Clinical Trial

Disease: Amyotrophic Lateral Sclerosis, ALS, or Frontotemporal Dementia FTD, (NCT04931862)

Disease info:

Frontotemporal dementia (FTD) is a rare form of dementia affecting specific brain areas, characterized by abnormal substances like tau proteins within nerve cells. It usually manifests between ages 40 and 60, with an average onset at 54, and can be hereditary. Symptoms progressively worsen and include behavioral changes, speech difficulties, and cognitive decline. Diagnosing FTD involves neuropsychological assessments, brain imaging, and sometimes cerebrospinal fluid examination. There is no cure, but medications may manage symptoms. Treatment focuses on managing mood swings, behavioral modifications, and addressing any contributing disorders. Advanced care often necessitates legal planning and comprehensive support services.

Frontotemporal dementia (FTD) is a rare form of dementia affecting specific brain areas, characterized by abnormal substances like tau proteins within nerve cells. It typically manifests between ages 40 and 60, with an average onset at 54, and can occur as early as 20. FTD can be hereditary, though its exact prevalence is low compared to other forms of dementia. Symptoms progressively worsen and include behavioral changes, speech difficulties, and cognitive decline. Diagnosing FTD involves neuropsychological assessments, brain imaging, and sometimes cerebrospinal fluid examination. There is no cure, but medications may manage symptoms. Treatment focuses on managing mood swings, behavioral modifications, and addressing any contributing disorders. Advanced care often necessitates legal planning and comprehensive support services.

Frequency:
FTD has a prevalence of 1-9 / 100,000 and 1 / 20,000 for ALS.
Official title:
A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-004 Administered Intrathecally to Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD)
Who:
Partners:
Locations:

Australia

New South Wales
Macquarie University, North Ryde, New South Wales, Australia, 2109

Queensland
The Wesley Hospital, Brisbane, Queensland, Australia, QLD 4066

Western Australia
Perron Institute, Nedlands, Western Australia, Australia, 6009

Belgium

UZ Leuven, Leuven, Belgium, 3000

Canada
Ontario
Sunnybrook Health Sciences Centre, Toronto, Ontario, Canada, M4N 3M5

Quebec
McGill University Health Center - Research Institute, Montréal, Quebec, Canada

Ireland

St James Hospital - Ireland, Dublin, Ireland, D08 NHY1

Netherlands

Erasmus University MC, Rotterdam, Netherlands, 3015

Universitair Medisch Centrum Utrecht, Utrecht, Netherlands

New Zealand

Auckland City Hospital, Auckland, New Zealand, 1023

New Zealand Brain Research Institute, Christchurch, New Zealand, 8011

Sweden

Karolinska University Hospital, Solna, Sweden

United Kingdom

University of Cambridge, Cambridge, United Kingdom

University College London Hospital, London, United Kingdom, WC1N 3BG

King's College Hospital, London, United Kingdom, WC2R 2LS

University of Oxford - Nuffield Department of Clinical Neurosciences, Oxford, United Kingdom, OX3 7LF

University of Sheffield, Sheffield, United Kingdom, S10 2TN

Study start:
Jun. 28, 2021
Enrollment:
35 participants
Gene editing method:
RNA editing oligonucleotide
Type of edit:
Gene silence
Gene:
C9orf72 hexanucleotide repeat expansion
Delivery method:
- In-vivo
Note:
Despite robust, sustained reductions in poly(GP), no clinical benefit was seen at 24 weeks, and reductions in poly(GP) were not associated with stabilization in functional outcomes. Based on these data, Wave decided to stop development of WVE-004. Source: https://classic.clinicaltrials.gov/ct2/show/NCT04931862
IndicatorIndicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Terminated

Description

This is a Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, and PD of intrathecal (IT) WVE-004 in adult patients with C9orf72-associated ALS or FTD. To participate in the study, patients must have a documented mutation (GGGGCC [G4C2] repeat expansion) in the first intronic region of the C9orf72 gene and be diagnosed with ALS or FTD.

Last updated: May. 29, 2024
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