The purpose is to assess safety and tolerability of votoplam and to determine whether votoplam slows disease progression in patients with early symptomatic Huntington's disease (HD) compared to the control arm.
HTT227 - current compound code (former code is PTC518 from PTC Therapeutics), HTT227 is Novartis code under Novartis sponsorship.
Signed informed consents must be obtained prior to participation in the study
Ambulatory male or female participants between 21 to 70 years of age, inclusive, on the day of Informed Consent signature
Genetically confirmed HD diagnosis with a cytosine-adenine-guanine (CAG) repeat length of 40 or above. Participants must have prior genetic confirmation and known CAG repeat length obtained prior to screening.
UHDRS IS score ≥90
History of gene therapy or cell transplantation or any other experimental brain surgery for the treatment of HD
positive anti-HBc IgM
positive anti-HBc IgG confirmed by positive HBsAg and/or HBV DNA
positive HCV ab test confirmed by positive HCV RNA