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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 : January 19, 2021
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

Tracking Information
First Submitted Date  ICMJE October 31, 2017
First Posted Date  ICMJE November 14, 2017
Results First Submitted Date  ICMJE October 7, 2020
Results First Posted Date  ICMJE January 19, 2021
Last Update Posted Date January 19, 2021
Actual Study Start Date  ICMJE January 2, 2018
Actual Primary Completion Date October 8, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 8, 2020)
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.
Original Primary Outcome Measures  ICMJE
 (submitted: November 13, 2017)
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
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 15, 2021)
  • 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 [ Time Frame: Baseline up to 15 months ]
    Data on mHTT protein levels in CSF were not available at the time of writing as validation of the mHTT assay in a regulated environment was ongoing and sample analysis was pending successful validation of the assay. results of the planned analysis related to mHTT protein levels in CSF will be reported once available
  • 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.
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures
 (submitted: November 13, 2017)
  • CSF huntingtin protein concentration (fM) [ Time Frame: 74 weeks ]
  • Ventricular volume (mL) [ Time Frame: 74 weeks ]
  • Huntington's disease cognitive assessment battery composite score (unitless) [ Time Frame: 74 weeks ]
 
Descriptive Information
Brief Title  ICMJE 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  ICMJE 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)
Brief Summary This study will test the safety, tolerability, pharmacokinetics and pharmacodynamics of RO7234292 administered intrathecally to adult patients with Huntington's Disease.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Huntington's Disease
Intervention  ICMJE Drug: RO7234292 (RG6042)
Intrathecal injection
Other Name: Tominersen
Study Arms  ICMJE
  • Experimental: RO7234292 Monthly
    RO7234292 is administered every 28 days intrathecally for 14 months.
    Intervention: Drug: RO7234292 (RG6042)
  • Experimental: RO7234292 Bimonthly
    RO7234292 is administered every 56 days intrathecally for 14 months following 2 monthly doses to serve as a loading dose.
    Intervention: Drug: RO7234292 (RG6042)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: November 13, 2017)
46
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE October 8, 2019
Actual Primary Completion Date October 8, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 25 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada,   Germany,   United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03342053
Other Study ID Numbers  ICMJE BN40697
Has Data Monitoring Committee No
U.S. FDA-regulated Product
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
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Hoffmann-La Roche
Study Sponsor  ICMJE Hoffmann-La Roche
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Clinical Trials Hoffmann-La Roche
PRS Account Hoffmann-La Roche
Verification Date January 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP