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Huntingtons DiseasePhase 1NCT04000594

A Study to Investigate the Pharmacokinetics and Pharmacodynamics of RO7234292 (RG6042) in CSF and Plasma, and Safety and Tolerability Following Intrathecal Administration in Patients With Huntington's Disease

A Phase 1 study testing RO7234292 (RG6042) in people with Huntingtons Disease, run by Hoffmann-La Roche at 3 sites in 2 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

Study BP40410 is an open-label, adaptive multiple-dose clinical study designed to characterize the PK of RO7234292 (RG6042) in plasma and CSF as well as the acute time course and recovery profile of CSF mHTT lowering in response to RO7234292 (RG6042) treatment after intrathecal (IT) administration of RO7234292… Read more about this study

Who is it for?
Ages 25 to 65 years
Conditions the study is for
Huntingtons 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
12 participants
Where
3 sitesin 2 countries
Study dates
2 Sep 2019to 18 Jan 2022
Phase
Phase 1
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 to 65 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Manifest HD diagnosis
  • Independence Scale score of >=70.
  • Genetically confirmed disease by direct deoxyribonucleic acid testing with a cytosine, adenine, and guanine base sequence found in DNA which is translated into glutamine (CAG) age product (CAP) score > 400.
  • Ability to read the words "red," "blue," and "green" in the patient's native language.
  • Ability to walk unassisted without a cane or walker and move about without a wheelchair on a daily basis as reviewed at screening and baseline visit.
  • Ability to undergo and tolerate MRI scans.

Who cannot join

  • History of attempted suicide or suicidal ideation with plan (i.e., active suicidal ideation) that required hospital visit and/or change in level of care within 12 months prior to screening.
  • Current active psychosis, confusional state, or violent behavior.
  • Any serious medical condition or clinically significant laboratory, vital signs, or ECG abnormalities at screening that, in the Investigator's judgment, precludes the patient's safe participation in and completion of the study.
  • Clinical diagnosis of chronic migraines or history of low pressure headache after lumbar puncture requiring hospitalization or blood patch.
  • Treatment with investigational therapy within 4 weeks prior to screening or 5 drug elimination half-lives of investigational therapy, whichever is longer.
  • Concurrent or planned concurrent participation in any interventional clinical study, including explicit pharmacological and non-pharmacological interventions. Observational studies are acceptable.
Show all 11Show fewer
  • Unable or unsafe to perform lumbar puncture on the patient.
  • Previous lumbar surgery that is likely, in the opinion of the Investigator or surgical team, to make IT catheter insertion or IT injection unduly difficult or hazardous.
  • Poor peripheral venous access.
  • Scoliosis or spinal deformity making IT injection not feasible in the outpatient setting.
  • Preexisting intra-axial or extra-axial lesions as assessed by a centrally read MRI scan during the screening period.

Trial locations and contacts

3 sites in 2 countries.

3 sites in 2 countries

United Kingdom2 sites

  • Leonard Wolfson Experimental Neurology Centre

    London, WC1N 3BG

  • Manchester University NHS Foundation Trust (MFT)

    Manchester, M13 9WL

Other trials in United Kingdom

Netherlands1 site

  • Centre For Human Drug Research; Research

    Leiden, 2333

Other trials in Netherlands

About this study

Official title: An Open-Label Adaptive Multiple-Dose Study to Investigate the Pharmacokinetics and Pharmacodynamics of RO7234292 in CSF and Plasma, and Safety and Tolerability Following Intrathecal Administration in Patients With Huntington's Disease

Study BP40410 is an open-label, adaptive multiple-dose clinical study designed to characterize the PK of RO7234292 (RG6042) in plasma and CSF as well as the acute time course and recovery profile of CSF mHTT lowering in response to RO7234292 (RG6042) treatment after intrathecal (IT) administration of RO7234292 (RG6042) to patients with manifest Hungtington's disease (HD).

Being tested
RO7234292 (RG6042)
Medical subject headings
Chorea

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

    RO7234292 (RG6042) will be administered in two IT doses of the same dose level at an interval of 28 days during the treatment period (Day 1 and Day 29). Each dose of RO7234292 (RG6042) will be administered as a single IT bolus injection.

