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Tests treatment safety and results for Huntington's Disease
Official title A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RG6496 in Huntington's Disease
ClinicalTrials.gov ID: NCT07246941
What this study is testing
What is RG6496?
RG6496 is an investigational medicine, being studied as a potential treatment for huntington's disease.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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)].
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 25 to 65
You may be able to join if
- 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...
You likely can't join if
- Part 1
- Concurrent or planned participation in any treatment clinical study, including current use of an ASO or any HTT-lowering therapy or treatment with...
- 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
See the full eligibility 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.
- Part 1
- Concurrent or planned participation in any treatment 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...
- 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 treatment 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.
The study team makes the final eligibility decision.
Where it's taking place
- Ciudad Autonoma Bs As, Argentina
- Westmead, New South Wales, Australia
- Clayton, Victoria, Australia
- Parkville, Victoria, Australia
- Christchurch, New Zealand
- Cambridge, Cambridgeshire, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 25 years to 65 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Ciudad Autonoma Bs As, Argentina; Westmead, New South Wales, Australia; Clayton, Victoria, Australia; Parkville, Victoria, Australia; Christchurch, New Zealand; Cambridge, Cambridgeshire, United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.