Study to Measure Cerebrospinal Fluid Mutant Huntingtin Protein in Participants With Early Manifest Stage I or Stage II Huntington's Disease

Completed · Phase 1

Conditions studied: Huntington's Disease

In brief

The study is designed as a multi-site, prospective, 15-month longitudinal, cohort study measuring CSF mHTT in participants with early manifest Stage I or Stage II Huntington's Disease (HD).

Key facts

Study ID: NCT03664804
Run by: Hoffmann-La Roche
People needed: 95
Starts: 2018-12-05
Expected to finish: 2022-05-07
Last updated by the study team: 2025-01-03

Who can join

Age: 25 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Capacity to consent to participate in the study as assessed using the Evaluation to Sign Consent tool and investigator judgment
Age 25 to 65 years, inclusive, at the time of signing Informed Consent Form
Early manifest, Stage I or Stage II HD (defined as TFC of 7-13, inclusive)
Genetically confirmed disease (CAG repeat length ≥ 36 in huntingtin gene by direct DNA testing)
Body mass index ≥18 and ≤32 kg/m2; total body weight >50 kg
Ability to undergo and tolerate MRI scans
Ability to tolerate blood draws and lumbar puncture
Ability and willingness to comply with all aspects of the protocol, including completion of interviews and questionnaires and carrying/wearing of a digital monitoring device
Stable medical, psychiatric, and neurological status for at least 12 weeks prior to screening and at the time of enrollment
Signed study companion consent for participation, if a study companion is available
For women of childbearing potential: agreement to remain abstinent or use acceptable contraceptive methods during the observational period

You may not qualify if…

Any condition, including severe chorea, that would prevent either writing or performing pen and paper or smartphone-based tasks
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 sign, or electrocardiogram abnormalities at screening that, in the investigator's judgement, precludes the participant's safe participation in and completion of the study
Pregnant or breastfeeding, or intending to become pregnant during the study
Positive for hepatitis C virus antibody or hepatitis B surface antigen at screening
Known HIV infection
Current or previous use of an antisense oligonucleotide (including small interfering RNA)
Current use of antipsychotics prescribed for psychosis, cholinesterase inhibitors, memantine, amantadine, or riluzole including use within 12 weeks of enrollment
Treatment with an investigational drug within 30 days prior to screening or 5 half-lives of the investigational drug, whichever is longer
Antiplatelet or anticoagulant therapy within the 14 days prior to screening or anticipated use during the study, including, but not limited, to aspirin (unless ≤81mg/day), clopidogrel, dipyridamole, warfarin, dabigatran, rivaroxaban, and apixaban
History of bleeding diathesis or coagulopathy; platelet count < lower limit of normal unless stable and assessed by the Investigator and Sponsor Medical Monitor to be not clinically significant
Malignancy within 5 years prior to screening, except basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated
History of gene therapy or cell transplantation or any other experimental brain surgery
Concurrent or planned concurrent participation in any clinical study without approval of the Medical Monitor
Presence of implanted shunt for the drainage of CSF or an implanted CNS catheter
Pre-existing structural brain lesion as assessed by MRI scan

Where it is running

CenExel Rocky Mountain Clinical Research, LLC — Englewood, Colorado, United States
Georgetown University; Research Division, Psychiatry — Washington D.C., District of Columbia, United States
Hereditary Neurological Disease Centre (HNDC) — Wichita, Kansas, United States
John Hopkins University School of Medicine — Baltimore, Maryland, United States
Columbia University — New York, New York, United States
The University of Texas Health Science Center at Houston; McGovern Medical School — Houston, Texas, United States
The University of British Columbia; The Centre for Huntington Disease — Vancouver, British Columbia, Canada
Centre for Movement Disorders (Neuropharm Consulting Inc.) — Markham, Ontario, Canada
Charité - Universitätsmedizin Berlin, Campus Charité Mitte; Klinik für Psychiatrie und Psychotherapi — Berlin, Germany
St. Josef and St. Elisabeth gGmbH ; St. Josef Hospital Bochum; Neurologisches Forschungszentrum — Bochum, Germany
Universitätsklinikum Ulm; Klinik für Neurologie — Ulm, Germany
NIHR Welcome Trust Birmingham CRF - University Hospitals Birmingham; Department of Neuropsychiatry — Birmingham, United Kingdom
Cardiff University School of Medicine; Institute of Psychological Medicine Clinical Neurosciences — Cardiff, United Kingdom
National Hospital For Neurology and Neurosurgery — London, United Kingdom
Central Manchester University Hospitals NHS Foundation Trust; Manchester Centre for Genomic Medicine — Manchester, United Kingdom

Full record on ClinicalTrials.gov

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