ProjectWave C9orf72 Trial – A multicenter, randomized, double-blind, placebo-controlled, Phase 1b/2a Study of WVE-004…

Basic data

Acronym:
Wave C9orf72 Trial
Title:
A multicenter, randomized, double-blind, placebo-controlled, Phase 1b/2a Study of WVE-004 administered intrathecally to patients with C9orf72-associated Amyothrophic Lateral Sclerosis (ALS) of Frontotemporal Dementia (FTD)
Duration:
01/07/2021 to 30/06/2024
Abstract / short description:
Diese pharma-initiierte Phase 1b/2a Studie untersucht die Wirksamkeit eines intrathekal applizierten Antisense-Oligonukleotids (ASOs) gegen die C9orf72-Repeatexpansion bei Mutationsträgern mit C9orf72 Repeatexpansion und klinischer Erkrankung einer Amyotrophen Lateralsklerose (ALS) oder Frontotemporalen Demenz (FTD).
Keywords:
Amyotrophe Lateralsklerose
Frontotemporale Demenz
Antisense Oligonukleotide
Therapiestudie
clinical trial
klinische Studie
gene therapy
Gentherapie

Involved staff

Managers

Faculty of Medicine
University of Tübingen
University Department of Neurology
Hospitals and clinical institutes, Faculty of Medicine

Contact persons

University Department of Neurology
Hospitals and clinical institutes, Faculty of Medicine

Local organizational units

Department of Neurology with Focus on Neurodegenerative Disorders
University Department of Neurology
Hospitals and clinical institutes, Faculty of Medicine

Funders

Birmingham, United Kingdom
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