Phase 2
Completed N=46
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RO7234292 (ISIS 443139) in Huntington's Disease Patients Who Participated in Prior Investigational Studies of RO7234292 (ISIS 443139)
Source: ClinicalTrials.gov NCT03342053 ↗Enrolled (actual)
46
Serious AEs
15.2%
Results posted
Jan 2021
Primary outcomePrimary: Percentage of Participants With Adverse Events — 100; 95.7 Percentage of participants
Summary
This study will test the safety, tolerability, pharmacokinetics and pharmacodynamics of RO7234292 administered intrathecally to adult patients with Huntington's Disease.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With Adverse Events |
100; 95.7 | — |
| SECONDARY RO7234292 CSF Trough Concentrations by Study Day Prior to Monthly and Bimonthly IT Administration of 120 mg RO7234292 (Primary Analysis) |
2.59; 2.52; 3.47; 3.70; 1.39; 3.97 | — |
| SECONDARY CSF mHTT Protein Concentration Logarithmic Value Change in Geometric Mean (95%CI) From Baseline |
-27.95; -21.84; -26.90; 10.11; -50.30; -32.63 | — |
| SECONDARY Mean Percentage Change in Ventricular Volume Boundary Shift Integral From Baseline to 15 Months |
46.09; 18.77 | — |
| SECONDARY Mean Percentage Change in Caudate Volume Boundary Shift Integral From Baseline to 15 Months |
8.64; 5.67 | — |
| SECONDARY Mean Percentage Change in Whole Brain Volume Boundary Shift Integral From Baseline to 15 Months |
1.63; 0.89 | — |
| SECONDARY EEG Parameters: Mean Change From Baseline to 15 Months in Absolute Power [8-12Hz] |
0.11; 0.02 | — |
| SECONDARY Mean Change From Baseline in Huntington's Disease Cognitive Assessment Battery Composite Score |
-0.33; -0.15 | — |
Eligibility Criteria
Key Inclusion Criteria
- Must have completed dosing in ISIS 443139-CS1
Key Exclusion Criteria
- Any new condition or worsening of existing condition that could make the patient unsuitable for participation or interfere with the patient participating in and/or completing the study
Data sourced from ClinicalTrials.gov (NCT03342053). 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. Informational only — not medical advice.