A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants with Prodromal and Early Manifest Huntington’s Disease

Brief summary (registry, in English)

To evaluate the safety of tominersen compared with placebo on the basis of incidence and severity of adverse events, with severity determined according to the Adverse Event Severity Grading Scale, change from baseline in clinical laboratory results [cerebrospinal fluid (CSF) white blood cell (WBC) and protein], and safety magnetic resonance imaging (MRI) (DB Period) To evaluate CSF mutant huntingtin (mHTT) protein levels in response to tominersen compared with placebo at 9 months (DB Period) To evaluate the efficacy of tominersen compared with placebo on the basis of change from baseline in composite unified huntington's disease rating scale (cUHDRS) (non-U.S.) and total functional capacity (TFC) (U.S.) at 16 months (DB Period) To evaluate the safety of long-term tominersen administration on the basis incidence and severity of adverse events, with severity determined according to the Adverse Event Severity Grading Scale, Change over time in clinical laboratory results (CSF, WBC and protein), and safety MRI (OLE Period)

What you commit to

• Study type: Interventional · Phase 2
• Planned participants: 162
• 32 sites in 8 countries
• Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of EU CTIS (euclinicaltrials.eu). It describes the study, not your situation.

Who cannot take part

Copied verbatim from EU CTIS (euclinicaltrials.eu) and grouped by topic. Only the trial team decides who can take part.

Who the study is looking for

Age in the registry: 18 years to 64 years · Sex: All · Does not accept healthy volunteers

Full criteria on EU CTIS (euclinicaltrials.eu)

Sites (32)

Sites in registry order. Pick your city on the disease page to sort them by distance.