Study to Measure Cerebrospinal Fluid Mutant Huntingtin Protein in Participants With Early Manifes… (NCT03664804) | Clinical Trial Compass
CompletedPhase 1
Study to Measure Cerebrospinal Fluid Mutant Huntingtin Protein in Participants With Early Manifest Stage I or Stage II Huntington's Disease
United States95 participantsStarted 2018-12-05
Plain-language summary
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).
Who can participate
Age range25 Years – 65 Years
SexALL
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Inclusion Criteria:
* 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
Exclusion Criteria:
* 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 behav…
What they're measuring
1
Change From Baseline in the Following Clinical Endpoints at 3, 9, and 15 Months: cUHDRS, TFC, TMS, SDMT, SWR Test and IS
Timeframe: Baseline to 15 Months
2
Change From Baseline in Biomarkers of Neuronal Injury (CSF NfL and Tau) at 3, 9, and 15 Months
Timeframe: Baseline to 15 Months
3
Change From Baseline in Brain Atrophy Endpoints (Whole Brain Volume Decline, Caudate Volume Decline) as Determined by Brain MRI, at 3, 9, and 15 Months