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Huntington's DiseasePhase 2NCT03342053

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)

A Phase 2 study testing RO7234292 (RG6042) in people with Huntington's Disease, run by Hoffmann-La Roche at 9 sites in 3 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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

Who is it for?
Ages 25 years and older
Conditions the study is for
Huntington's Disease
What is being tested?
RO7234292 (RG6042)
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
46 participants
Where
9 sitesin 3 countries
Study dates
2 Jan 2018to 8 Oct 2019
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 25 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Must have completed dosing in ISIS 443139-CS1

Who cannot join

  • 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

Trial locations and contacts

9 sites in 3 countries.

9 sites in 3 countries

United Kingdom5 sites

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

    Birmingham, B15 2FG

  • University of Cambridge - John van Geest Centre for Brain Repair

    Cambridge, CB2 0PY

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

    Cardiff, CF24 4HQ

  • Leonard Wolfson Experimental Neurology Centre

    London, WC1N 3BG

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

    Manchester, M13 9WL

Other trials in United Kingdom

Germany3 sites

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

    Berlin, 10117

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

    Bochum, 44791

  • Universitaetsklinikum Ulm

    Ulm, 89081

Other trials in Germany

Canada1 site

  • The University of British Columbia; The Centre for Huntington Disease

    Vancouver, British Columbia, V6T 2B5

Other trials in Canada

About this study

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)

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

Being tested
RO7234292 (RG6042)
Medical subject headings
Huntington Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • RO7234292 (RG6042)Drug

    Also called Tominersen

    Intrathecal injection

Study groups in NCT03342053
Study groupRoleWhat they receive
RO7234292 MonthlyExperimentalRO7234292 is administered every 28 days intrathecally for 14 months.
Receives: Drug: RO7234292 (RG6042)
RO7234292 BimonthlyExperimentalRO7234292 is administered every 56 days intrathecally for 14 months following 2 monthly doses to serve as a loading dose.
Receives: Drug: RO7234292 (RG6042)

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: From baseline up to 18 months

Secondary outcomes (7)

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

    Measured: From baseline to Day 421

  2. 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

    Measured: From Baseline to Day 421

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

    Measured: Baseline up to 15 months

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

    Measured: Baseline up to 15 months

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

    Measured: Baseline up to 15 months

  6. EEG Parameters: Mean Change From Baseline to 15 Months in Absolute Power [8-12Hz]

    Measured: Baseline to 15 Months

  7. 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.

    Measured: Baseline to 15 Months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted14 Nov 2017
  2. Study start2 Jan 2018
  3. Primary completion8 Oct 2019
  4. Study completion8 Oct 2019

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
46 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03342053
Sponsor's protocol ID
BN40697
Status last verified
Mar 2022
First submitted
31 Oct 2017
Last updated
24 Mar 2022
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing RO7234292 (RG6042) in people with Huntington's Disease, run by Hoffmann-La Roche at 9 sites in 3 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 24 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 sites in United Kingdom, Germany, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 19 Jan 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percentage of Participants With Adverse Events (time frame: From baseline up to 18 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03342053.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Mar 2022.

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