ONGOING INTERVENTIONAL TRIALS
Before participating in a study, talk to your health care provider and learn about the risks and potential benefits.
The purpose of this study is to look at the safety, how well the drug is tolerated, the pharmacodynamics(PD- the body’s biological response to drugs) and pharmacokinetics (PK-the movement of drugs through the body) of a single dose of ALN-HTT02
AB-1001 is an investigational gene therapy originally developed by BrainVectis, which was acquired by AskBio in 2020. AB-1001 was previously called BV-101. This is Phase I/II dose-finding study to evaluate the safety, tolerability, and the ability to produce the desired result (i.e., efficacy) of a one-time intracerebral injection of AB-1001 (previously BV-101) within the striatum of adults with early manifest Huntington’s disease.
This study evaluates the safety, biomarkers, and efficacy trends of different dose levels of investigational drug tominersen in people aged 25 to 50 with prodromal (very early subtle signs of HD) or early manifest HD. Tominersen is an investigational drug that has been studied in several clinical trials since 2015, including in a Phase III study called GENERATION HD1 that tested two different dose regimens of tominersen in adults with manifest HD.
The European Phase Ib/II clinical trial of AMT-130 for the treatment of HD will explore the safety, proof of concept, and dosing in 15 total patients with with early manifest Huntington’s disease split into a five person manifest Huntington’s disease split into a five person, low-dose open-label(patients know what they are getting) cohort, followed by a nine patient, higher-dose open-label cohort. All patients will be dosed with AMT-130. The therapeutic goal is to inhibit the production of the mHTT. Using AAV vectors to deliver micro-RNAs directly to the brain. A one time injection.
The PIVOT-HD study is a Phase II trial looking at safety of Votoplam (PTC518), a new Huntington-Lowering drug, in patients with HD. Votoplam is an oral tablet (taken by mouth every day) that not only tries to lower huntingtin (HTT) in the brain but also treat HD everywhere in the body (brain, muscle, immune system…).
The goal of this first-in-human clinical trial is to assess the safety and tolerability of four doses of a new study drug called VO659 in people with genetic disorders called spinocerebellar ataxia type 1, type 3 or Huntington’s disease. Another aim is to determine the concentrations of the study drug in the cerebral spinal fluid and blood.