GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington's Disease
- Conditions
- Huntington Disease
- Interventions
- Registration Number
- NCT05686551
- Lead Sponsor
- Hoffmann-La Roche
- Brief Summary
This study will evaluate the safety, biomarkers, and efficacy of tominersen compared with placebo in participants with prodromal and early manifest Huntington's Disease (HD).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 301
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Tominersen 60 milligrams (mg) Tominersen 60 mg - Placebo Placebo - Tominersen 100 mg Tominersen 100 mg -
- Primary Outcome Measures
Name Time Method Incidence and Severity of Adverse Events (AEs), With Severity Determined According to the AE Severity Grading Scale Up to approximately 24 months Change From Baseline in Clinical Laboratory Results - Cerebrospinal Fluid (CSF) White Blood Cell (WBC) From baseline visit (Day 1), and months 4, 8, 9, 12, 16 Change From Baseline in Clinical Laboratory Results CSF Protein From baseline visit (Day 1), and months 4, 8, 9, 12, 16 Change From Baseline in Structural Magnetic Resonance Imaging (MRI) Assessing Any New Abnormalities Including Radiographic Features Consistent With Hydrocephalus and Other Relevant MRI Safety Findings From baseline, months 4, 8, 12, 16 and up to approximately month 24 Percentage Change From Baseline in Geometric Means of CSF Mutant Huntingtin (mHTT) Protein Levels at Month 9 Baseline, Month 9 Change From Baseline in Composite Unified Huntington's Disease Rating Scale (cUHDRS) Scores (non-U.S. Sites) at 16 Months Baseline to 16 months Change in scores on the scale.
Change From Baseline in Total Functional Capacity (TFC) Scores (U.S. Sites) at 16 Months Baseline to 16 months Change in scores on the scale.
- Secondary Outcome Measures
Name Time Method Change From Baseline in Montreal Cognitive Assessment (MoCA) Scores From baseline, months 4, 8, 12, 16 and up to approximately month 24 Percentage of Participants With Suicidal Ideation or Behavior (I/B) as Assessed by Columbia-Suicide Severity Rating Scale (C-SSRS) at Each Visit, Including Detailed Focus on Any Individual Cases Identified as Having Severe I/B During the Study Conduct Up to approximately 24 months Change From Baseline at 16 Months for the Assessments of TFC (non-U.S. Sites) Scores Baseline to 16 months Change From Baseline at 16 Months for the Assessments of cUHDRS (U.S. Sites) Scores Baseline to 16 months Change From Baseline at 16 Months for the Assessments of Symbol Digit Modalities Test (SDMT) Scores Baseline to 16 months Change From Baseline at 16 Months for the Assessments of Stroop Word Reading (SWR) Score Baseline to 16 months Change From Baseline at 16 Months for the Assessment of Total Motor Score (TMS) Baseline to 16 months Change From Baseline in CSF Neurofilament Light Chain (NfL) Levels at 16 Months Baseline to 16 months Incidence of Anti-drug Antibodies (ADAs) at Specified Timepoints Relative to the Prevalence of ADAs at Baseline From baseline, months 4, 8, 12, 16 and up to approximately month 24 Titers Determined if ADAs are Identified From baseline, months 4, 8, 12, 16 and up to approximately month 24
Trial Locations
- Locations (72)
Uab Medicine
🇺🇸Birmingham, Alabama, United States
Barrow Neurological Institute
🇺🇸Phoenix, Arizona, United States
University of California Davis Medical System
🇺🇸Sacramento, California, United States
CenExel Rocky Mountain Clinical Research, LLC
🇺🇸Englewood, Colorado, United States
Georgetown University
🇺🇸Washington, District of Columbia, United States
University of Florida
🇺🇸Gainesville, Florida, United States
University of South Florida
🇺🇸Tampa, Florida, United States
Northwestern University
🇺🇸Chicago, Illinois, United States
University of Iowa Hospitals and Clinics
🇺🇸Iowa City, Iowa, United States
John Hopkins University School of Medicine
🇺🇸Baltimore, Maryland, United States
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