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

Sponsor
Hoffmann-La Roche
Study ID
NCT07246941
Phase
PHASE1
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
25 Years - 65 Years
Healthy Volunteers
Not accepted

Interventions

  • RG6496 — DRUG
    RG6496 will be administered as per the schedule specified in the respective arms.
  • Placebo — DRUG
    RG6496 matching placebo will be administered as per the schedule specified in placebo arm.

Study Details

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

Key Dates

Start date
Nov 19, 2025
Status verified
May 2026
Primary completion
Feb 28, 2028
Completion
May 23, 2029

Study Design

Enrollment
40 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Part 1: RG6496
    Participants will receive single dose of RG6496. Participants will be enrolled in planned sequential cohorts associated with ascending dose levels.
  • Placebo Comparator: Part 1: Placebo
    Participants will receive a single dose of RG6496 matching placebo.
  • Experimental: Part 2: OLE
    All participants who have completed Part 1 and are eligible for Part 2 will receive one open-label dose of RG6496.

Primary Outcome Measure

Part 1: Number of Participants With Adverse Events (AEs) [ Time Frame: Up to approximately 24 months ]

Central Contacts

  • Reference Study ID Number: BP45378 https://forpatients.roche.com/
    888-662-6728 (U.S. Only)
    [email protected]
  • Fastest response: use the inquiry form. No email attachments. https://www.gene.com/contact-us/submit-medical-inquiry

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