Study to Measure Cerebrospinal Fluid Mutant Huntingtin Protein in Participants With Early Manifest Stage I or Stage II Huntington's Disease
NCT ID: NCT03664804
Last Updated: 2025-01-03
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE1
95 participants
INTERVENTIONAL
2018-12-05
2022-05-07
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
OTHER
NONE
Study Groups
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Participants with Early Manifest Stage I or II HD
No study drug was administered in this study
No Study Drug was Administered in this Study
No study drug was administered in this study
Interventions
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No Study Drug was Administered in this Study
No study drug was administered in this study
Eligibility Criteria
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Inclusion Criteria
* 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
* 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
25 Years
65 Years
ALL
No
Sponsors
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Hoffmann-La Roche
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Trials
Role: STUDY_DIRECTOR
Hoffmann-La Roche
Locations
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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
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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BN40422
Identifier Type: -
Identifier Source: org_study_id
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