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This research aims to evaluate the safety, tolerability, pharmacodynamics, and pharmacokinetics of single or repeat doses of ALN-HTT02 in adults with Huntington's Disease, specifically those in stage 2 or early stage 3 according to the Huntington's Disease Integrated Staging System. The study is a Phase 1 trial focusing on this neurodegenerative condition to better understand the investigational treatment's effects. Participants will receive either ALN-HTT02 or a placebo, both administered intrathecally. The study is randomized, double-blind, and placebo-controlled, with doses given through the spinal canal. The trial includes a double-blind part as well as an open-label extension lasting up to 36 months to monitor longer-term safety. Throughout the study, researchers will closely monitor the frequency of adverse events in all study phases, including the double-blind, open-label, and extension periods. Participants will undergo safety and tolerability assessments, pharmacodynamic and pharmacokinetic evaluations, and ongoing observation to gather comprehensive data over time.

Researchers are conducting Enroll-HD, a large, long-term observational study involving individuals affected by Huntington's Disease (HD), those at risk, and control participants. This study combines data from previous registries in Europe, North America, Australasia, and now includes Latin America. It aims to build a comprehensive database of clinical information and biological samples to support research on disease progression, prognosis, and clinical characteristics, as well as to establish clear endpoints for future interventional studies. Enroll-HD collects detailed clinical and genetic information along with blood samples from participants categorized as carriers of the HD gene mutation, controls without the mutation, and family or community controls. The study enrolls participants from over 150 sites in 23 countries and conducts annual assessments without a set end date. Participant groups include those with manifest HD symptoms, pre-manifest carriers, relatives with unknown genotype, and genotype-negative relatives. Participants undergo yearly evaluations including motor, functional, behavioral, and cognitive assessments using standardized scales such as the Unified Huntington's Disease Rating Scale and the Problem Behaviors Assessment-Short. Researchers track changes over an average of one year or longer through this ongoing registry. Data collected supports multiple research efforts and is accessible to qualified researchers worldwide.

Researchers are collecting cerebrospinal fluid (CSF) and blood plasma samples from people at various stages of Huntington's disease (HD) to help develop new treatments. This study aims to gather high-quality CSF samples for biomarker analysis and to better understand disease pathways. It also seeks to collect matching plasma samples to support HD research and therapeutic development. Participants will take part in an ongoing observational study with annual visits. Each year, they have a Screening Visit to provide medical history and clinical data, followed by a Sampling Visit where blood and CSF samples are collected after fasting for at least six hours. Some participants may be invited to an optional repeat Sampling Visit within 4 to 8 weeks of their first sampling during their first year in the study. Visits continue annually for multiple years with flexibility to skip visits without leaving the study. During each visit, researchers will gather detailed health information and biological samples through blood draws and lumbar punctures. Participants' participation is monitored over time, with the option to re-enroll if they stop attending visits. The main goal is to measure biomarkers relevant to HD progression and treatment development, with careful attention to safety and participant willingness throughout the study duration.