Study to Evaluate LMI070/branaplam Treatment Medication in Participants

Recruiting

18 years - 99 years

All

75 participants needed

1 Location

Brief description of study

This is a dose range finding safety study of Branaplam administered as weekly oral doses in participants with early manifest HD. The study aims to explore a range of doses to select a safe and tolerable dose that lowers mutant huntingtin protein (mHTT) levels in the CSF to a degree expected to be efficacious over longer periods of time. Participants will undergo MRI, blood draws/CSF collection, physical examination, functional testing and will complete questionnaires.

Detailed description of study

Eligibility of study

You may be eligible for this study if you meet the following criteria:

Conditions: Medical Research, Huntington's Disease
Age: 18 years - 99 years
Gender: All

Patients with Huntington’s Disease

Updated on 04 Aug 2024. Study ID: 850637

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Study to Evaluate LMI070/branaplam Treatment Medication in Participants with Huntington's Disease

Brief description of study

Detailed description of study

Eligibility of study

Find a site

Interested in the study