A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS)

PHASE1CompletedINTERVENTIONAL
Enrollment

74

Participants

Timeline

Start Date

August 19, 2020

Primary Completion Date

July 31, 2025

Study Completion Date

July 31, 2025

Conditions
Angelman Syndrome
Interventions
DRUG

RO7248824

In the MAD part, RO7248824 will be administered as IT injection of varing dose levels over a period of 8 weeks, with a minimum of approximately 4 weeks between each dose administration. In the LTE part, RO7248824 will be administered as IT injection of varying dose levels over a period of 144 weeks, with a minimum of approximately 16 weeks between each dose administration. In the OOE part, participants will receive RO7248824 as an IT injection with the same dosing regime as the LTE part over a period of up to 48 weeks.

Trial Locations (12)

10032

Columbia University Medical Center, New York

27510

Carolina Institute for Development DisabilitiesUniversity of North Carolina/School of Medicine, Carrboro

41013

Hospital Universitario Virgen del Rocío, Seville

55905

Mayo Clinic - Rochester, Rochester

60612

Rush Medical Center, Chicago

77030

Baylor College of Med, Houston

90024

UCLA Neuropsychiatric Institute, Los Angeles

92123

Rady Children's Hospital - San Diego, San Diego

00165

Ospedale Pediatrico Bambino Gesù, Rome

3015 GJ

Erasmus MC / location Sophia Kinderziekenhuis, Rotterdam

08950

Hospital Sant Joan De Deu, Esplugues de Llobregas

08208

Corporacio Sanitaria Parc Tauli, Sabadell

All Listed Sponsors
lead

Hoffmann-La Roche

INDUSTRY

NCT04428281 - A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS) | Biotech Hunter | Biotech Hunter