Araştırma
Huntington araştırmaları için özellikle heyecan verici bir dönem. Dünya çapında sayısız doktor ve bilim insanı, genel olarak nöroloji ve özel olarak Huntington Hastalığı alanındaki araştırma projelerinde ve klinik çalışmalarda aktif olarak yer almaktadır.
En son araştırma haberlerini aşağıdaki kaynaklarda bulabilirsiniz:
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 Huntington Hastalığı
Location: Argentina, Australia, Austria, Canada, Denmark, France, Germany, Italy, New Zealand, Poland, Portugal, Spain, Switzerland, United Kingdom, Amerika Birleşik Devletleri
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, Birleşik Krallık
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.
Araştırma Güncellemeleri
Ongoing clinical trial updates from EHDN & Enroll-HD 2024 Strasbourg
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