UMass HSDA Center of Excellence Research in partnership with BIDMC Samuel Frank Laboratory

Collaborative Clinical Trials and Study Projects through BIDMC, Samuel Frank Laboratory

• A Randomized, Multicenter, Double-Blind, Placebo-Controlled, Phase III Clinical Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 in Patients with Manifest Huntington's Disease
  • Purpose: The purpose of this study is to compare the effects, good and/or bad, of RO7234292, in patients with Huntington's disease. In previous studies, RO7234292 has been shown to lower toxic protein levels that may underlie the disease process. By reducing this protein, disease progression may be slowed or stopped.
• A Phase II, Multicenter, Open-Label Study to Evaluate Safety/Tolerability of Intrathecally Administered Tominersen in Patients with Late-Onset Manifest Huntington's Disease
  • Purpose: The purpose of this study is to evaluate the effects, good and/or bad, of tominersen on patients with late-onset manifest Huntington's disease. In previous studies, tominersen has been shown to lower levels of toxic protein that may underlie the disease process. By reducing this protein, progression of disease may be slowed or stopped.
• A Prospective Registry Study in a Global Huntington's Disease Cohort -- A CHDI Foundation Project
  • Purpose: The purpose of this study is to collect clinical information about, and biological samples from, individuals with Huntington's disease. The information and samples will be used to learn more about Huntington's disease and to try to find new treatments for the disease.
• A Randomized, Double-Blind, Placebo Controlled, 52-week Phase II Study to Evaluate the Efficacy of Intravenous RO7046015 in Participants with Early Parkinson's Disease with a 52-week Blinded Extension
  • Purpose: The purpose of this study is to evaluate the effects, good and/or bad, of RO7046015, in patients with Parkinson's disease. RO7046015 may bind to and clear pathological protein in the brain and prevent spread of this protein, potentially slowing the course of Parkinson's disease.
• A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel Arm, Multicenter Study Evaluating the Efficacy and Safety of Pridopidine in Patients with Early Stage of Huntington Disease
  • Purpose: The purpose of this study is to evaluate the effect of pridopidine on everyday functioning and daily activities, as well as movement and behavior in participants with early stage Huntington's disease.
• A Phase I/II, Randomized, Double-Blind, Sham Control Study to Explore Safety, Tolerability, and Efficacy Signals of Multiple Ascending Doses of Striatally-Administered rAAV5-miHTT Total Huntingtin Gene (HTT) Lowering Therapy (AMT-130) in Early Manifest Huntington Disease
  • Purpose: The purpose of this study is to evaluate how AMT-130 affects the progression of early stage Huntington's disease. At this time, AMT-130 has only been studied in animals. These animals studies showed that AMT-130 lowered protein associated with symptoms of Huntington's disease.