Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
RO7234292 · 4 trials · 3 indications
The reported are the treatment-emergent AEs with an onset date up to 5 months after last study drug intake.
C-SSRS is to assess suicidal ideation and behavior. 4 constructs measured: severity of ideation, intensity of ideation, behavior, and lethality of actual suicide attempts. Yes/No data collected for 10 categories, composite endpoints based on the categories are followed over time to monitor safety. Composite endpoint of suicidal ideation (1-5), n and (%) are the number and % of who experience any of the five suicidal ideation events at least once after receiving the first dose of study medication. Composite endpoint of suicidal behavior (6-10), n and (%) are the number and percent of patients who experience any 1of 5 suicidal behavior events at least 1 after receiving the 5 dose of study medication. Composite endpoint of suicidal ideation or behavior (1-10), n and (%) are the number and % of patients who experience any 1 the 10 suicidal ideation or behavior events at least once after receiving the first dose of study medication.
MoCA contains a series of basic assessments, including attention and visuospatial tasks. The total score ranges from 0-30, where lower scores indicate greater impairment. MOCA01-Total scores are reported. The data presented are absolute scores for baseline and change from baseline for post-baseline assessments. All Arms except Tominersen 120mg Q4W (Period 1) belong to the Milestone Period 2.
cUHDRS includes the Total Functional Capacity (range, 0-13; higher score means better functioning), Total Motor Score (range, 0-124; higher score means worse motor severity), Symbol Digit Modality Test (range, 0-110, correctly paired numbers-symbols in 90 seconds; higher score means better cognitive performance), and Stroop Word Reading (range, 0-no max value, correctly read colour words in 45 seconds; higher score means better cognitive performance) scores. A z-score for each test is calculated, which alone can be used to describe relationship between an individual's test score and the mean score of a target population. A z-score of 0 is the mean, and ±1 is 1 standard deviation from the mean. For cUHDRS, z-scores of each test are summed, whereby a higher cUHDRS score is better (score of -3.06-no max value) and a change of ≥1.2 is a meaningful worsening, shown to track functional decline.
Total Functional Capacity (TFC) Scores are reported at Weeks 21 and 69. Total Functional Capacity Score ranges from 0 to 13, with a higher score representing better functioning.
Adverse Events include Adverse Events that started at or after Date/Time of First Exposure to Treatment and procedure-related Adverse Events occurring before the start of treatment.
NA represents: insufficient number of participants with events.
NA represents:insufficient number of participants with events
CSF Mutant Huntingtin Protein (fmol/L) values at time point visits are reported.
| Arm | Type | Description |
|---|---|---|
| RO7234292 (RG6042) Q8W | EXPERIMENTAL | Participants who received open-label RO7234292 Q4W in a preceding study or who received RO7234292 Q4W in this study may be randomly allocated to receive RO7234292 Q8W. Participants who previously received open-label of RO7234292 Q8W in a preceding study or are currently receiving RO7234292 Q8W in this study will receive RO7234292 Q8W. Participants who previously received placebo, or did not previously receive treatment with RO7234292 or received short-term treatment with a treatment-free follow-up period may be randomly allocated to receive RO7234292 Q8W. Participants who previously received blinded placebo Q8W may receive RO7234292 Q8W. Participants who received blinded RO7234292 Q8W will receive open-label RO7234929 Q8W. Participants who received blinded placebo Q8W may receive RO7234292 Q8W. Participants who received blinded RO7234292 Q4W or blinded placebo Q4W may be randomly allocated to receive open-label of RO7234292 Q8W. |
