ONGOING INTERVENTIONAL TRIALS
Before participating in a study, talk to your health care provider and learn about the risks and potential benefits.
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.
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 is to look at whether so-called radioligands (small radioactive particles) can detect mutant Huntingtin (mHTT) in the brain. The imaging study uses PET scans of the brain to see if when the radioligands are present is related with the expression of mHTT in brain cells. If the relationship is proven correct then it will a valuable way to measure mHTT and track if huntingtin lowering therapies actually lower mHTT in the brain.
The PIVOT-HD study is a Phase II trial looking at how safety is PTC518, a new Huntington-Lowering drug, in patients with HD. PTC518 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 study is a phase 1/2 and will look at the safety and how participants tolerate a new antisense oligonucleotide(ASO) in early stage disease, with a drug called WVE-003.
The main purpose of this trial is to study the efficacy and safety of SOM3355 in patients with Huntington’s disease. SOM3355, which has been used for hypertension, will be looked at in this phase 2b trial to establish if it helps with chorea in HD. 2 doses and a placebo group will receive the drug orally twice a day for 12 weeks. 129 participants will be recruited.
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.