Safety and Tolerability of RG6496 in Huntington’s Disease Gene Expansion Carriers with the Selected Genetic Variant (POINT‑HD)

The primary objective is to assess the safety of RG6496 in individuals with Huntington’s disease gene expansion carriers with the selected genetic variant. No secondary objectives are specified in the source data.

The trial enrolled 36 participants identified as Huntington’s Disease Gene Expansion Carriers. Both female and male individuals were included, representing a vulnerable patient group. Subjects were selected from the patient population and assigned to the study based on the age‑range code “3”, corresponding to the predefined age bracket used by the protocol. All participants were carriers of the pathogenic gene expansion, and no additional health status or lifestyle restrictions were specified in the provided information.

The study is a Phase 1 safety assessment of RG6496 in individuals identified as Huntington’s disease gene expansion carriers, with enrollment projected to begin in June 2026 and conclude in May 2029. Eligible participants will first attend a screening visit to confirm genetic status, eligibility criteria, and baseline assessments. Following successful screening, participants will receive the investigational product and attend scheduled follow‑up visits at predetermined intervals to monitor safety parameters, laboratory values, and adverse events. The trial will culminate in an end‑of‑study visit during which final safety evaluations and study completion procedures are performed. Participant involvement extends from the initial screening through the end‑of‑study visit, encompassing the entire study duration. Early termination may occur if a participant experiences a serious adverse event, fails to comply with protocol requirements, or withdraws consent.