| RO7234292 (RG6042) Q16W | EXPERIMENTAL | Participants who previously received open-label RO7234292 Q4W in a preceding study or who received RO7234292 Q4W in this study may be randomly allocated to receive RO7234292 Q16W. Participants who previously received placebo in a preceding study or did not previously receive treatment with RO7234292 or received short-term treatment with a treatment-free follow-up period may be randomly allocated to receive RO7234292 Q16W. Participants who previously received blinded placebo Q8W will receive RO7234292 Q8W. Participants who previously received blinded RO7234292 Q16W will receive open-label RO7234292 Q16W. Participants who received blinded RO7234292 Q4W or blinded placebo Q4W may be randomly allocated to receive open-label of RO7234292 Q16W. Participants who previously received open-label RO7234292 Q16W will receive open-label RO7234292 Q16W. |
| RO7234292 Q8W | EXPERIMENTAL | RO4234292 is administered intrathecally every 8 weeks. |
| RO7234292 Q16W | EXPERIMENTAL | RO7234292 is administered intrathecally every 16 weeks. Participants in this arm will also receive placebo at alternate weeks to keep the blind. |
| Placebo | PLACEBO_COMPARATOR | Placebo will be administered every 8 weeks by IT injection. |
| RO7234292 Monthly | EXPERIMENTAL | RO7234292 is administered every 28 days intrathecally for 14 months. |
| RO7234292 Bimonthly | EXPERIMENTAL | RO7234292 is administered every 56 days intrathecally for 14 months following 2 monthly doses to serve as a loading dose. |
| Dose level 1 of RO7234292 (RG6042) | EXPERIMENTAL | Participants will receive dose level 1 of RO7234292 (RG6042) intrathecally on Day 1 and Day 29. |
| Dose level 2 of RO7234292 (RG6042) | EXPERIMENTAL | Participants will receive dose level 2 of RO7234292 (RG6042) intrathecally on Day 1 and Day 29. |
| Dose level 3 of RO7234292 (RG6042) | EXPERIMENTAL | Participants will receive dose level 3 of RO7234292 (RG6042) intrathecally on Day 1 and Day 29. |
| Name | Type | Description |
|---|---|---|
| RO7234292 (RG6042) | DRUG | Intrathecal injection |
| RO7234292 | DRUG | Intrathecal injection |
| Placebo | DRUG | Intrathecal injection |
Inclusion Criteria: * Prior enrollment in a Roche-sponsored or Genentech-sponsored study in HD for the RO7234292 (RG6042) development program that made provision for entry into an OLE study * For women of childbearing potential: agreement to remain abstinent or use contraceptive measures * For men:...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novartis AG Sponsored ADR | NVS | 2 | PHASE3 | Votoplam |
| Neurocrine Biosciences, Inc. | NBIX | 1 | PHASE3 | Valbenazine |
| Alnylam Pharmaceuticals, Inc | ALNY | 1 | PHASE1 | ALN-HTT02 |
| uniQure N.V. | QURE | 2 | PHASE1 | intra-striatal rAAV5-miHTT |
| Sarepta Therapeutics, Inc. | SRPT | 1 | PHASE1 | SRP-1005 |
RO7234292 is an investigational small molecule being developed for Huntington's disease, a progressive neurological disorder. It is administered intrathecally and is currently in clinical development, though no regulatory approval has been granted. The drug has been studied in patients with manifest Huntington's disease.
RO7234292 is being developed by Roche Holding AG, traded on the OTC market under the ticker RHHBY. The drug is an investigational therapy for Huntington's disease and has not received regulatory approval. Roche has sponsored multiple clinical trials evaluating its safety and efficacy.
RO7234292 is in Phase 3 clinical development for Huntington's disease. It has completed two Phase 3 trials, including a large efficacy study with 899 participants. However, the drug remains investigational and has not been approved by regulatory authorities.
RO7234292 has been studied in several completed trials, including NCT03761849, a Phase 3 efficacy study in 899 patients with manifest Huntington's disease, and NCT03342053, a Phase 2 rollover study. Additional trials include NCT03842969, an open-label extension, and NCT04000594, a Phase 1 pharmacokinetic study.
Yes, RO7234292 is also known as RG6042 and ISIS 443139. These names refer to the same investigational drug developed by Roche for Huntington's disease. In clinical trial records, the drug is sometimes listed under these alternative identifiers.