Phase 1 study
Testing the safety and tolerability of RO7248824, administered via IT injection.
RO7248824 is an antisense oligonucleotides (ASO) which aims to unsilence UBE3A production.
If you were participating in the trial you can expect to be contacted by the PI who can share more information and support.
Below is a preliminary list of inclusion criteria.
See the full list of eligibility criteria on the clinical trials website.
- Diagnosis of Angelman syndrome
- Between 1 and 12 years old
- Stable medical status for 4 weeks prior to screening visit
- Ability to tolerate blood draws
- Able to undergo LP and IT injection, under sedation or anesthesia 3 times during an 8-week period
Locations & Contacts
Los Angeles, CA
UCLA Neuropsychiatric Institute
San Diego, CA
Rady Children’s Hospital – San Diego
Lynne Bird, MD firstname.lastname@example.org
Rush University Medical Center
Kathryn Kudlacz email@example.com
Carrboro, North Carolina
University of North Carolina
Hannah Riehl firstname.lastname@example.org
Baylor College of Medicine/ Texas Children’s Hospital
Beverly Feldman email@example.com
New York, NY
Columbia University Irving Medical Center
Jennifer Bain, MD firstname.lastname@example.org
Roma, Lazio, Italy
Ospedale Pediatrico Bambino Gesù; Dip. Neuroscienze e Neuroriabilitazione
Erasmus MC / location Sophia Kinderziekenhuis
Hospital Sant Joan De Deu
Esplugues De Llobregas
Sabadell, Barcelona, Spain
Corporacio Sanitaria Parc Tauli
Hospital Universitario Virgen del Rocío