NCT03342053CompletedPhase 2
An Open-Label Extension 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)
Conditions
Interventions
Drugs
Trial Snapshot
- Phase
- Phase 2
- Status
- Completed
- Sponsor
- Hoffmann-La Roche
- Enrollment
- 46
- Locations
- 9
- Primary Endpoint
- Percentage of Participants With Adverse Events
Study Overview
Brief Summary
This study will test the safety, tolerability, pharmacokinetics and pharmacodynamics of RO7234292 administered intrathecally to adult patients with Huntington's Disease.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 25 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Must have completed dosing in ISIS 443139-CS1
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
Arms & Interventions
RO7234292 Monthly
Experimental
RO7234292 is administered every 28 days intrathecally for 14 months.
Intervention: RO7234292 (RG6042) (Drug)
RO7234292 Bimonthly
Experimental
RO7234292 is administered every 56 days intrathecally for 14 months following 2 monthly doses to serve as a loading dose.
Intervention: RO7234292 (RG6042) (Drug)
Outcomes
Primary Outcomes
Percentage of Participants With Adverse Events
Time Frame: From baseline up to 18 months
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.
Secondary Outcomes
- RO7234292 CSF Trough Concentrations by Study Day Prior to Monthly and Bimonthly IT Administration of 120 mg RO7234292 (Primary Analysis)(From baseline to Day 421)
- Mean Percentage Change in Caudate Volume Boundary Shift Integral From Baseline to 15 Months(Baseline up to 15 months)
- Mean Percentage Change in Whole Brain Volume Boundary Shift Integral From Baseline to 15 Months(Baseline up to 15 months)
- CSF mHTT Protein Concentration Logarithmic Value Change in Geometric Mean (95%CI) From Baseline(From Baseline to Day 421)
- Mean Percentage Change in Ventricular Volume Boundary Shift Integral From Baseline to 15 Months(Baseline up to 15 months)
- EEG Parameters: Mean Change From Baseline to 15 Months in Absolute Power [8-12Hz](Baseline to 15 Months)
- Mean Change From Baseline in Huntington's Disease Cognitive Assessment Battery Composite Score(Baseline to 15 Months)
Investigators
Study Sites (9)
Loading locations...
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