Study groups in NCT04000594
Study groupRoleWhat they receive
Dose level 1 of RO7234292 (RG6042)ExperimentalParticipants will receive dose level 1 of RO7234292 (RG6042) intrathecally on Day 1 and Day 29.
Receives: Drug: RO7234292 (RG6042)
Dose level 2 of RO7234292 (RG6042)ExperimentalParticipants will receive dose level 2 of RO7234292 (RG6042) intrathecally on Day 1 and Day 29.
Receives: Drug: RO7234292 (RG6042)
Dose level 3 of RO7234292 (RG6042)ExperimentalParticipants will receive dose level 3 of RO7234292 (RG6042) intrathecally on Day 1 and Day 29.
Receives: Drug: RO7234292 (RG6042)

What is being measured

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

  1. Concentrations of RO7234292 in CSF (Cerebrospinal Fluid)

    NA represents: insufficient number of participants with events.

    Measured: Day 1, 2, 3, 4, 29, 43, 71, 127, and follow-up visit (6 months after last study drug administration)

  2. Concentrations of RO7234292 in Plasma

    NA represents:insufficient number of participants with events

    Measured: Day 1, 2, 3, 4, 5, 28, 29, 30, 43, 71, 127, and follow-up visit (6 months after last study drug administration)

  3. mHTT (Mutant Huntingtin) Concentration in CSF

    CSF Mutant Huntingtin Protein (fmol/L) values at time point visits are reported.

    Measured: Days 1, 2, 3, 29, 43, 71, 127 and follow-up visit (6 months after last study drug administration)

Secondary outcomes (5)

  1. Percentage of Participants With Adverse Events According to NCI-CTCAE Grading System

    Severity levels levels: 1 = mild; 2 = moderate; 3 = severe used according to NCI-CTCAE grading system.

    Percentage of participants with 1-3 levels of severities are reported.

    Measured: Up to 6 months

  2. Percentage of Participants With Suicidal Ideation or Behavior, as Assessed by Columbia-Suicide Severity Rating Scale (C-SSRS) Score

    The Columbia-Suicide Severity Rating Scale (C-SSRS) is a structured tool to assess suicidal ideation and behavior.

    Four constructs are measured: severity of ideation, intensity of ideation, behavior, and lethality of actual suicide attempts. Binary (yes/no) data are collected for 10 categories, and composite endpoints based on the categories are followed over time to monitor patient safety. Scores 1-10 is used, 1 being less and to 10 being severity increasing.

    Only one time frame was used per score category: from screening to follow-up visit.

    Measured: From Screening Day Up To Follow-up Visit (6 months after the last Dosing Day-Day 127)

  3. Incidence of Anti-Drug Antibodies (ADAs)

    Percentage of participants who have negative or positive anti-drug antibody affects are reported.

    Measured: Day 1, Day 28, and follow-up visit (6 months after last study drug administration)

  4. Titer and Antibody Subtype, Determined if ADAs Are Identified

    The data cannot be reported and the outcome measure is not applicable as no ADA sample was positive. As no ADA, titer and subtype could not be identified

    Measured: Day 1, Day 28, and follow-up visit (6 months after last study drug administration)

  5. Amount of RO7234292 in Urine Ae (Micrograms)

    Measured: Up to 72 hours

Timeline

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

  1. First posted27 Jun 2019
  2. Study start2 Sep 2019
  3. Primary completion18 Jan 2022
  4. Study completion18 Jan 2022

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 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
12 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04000594
Sponsor's protocol ID
BP40410
Other IDs
GEN-PEAK
Status last verified
Jun 2024
First submitted
26 Jun 2019
Last updated
3 Oct 2024
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No: Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.clinicalstudydatarequest.com). Further details on Roche's criteria for eligible studies are available here (https://clinicalstudydatarequest.com/Study-Sponsors/Study-Sponsors-Roche.aspx). For further details on Roche's Global Policy on Sharing of Clinical Study Information and how to request access to related clinical study documents, see here (https://www.roche.com/research\_and\_development/who\_we\_are\_how\_we\_work/clinical\_trials/our\_commitment\_to\_data\_sharing.htm).

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing RO7234292 (RG6042) in people with Huntingtons Disease, run by Hoffmann-La Roche at 3 sites in 2 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 6 inclusion and 11 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 3 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in United Kingdom, Netherlands. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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 3 Oct 2024). 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?

Concentrations of RO7234292 in CSF (Cerebrospinal Fluid) (time frame: Day 1, 2, 3, 4, 29, 43, 71, 127, and follow-up visit (6 months after last study drug administration)). The study lists 3 primary outcomes in all. 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 NCT04000594.

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 3 Oct 2024.

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