Phase 3
N=899
A Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Participants With Manifest Huntington's Disease
Huntingtons Disease
Bottom Line
View on ClinicalTrials.gov: NCT03761849 ↗Enrolled (actual)
899
Serious AEs
12.9%
Results posted
Nov 2023
Primary outcome: Primary: Change From Baseline in the Composite Unified Huntington's Disease Rating Scale (cUHDRS) Score-Z Score — 0.103; -0.367; -0.139; -1.173 Scores on a Scale
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- RO7234292 (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 25+ yrs
- Sex
- All
- Sponsor
- Hoffmann-La Roche
- Primary completion
- Mar 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in the Composite Unified Huntington's Disease Rating Scale (cUHDRS) Score-Z Score |
0.103; -0.367; -0.139; -1.173; -0.793; -0.630 | — |
| PRIMARY Change From Baseline in the Total Functional Capacity (TFC) Score |
0.150; -0.463; -0.460; -1.284; -0.921; -0.883 | — |
| SECONDARY Change From Baseline in Total Motor Score (TMS) |
-1.977; 2.570; 1.493; 4.028; 3.524; 3.513 | — |
| SECONDARY Change From Baseline in Symbol Digit Modalities Test (SDMT) Scores' Least Squares Mean Values |
1.865; -1.166; 1.204; -2.641; -0.996; -0.216 | — |
| SECONDARY Change From Baseline in Stroop Word Reading (SWR) Test Scores' Least Squares Mean Values |
-1.397; 0.469; -1.036; -5.224; -3.949; -2.555 | — |
| SECONDARY Change From Baseline in the Clinical Global Impression, Severity Scale (CGI-S) Scores' Least Squares Mean Values |
0.662; 0.640; 0.545 | — |
| SECONDARY Percentage of Patients With a Decrease From Baseline of >=1 Point on the Total Functional Capacity (TFC) Score |
55.7; 50.3; 49.5; 44.3; 49.7; 43.7 | — |
| SECONDARY Percentage of Patients With a Decline From Baseline of >=1.2 Points on the Composite Unified Huntington's Disease Rating Scale-cUHDRS Score |
44.5; 32.1; 29.7; 55.5; 67.9; 70.3 | — |
| SECONDARY Percentage of Patients With an Unchanged or Improved Score on the Clinical Global Impression, Change Scale Score |
55.4; 59.6; 56.3; 44.6; 40.4; 43.7 | — |
| SECONDARY Percentage of Participants With Adverse Events |
77.8; 80.0; 61.1; 92.3; 90.0; 93.1 | — |
| SECONDARY Change From Baseline in Montreal Cognitive Assessment (MoCA) |
-0.07; 0.10; 0.33; 0.44; 0.39; 0.45 | — |
| SECONDARY Percentage of Participants With Suicidal Ideation or Behavior, as Assessed by Columbia-Suicide Severity Rating Scale (C-SSRS) Score |
0; 8.6; 2.8; 9.7; 11.2; 10.4 | — |
| SECONDARY Concentration of RO7234292 in Plasma |
0.511; 0.203; 0.20; 0.06; 1.63; 0.28 | — |
| SECONDARY Trough Concentration of RO7234292 in Cerebrospinal Fluid (CSF) |
4.44; 1.51; 1.54; 0.70; 1.74; 0.55 | — |
| SECONDARY Incidence of Anti-Drug Antibodies (ADAs). |
94.3; 91.7; 91.4; 91.7; 2.9; 0 | — |
| SECONDARY Titer and Antibody Subtype, Determined if ADAs Are Identified |
— | — |
| SECONDARY Change From Baseline in CSF mHTT Protein Level |
— | — |
| SECONDARY Change From Baseline in Whole and Regional Brain Volumes, as Detrmined by Structural Magnetic Resonance Imaging (MRI) |
0.996; 0.330; 1.124; 1.537; 3.785; 2.011 | — |
| SECONDARY Change From Baseline in CSF Neurofilament Light Chain (NfL) Proteint Level |
12.233; 34.548; 16.553; 31.893; 5.406; 3.365 | — |
Summary
This study will evaluate the efficacy, safety, and biomarker effects of RO7234292 (RG6042) compared with placebo in participants with manifest Huntington's disease (HD)
Eligibility Criteria
Inclusion Criteria
- Manifest HD diagnosis, defined as a DCL score of 4
- Independence Scale (IS) score >= 70
- Genetically confirmed disease by direct DNA testing with a CAP score >400
- Clinical assessment to ensure individual has intact functional independence at baseline to maintain self-care and core activities of daily living (ADLs).
Exclusion Criteria
- Any serious medical condition or clinically significant laboratory, or vital sign abnormality or claustrophobia at screening that, in the investigator's judgment, precludes the patient's safe participation in and completion of the study
- Pregnant or breastfeeding, or intending to become pregnant during the study or within 5 months after the final dose of study drug
Data sourced from ClinicalTrials.gov (NCT03761849). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.