Trial · NCT03761849
A Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Participants With Manifest Huntington's Disease
NCT03761849, Phase 3, Completed, Hoffmann-La Roche; results first posted 2023-11-07
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Participants With Manifest Huntington's Disease. Trials, record NCT03761849. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03761849
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Participants With Manifest Huntington's Disease
- NCT number
- NCT03761849
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 899 participants, actual
- Lead sponsor
- Hoffmann-La Roche
- Conditions
- Huntingtons Disease
- Interventions
- RO7234292, Placebo
- First posted
- 2018-12-03
- Last updated
- 2024-03-01
- Results first posted
- 2023-11-07
Source ClinicalTrials.gov
Registered outcomes19
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in the Composite Unified Huntington's Disease Rating Scale (cUHDRS) Score-Z Score | Weeks 21 for ODC and 69 for NDC | 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. |
| Primary | Change From Baseline in the Total Functional Capacity (TFC) Score | Weeks 21 for ODC and 69 for NDC | 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. |
| Secondary | Change From Baseline in Total Motor Score (TMS) | Weeks 21 for ODC and 69 for NDC | The TMS score is the sum of the individual motor ratings obtained from administration of the 31-item motor assessment. The score ranges from 0 to 124, with a higher score representing more severe impairment. |
| Secondary | Change From Baseline in Symbol Digit Modalities Test (SDMT) Scores' Least Squares Mean Values | Weeks 21 for ODC and 69 NDC | Symbol Digit Modality Test -SDMT test measures the number of items correctly paired maximum of 110 correct pairs in 90 seconds, more correctly paired items representing less impairment. The differences in LS mean ( +/-SE) change from baseline SDMT score between the active groups compared with the placebo group at Weeks 21 and 69 were reported. A negative change from baseline in the SDMT indicates disease progression. The Least Square Mean values of Symbol Digit Modality Test Scores are reported below. The minimum range for the SDMT scale is 0, indicating highest severity. A max number is not possible as it is a time based task, based on the number of correct answers within a set time frame. There are no validated SDMT score thresholds to indicate the level of HD symptom severity. |
| Secondary | Change From Baseline in Stroop Word Reading (SWR) Test Scores' Least Squares Mean Values | Weeks 21 for ODC and 69 for NDC | Stroop Word Reading-SWR number of words and colors read correctly is counted, with a higher score indicating better cognitive performance scores. There is no upper limit for SWR as it is a time based task. The lower limit (worst possible) however is 0; higher score is better meaning less severity. The differences in LS mean ( +/-SE) change from baseline SWR score between the active groups compared with the placebo group at Weeks 21 and 69 were reported. A negative change from baseline in the SWR indicates disease progression. The Least Square Mean values of Stroop Word Reading (SWR) Test Scores are reported below. |
| Secondary | Change From Baseline in the Clinical Global Impression, Severity Scale (CGI-S) Scores' Least Squares Mean Values | Week 69 for NDC Only | The CGI-S is a single-item measure of current global severity and is completed by the clinician at specified clinic visits. The CGI-S is assessed using an 11-point numeric rating scale (NRS), where higher scores indicate greater severity. Only NDC participants data were reported, all other data were not available. CGI-S) Scores range from 0 (not at all severe) to 10 (Extremely severe); lower score is better meaning less severity. |
| Secondary | Percentage of Patients With a Decrease From Baseline of >=1 Point on the Total Functional Capacity (TFC) Score | Week 69 for NDC only | Only NDC participant data are available and reported. Total Functional Capacity-TFC score ranges from 0 to 13, with a higher score representing better functioning. In this outcome measure, participants with 1 or higher point score decrease from the Baseline TFC Total Score was considered. The Percentage of these participants with such a change was calculated. |
