Phase 1 Huntington's Disease Trial, Recruiting
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:
Part 1 (first phase):
- You have been confirmed to have a genetic change linked to Huntington's disease, with a specific gene repeat number greater than 39
- You carry a particular genetic variant (called a SNP) that this treatment is designed to target
- Your scores on standard Huntington's disease assessments show you are still largely independent and functioning at a certain level (confirm with trial site)
- You are able to read the words "red," "blue," and "green," and you speak the language used at the study site fluently
- You are able to walk without any assistance
- You weigh more than 40 kg (about 88 lbs) and your body mass index (a measure of weight relative to height) falls between 18 and 32
- You are able to have MRI scans (brain imaging) without difficulty
Part 2 (second phase):
- You have already completed the safety follow-up period after receiving the treatment in Part 1 of this study
- In the doctor's opinion, your health has not gotten worse in a way that would make it unsafe for you to continue
Who may not be able to join:
Part 1 (first phase):
- You are currently taking part in, or plan to take part in, another clinical trial, or have used certain gene-silencing or Huntington's-related experimental treatments recently
- You are pregnant, breastfeeding, or planning to become pregnant during the study or during the required period of contraception
- You have been diagnosed with cancer within the last 5 years
- You have planned brain surgery scheduled during the study
- You have tested positive for HIV, or certain types of hepatitis B at your screening appointment
- You have experienced serious mental health episodes such as psychosis, severe confusion, or violent or aggressive behaviour in the past 12 weeks
- You have a current or past diagnosis of a primary psychotic disorder (a serious mental health condition involving loss of touch with reality)
- You have a spine condition or have had spinal surgery that would make it impossible to safely receive an injection into the spinal fluid as an outpatient
- You have had a suicide attempt, or serious thoughts of suicide that required a hospital visit or change in care, within the past 12 months
Part 2 (second phase):
- You left Part 1 of the study early for any reason before completing the full follow-up period
- You are pregnant, breastfeeding, or planning to become pregnant during the study or during the required period of contraception
- You are currently taking part in, or plan to take part in, another clinical trial, or are using certain gene-silencing or Huntington's-related experimental treatments
- You have received any other experimental treatment (other than the one used in this trial) during or since completing Part 1
- You experienced a serious side effect during Part 1 that met the trial's definition of a dose-limiting event (confirm with trial site)
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: Clinical Trials, Hoffmann-La Roche
Phone: 888-662-6728 (U.S. Only)
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
Part 1: Number of Participants With Adverse Events (AEs); Part 1: Change From Baseline in Neurological Assessment; Part 1: Percentage of Participants With Suicidal Ideation or Behavior, as Assessed by Columbia-Suicide Severity Rating Scale (C-SSRS) Score; Part 1: Change from Baseline in Montreal Cognitive Assessment (MoCA); Part 2: Number of Participants With AEs; Part 2: Change From Baseline in Neurological Assessment; Part 2: Number of Participants With Suicidal Ideation or Behavior, as Assessed by C-SSRS Score; Part 2: Change from Baseline in MoCA
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.