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
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ClinicalTrials.gov Identifier: NCT04000594 |
Recruitment Status :
Completed
First Posted : June 27, 2019
Last Update Posted : May 31, 2022
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Huntingtons Disease | Drug: RO7234292 (RG6042) | Phase 1 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 12 participants |
Allocation: | Non-Randomized |
Intervention Model: | Parallel Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
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 |
Actual Study Start Date : | September 2, 2019 |
Actual Primary Completion Date : | January 18, 2022 |
Actual Study Completion Date : | January 18, 2022 |

Arm | Intervention/treatment |
---|---|
Experimental: Dose level 1 of RO7234292 (RG6042)
Participants will receive dose level 1 of RO7234292 (RG6042) intrathecally on Day 1 and Day 29.
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Drug: RO7234292 (RG6042)
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.
Other Name: Tominersen |
Experimental: Dose level 2 of RO7234292 (RG6042)
Participants will receive dose level 2 of RO7234292 (RG6042) intrathecally on Day 1 and Day 29.
|
Drug: RO7234292 (RG6042)
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.
Other Name: Tominersen |
Experimental: Dose level 3 of RO7234292 (RG6042)
Participants will receive dose level 3 of RO7234292 (RG6042) intrathecally on Day 1 and Day 29.
|
Drug: RO7234292 (RG6042)
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.
Other Name: Tominersen |
- 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) ]
- Concentrations of RO7234292 in Plasma [ Time Frame: Day 1, 2, 3, 4, 5, 28, 29, 30, 43, 71, 127, and follow-up visit (6 months after last study drug administration) ]
- mHTT (mutant Huntingtin) Concentration in CSF [ Time Frame: Days 1, 2, 3, 29, 43, 71, 127 and follow-up visit (6 months after last study drug administration) ]
- Incidence and Severity of Adverse Events [ Time Frame: Up to 6 months ]
- Percentage of Participants With Suicidal Ideation or Behavior, as Assessed by Columbia-Suicide Severity Rating Scale (C-SSRS) Score [ Time Frame: Screening, Day -1, Day 28, Day 71, Day 127, and follow-up visit (6 months after last study drug administration) ]
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.
- Incidence of Anti-Drug Antibodies (ADAs) [ Time Frame: Day 1, Day 28, and follow-up visit (6 months after last study drug administration) ]
- Titer and Antibody Subtype, determined if ADAs are Identified [ Time Frame: Day 1, Day 28, and follow-up visit (6 months after last study drug administration) ]
- Concentrations of RO7234292 in Urine [ Time Frame: Up to 72 hours ]

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 25 Years to 65 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
- 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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04000594
Netherlands | |
Centre For Human Drug Research; Research | |
Leiden, Netherlands, 2333 | |
United Kingdom | |
Leonard Wolfson Experimental Neurology Centre | |
London, United Kingdom, WC1N 3BG | |
Manchester University NHS Foundation Trust (MFT) | |
Manchester, United Kingdom, M13 9WL |
Study Director: | Clinical Trials | Hoffmann-La Roche |
Responsible Party: | Hoffmann-La Roche |
ClinicalTrials.gov Identifier: | NCT04000594 |
Other Study ID Numbers: |
BP40410 GEN-PEAK ( Other Identifier: Hoffmann-La Roche ) |
First Posted: | June 27, 2019 Key Record Dates |
Last Update Posted: | May 31, 2022 |
Last Verified: | May 2022 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Plan Description: | 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). |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
Huntington Disease Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Nervous System Diseases Dementia Chorea Dyskinesias |
Movement Disorders Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Genetic Diseases, Inborn Cognition Disorders Neurocognitive Disorders Mental Disorders |