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Clinical Trial NCT07246941 (POINT-HD) for Huntington's Disease is recruiting. See the Trial Radar Card View and AI discovery tools for all the details. Or ask anything here. | ||
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Hoffmann-La RocheA Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RG6496 in Huntington's Disease (POINT-HD) Phase 1 40 First-in-Human Open-Label
Clinical Trial NCT07246941 (POINT-HD) is designed to study Treatment for Huntington's Disease. It is a Phase 1 interventional study that is recruiting, having started on 19 November 2025, with plans to enroll 40 participants. Led by Hoffmann-La Roche, it is expected to complete by 5 March 2029. The latest data from ClinicalTrials.gov was last updated on 27 February 2026.
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 \[open-label extension (OLE)\].
Official Title
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
Conditions
Huntington's DiseaseOther Study IDs
- POINT-HD
- BP45378
- 2025-520631-17-00 (EU Study (CTIS) Number)
NCT ID Number
Start Date (Actual)
2025-11-19
Last Update Posted
2026-02-27
Completion Date (Estimated)
2029-03-05
Enrollment (Estimated)
40
Study Type
Interventional
PHASE
Phase 1
Status
Recruiting
Primary Purpose
Treatment
Design Allocation
Randomized
Interventional Model
Parallel
Masking
Double
Arms / Interventions
| Participant Group/Arm | Intervention/Treatment |
|---|---|
ExperimentalPart 1: RG6496 Participants will receive single dose of RG6496. Participants will be enrolled in planned sequential cohorts associated with ascending dose levels. | RG6496 RG6496 will be administered as per the schedule specified in the respective arms. |
Placebo ComparatorPart 1: Placebo Participants will receive a single dose of RG6496 matching placebo. | Placebo RG6496 matching placebo will be administered as per the schedule specified in placebo arm. |
ExperimentalPart 2: OLE All participants who have completed Part 1 and are eligible for Part 2 will receive one open-label dose of RG6496. | RG6496 RG6496 will be administered as per the schedule specified in the respective arms. |
Primary Outcome Measures
Secondary Outcome Measures
| Outcome Measure | Measure Description | Time Frame |
|---|---|---|
Part 1: Number of Participants With Adverse Events (AEs) | Up to approximately 24 months | |
Part 1: Change From Baseline in Neurological Assessment | 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. | Up to approximately 24 months |
Part 1: Percentage of Participants With Suicidal Ideation or Behavior, as Assessed by Columbia-Suicide Severity Rating Scale (C-SSRS) Score | 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. | Up to approximately 24 months |
Part 1: Change from Baseline in Montreal Cognitive Assessment (MoCA) | 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. | Up to approximately 24 months |
Part 2: Number of Participants With AEs | Up to approximately 38 months | |
Part 2: Change From Baseline in Neurological Assessment | 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. | Up to approximately 38 months |
Part 2: Number of Participants With Suicidal Ideation or Behavior, as Assessed by C-SSRS Score | 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. | Up to approximately 38 months |
Part 2: Change from Baseline in MoCA | 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. | Up to approximately 38 months |
| Outcome Measure | Measure Description | Time Frame |
|---|---|---|
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 |
Participation Assistant
Eligibility Criteria
Eligible Ages
Adult, Older Adult
Minimum Age
25 Years
Eligible Sexes
All
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.
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.
Hoffmann-La Roche289 active studies to exploreStudy Central Contact
Contact: Reference Study ID Number: BP45378 https://forpatients.roche.com/, 888-662-6728 (U.S. Only), [email protected]
3 Study Locations in 3 Countries
New South Wales
Westmead Hospital, Westmead, New South Wales, 2145, Australia
Recruiting
New Zealand Brain Research Institute, Christchurch, 8011, New Zealand
Recruiting
Hospital Britanico de Buenos Aires, Ciudad Autonoma Bs As, C1280AEB, Argentina
Recruiting