| Secondary | Percentage of Patients With a Decline From Baseline of >=1.2 Points on the Composite Unified Huntington's Disease Rating Scale-cUHDRS Score | Week 69 for NDC Only | Only NDC participant data are available and reported. The cUHDRS is comprised of the sum scores of the subscales, score ranges and severities mentioned in the Outcome Measure Description 1 (please see above). In this outcome measure, participants with 1.2 or higher point score decrease from the Baseline Composite Unified Huntington's Disease Rating Scale- cUHDRS Total Score was considered. The Percentage of these participants with such a change was calculated. cUHDRS lowest (worst) score possible value is -3.06 but no upper limit as it involves SWR; higher score is better meaning less severity. |
| Secondary | Percentage of Patients With an Unchanged or Improved Score on the Clinical Global Impression, Change Scale Score | Weeks 53 and 69 NDC only | The Clinical Global Impression, Change - CGI-C Scale is a single-item measure of change in global status scale and total scores are summed and reported. The CGI-C has 7 response options: "very much worse," "much worse," "minimally worse," "no change," "minimally improved," "much improved," and "very much improved." "Yes", "No" responses collected and total scores are summed and reported below. Percentage of participants who have unchanged or improved scores from the Baseline CGI-C Scores are calculated and reported here. Total CGI-C scores range from 1 (Very much improved) to 7 (Very much worse); lower score is better meaning less severity. Only NDC Arms data were available. Minimum and maximum values are 1 and 7 respectively. |
| Secondary | Percentage of Participants With Adverse Events | Up to 117 Weeks (29 months) |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 registered outcomesOutcomes19
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in the Composite Unified Huntington's Disease Rating Scale (cUHDRS) Score-Z Score | Weeks 21 for ODC and 69 for NDC | ITT Population. Only those participants who contributed to data reported in the table. All other data were not evaluable. | Scores on a Scale | LEAST_SQUARES_MEAN |
| PRIMARY | Change From Baseline in the Total Functional Capacity (TFC) Score | Weeks 21 for ODC and 69 for NDC | ITT Population. Only those participants who contributed to data reported in the table. All other data were not evaluable. | Scores on a Scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Total Motor Score (TMS) | Weeks 21 for ODC and 69 for NDC | ITT Population. Only those participants who contributed to data reported in the table. All other data were not evaluable. | Score on a Scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Symbol Digit Modalities Test (SDMT) Scores' Least Squares Mean Values | Weeks 21 for ODC and 69 NDC | ITT Population. Only those participants who contributed to data reported in the table. All other data were not evaluable. | Scores on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Stroop Word Reading (SWR) Test Scores' Least Squares Mean Values | Weeks 21 for ODC and 69 for NDC | ITT Population. Only those participants who contributed to data reported in the table. All other data were not evaluable. | Scores on a Scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in the Clinical Global Impression, Severity Scale (CGI-S) Scores' Least Squares Mean Values | Week 69 for NDC Only | ITT Population. Only those participants who were contributed data reported in the table. All other data were not evaluable. No data could be reported in ODC Arms due to no data were collected. | Scores on a Scale | LEAST_SQUARES_MEAN |
| SECONDARY | Percentage of Patients With a Decrease From Baseline of >=1 Point on the Total Functional Capacity (TFC) Score | Week 69 for NDC only | ITT Population. Only those participants who contributed to data reported in the table. All other data were not evaluable. No data could be reported in ODC Arms due to no data were collected. | Percentage | NUMBER |
| SECONDARY | Percentage of Patients With a Decline From Baseline of >=1.2 Points on the Composite Unified Huntington's Disease Rating Scale-cUHDRS Score | Week 69 for NDC Only | ITT Population. Only those participants who were contributed data reported in the table. All other data were not evaluable. No data could be reported in ODC Arms due to no data were collected. | Percentage of Participants | NUMBER |
| SECONDARY | Percentage of Patients With an Unchanged or Improved Score on the Clinical Global Impression, Change Scale Score | Weeks 53 and 69 NDC only | ITT Population. Only those participants who were contributed data reported in the table. All other data were not evaluable. No data could be reported in ODC Arms due to no data were collected. | Percentage of Participants | NUMBER |
