Trials / Terminated
TerminatedNCT04931862
Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD)
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)
- Status
- Terminated
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 35 (actual)
- Sponsor
- Wave Life Sciences Ltd. · Industry
- Sex
- All
- Age
- 18 Years – 80 Years
- Healthy volunteers
- Not accepted
Summary
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | WVE-004 | WVE-004 is a stereopure antisense oligonucleotide (ASO) |
| DRUG | Placebo | Artificial cerebrospinal fluid (aCSF) |
Timeline
- Start date
- 2021-06-28
- Primary completion
- 2023-06-27
- Completion
- 2023-06-27
- First posted
- 2021-06-18
- Last updated
- 2023-10-23
Locations
17 sites across 8 countries: Australia, Belgium, Canada, Ireland, Netherlands, New Zealand, Sweden, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04931862. Inclusion in this directory is not an endorsement.