NCT04849741 - A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD) | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

June 1, 2021

Primary Completion Date

September 30, 2025

Study Completion Date

September 30, 2029

Conditions

Alexander Disease

Interventions

DRUG

zilganersen

zilganersen will be administered by ITB injection.

DRUG

Placebo

zilganersen-matching placebo will be administered by ITB injection.

Trial Locations (13)

Ospedale Pediatrico Bambino Gesù, Roma

Murdoch Children's Research Institute, Parkville

Children's Hospital of Philadelphia, Philadelphia

Ospedale dei Bambini Vittore Buzzi, Milan

Children's Hospital of Atlanta, Atlanta

Lucile Packard Children's Hospital Stanford, Palo Alto

Pediatric Neurology Institute, Dana-Dwek Children's Hospital, Tel Aviv Sourasky Medical Center, Tel Aviv

Massachusetts General Hospital, Boston

McGill University Health Centre, Montreal

National Center of Neurology and Psychiatry, Tokyo

Amsterdam Universitair Medische Centra - Academisch Medisch Centrum, Amsterdam

University College London Hospitals NHS Foundation Trust, London

Great Ormond Street Hospital for Children NHS Foundation Trust, London