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Antisense Oligonucleotide

WVE-003 for Huntington's Disease

Phase 1 & 2
Waitlist Available
Research Sponsored by Wave Life Sciences Ltd.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Ambulatory, male or female patients aged ≥25 to ≤60 years
Presence of the A variant of SNP3 on the same allele as the pathogenic CAG triplet expansion
Must not have
Implantable CNS device that may interfere with ability to administer study drug via lumbar puncture or undergo MRI scan
Bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or unsuccessful lumbar puncture
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a new drug called WVE-003 in adults with early-stage Huntington's Disease who have a specific genetic marker. The goal is to see if the drug is safe and effective by changing how certain genes work.

Who is the study for?
This trial is for adults aged 25-60 with early-manifest Huntington's Disease, who can walk and have certain scores on the UHDRS scale. They must carry a specific genetic marker (SNP3-A variant) linked to their condition. People who've had other experimental drugs recently or have conditions that make lumbar punctures risky can't join.
What is being tested?
The study tests WVE-003, a potential treatment for Huntington's Disease. Participants are randomly assigned to receive either WVE-003 or a placebo without knowing which one they're getting. The trial will assess safety, how well the body handles the drug (PK), and its effect on disease markers (PD).
What are the potential side effects?
Since this is an early-phase trial for WVE-003, detailed side effects aren't fully known yet. Generally, participants may experience issues related to the administration of the drug via lumbar puncture and possible reactions similar to other oligonucleotides.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am between 25 and 60 years old and can walk.
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My genetic test shows I have the A variant of SNP3 linked to my condition.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I do not have any implants in my brain or spine that could interfere with spinal injections or MRI scans.
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I have a condition that could make a spinal tap risky or difficult.
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I have previously been treated with tominersen.
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I haven't had cancer or cancer treatment in the last 5 years, except for skin cancer.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups
Experimental Treatment
Group I: WVE-003 (Dose C) or placeboExperimental Treatment1 Intervention
Group II: WVE-003 (Dose B) or placeboExperimental Treatment1 Intervention
Group III: WVE-003 (Dose A) or placeboExperimental Treatment1 Intervention
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
WVE-003
2021
Completed Phase 2
~50

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The most common treatments for Huntington's Disease (HD) that focus on gene expression modulation, such as WVE-003, work by targeting specific single nucleotide polymorphisms (SNPs) to reduce the production of the mutant huntingtin protein. This approach involves the use of antisense oligonucleotides (ASOs) or small interfering RNAs (siRNAs) that bind to the mutant mRNA, promoting its degradation or preventing its translation. By lowering the levels of the toxic protein, these treatments aim to slow disease progression and alleviate symptoms. This is crucial for HD patients as it addresses the root cause of the disease at the genetic level, potentially offering more effective and long-lasting benefits compared to symptomatic treatments.
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Find a Location

Who is running the clinical trial?

Wave Life Sciences Ltd.Lead Sponsor
12 Previous Clinical Trials
448 Total Patients Enrolled
Medical Director, MDStudy DirectorWave Life Sciences
81 Previous Clinical Trials
15,956 Total Patients Enrolled
~11 spots leftby Dec 2025