Recent Updates
Recently added Catalysts

SAD: WVE-003

Phase 1

Huntington Disease | Small molecule | Neurology |Wave Life Sciences Ltd.|Last Updated: Aug 12, 2025

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment47

FDA Designations

No designations recorded

Clinical trial landscape

SAD: WVE-003 · 1 trial · 1 indication

Phase 1 1
NCT05032196Study of WVE-003 in Patients With Huntington's DiseaseHuntington Disease
COMPLETED47 Analytics
PHASE1COMPLETED
Study of WVE-003 in Patients With Huntington's Disease
Huntington DiseaseUnlock trial analytics

Study Endpoints

Primary Endpoints

Safety: Proportion of Patients With Treatment Emergent Adverse Events (TEAEs) Related to Study Drug
Day 1 through Week 24 (single ascending dose Period 1); Day 1 through Week 28 (multi dose Period 2)

The primary outcome for this study was safety and is reported as the proportion of patients with TEAEs related to study drug.

Secondary Endpoints

Pharmacokinetics of WVE-003 in Plasma
Day 1 (single ascending dose Period 1); Day 1 and Day 113 (multi dose Period 2)
Concentration of WVE-003 in Cerebrospinal Fluid (CSF)
28 days post-dose during Period 1 (P1:Day29); 28 days post last dose during Period 2 (P2: Day141)
Unlock Study Endpoints

Study Design & Arms

AllocationRANDOMIZED
MaskingDOUBLE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
SAD: Pooled PlaceboPLACEBO_COMPARATORPlacebo
SAD: 30mg WVE-003EXPERIMENTALSingle Ascending Dose - 30mg WVE-003
SAD: 60mg WVE-003EXPERIMENTALSingle Ascending Dose - 60mg WVE-003
SAD: 90mg WVE-003EXPERIMENTALSingle Ascending Dose - 90mg WVE-003
MD: PlaceboPLACEBO_COMPARATORPlacebo
MD: 30mg WVE-003EXPERIMENTALMultiple Dose - 30mg WVE-003

Interventions

NameTypeDescription
SAD: 30mg WVE-003DRUGSingle ascending dose of 30mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO)
SAD: 60mg WVE-003DRUGSingle ascending dose of 60mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO)
SAD: 90mg WVE-003DRUGSingle ascending dose of 90mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO)
SAD: Pooled PlaceboDRUGSingle dose of placebo
MD: 30mg WVE-003DRUGThree doses of 30mg WVE-003 Q8WK an allele-selective stereopure, antisense oligonucleotide (ASO)
MD: PlaceboDRUGThree doses of placebo Q8WK
Unlock Study Design Details

Eligibility Criteria

Age Range25 Years to 60 Years
SexALL
Healthy VolunteersNo
Study Sites23

Inclusion Criteria: 1. Presence of the A variant of SNP3 on the same allele as the pathogenic CAG triplet expansion 2. Ambulatory, male or female patients aged ≥25 to ≤60 years 3. Clinical diagnostic motor features of HD, defined as Unified Huntington's Disease Rating Scale (UHDRS) Diagnostic Confi...

Countries:AustraliaCanadaDenmarkFranceGermanyItalyNetherlandsPolandSpainUnited Kingdom
Unlock Eligibility Criteria

Competitive Landscape -Huntington's Disease 8 trials (matched to "Huntington Disease")

Frequently asked questions about SAD: WVE-003

What is WVE-003 used for in Huntington Disease?

WVE-003 is an investigational small molecule being developed for the treatment of Huntington Disease. It is currently in Phase 1 clinical development and has not been approved by the FDA. The drug is being studied in patients with Huntington Disease to evaluate its safety and efficacy.

Who makes WVE-003?

WVE-003 is being developed by Wave Life Sciences Ltd., a biopharmaceutical company traded on the stock exchange under the ticker symbol WVE. The company is conducting clinical research on this investigational drug for Huntington Disease.

What phase is WVE-003 in?

WVE-003 is in Phase 1 clinical development. It is an investigational drug and has not yet received regulatory approval. The Phase 1 study has been completed, and the drug remains in early-stage clinical testing for Huntington Disease.

What clinical trials is WVE-003 in?

WVE-003 has been studied in a Phase 1 clinical trial with the identifier NCT05032196, titled "Study of WVE-003 in Patients With Huntington's Disease." This trial enrolled 47 participants and was conducted across multiple countries including Australia, Canada, Denmark, France, Germany, Italy, Netherlands, Poland, Spain, and the United Kingdom.

Is WVE-003 a randomized controlled trial drug?

Yes, the Phase 1 clinical trial of WVE-003 was a randomized, double-blind, placebo-controlled study. This design helps to assess the drug's effects compared to a placebo in patients with Huntington Disease, providing valuable data on its safety and potential efficacy.