- 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.
About
The main purpose of the study is to characterize the movement of the drug RO7234292 (RG6042) into, through, and out of the Cerebrospinal fluid( CSF)and plasma in addition to their reaction to the drug. The study will also look at the acute time course and recovery profile of CSF, mHHT lowering in response to RO7234292 (RG6042) treatment after intrathecal (IT) administration of RO7234292 (RG6042) to patients with manifest Huntington’s disease (HD).
Sponsored by
Hoffmann-La Roche
Countries
Netherlands
United Kingdom
Number of participants
20 participants
Recruitment status
Recruiting
Eligibility Criteria
Inclusion Criteria
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.
TRIAL SITES
UNITED KINGDOM
| Trial site: Leonard Wolfson Experimental Neurology Centre | Status: [maxbutton id=”1″ ] | |
| Address: London, United Kingdom, WC1N 3BG | ||
Contact: Not available at the moment
| Telephone: Not available at the moment
| Email: Not available at the moment
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| Trial site: Manchester University NHS Foundation Trust (MFT) | Status: [maxbutton id=”1″ ] | |
| Address: Manchester, United Kingdom, M13 9WL | ||
Contact: Not available at the moment
| Telephone: Not available at the moment
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