A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RG
This is a first-in-human (FIH) study of RG6496 that will assess the safety and tolerability of single-ascending doses of RG6496 administered to huntington's disease gene expansion carriers (HDGECs). The study consists of two parts: Part 1 \[single-ascending dose\] followed by Part 2 \[open-label extension (OLE)\].
| Condition(s) | Huntington's Disease |
|---|---|
| Status | Recruiting |
| Phase | Phase 1 |
| Study type | Interventional |
| Summary | This is a first-in-human (FIH) study of RG6496 that will assess the safety and tolerability of single-ascending doses of RG6496 administered to huntington's disease gene expansion carriers (HDGECs). The study consists of two parts: Part 1 \[single-ascending dose\] followed by Part 2 \[open-label extension (OLE)\]. |
| Who can participate | Inclusion Criteria: Part 1 * Confirmation of HDGEC status with cytosine-adenine-guanine (CAG) expansion \> 39. * Confirmation of SNP carrier status of the target SNP * Independence Scale (IS) score of ≥70, total functional capacity (TFC) ≥10, total motor score (TMS) \>6. * Ability to read the words "red," "blue," and "green" and be fluent in the language of the informed consent form (ICF) and the tests used at the study site. * Ability to walk unassisted. * Total body weight \> 40 kilogram (kg) and body mass index (BMI) within the range 18-32 kilogram per square meter (kg/m\^2) (inclusive) at baseline. * Ability to undergo and tolerate MRI scans. Part 2 * Completed the post-dose safety follow-up period in the Part 1 of the study. * In the opinion of the Investigator, the participant has no |
| Ages | 25 Years to 65 Years |
| Sex | All |
| Lead sponsor | Hoffmann-La Roche |
| Locations | Ciudad Autonoma Bs As, Argentina; Westmead, New South Wales, Australia; Parkville, Victoria, Australia; Christchurch, New Zealand |
| Start date | 2025-11-19 |
| NCT ID | NCT07246941 |
| Official listing | https://clinicaltrials.gov/study/NCT07246941 |