Phase 2 Huntington's Disease Trial, Terminated NCT03225846 Sponsor: Wave Life Sciences USA, Inc. Condition: Huntington's Disease
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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 (NCT03225846) enrolled 88 people in total across seven groups. Twenty-two participants received a pooled placebo (a dummy treatment), while the remaining 66 received one of six different doses of an investigational drug called WVE-120102, ranging from 2 mg up to 32 mg. The trial was primarily set up to track the number of participants who experienced unwanted health events (called "adverse events") during the study, including those that were severe or serious. It also measured how the drug moved through the body — for example, how high the drug's level in the blood got, and how quickly that peak level was reached. The reported data shows that in the primary outcomes, most participants across all groups — including those on placebo — experienced at least one adverse event during the trial. For example, 20 out of 22 placebo participants had an adverse event, compared to numbers ranging from 4 to 13 out of each active-dose group. Regarding severe adverse events, the highest-dose group (32 mg) had 9 out of 13 participants affected, compared to 2 out of 22 in the placebo group. Serious adverse events (those involving hospitalisation or other significant consequences) were reported for 9 participants in the 32 mg group, compared to none in the placebo group. Six participants in the 32 mg group also withdrew from the trial due to adverse events, while no withdrawals for this reason were reported in any other group. The reported data for the secondary outcomes shows that as the dose increased, the peak level of WVE-120102 measured in the blood generally rose — from approximately 1.35 ng/mL at the 2 mg dose up to 96.21 ng/mL at the 16 mg dose (figures for the 32 mg dose were not reported in the submitted data). The time taken to reach that peak level generally ranged from roughly 1.2 to 3.3 hours across the different dose groups. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

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

NCT03225846
Terminated Phase 2 🇦🇺 Australian site

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 (a DNA variation) that is on the same gene as the Huntington's disease (HD) mutation (confirm with trial site)
  • You are male or female, aged 25 to 65 years old, and are able to walk
  • You have been clinically diagnosed with Huntington's disease based on a specific, standardised rating scale used by doctors (called the UHDRS), with a confirmed diagnosis score
  • You are in the early stages of HD (Stage I or Stage II), meaning your ability to carry out daily activities falls within a certain range on the UHDRS scoring system

Who may not be able to join:

  • You have had cancer, or received treatment for cancer, within the last 5 years — although having had a treated skin cancer (basal cell or squamous cell) may not necessarily exclude you (confirm with trial site)
  • You have taken part in another clinical trial involving an experimental drug or implanted device in the last 3 months, or an experimental gene-silencing type drug (oligonucleotide) in the last 6 months or longer
  • You have a serious unstable 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 any other issue that could make a lumbar puncture (spinal fluid sample) risky or difficult to perform

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.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.
Last synced 28 July 2026
Phase 2: approximately ~30% of drugs entering this phase reach regulatory approval, based on published industry-wide historical data. This is not specific to this trial.
Phase success rates shown are historical industry-wide averages based on published data. They are not a prediction for this specific trial or your individual situation.

Contact this trial

Principal Investigator: Medical Director, MD, Wave Life Sciences

Australian sites

Westmead Hospital, Sydney, New South Wales
Royal Brisbane & Women's Hospital, Herston, Queensland
Royal Melbourne Hospital, Carlton, Victoria
Monash Health, Clayton, Victoria
Alfred Health, Melbourne, Victoria
Calvary Health Care Bethlehem, Parkdale, Victoria
North Metropolitan Health Service, Perth, Western Australia

Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.

GP referral letter

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Trial details

Status
Terminated
Phase
Phase 2
Sponsor
Wave Life Sciences USA, Inc.
Registry
ClinicalTrials.gov
Start date
17 July 2017
Est. completion
10 May 2021

Where this trial is recruiting

🇦🇺 Australia 🇨🇦 Canada 🇩🇰 Denmark 🇫🇷 France 🇩🇪 Germany 🇵🇱 Poland 🇬🇧 United Kingdom 🇺🇸 United States

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: Number of Patients Who Experienced Severe TEAEs; Safety: Number of Patients With Serious TEAEs; Safety and Tolerability: Number of Patients Who Withdraw Due to TEAEs

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

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.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov