NCT03342053

Brief Summary

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

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
46

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Jan 2018

Geographic Reach
3 countries

9 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

October 31, 2017

Completed
14 days until next milestone

First Posted

Study publicly available on registry

November 14, 2017

Completed
2 months until next milestone

Study Start

First participant enrolled

January 2, 2018

Completed
1.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 8, 2019

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 8, 2019

Completed
1.3 years until next milestone

Results Posted

Study results publicly available

January 19, 2021

Completed
Last Updated

March 24, 2022

Status Verified

March 1, 2022

Enrollment Period

1.8 years

First QC Date

October 31, 2017

Results QC Date

October 7, 2020

Last Update Submit

March 1, 2022

Conditions

Huntington's Disease

Outcome Measures

Primary Outcomes (1)

  • 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.

    From baseline up to 18 months

Secondary Outcomes (7)

  • RO7234292 CSF Trough Concentrations by Study Day Prior to Monthly and Bimonthly IT Administration of 120 mg RO7234292 (Primary Analysis)

    From baseline to Day 421

  • CSF mHTT Protein Concentration Logarithmic Value Change in Geometric Mean (95%CI) From Baseline

    From Baseline to Day 421

  • Mean Percentage Change in Ventricular Volume Boundary Shift Integral From Baseline to 15 Months

    Baseline up to 15 months

  • Mean Percentage Change in Caudate Volume Boundary Shift Integral From Baseline to 15 Months

    Baseline up to 15 months

  • Mean Percentage Change in Whole Brain Volume Boundary Shift Integral From Baseline to 15 Months

    Baseline up to 15 months

  • +2 more secondary outcomes

Study Arms (2)

RO7234292 Monthly

EXPERIMENTAL

RO7234292 is administered every 28 days intrathecally for 14 months.

Drug: RO7234292 (RG6042)

RO7234292 Bimonthly

EXPERIMENTAL

RO7234292 is administered every 56 days intrathecally for 14 months following 2 monthly doses to serve as a loading dose.

Drug: RO7234292 (RG6042)

Interventions

RO7234292 (RG6042)DRUG

Intrathecal injection

Also known as: Tominersen
RO7234292 BimonthlyRO7234292 Monthly

Eligibility Criteria

Age25 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Must have completed dosing in ISIS 443139-CS1

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (9)

The University of British Columbia; The Centre for Huntington Disease

Vancouver, British Columbia, V6T 2B5, Canada

Location

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

Berlin, 10117, Germany

Location

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

Bochum, 44791, Germany

Location

Universitaetsklinikum Ulm

Ulm, 89081, Germany

Location

NIHR Welcome Trust Birmingham CRF - University Hospitals Birmingham; Department of Neuropsychiatry

Birmingham, B15 2FG, United Kingdom

Location

University of Cambridge - John van Geest Centre for Brain Repair

Cambridge, CB2 0PY, United Kingdom

Location

Cardiff University School of Medicine; Institute of Psychological Medicine Clinical Neurosciences

Cardiff, CF24 4HQ, United Kingdom

Location

Leonard Wolfson Experimental Neurology Centre

London, WC1N 3BG, United Kingdom

Location

Central Manchester University Hospitals NHS Foundation Trust; Manchester Centre for Genomic Medicine

Manchester, M13 9WL, United Kingdom

Location

MeSH Terms

Conditions

Huntington Disease

Interventions

tominersen

Condition Hierarchy (Ancestors)

Basal Ganglia DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesDementiaChoreaDyskinesiasMovement DisordersHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesCognition DisordersNeurocognitive DisordersMental Disorders

Results Point of Contact

Title
Medical Communications
Organization
Hoffmann-La Roche
genentech@druginfo.com
800 821-8590

Study Officials

  • Clinical Trials

    Hoffmann-La Roche

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 31, 2017

First Posted

November 14, 2017

Study Start

January 2, 2018

Primary Completion

October 8, 2019

Study Completion

October 8, 2019

Last Updated

March 24, 2022

Results First Posted

January 19, 2021

Record last verified: 2022-03

Locations

DE(3)CA(1)GB(5)