- recruitingPHASE1, PHASE2
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
This study, called HALOS, is looking at a new medicine called ION582 for Angelman syndrome. It aims to see if different doses are safe, how the body handles the medicine, and how it affects the condition. It includes children and adults up to 50 years old.
United States · Australia · France - recruitingPHASE2
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
This study is looking at a new treatment called GTX-102 for Angelman Syndrome. It aims to see if the treatment is safe and if it helps improve the condition in people of different ages and genetic types. Some people will get the treatment, and some won't at first.
United States · Argentina · Brazil - recruitingPHASE3
REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome
This study is testing a new medicine, ION582, for Angelman syndrome. It wants to see if the medicine is safe and helps children and adults with the condition, which affects development and causes seizures.
United States · Australia · Canada - active not recruitingPHASE3
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
This study is testing a new medicine called GTX-102 for children with Angelman Syndrome. It will look at how well the medicine helps with thinking skills. Children aged 4 to 17 with a specific type of Angelman Syndrome can take part.
United States · Canada · Germany - enrolling by invitationPHASE3
Long-term Extension of GTX-102 in Angelman Syndrome
This study is looking into the long-term safety and effects of a treatment called GTX-102 for people with Angelman Syndrome. It's for those who have already been in an earlier study with this treatment.
United States · Australia · Canada