Výzkum

Budoucnost nikdy nebyla nadějnější

Pro výzkum Huntingtonovy choroby je to obzvlášť vzrušující období. Nespočet lékařů a vědců po celém světě se aktivně podílí na výzkumných projektech a klinických studiích v oblasti neurologie obecně a Huntingtonovy choroby zvláště.

Nejnovější zprávy o výzkumu najdete v následujících zdrojích:

Clinical Trials

DIMENSION

Status: Active and recruiting

Aims and goal: The primary purpose of this study is to evaluate the effect of SAGE-718 on cognitive performance and functioning in participants with HD.

Location: United States of America, Australia, Canada and United Kingdom

KINECT-HD2

Status: Enrollment closed

Aims and goal: KINECT-HD2 is an Open-Label Rollover Study for Continuing Valbenazine Administration for the Treatment of Chorea Associated with Huntington’s Disease.

Location: United States of America and Canada

Phase I/II Clinical Trial of AMT-130

Status: Recruiting

Aims and goal: This is the first study testing AMT-130 in humans. The main purpose is to find a safe dose of AMT-130 in adults who have tested positive for the HD gene and who have early-stage HD. The study will also look at how the body processes AMT-130 and will explore how it might affect how the disease progresses.

Location: Germany, Poland and United Kingdom

GENERATION HD2

Status: Recruiting

Aims and goal: This study will evaluate the safety, biomarkers, and efficacy of tominersen compared with placebo in participants with prodromal and early manifest Huntingtonova choroba

Location: Argentina, Australia, Austria, Canada, Denmark, France, Germany, Italy, New Zealand, Poland, Portugal, Spain, Switzerland, United Kingdom, Spojené státy americké

SOM3355 for Huntington’s disease

Status: Completed

Aims and goal: SOM3355 has successfully completed a Phase 2a trials in HD and shown a favorable safety profile with no neurological or neuropsychiatric side effects. The Phase 2b dose-range finding trial program received guidance from Pre-IND discussions with the US Food and Drug Administration (FDA) and Scientific Advice from the European Medicines Agency (EMA). 

Location: France, Germany, Italy, Poland, Spain, Switzerland, United Kingdom

PIVOT-HD

Status: Active

Aims and goal: The primary goal of this study is to evaluate the safety and pharmacodynamic effects of PTC518 compared with placebo in participants with HD.

Location: Australia, Austria, Canada, France, Germany, Italy, Netherlands, New Zealand, Spain, Spojené království

SELECT-HD

Status: Completed

Aims and goal: The SELECT-HD trial was a global, multicenter, randomized, double-blind, placebo-controlled Phase 1b/2a clinical trial to assess the safety and tolerability of single- and multiple-ascending intrathecal doses of WVE-003 in people with a confirmed diagnosis of HD who are in the early stages of the disease and carry SNP3 in association with their cytosine-adenine-guanine (CAG) expansion. Additional objectives include assessing pharmacokinetics and exploratory pharmacodynamic and clinical endpoints. 

Location: Australia, Canada, Denmark, France, Germany, Italy, Netherlands, Poland, Spain, United Kingdom

PROOF-HD

Status: Completed – waiting for pridopidine approval

Aims and goal: PROOF-HD is a Phase 3, randomized, double-blind, placebo-controlled study evaluating the efficacy and safety of pridopidine in patients with early stage of Huntington disease.

Aktualizace výzkumu

Ongoing clinical trial updates from EHDN & Enroll-HD 2024 Strasbourg

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