| SECONDARY | Percentage of Participants With Adverse Events | Up to 117 Weeks (29 months) | Safety Population | Percentage | NUMBER |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 outcomesResults by group186
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in the Composite Unified Huntington's Disease Rating Scale (cUHDRS) Score-Z Score | Placebo ODC | Week 21 | 0.103 | Standard Error 0.193 | Scores on a Scale |
| Change From Baseline in the Composite Unified Huntington's Disease Rating Scale (cUHDRS) Score-Z Score | RO7234292 Q4W ODC | Week 21 | -0.367 | Standard Error 0.189 | Scores on a Scale |
| Change From Baseline in the Composite Unified Huntington's Disease Rating Scale (cUHDRS) Score-Z Score | RO7234292 Q8W ODC | Week 21 | -0.139 | Standard Error 0.197 | Scores on a Scale |
| Change From Baseline in the Composite Unified Huntington's Disease Rating Scale (cUHDRS) Score-Z Score | Tomi 120 mg Q8W NDC | Week 69 | -1.173 | Standard Error 0.091 | Scores on a Scale |
| Change From Baseline in the Composite Unified Huntington's Disease Rating Scale (cUHDRS) Score-Z Score | Tomi 120mg Q16W NDC | Week 69 | -0.793 | Standard Error 0.093 | Scores on a Scale |
| Change From Baseline in the Composite Unified Huntington's Disease Rating Scale (cUHDRS) Score-Z Score | Placebo NDC | Week 69 | -0.630 | Standard Error 0.091 | Scores on a Scale |
| Change From Baseline in the Total Functional Capacity (TFC) Score | Placebo ODC | Week 21 | 0.150 | Standard Error 0.246 | Scores on a Scale |
| Change From Baseline in the Total Functional Capacity (TFC) Score | RO7234292 Q4W ODC | Week 21 | -0.463 | Standard Error 0.242 | Scores on a Scale |
| Change From Baseline in the Total Functional Capacity (TFC) Score | RO7234292 Q8W ODC | Week 21 | -0.460 | Standard Error 0.255 | Scores on a Scale |
| Change From Baseline in the Total Functional Capacity (TFC) Score | Tomi 120 mg Q8W NDC | Week 69 | -1.284 | Standard Error 0.124 | Scores on a Scale |
1 to 10 of 186Source ClinicalTrials.gov
See all 186 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Placebo ODC | 36 | |
| Overall Study | STARTED | RO7234292 Q4W ODC | 36 | |
| Overall Study | STARTED | RO7234292 Q8W ODC | 36 | |
| Overall Study | STARTED | Tomi 120 mg Q8W NDC | 263 | |
| Overall Study | STARTED | Tomi 120mg Q16W NDC | 264 | |
| Overall Study | STARTED | Placebo NDC | 264 | |
| Overall Study | COMPLETED | Placebo ODC | 0 | |
| Overall Study | COMPLETED | RO7234292 Q4W ODC | 0 | |
| Overall Study | COMPLETED | RO7234292 Q8W ODC | 0 | |
| Overall Study | COMPLETED | Tomi 120 mg Q8W NDC | 216 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events864
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Diarrhoea | Gastrointestinal disorders | PLB ODC | 2 | 36 | 2 |
| other | Diarrhoea | Gastrointestinal disorders | Q4W ODC | 2 | 36 | 3 |
| other | Diarrhoea | Gastrointestinal disorders | Q8W ODC | 2 | 36 | 2 |
| other | Diarrhoea | Gastrointestinal disorders | Q8W NDC | 17 | 263 | 20 |
| other | Diarrhoea | Gastrointestinal disorders | Q16W NDC | 15 | 264 | 16 |
| other | Diarrhoea | Gastrointestinal disorders | PLB NDC | 19 | 264 | 20 |
| other | Nausea | Gastrointestinal disorders | PLB ODC | 1 | 36 | 1 |
| other | Nausea | Gastrointestinal disorders | Q4W ODC | 1 | 36 | 1 |
| other | Nausea | Gastrointestinal disorders | Q8W ODC | 0 | 36 | 0 |
| other | Nausea | Gastrointestinal disorders | Q8W NDC | 12 | 263 | 14 |
1 to 10 of 864Source ClinicalTrials.gov
See all 864 adverse eventsAdverse event totals18
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | PLB ODC | 0 | 36 |
| Total, all-cause mortality | Q4W ODC | 0 | 36 |
| Total, all-cause mortality | Q8W ODC | 0 | 36 |
| Total, all-cause mortality | Q8W NDC | 1 | 263 |
| Total, all-cause mortality | Q16W NDC | 2 | 264 |
| Total, all-cause mortality | PLB NDC | 3 | 264 |
| Total, other adverse events | PLB ODC | 24 | 36 |
| Total, other adverse events | Q4W ODC | 23 | 36 |
| Total, other adverse events | Q8W ODC | 19 | 36 |
| Total, other adverse events | Q8W NDC | 204 | 263 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 adverse event totalsPapers3
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests1
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
| Test | Named as |
|---|---|
| Symbol Coding | Secondary outcome |
Source ConductCognition
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03761849
GET https://conductscience.org/api/trials/v1/NCT03761849/results?page=2