Clinical trial NCT03342053: 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)
Official title: 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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Huntington's Disease
Lead sponsor: Hoffmann-La Roche
Sponsor class: INDUSTRY
First posted: 2017-11-14
Enrollment: 46
Sex: ALL
Minimum age (years): 25
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study will test the safety, tolerability, pharmacokinetics and pharmacodynamics of RO7234292 administered intrathecally to adult patients with Huntington's Disease.


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

Primary outcomes:
measure: Percentage of Participants With Adverse Events
timeFrame: From baseline up to 18 months
Secondary outcomes:
measure: RO7234292 CSF Trough Concentrations by Study Day Prior to Monthly and Bimonthly IT Administration of 120 mg RO7234292 (Primary Analysis)
timeFrame: From baseline to Day 421
measure: CSF mHTT Protein Concentration Logarithmic Value Change in Geometric Mean (95%CI) From Baseline
timeFrame: From Baseline to Day 421
measure: Mean Percentage Change in Ventricular Volume Boundary Shift Integral From Baseline to 15 Months
timeFrame: Baseline up to 15 months
measure: Mean Percentage Change in Caudate Volume Boundary Shift Integral From Baseline to 15 Months
timeFrame: Baseline up to 15 months
measure: Mean Percentage Change in Whole Brain Volume Boundary Shift Integral From Baseline to 15 Months
timeFrame: Baseline up to 15 months
measure: EEG Parameters: Mean Change From Baseline to 15 Months in Absolute Power [8-12Hz]
timeFrame: Baseline to 15 Months
measure: Mean Change From Baseline in Huntington's Disease Cognitive Assessment Battery Composite Score
timeFrame: Baseline to 15 Months
Other outcomes:
Locations:
city: Vancouver
country: Canada
facility: The University of British Columbia; The Centre for Huntington Disease
state: British Columbia
city: Berlin
country: Germany
facility: Charité - Universitätsmedizin Berlin, Campus Charité Mitte; Klinik für Psychiatrie und Psychotherapi
city: Bochum
country: Germany
facility: St. Josef and St. Elisabeth GmbH ; Klinikum Bochum, Zentralapotheke
city: Ulm
country: Germany
facility: Universitaetsklinikum Ulm
city: Birmingham
country: United Kingdom
facility: NIHR Welcome Trust Birmingham CRF - University Hospitals Birmingham; Department of Neuropsychiatry
city: Cambridge
country: United Kingdom
facility: University of Cambridge - John van Geest Centre for Brain Repair
city: Cardiff
country: United Kingdom
facility: Cardiff University School of Medicine; Institute of Psychological Medicine Clinical Neurosciences
city: London
country: United Kingdom
facility: Leonard Wolfson Experimental Neurology Centre
city: Manchester
country: United Kingdom
facility: Central Manchester University Hospitals NHS Foundation Trust; Manchester Centre for Genomic Medicine
Study officials:
affiliation: Hoffmann-La Roche
name: Clinical Trials
role: STUDY_DIRECTOR
References: