HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome

PHASE1/PHASE2RecruitingINTERVENTIONAL
Enrollment

70

Participants

Timeline

Start Date

December 22, 2021

Primary Completion Date

March 31, 2029

Study Completion Date

March 31, 2029

Conditions
Angelman Syndrome
Interventions
DRUG

ION582

ION582 will be administered by IT injection.

Trial Locations (11)

27510

RECRUITING

University of North Carolina at Chapel Hill School of Medicine, Carrboro

56126

RECRUITING

Azienda Ospedaliera Universitaria Pisana, Pisa

60612

RECRUITING

Rush University Medical Center, Chicago

75015

ACTIVE_NOT_RECRUITING

Necker-Enfants Malades Hospital, Paris

77030

ACTIVE_NOT_RECRUITING

Texas Children's Hospital, Houston

80045

RECRUITING

Colorado Children's Hospital Research Institute, Aurora

92123

ACTIVE_NOT_RECRUITING

Rady Children's Hospital, San Diego

5262100

RECRUITING

Sheba Medical Center, Ramat Gan

02215

RECRUITING

Boston Children's Hospital, Boston

NSW 2031

ACTIVE_NOT_RECRUITING

Sydney Children's Hospital, Kids Cancer Centre, Randwick

OX3 9DU

ACTIVE_NOT_RECRUITING

STRONG Group University of Oxford, Oxford

Sponsors
All Listed Sponsors
lead

Ionis Pharmaceuticals, Inc.

INDUSTRY