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).
Age range
25 Years – 50 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
DB Period: Incidence and Severity of Adverse Events (AEs), With Severity Determined According to the AE Severity Grading Scale
Timeframe: Up to approximately 36 months
DB Period: Change From Baseline in Clinical Laboratory Results - Cerebrospinal Fluid (CSF) White Blood Cell (WBC)
Timeframe: Baseline visit (Day 1), and Months 4, 8, 9, 12, 16
DB Period: Change From Baseline in Clinical Laboratory Results - CSF Protein
Timeframe: Baseline visit (Day 1), and Months 4, 8, 9, 12, 16
DB Period: 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
Timeframe: Baseline, Months 4, 8, 12, 16 and up to approximately 36 months
DB Period: Percentage Change From Baseline in Geometric Means of CSF Mutant Huntingtin (mHTT) Protein Levels at Month 9
Timeframe: Baseline, Month 9
DB Period: Change From Baseline in Composite Unified Huntington's Disease Rating Scale (cUHDRS) Scores (non-U.S. Sites) at 16 Months
Timeframe: Baseline to 16 months
DB Period: Change From Baseline in Total Functional Capacity (TFC) Scores (U.S. Sites) at 16 Months
Timeframe: Baseline to 16 months
OLE Period: Incidence and Severity of AEs, With Severity Determined According to the AE Severity Grading Scale
Timeframe: Up to approximately 29 months
OLE Period: Change Over Time in Clinical Laboratory Results - CSF WBC
Timeframe: Up to approximately 24 months
OLE Period: Change Over Time in Clinical Laboratory Results - CSF Protein
Timeframe: Up to approximately 24 months
OLE Period: Change From Baseline in Structural MRI Assessing Any New Abnormalities, Including Radiographic Features Consistent With Hydrocephalus and Other Relevant MRI Safety Findings
Timeframe: Up to approximately 29 months