A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RG6496 in… (NCT07246941) | Clinical Trial Compass
RecruitingPhase 1
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RG6496 in Huntington's Disease
Argentina, Australia, New Zealand40 participantsStarted 2025-11-19
Plain-language 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
Age range25 Years – 65 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 not experienced a worsening in health that precludes their safe continued participation in the study.
Exclusion Criteria:
Part 1
* Concurrent or planned participation in any interventional clinical study, including current use of an ASO or any HTT-lowering therapy or treatment with investigational therapy within 90 days or 5 drug-elimination half-lives, whichever is longer prior to screening
* Pregnant or breastfeeding, or with the intention of becoming pregnant during the study or within the timeframe in which contraception is required
* Malignancy within 5 years prior to screening
* Planned brain surgery during the study
* Positive HIV test, hepatitis B surface antigen and hepatitis B…
What they're measuring
1
Part 1: Number of Participants With Adverse Events (AEs)
Timeframe: Up to approximately 24 months
2
Part 1: Change From Baseline in Neurological Assessment
Timeframe: Up to approximately 24 months
3
Part 1: Percentage of Participants With Suicidal Ideation or Behavior, as Assessed by Columbia-Suicide Severity Rating Scale (C-SSRS) Score
Timeframe: Up to approximately 24 months
4
Part 1: Change from Baseline in Montreal Cognitive Assessment (MoCA)
Timeframe: Up to approximately 24 months
5
Part 2: Number of Participants With AEs
Timeframe: Up to approximately 38 months
6
Part 2: Change From Baseline in Neurological Assessment
Timeframe: Up to approximately 38 months
7
Part 2: Number of Participants With Suicidal Ideation or Behavior, as Assessed by C-SSRS Score
Timeframe: Up to approximately 38 months
8
Part 2: Change from Baseline in MoCA
Trial details
NCT IDNCT07246941
SponsorHoffmann-La Roche
Sponsor typeINDUSTRY
Study typeINTERVENTIONAL
Primary completion2027-12-11
Contact for this trial
Reference Study ID Number: BP45378 https://forpatients.roche.com/