Actively Recruiting
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RG6496 in Huntington's Disease
Led by Hoffmann-La Roche · Updated on 2026-04-27
40
Participants Needed
3
Research Sites
171 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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 \[open-label extension (OLE)\].
CONDITIONS
Official Title
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RG6496 in Huntington's Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Confirmed Huntington's disease gene expansion carrier status with CAG repeat > 39
- Confirmation of target SNP carrier status
- Independence Scale score of 70 or higher
- Total functional capacity score of 10 or higher
- Total motor score greater than 6
- Ability to read "red," "blue," and "green" and fluency in the study language
- Ability to walk unassisted
- Body weight over 40 kilograms and BMI between 18 and 32 kg/m²
- Ability to undergo and tolerate MRI scans
- Completed post-dose safety follow-up in Part 1 to join Part 2
- No health worsening that would prevent safe participation as judged by investigator
You will not qualify if you...
- Current or planned participation in other interventional clinical studies, including use of ASO or HTT-lowering therapy within 90 days or 5 drug half-lives prior to screening
- Pregnant, breastfeeding, or planning pregnancy during the study or contraceptive period
- History of malignancy within 5 years prior to screening
- Planned brain surgery during the study
- Positive tests for HIV or hepatitis B at screening
- Active psychosis, confusion, violent behavior, or recent history of primary psychotic disorder
- Scoliosis, spinal deformity, or surgery preventing intrathecal injection in outpatient setting
- History of suicide attempt or active suicidal ideation requiring hospital or care change within 12 months prior to screening
- Premature discontinuation from Part 1
- Received other investigational treatments besides RG6496 since Part 1
- Confirmed dose-limiting adverse events in Part 1
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 3 locations
1
Hospital Britanico de Buenos Aires
Ciudad Autonoma Bs As, Argentina, C1280AEB
Actively Recruiting
2
Westmead Hospital
Westmead, New South Wales, Australia, 2145
Actively Recruiting
3
New Zealand Brain Research Institute
Christchurch, New Zealand, 8011
Actively Recruiting
Research Team
R
Reference Study ID Number: BP45378 https://forpatients.roche.com/
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
DOUBLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
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