Stopped: The trial was halted prematurely due to signs and symptoms that suggested the possibility of peripheral neuropathy. All participants who received branaplam continued to undergo routine (safety) evaluations for up to a year following their final dose
This is the first study of branaplam in adults with Huntington's Disease (HD) to determine the correct dose required to lower mutant huntingtin protein (mHTT) levels in the cerebrospinal fluid (CSF) to a degree expected to be efficacious over longer periods of time.
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Percentage Change From Baseline to Week 17 in mHTT Protein in CSF
Timeframe: Baseline, Week 17
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timeframe: From first dose of study treatment up to Week 69