About Clinical Trials
Clinical trials test the safety and effectiveness of a therapy or drug. After drugs or procedures are tested in the lab and in animals, the most promising treatments are moved into clinical trials to find out how well they work in humans.
The development, research and testing of a drug or treatment can take many years before getting to the clinical trial stage.
There are three phases of clinical trials. If these phases are completed and the safety and effectiveness of a drug or treatment is proven, the drug or treatment can be available to the public.
Clinical Trials in Angelman Syndrome
Below you will find information about active or upcoming clinical trials in Angelman syndrome. Please see the participant age and locations column to determine which trials may be most appropriate for your family.
Halos
Sponsor
Ionis Pharmaceuticals
Phase
1/2
Participant Age
2 – 50 years
Locations
US, Australia, Israel, Italy, UK
KIK-AS
Sponsor
Ultragenyx
Phase
1/2
Participant Age
4 – 17 years
Locations
US, Canada, UK
Tangelo
Sponsor
Hoffman La Roche
Phase
1
Participant Age
1 – 12 years
Locations
US, Italy, Netherlands, Spain
Angelman Syndrome Study
Sponsor
Neuren Pharmaceuticals
Phase
2
Participant Age
3 – 17 years
Locations
Australia
Upcoming Clinical Trials
Alogabat
This study is not yet recruiting.
Alogabat is a GABA-modulator, taken as a once-daily oral tablet, is a small molecule positive allosteric modulator of the GABA a5 receptors. Individuals with deletion subtype Angelman syndrome have 50 percent fewer of these receptors expressed in the brain, which may contribute to some symptoms. Alogabat may restore GABA functioning in AS individuals with deletion genotype and may improve some of the symptoms and potentially aid brain development. This has the potential to elicit a benefit in deletion patients independent of and in addition to the benefit expected from treatments aimed purely at restoring UBE3A function.
Sponsor
Roche/Genentech
Phase
2
Participant
5 – 17 years, Deletion genotype