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Trial details imported from

For full trial details, please see the original record at

Registration number
Ethics application status
Date submitted
Date registered
Date last updated

Titles & IDs
Public title
Study of WVE-003 in Patients With Huntington's Disease
Scientific title
A Multicenter, Randomized, Double-blind, Placebo Controlled, Phase 1b/2a Study of WVE-003 Administered Intrathecally in Patients With Huntington's Disease
Secondary ID [1] 0 0
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Huntington Disease 0 0
Condition category
Condition code

Study type
Description of intervention(s) / exposure
Treatment: Drugs - WVE-003

Experimental: WVE-003 (Dose A) or placebo -

Experimental: WVE-003 (Dose B) or placebo -

Experimental: WVE-003 (Dose C) or placebo -

Experimental: WVE-003 (Dose D) or placebo -

Treatment: Drugs: WVE-003
WVE-003 is a stereopure antisense oligonucleotide (ASO)

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Primary outcome [1] 0 0
Safety: Proportion of patients with adverse events (AEs)
Timepoint [1] 0 0
Day 1 through end of study (minimum of 36 weeks)
Secondary outcome [1] 0 0
Maximum concentration (Cmax) of WVE-003 in plasma
Timepoint [1] 0 0
Day 1 through end of study (minimum of 36 weeks)
Secondary outcome [2] 0 0
Area under the plasma concentration time curve for WVE-003 from time 0 to last quantifiable concentration (AUC0-t)
Timepoint [2] 0 0
Day 1 through end of study (minimum of 36 weeks)
Secondary outcome [3] 0 0
Terminal half-life (t1/2) of WVE-003 in cerebrospinal fluid (CSF)
Timepoint [3] 0 0
Day 1 through end of study (minimum of 36 weeks)

Key inclusion criteria
1. Presence of the A variant of SNP3 on the same allele as the pathogenic CAG triplet

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 Confidence Score = 4

4. UHDRS Total Functional Capacity Scores =9 and =13
Minimum age
25 Years
Maximum age
60 Years
Both males and females
Can healthy volunteers participate?
Key exclusion criteria
1. Malignancy or received treatment for malignancy, other than treated basal cell or
squamous cell carcinoma of the skin, within the previous 5 years

2. Received any other study drug, including an investigational oligonucleotide, within
the past 1 year or 5 half-lives of the drug, whichever is longer, with the exception
of the following:

a. Received WVE-120101 or WVE-120102 within the last 3 months

3. Implantable CNS device that may interfere with ability to administer study drug via
lumbar puncture or undergo MRI scan

4. Inability to undergo brain MRI (with or without sedation)

5. Bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or
unsuccessful lumbar puncture

Study design
Purpose of the study
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s

The people analysing the results/data
Intervention assignment
Other design features
Phase 1/Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment status
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Date of last participant enrolment
Date of last data collection
Sample size
Accrual to date
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Monash Health - Clayton
Recruitment hospital [2] 0 0
Royal Melbourne Hospital - Melbourne
Recruitment hospital [3] 0 0
North Metropolitan Health Service - Perth
Recruitment postcode(s) [1] 0 0
3168 - Clayton
Recruitment postcode(s) [2] 0 0
3050 - Melbourne
Recruitment postcode(s) [3] 0 0
6910 - Perth
Recruitment outside Australia
Country [1] 0 0
State/province [1] 0 0
Country [2] 0 0
State/province [2] 0 0
Country [3] 0 0
State/province [3] 0 0
Country [4] 0 0
State/province [4] 0 0
Country [5] 0 0
State/province [5] 0 0
Country [6] 0 0
United Kingdom
State/province [6] 0 0

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Wave Life Sciences Ltd.

Ethics approval
Ethics application status

Brief summary
This is a Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to
evaluate the safety, tolerability, PK, and PD of WVE-003 in adult patients with
early-manifest HD who carry the targeted single nucleotide polymorphism (SNP) - SNP3.
Trial website
Trial related presentations / publications
Public notes

Principal investigator
Name 0 0
Medical Director, MD
Address 0 0
Wave Life Sciences
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Clinical Operations
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
For IPD and results data, please see