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RG6496

Phase 1

Huntington's Disease | Small molecule | Neurology |Roche Holding AG|Last Updated: Sep 2, 2026

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment40

FDA Designations

No designations recorded

Clinical trial landscape

RG6496 · 1 trial · 1 indication

Phase 1 1
NCT07246941A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RG6496 in Huntington's DiseaseHuntington's Disease
ACTIVE NOT_RECRUITING3 Analytics
PHASE1ACTIVE NOT_RECRUITING
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RG6496 in Huntington's Disease
Huntington's DiseaseUnlock trial analytics

Study Endpoints

Primary Endpoints

Part 1: Number of Participants With Adverse Events (AEs)
Up to approximately 24 months
Part 1: Change From Baseline in Neurological Assessment
Up to approximately 24 months

A neurologic examination including fundoscopy, will be performed by a neurologist or an ophthalmologist depending on local practice. A neurologic examination will include an assessment of mental status, level of consciousness, cranial nerve function, motor function, sensory function, reflexes, gait, and coordination.

Part 1: Percentage of Participants With Suicidal Ideation or Behavior, as Assessed by Columbia-Suicide Severity Rating Scale (C-SSRS) Score
Up to approximately 24 months

C-SSRS measures 4 constructs as follows: severity of ideation, intensity of ideation, behavior, and lethality of actual suicide attempts. Binary (yes/no) data are collected for 10 categories. Numeric ratings are provided for intensity of ideation (if present), from 1 to 5, with 1 being the least severe and 5 being the most severe.

Part 1: Change from Baseline in Montreal Cognitive Assessment (MoCA)
Up to approximately 24 months

MoCA is a rater-administered, participant completed assessment (PerfO instrument) used to detect cognitive impairment. It contains a series of basic assessments, including attention and visuospatial tasks. The total score ranges from 0 to 30, where lower scores indicate greater impairment.

Part 2: Number of Participants With AEs
Up to approximately 38 months
Part 2: Change From Baseline in Neurological Assessment
Up to approximately 38 months

A neurologic examination including fundoscopy, will be performed by a neurologist or an ophthalmologist depending on local practice. A neurologic examination will include an assessment of mental status, level of consciousness, cranial nerve function, motor function, sensory function, reflexes, gait, and coordination.

Part 2: Number of Participants With Suicidal Ideation or Behavior, as Assessed by C-SSRS Score
Up to approximately 38 months

C-SSRS measures 4 constructs as follows: severity of ideation, intensity of ideation, behavior, and lethality of actual suicide attempts. Binary (yes/no) data are collected for 10 categories. Numeric ratings are provided for intensity of ideation (if present), from 1 to 5, with 1 being the least severe and 5 being the most severe.

Part 2: Change from Baseline in MoCA
Up to approximately 38 months

MoCA is a rater-administered, participant completed assessment (PerfO instrument) used to detect cognitive impairment. It contains a series of basic assessments, including attention and visuospatial tasks. The total score ranges from 0 to 30, where lower scores indicate greater impairment.

Secondary Endpoints

Part 1: Plasma Concentration of RG6496
Up to approximately 24 months
Part 1 and Part 2: Number of Participants With Anti-drug Antibody (ADA) in Plasma to RG6496
Part 1: Up to approximately 24 months; Part 2: Up to approximately 38 months
Part 1 and Part 2: Change From Baseline of Mutant Huntingtin Protein (mHTT) Concentrations in Cerebrospinal Fluid (CSF) Over Time
Part 1: Up to approximately 24 months; Part 2: Up to approximately 38 months
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Study Design & Arms

AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Part 1: RG6496EXPERIMENTALParticipants will receive single dose of RG6496. Participants will be enrolled in planned sequential cohorts associated with ascending dose levels.
Part 1: PlaceboPLACEBO_COMPARATORParticipants will receive a single dose of RG6496 matching placebo.
Part 2: OLEEXPERIMENTALAll participants who have completed Part 1 and are eligible for Part 2 will receive one open-label dose of RG6496.

Interventions

NameTypeDescription
RG6496DRUGRG6496 will be administered as per the schedule specified in the respective arms.
PlaceboDRUGRG6496 matching placebo will be administered as per the schedule specified in placebo arm.
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Eligibility Criteria

Age Range25 Years to 65 Years
SexALL
Healthy VolunteersNo
Study Sites7

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 word...

Countries:ArgentinaAustraliaCanadaNew ZealandUnited Kingdom
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Competitive Landscape -Huntington's Disease 8 trials

Recent Changes (Last 90 Days)

HIGHSep 2, 2026NCT07246941Status: RECRUITING → ACTIVE_NOT_RECRUITING
HIGHSep 2, 2026NCT07246941Status: RECRUITING → ACTIVE_NOT_RECRUITING
HIGHSep 2, 2026NCT07246941Status: RECRUITING → ACTIVE_NOT_RECRUITING
LOWAug 28, 2026NCT07246941Status: ACTIVE_NOT_RECRUITING → RECRUITING
LOWAug 28, 2026NCT07246941Status: ACTIVE_NOT_RECRUITING → RECRUITING
MEDIUMAug 3, 2026NCT07246941Status: RECRUITING → ACTIVE_NOT_RECRUITING

Frequently asked questions about RG6496

What is RG6496 used for in Huntington's Disease?

RG6496 is an investigational small molecule being developed for the treatment of Huntington's Disease. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities. The drug is being studied to evaluate its safety, tolerability, pharmacokinetics, and pharmacodynamics in patients with this condition.

Who makes RG6496?

RG6496 is being developed by Roche Holding AG, a multinational healthcare company. Roche's stock is traded under the ticker symbol RHHBY. The company is conducting clinical trials to evaluate the drug's potential as a treatment for Huntington's Disease.

What phase is RG6496 in?

RG6496 is currently in Phase 1 clinical development. It is an investigational drug and has not received regulatory approval. The ongoing Phase 1 trial is designed to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of RG6496 in patients with Huntington's Disease.

What clinical trials is RG6496 in?

RG6496 is being evaluated in a Phase 1 clinical trial registered as NCT07246941. This study is active but not recruiting participants and is assessing the safety, tolerability, pharmacokinetics, and pharmacodynamics of RG6496 in Huntington's Disease. The trial is randomized, double-blind, and placebo-controlled, with an enrollment of 3 participants.

Is RG6496 FDA approved?

RG6496 is not FDA approved. It is an investigational drug currently in Phase 1 clinical development for Huntington's Disease. The drug has not yet completed the clinical trials necessary to establish its safety and efficacy for regulatory approval.