Recruitment is still open for the Aldebaran study, sponsored by Roche, to assess an investigational drug (called Alogabat) to potentially treat Angelman syndrome. While it does not involve replacing or unsilencing the UBE3Agene in individuals living with AS, it’s a mechanism aimed to help improve various symptoms of AS such as learning, sleep, and seizure control.
The study is looking for approximately 56 children and adolescents diagnosed with (genetically confirmed) deletion AS to participate in this study. Each child must be between the ages of 5 and 17 years old at the time they enter the study.
Participation will last for up to approximately 24 weeks in total, including screening, treatment, and follow-up periods. The treatment period will last for 12 weeks. The study consists of two consecutive parts – Part 1 and Part 2. Both parts will assess how safe and well-tolerated the investigational drug is, as well as how the body processes the drug.
Data from Part 1 will be used to determine which dose is used for each age group in Part 2. In addition, Part 2 will focus on assessing the investigational drug’s impact on brain waves. In both parts, participants will take the investigational drug once a day by mouth (tablet form), with or shortly after food. Tablets can be dispersed or hidden in food.
Please consult your physician if you are interested in participating in the study. Currently sites are open in New York, North Carolina, Illinois, Australia, France, Spain and Italy.