Trial results
AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
According to the results reported on ClinicalTrials.gov, this trial tested an investigational drug called WVE-003, which is being studied in the context of Huntington's disease. The trial was set up in two parts: a single-dose phase, where participants received one dose at one of three different dose levels (30 mg, 60 mg, or 90 mg) or a placebo (an inactive substance); and a multiple-dose phase, where participants received repeated doses of 30 mg or a placebo. In total, 47 people took part in the single-dose phase and 23 people took part in the multiple-dose phase. The main thing the trial was measuring was the number of participants who experienced adverse events (unwanted or unexpected health changes) that appeared to be related to the study drug. The reported data shows that in the single-dose phase, 2 out of 16 placebo participants, 1 out of 12 in the 30 mg group, 3 out of 11 in the 60 mg group, and 3 out of 8 in the 90 mg group had adverse events considered related to the study drug. In the multiple-dose phase, none of the 7 placebo participants had such events, while 8 out of 16 participants in the 30 mg WVE-003 group did. The trial also measured how much of the drug appeared in the blood and in the cerebrospinal fluid (the fluid surrounding the brain and spine). The reported data shows that drug levels in the blood and cerebrospinal fluid generally increased with higher doses in the single-dose phase, and that the drug was detectable in the cerebrospinal fluid across all dose groups tested. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
View full results on ClinicalTrials.gov ↗ · Read the linked publication on PubMed (PMID 39027419) ↗
These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.
Phase 2 Huntington's Disease Trial, Completed
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- You have a specific genetic marker (called the "A variant of SNP3") on the same gene affected by Huntington's disease — your doctor can check this through genetic testing
- You are a man or woman aged between 25 and 60 years old, and you are able to walk
- You have been clinically diagnosed with Huntington's disease, confirmed using a standard medical rating scale (confirm with trial site)
- Your ability to carry out daily activities falls within a specific range on a Huntington's disease rating scale (confirm with trial site)
Who may not be able to join:
- You have been diagnosed with cancer, or received cancer treatment, in the past 5 years — except for certain treated skin cancers (basal cell or squamous cell)
- You have taken part in another clinical trial or taken an experimental drug within the past year, or within a certain time period based on that drug — with some exceptions for two specific experimental drugs taken more than 3 months ago (confirm with trial site)
- You have an implanted device in your brain or nervous system that could interfere with receiving the study drug by lumbar puncture (spinal injection) or with having an MRI scan
- You are unable to have a brain MRI scan, even with sedation if needed
- You have a condition affecting your bones, spine, or blood clotting that would make a lumbar puncture (spinal injection) risky or unlikely to succeed
- You have previously received a drug called tominersen
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Medical Director, MD, Wave Life Sciences
Australian sites
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
3 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Safety: Proportion of Patients With Treatment Emergent Adverse Events (TEAEs) Related to Study Drug
Can't join this trial?
Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.