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)
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ClinicalTrials.gov Identifier: NCT03342053 |
Recruitment Status :
Completed
First Posted : November 14, 2017
Results First Posted : January 19, 2021
Last Update Posted : March 24, 2022
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Huntington's Disease | Drug: RO7234292 (RG6042) | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 46 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
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) |
Actual Study Start Date : | January 2, 2018 |
Actual Primary Completion Date : | October 8, 2019 |
Actual Study Completion Date : | October 8, 2019 |

Arm | Intervention/treatment |
---|---|
Experimental: RO7234292 Monthly
RO7234292 is administered every 28 days intrathecally for 14 months.
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Drug: RO7234292 (RG6042)
Intrathecal injection
Other Name: Tominersen |
Experimental: RO7234292 Bimonthly
RO7234292 is administered every 56 days intrathecally for 14 months following 2 monthly doses to serve as a loading dose.
|
Drug: RO7234292 (RG6042)
Intrathecal injection
Other Name: Tominersen |
- 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.
- RO7234292 CSF Trough Concentrations by Study Day Prior to Monthly and Bimonthly IT Administration of 120 mg RO7234292 (Primary Analysis) [ Time Frame: From baseline to Day 421 ]
- CSF mHTT Protein Concentration Logarithmic Value Change in Geometric Mean (95%CI) From Baseline [ Time Frame: From Baseline to Day 421 ]The results of the planned analysis related to mHTT protein levels in CSF are reported
- Mean Percentage Change in Ventricular Volume Boundary Shift Integral From Baseline to 15 Months [ Time Frame: Baseline up to 15 months ]
- Mean Percentage Change in Caudate Volume Boundary Shift Integral From Baseline to 15 Months [ Time Frame: Baseline up to 15 months ]
- Mean Percentage Change in Whole Brain Volume Boundary Shift Integral From Baseline to 15 Months [ Time Frame: Baseline up to 15 months ]
- EEG Parameters: Mean Change From Baseline to 15 Months in Absolute Power [8-12Hz] [ Time Frame: Baseline to 15 Months ]
- Mean Change From Baseline in Huntington's Disease Cognitive Assessment Battery Composite Score [ Time Frame: Baseline to 15 Months ]HD Cognitive Assessment Battery (HD-CAB) was developed to assess cognitive dysfunction in late premanifest and early manifest HD patients. HD-CAB combines scores from six cognitive tests: SDMT, Self-Paced Tapping, Emotional Recognition, CANTAB One Touch Stocking, Hopkins Verbal Learning Test - Revised, and Trail-Making Test. A multi-component score is derived by transforming the subject's score on each cognitive test to a z-score. Using z-scores permits the combination of test scores with different scales. Unlike other measures that use an external reference population to create z-scores, HD-CAB uses the baseline data of the study. Individually, for each of the six cognitive tests, the study baseline mean is subtracted from the subject's score, and this value is divided by the study baseline standard deviation. The six z-scores are averaged to produce the HD-CAB score. A positive change from baseline indicates improvement in cognitive function; a negative change indicates worsening.

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Ages Eligible for Study: | 25 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03342053
Canada, British Columbia | |
The University of British Columbia; The Centre for Huntington Disease | |
Vancouver, British Columbia, Canada, V6T 2B5 | |
Germany | |
Charité - Universitätsmedizin Berlin, Campus Charité Mitte; Klinik für Psychiatrie und Psychotherapi | |
Berlin, Germany, 10117 | |
St. Josef and St. Elisabeth GmbH ; Klinikum Bochum, Zentralapotheke | |
Bochum, Germany, 44791 | |
Universitaetsklinikum Ulm | |
Ulm, Germany, 89081 | |
United Kingdom | |
NIHR Welcome Trust Birmingham CRF - University Hospitals Birmingham; Department of Neuropsychiatry | |
Birmingham, United Kingdom, B15 2FG | |
University of Cambridge - John van Geest Centre for Brain Repair | |
Cambridge, United Kingdom, CB2 0PY | |
Cardiff University School of Medicine; Institute of Psychological Medicine Clinical Neurosciences | |
Cardiff, United Kingdom, CF24 4HQ | |
Leonard Wolfson Experimental Neurology Centre | |
London, United Kingdom, WC1N 3BG | |
Central Manchester University Hospitals NHS Foundation Trust; Manchester Centre for Genomic Medicine | |
Manchester, United Kingdom, M13 9WL |
Study Director: | Clinical Trials | Hoffmann-La Roche |
Documents provided by Hoffmann-La Roche:
Responsible Party: | Hoffmann-La Roche |
ClinicalTrials.gov Identifier: | NCT03342053 |
Other Study ID Numbers: |
BN40697 |
First Posted: | November 14, 2017 Key Record Dates |
Results First Posted: | January 19, 2021 |
Last Update Posted: | March 24, 2022 |
Last Verified: | March 2022 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Product Manufactured in and Exported from the U.S.: | Yes |
Huntington Disease Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Nervous System Diseases Dementia Chorea Dyskinesias |
Movement Disorders Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Genetic Diseases, Inborn Cognition Disorders Neurocognitive Disorders Mental Disorders |