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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
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

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

Condition or disease Intervention/treatment Phase
Huntington's Disease Drug: RO7234292 (RG6042) Phase 2

Study Design
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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

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: RO7234292 Monthly
RO7234292 is administered every 28 days intrathecally for 14 months.
 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


Outcome Measures
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Primary Outcome Measures :
  1. 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 Outcome Measures :
  1. 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 ]
  2. 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

  3. Mean Percentage Change in Ventricular Volume Boundary Shift Integral From Baseline to 15 Months [ Time Frame: Baseline up to 15 months ]
  4. Mean Percentage Change in Caudate Volume Boundary Shift Integral From Baseline to 15 Months [ Time Frame: Baseline up to 15 months ]
  5. Mean Percentage Change in Whole Brain Volume Boundary Shift Integral From Baseline to 15 Months [ Time Frame: Baseline up to 15 months ]
  6. EEG Parameters: Mean Change From Baseline to 15 Months in Absolute Power [8-12Hz] [ Time Frame: Baseline to 15 Months ]
  7. 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.


Eligibility Criteria
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Ages Eligible for Study:   25 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

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


Locations
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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
Sponsors and Collaborators
Hoffmann-La Roche
Investigators
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Study Director: Clinical Trials Hoffmann-La Roche
  Study Documents (Full-Text)

Documents provided by Hoffmann-La Roche:
Study Protocol and Statistical Analysis Plan  [PDF] August 30, 2018

More Information
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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

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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
Additional relevant MeSH terms:
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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