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Completed

NCT Number: 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)

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

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Key information

Conditions

Huntington's Disease Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Chorea Cognition Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dementia Dyskinesias Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Huntington Disease Mental Disorders Movement Disorders Nervous System Diseases Neurocognitive Disorders Neurodegenerative Diseases

Age range

25 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

The University of British Columbia; The Centre for Huntington Disease, Vancouver, British Columbia, Canada

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

RO7234292 (RG6042)

Drug

Intrathecal injection

Other names: Tominersen

Primary outcomes

  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 outcomes

  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.

Sponsors and collaborators

Lead sponsor

Hoffmann-La Roche

Industry

Registry information

Official study 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)

Important dates

Study start
2018
Primary completion
2019
Study completion
2019
First posted
Nov 14, 2017
Registry last updated
Mar 24, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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