NCT05683860 is a Phase 1, PHASE2 clinical trial of WVE-004 in ALS, sponsored by Wave Life Sciences Ltd..

Who is sponsoring NCT05683860?

NCT05683860 is sponsored by Wave Life Sciences Ltd..

What drugs are being tested in NCT05683860?

Interventions in NCT05683860: WVE-004.

Is NCT05683860 still recruiting?

NCT05683860 is currently terminated. Last updated 23 October 2023.

Last reviewed 2023-10-23 · How we verify

NCT05683860

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Open-label Extension (OLE) Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)

Terminated Phase 1, PHASE2 Last updated 23 October 2023

What this trial tests

Phase 1, PHASE2 trial testing WVE-004 in ALS in 8 participants. Terminated before completion.

Timeline

14 December 2022

Primary endpoint
30 June 2023

30 June 2023

Quick facts

Lead sponsor	Wave Life Sciences Ltd.
Phase	Phase 1, PHASE2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	8
Start date	14 December 2022
Primary completion	30 June 2023
Estimated completion	30 June 2023
Sites	3 locations across United Kingdom, Netherlands

Drugs / interventions tested

WVE-004 — full drug profile →

Conditions studied

ALS — all drugs for ALS →
FTD — all drugs for FTD →

Sponsor

Wave Life Sciences Ltd. — full company profile →

Who can join

Eligibility, any sex, with ALS or FTD. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own 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.

Publications & conference data

5 peer-reviewed publications reference this trial (live from Europe PMC):

Axonopathy Underlying Amyotrophic Lateral Sclerosis: Unraveling Complex Pathways and Therapeutic Insights.
Luan T, Li Q, Huang Z, Feng Y, et al · · 2024 · cited 9× · PMID 39097850 · DOI 10.1007/s12264-024-01267-2
Gene therapy breakthroughs in ALS: a beacon of hope for 20% of ALS patients.
Xie Q, Li K, Chen Y, Li Y, et al · · 2025 · cited 5× · PMID 40234983 · DOI 10.1186/s40035-025-00477-6
CRISPR-Cas technologies in neurodegenerative disorders: mechanistic insights, therapeutic potential, and translational challenges.
Yashooa RK, Nabi AQ, Smail SW, Azeez SS, et al · · 2025 · cited 3× · PMID 41674784 · DOI 10.3389/fneur.2025.1737468
Antisense Oligonucleotides: Technological Advances, Clinical Progress, and Expanding Therapeutic Frontiers.
Xu L, Zhang H, Jiang B, Jiang Y, et al · · 2026 · PMID 42076098 · DOI 10.3390/pharmaceutics18040446
Amyotrophic Lateral Sclerosis (ALS) Genetics and Microbiota: A Comprehensive Review.
Kurdi MA, Alotaibi H, Alkhuraymi AT, Aldahery LN, et al · · 2026 · PMID 41752118 · DOI 10.3390/ijms27041978

Verify or expand the search:

Other trials of WVE-004

Trials testing the same drug.

NCT04931862 — Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD · Phase 1, PHASE2 · terminated

Other recruiting trials for ALS

Currently open trials in the same condition.

NCT07460037 — Chinese-Specific Speech Imagery Coding Using High-Density ECoG · NA · recruiting
NCT07006571 — At-home Treatment With Cortico-spinal tDCS for Amyotrophic Lateral Sclerosis · NA · recruiting
NCT07193953 — Intravenous Immunoglobulin (IVIG) and Blood-Brain Barrier Disruption in Amyotrophic Lateral Sclerosis (ALS) · Phase 1 · recruiting
NCT06735014 — Multicenter ALS Imaging Study · recruiting
NCT06511934 — Feasibility of the BrainGate2 Neural Interface System in Persons With Tetraplegia (BG-Tablet-01) · NA · recruiting

Other Wave Life Sciences Ltd. trials

Trials by the same sponsor.

NCT06186492 — A Phase 1 Research Study to Evaluate Safety, Tolerability, and Pharmacokinetics of WVE-006 in Healthy Participants With · Phase 1 · completed
NCT04906460 — Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53) · Phase 1, PHASE2 · recruiting
NCT05032196 — Study of WVE-003 in Patients With Huntington's Disease · Phase 1, PHASE2 · completed
NCT04931862 — Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD · Phase 1, PHASE2 · terminated
NCT04617847 — Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120101 in Patients With Huntington's Disease · Phase 1, PHASE2 · terminated

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT05683860 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Wave Life Sciences Ltd.
Last refreshed: 23 October 2023

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT05683860.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing