A Study to Investigate the Pharmacokinetics (PK) and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype.

PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

48

Participants

Timeline

Start Date

July 27, 2023

Primary Completion Date

December 4, 2025

Study Completion Date

December 4, 2025

Conditions
Angelman Syndrome
Interventions
DRUG

Alogabat

Alogabat will be administered QD with dose depending on cohort and age of the participant.

Trial Locations (20)

4101

Queensland Children?s Hospital, South Brisbane

10032

Columbia University Medical Center, New York

13005

Hopital la Timone Enfants, Marseille

16147

IRCCS Istituto G. Gaslini, Genoa

21000

CHU Dijon Bourgogne Hôpital François Mitterand, Dijon

27510

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

28222

Hospital Universitario Puerta De Hierro Majadahonda, Madrid

29609

CHRU de Brest, Brest

31008

Hospital Universitario de Navarra;Unidad de Neuropediatría, Pamploa

31015

IRCCS Eugenio Medea, Conegliano Veneto (TV)

60612

Rush Medical Center, Chicago

75015

Groupe Hospitalier Necker Enfants Malades, Paris

80337

Dr. Von Haunersches Kinderspital, München

92123

Rady Children's Hospital - San Diego, San Diego

98405

Multicare Institute for Research and Innovation, Tacoma

02115

Boston Children's Hospital, Boston

37232-9119

Vanderbilt Children's Hospital, Nashville

00165

Ospedale Pediatrico Bambino Gesù, Rome

08950

Hospital Sant Joan de Deu, Esplugues de Llobregat · Barcelona

08208

Corporacio Sanitaria Parc Tauli, Sabadell

All Listed Sponsors
lead

Hoffmann-La Roche

INDUSTRY