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)

Phase 2CompletedNCT03342053

Hoffmann-La Roche46 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Huntington's Disease

Interventions

RO7234292 (RG6042)DRUG

Intrathecal injection

Eligibility

Sex: ALLMin age: 25 Years

Medical Language ↔ Plain English

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

Locations (9)

The University of British Columbia; The Centre for Huntington Disease

Vancouver, British Columbia, Canada

Charité - Universitätsmedizin Berlin, Campus Charité Mitte; Klinik für Psychiatrie und Psychotherapi

Berlin, Germany

St. Josef and St. Elisabeth GmbH ; Klinikum Bochum, Zentralapotheke

Bochum, Germany

Outcomes

Primary Outcomes

Percentage of Participants With Adverse Events

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.

Time frame: From baseline up to 18 months

Secondary Outcomes

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

The results of the planned analysis related to mHTT protein levels in CSF are reported

Time frame: From Baseline to Day 421

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

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.

Data from ClinicalTrials.gov

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