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

A Phase I, 2-Part Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-ascending Doses of Intrathecally Administered RG6496 in a Randomized, Placebo-controlled, Investigator/Participant-blind Study With an Open-label Extension in Huntington's Disease Gene Expansion Carriers

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RG6496 in Huntington's Disease (NCT07246941) is a Phase 1 interventional studying Huntington's Disease, sponsored by Hoffmann-La Roche. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

About This Trial

What Stage of Research Is This?

Phase 1 trials test a new treatment for the first time in humans, focusing on safety, dosing, and how the body processes the drug. For Huntington's Disease, a Phase 1 study typically enrolls a small number of participants — often healthy volunteers or patients who have exhausted standard treatment options. Phase 1 results determine whether a treatment moves into larger Phase 2 efficacy studies.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

With a target enrollment of 40 participants, this is a small study — typical of early-phase research, rare-disease trials, or pilot studies designed to generate preliminary signal before a larger study is launched.

Who May Be Eligible (Plain English)

Who May Qualify: 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 willing to sign a consent form 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. Who Should NOT Join This Trial: 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 ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language

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.

Treatments Being Tested

DRUG

RG6496

RG6496 will be administered as per the schedule specified in the respective arms.

DRUG

Placebo

RG6496 matching placebo will be administered as per the schedule specified in placebo arm.

Locations (3)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

Hospital Britanico de Buenos Aires

Ciudad Autonoma Bs As, Argentina

Westmead Hospital

Westmead, New South Wales, Australia

New Zealand Brain Research Institute

Christchurch, New Zealand

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT07246941), the sponsor (Hoffmann-La Roche), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT07246941 clinical trial studying?

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 \[open-label extension (OLE)\]. The full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Who can participate in NCT07246941?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT07246941?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT07246941. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT07246941. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-05-08 · Data from ClinicalTrials.gov.