and Safety of Circadin® in the Treatment of Sleep Disturbances in Children With Neurodevelopment DisabilitiesCompletedNCT01906866Phase 35 countries
- A Study of OV101 in Individuals With Angelman Syndrome (AS)CompletedNCT04106557Phase 35 countries
- Angelman Syndrome Video Assessment (ASVA) Source Material StudyCompletedNCT056376971 country
- Development of a Newborn Screening Assay for Angelman Syndrome and Prader-Willi SyndromeCompletedNCT057837911 country
- Minocycline in the Treatment of Angelman SyndromeCompletedNCT01531582Not applicable1 country
- A Study To Assess Distribution Of RO7248824 In The Central Nervous System Following Single Intrathecal Doses Of [89zr] Labeled RO7248824 In Healthy Male ParticipantsCompletedNCT04863794Phase 11 country
- Angelman Syndrome (AS) Biomarker StudyCompletedNCT04103333Early Phase 11 country
- An Exploratory Open Label Study of EPI-743 (Vincerinone TM) in Children With Autism Spectrum DisorderWithdrawnNCT02226458Phase 2
- Study on the Brain Network of Angelman SyndromeCompletedNCT033588231 country
- Characterization of Angelman SyndromeCompletedNCT002967641 country
- SNP-based Microdeletion and Aneuploidy RegisTry (SMART)CompletedNCT023814576 countries
- Nutritional Formulation for Angelman SyndromeCompletedNCT03644693Not applicable1 country
- A Trial of Levodopa in Angelman SyndromeCompletedNCT01281475Phase 2/31 country
- Clinical Trial of Levodopa/Carbidopa ( Sinemet) Therapy in Angel Man SyndromeCompletedNCT03235037Not applicable
- Single Dose Pharmacokinetic (PK) StudyCompletedNCT03109756Phase 11 country
- Study on Tolerability of Levodopa/Carbidopa in Children With Angelman SyndromeCompletedNCT00829439Phase 11 country
- Sleep Abnormalities in Rare Genetic Disorders: AS, RTT, and PWCompletedNCT026706941 country
- Study to Evaluate the Efficacy and Safety of Minocycline in Angelman SyndromeCompletedNCT02056665Phase 21 country
- Genomic Imprinting and Assisted Reproductive TechnologiesCompletedNCT007738251 country
- Assessment of the Pharmacokinetics of Circadin® in Children With Neurodevelopmental Disorders and Sleep DisturbancesCompletedNCT01903681Phase 11 country