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 enrolled 153 participants across two groups: 72 people in "Cohort 1 (Direct Rollover)" and 81 people in "Cohort 2 (Gap Rollover)." These labels suggest participants had previously been in a related study and either moved straight into this one or had a break in between. The trial's primary focus was on tracking unwanted medical events (called treatment-emergent adverse events, or TEAEs — meaning any new or worsening health issue that appeared after starting the study treatment) and on measuring changes in basic physical signs like blood pressure and heart rate. The reported data shows that in Cohort 1, 46 out of 72 participants experienced at least one such unwanted medical event, and the same number — 46 out of 81 — did so in Cohort 2. Breaking those down by how serious they felt: in Cohort 1, 21 participants had mild events, 22 had moderate events, and 3 had severe events; in Cohort 2, 24 had mild, 18 had moderate, and 4 had severe events. A small number of participants left the study because of these events — 2 in Cohort 1 and 3 in Cohort 2. For the physical measurements, the reported changes from the start of the study to the last recorded point were very small: blood pressure shifted by only a few millimetres of mercury (mmHg) in either direction depending on position and group, heart rate changed by roughly 2 beats per minute across both groups, and breathing rate was essentially unchanged (0.0 to −0.1 breaths per minute). It is worth noting that very few participants formally "completed" the study as defined by the trial — only 2 in Cohort 1 and 4 in Cohort 2 — though the data does not explain why the rest did not reach that milestone. No secondary outcome data appears to have been 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 ↗
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 3 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 either completed one of two specific earlier related studies (NCT05107128 or NCT05358821), or you meet the requirements to join as a new participant.
- You are willing to avoid recreational drugs for the whole study, and avoid alcohol for 48 hours before each visit.
- You are willing to invite a trusted person aged 18 or older (such as a family member or friend) to participate alongside you, if you have someone available.
- You are able to travel to the study centre regularly and are likely to be able to keep doing so for the full duration of the study.
For new participants only (not from the earlier studies), you must also:
- Be between 25 and 65 years old at the time of screening.
- Have a genetically confirmed Huntington's disease (HD) diagnosis with a specific gene expansion result of 40 or above, and no signs of the juvenile form of HD.
- Have a specific disease score of 90 or above, calculated using your age and gene test result (confirm with trial site).
- Meet one of two specific combinations of scores on standard HD assessment tests measuring daily functioning and memory/thinking ability (confirm with trial site).
Who may not be able to join:
- You have been diagnosed with another ongoing brain or nerve condition besides HD, such as Alzheimer's disease, Parkinson's disease, vascular dementia, or dementia with Lewy bodies.
- You have had gastric bypass surgery, currently have a gastric sleeve or lap band, or have had any procedure that affects how food moves through your digestive system.
- You have a known allergy to any ingredient in the study drug SAGE-718, including soy lecithin.
- You are taking or have taken certain medications not allowed in this study within the 30 days before screening, or during the study (confirm with trial site for the specific list).
For new participants only, you also may not be able to join if:
- You have previously received gene therapy, or have taken part in another HD experimental drug or device trial within the last 180 days, or another non-HD experimental trial within the last 30 days (or longer, depending on the drug).
- You have previously been treated with antisense oligonucleotides or a specific type of gene-modifying treatment called an mRNA splicing modifier (confirm with trial site).
- Note: If you were confirmed to have received a placebo (dummy treatment) in a previous trial, this would not exclude you.
For participants from the two earlier studies only:
- You have one or more serious ongoing side effects from the previous study.
- You have ongoing unresolved side effects that the study doctor believes could interfere with how the study is carried out.
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
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
Number of Participants With at Least One Treatment-Emergent Adverse Events (TEAEs); Number of Participants With at Least One TEAE by Severity; Number of Participants Who Withdrew From Study Due to TEAEs; Change From Baseline in Vital Signs: Blood Pressure; Change From Baseline in Vital Signs: Heart Rate; Change From Baseline in Vital Signs: Respiratory Rate; Change From Baseline in Vital Signs: Temperature; Change From Baseline in Clinical Laboratory Parameters: Hematology - Basophils, Eosinophils, Erythrocytes, Leukocytes, Lymphocytes, Monocytes, Neutrophils, Platelets; Change From Baseline i...
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.