Clinical Trial

Disease: Amyotrophic Lateral Sclerosis, ALS, and Frontotemporal Dementia, FTD, (NCT05683860)

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, Open-label Extension (OLE) Study to Evaluate the Safety, Pharmacodynamics, and Clinical Effects of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)
Who:
Partners:
Locations:

Netherlands

Erasmus MC, Rotterdam, Netherlands, 3015

Universitair Medisch Centrum Utrecht, Utrecht, Netherlands


United Kingdom

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

Study start:
Dec. 14, 2022
Enrollment:
8 participants
Gene editing method:
RNA editing oligonucleotide
Type of edit:
Gene silence
Gene:
C9orf72 hexanucleotide repeat expansion
Delivery method:
- In-vivo
IndicatorIndicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Terminated

Description

This is an OLE study conducted to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and clinical effects of WVE-004 in adult patients with ALS, FTD, or mixed ALS/FTD phenotype with a documented mutation in the C9orf72 gene. To participate in the study, patients must have successfully completed Phase 1b/2a WVE-004-001 study.

Last updated: May. 29, 2024
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