NCT07246941 A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RG6496 in Huntington's Disease

Eligibility & Interventions

Eligibility Fast-Check

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What to Expect as a Participant

You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.

Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.

This trial targets 40 participants in total. It began in 2025-11-19 with a primary completion date of 2028-02-28.

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

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.

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