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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)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03342053
Recruitment Status : Completed
First Posted : November 14, 2017
Last Update Posted : March 9, 2020
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 : November 27, 2017
Actual Primary Completion Date : October 8, 2019
Actual Study Completion Date : October 8, 2019

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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 including 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. Incidence of treatment-emergent adverse events [safety and tolerability] [ Time Frame: Participants will be followed for the duration of the study; an expected 74 weeks ]
    The number of participants with adverse events


Secondary Outcome Measures :
  1. CSF Huntingtin Protein Concentration (fM) [ Time Frame: 74 weeks ]
  2. Ventricular Volume (mL) [ Time Frame: 74 Weeks ]
  3. Quantitative Electroencephalography [ Time Frame: 74 weeks ]
  4. Huntington's Disease Cognitive Assessment Battery Composite Score (unitless) [ Time Frame: 74 weeks ]

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
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
Last Update Posted: March 9, 2020
Last Verified: March 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
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