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A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RG6496 in Huntington's Disease

Active Not RecruitingPhase 1

Sponsor: Hoffmann-La Roche

Conditions: Huntington's Disease

Interventions: RG6496, Placebo

Countries: Argentina, Australia, Canada, New Zealand, United Kingdom

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 \[open-label extension (OLE)\].

Eligibility overview

Sex: ALL

Age: 25 Years to 65 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria

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 surface antigen at screening
* Active psychosis, confusional state, or violent behavior, including aggression that could cause harm to self or others, over the 12 weeks prior to screening.
* Current or previous history of a primary independent psychotic disorder.
* Scoliosis or spinal deformity or surgery making IT injection not feasible in an outpatient setting
* History of attempted suicide or suicidal ideation with plan (i.e., active suicidal ideation) that required hospital visit and/or change in level of care within 12 months prior to screening.

Part 2

* Prematurely discontinued from Part 1 for any reason (i.e., before the completion of the postdose safety follow-up period of Part 1).
* Pregnant or breastfeeding, or with the intention of becoming pregnant during the study or within the timeframe in which contraception is required.
* Concurrent or planned participation in any interventional clinical study, including current use of an ASO or any HTT-lowering therapy
* Received any active investigational treatment other than RG6496 during or since completion of Part 1 of the study.
* Had confirmed Dose-Limiting Adverse Event (DLAE)(s) in Part 1 of the study.

Locations (7)

Ciudad Autonoma Bs As, Argentina
Westmead, New South Wales, Australia
Clayton, Victoria, Australia
Parkville, Victoria, Australia
Montral, Quebec, Canada
Christchurch, New Zealand
Cambridge, Cambridgeshire, United Kingdom