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 (NCT03225833) enrolled 61 people in total across six groups: 16 received a placebo (an inactive substance used for comparison), and the remaining 45 received one of five different doses of an experimental drug called WVE-120101 — either 2 mg, 4 mg, 8 mg, 16 mg, or 32 mg. The trial was primarily measuring how many participants experienced unwanted medical events (called adverse events) after receiving the drug or placebo, and also tracked how the drug moved through the body over time. The reported data shows that when it came to any adverse event during the study, 12 of the 16 placebo participants experienced at least one, compared to 8, 8, 9, 7, and 9 participants in the five WVE-120101 dose groups respectively. For serious adverse events — defined as those involving hospitalisation, life-threatening situations, or lasting disability — none occurred in the placebo group, while 2, 1, 0, 0, and 4 participants experienced them across the five dose groups from lowest to highest dose. Regarding severe adverse events specifically, the numbers reported were 1 in the placebo group and 2, 1, 1, 0, and 5 across the dose groups. A total of 0 placebo participants withdrew from the study due to adverse events, compared to 1, 2, 0, 0, and 2 participants in the WVE-120101 groups. The reported data also shows measurements of how much of the drug appeared in the bloodstream and how quickly. The peak blood concentration of WVE-120101 ranged from approximately 7.70 ng/mL (nanograms per millilitre, a measure of drug level in the blood) in the lowest dose group up to 229.01 ng/mL in the highest dose group. The time it took to reach that peak concentration ranged from about 1.34 hours in the lowest dose group to 4.61 hours in the highest dose group. Some additional measurements for repeated dosing were partially reported, but full data for all groups across all time points was not reported in the submitted results. 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 32250312) ↗
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, Terminated
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 been pre-screened and found to have a specific genetic marker linked to the Huntington's disease (HD) gene mutation (confirm with trial site)
- You are male or female, aged between 25 and 65 years old, and are able to walk
- You have been clinically diagnosed with Huntington's disease based on a standard HD assessment tool, with a confirmed diagnosis score
- You are in the early stages of HD (Stage I or Stage II), meaning you still have a relatively good level of daily functioning as measured by a standard HD scale
Who may not be able to join:
- You have had cancer, or received cancer treatment, in the last 5 years (note: previously treated minor skin cancers may be an exception — confirm with trial site)
- You have taken part in another clinical trial drug study in the past 3 months, or received a specific type of experimental drug called an oligonucleotide in the past 6 months or longer
- You have a significant ongoing medical condition, unstable mental health symptoms, a substance abuse problem, or are pregnant
- You are unable to have a brain MRI scan for any reason
- You have a condition affecting your bones, spine, or blood clotting, or anything else that could make a lumbar puncture (spinal fluid sample) risky or difficult
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
7 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Safety: Number of Patients With Treatment-emergent Adverse Events (TEAEs); Safety: Severity of Adverse Events; Safety: Number of Patients With Serious TEAEs; Safety and Tolerability: Number of Patients Who Withdraw Due to TEAEs
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.