Study of WVE-003 in Patients With Huntington's Disease

July 23, 2025 updated by: Wave Life Sciences Ltd.

A Multicenter, Randomized, Double-blind, Placebo Controlled, Phase 1b/2a Study of WVE-003 Administered Intrathecally in Patients With Huntington's Disease (SELECT-HD)

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

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
47

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Australia
    • New South Wales
      • Westmead, New South Wales, Australia, 2145
        • Westmead Hospital
    • Victoria
      • Clayton, Victoria, Australia, 3168
        • Monash Health
      • Melbourne, Victoria, Australia, 3050
        • Royal Melbourne Hospital
  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G 2G3
        • University of Alberta Hospital
    • Ontario
      • Ottawa, Ontario, Canada, K1H 8L6
        • The Ottawa Hospital
    • Quebec
      • Montréal, Quebec, Canada, H2X019
        • Centre Hospitalier de l-Universite de Montreal
  • Denmark
      • Copenhagen, Denmark, 2100
        • Rigshospitalet
  • France
      • Créteil, France, 94010
        • Hopital Henri Mondor - Hospital
      • Paris, France, 75646
        • Institut du Cerveau et de la Moelle Epinière
  • Germany
      • Bochum, Germany, 44791
        • Katholisches Klinikum Bochum gGmbH
      • Muenster, Germany, 48149
        • George-Huntington-Institut GmbH
      • Taufkirchen, Germany, 84416
        • Kbo-Isar-Amper-Klinikum Taufkirchen (Vils)
  • Italy
      • Verona, Italy
        • Centro Ricerche Cliniche di Verona
  • Netherlands
      • Leiden, Netherlands, 2333 ZA
        • Leiden University Medical Center
      • Maastricht, Netherlands, 6229 HX
        • Maastricht University Medical Center
  • Poland
      • Gdańsk, Poland, 80-462
        • Szpital Sw. Wojciecha
      • Warsaw, Poland, 02-957
        • Instytut Psychiatrii i Neurologii
  • Spain
      • Barcelona, Spain, 08041
        • Hospital de la Sanata Creu i Sant Pau
      • Madrid, Spain, 28034
        • Hospital Universitario Ramón y Cajal
  • United Kingdom
      • Liverpool, United Kingdom, L7 8XP
        • Royal Liverpool University Hospital
    • Devon
      • Exeter, Devon, United Kingdom, EX2 5DW
        • Royal Devon and Exeter Hospital NHS Trust
    • Glasgow City
      • Glasgow, Glasgow City, United Kingdom, G51 4TF
        • Royal Hospital for Children, Pharmacy Aseptic Unit
    • Wales
      • Cardiff, Wales, United Kingdom, CF14 4XW
        • Cardiff University, Schools of Medicine and Biosciences

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

25 years to 60 years (Adult)

Accepts Healthy Volunteers

No

Description

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 Confidence Score = 4
  4. UHDRS Total Functional Capacity Scores ≥9 and ≤13

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
  6. Previously received tominersen

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Sequential Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Placebo Comparator: SAD: Pooled Placebo
Placebo
Drug: SAD: Pooled Placebo
Single dose of placebo
Experimental: SAD: 30mg WVE-003
Single Ascending Dose - 30mg WVE-003
Drug: SAD: 30mg WVE-003
Single ascending dose of 30mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO)
Experimental: SAD: 60mg WVE-003
Single Ascending Dose - 60mg WVE-003
Drug: SAD: 60mg WVE-003
Single ascending dose of 60mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO)
Experimental: SAD: 90mg WVE-003
Single Ascending Dose - 90mg WVE-003
Drug: SAD: 90mg WVE-003
Single ascending dose of 90mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO)
Placebo Comparator: MD: Placebo
Placebo
Drug: MD: Placebo
Three doses of placebo Q8WK
Experimental: MD: 30mg WVE-003
Multiple Dose - 30mg WVE-003
Drug: MD: 30mg WVE-003
Three doses of 30mg WVE-003 Q8WK an allele-selective stereopure, antisense oligonucleotide (ASO)

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Safety: Proportion of Patients With Treatment Emergent Adverse Events (TEAEs) Related to Study Drug
Time Frame: 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.
Day 1 through Week 24 (single ascending dose Period 1); Day 1 through Week 28 (multi dose Period 2)

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Pharmacokinetics of WVE-003 in Plasma
Time Frame: Day 1 (single ascending dose Period 1); Day 1 and Day 113 (multi dose Period 2)
Parameter analyzed: AUC0-6 = area under the concentration-time curve from time 0 to 6 hrs
Day 1 (single ascending dose Period 1); Day 1 and Day 113 (multi dose Period 2)
Pharmacokinetics of WVE-003 in Plasma
Time Frame: Day 1 (single ascending dose Period 1); Day 1 and Day 113 (multi dose Period 2)
Parameter analyzed: Cmax = maximum observed concentration.
Day 1 (single ascending dose Period 1); Day 1 and Day 113 (multi dose Period 2)
Concentration of WVE-003 in Cerebrospinal Fluid (CSF)
Time Frame: 28 days post-dose during Period 1 (P1:Day29); 28 days post last dose during Period 2 (P2: Day141)
WVE-003 concentration in cerebrospinal fluid (CSF) is reported in ng/mL.
28 days post-dose during Period 1 (P1:Day29); 28 days post last dose during Period 2 (P2: Day141)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Wave Life Sciences Ltd.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Medical Director, MD, Wave Life Sciences

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
September 6, 2021

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
May 24, 2024

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
May 24, 2024

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
August 18, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 26, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
September 2, 2021

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 12, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 23, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
July 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • WVE-003-001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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