View clinical trials related to Huntington Disease.
Filter by:WVE-HDSNP2-002 is an open-label extension (OLE) study to evaluate the safety, tolerability, PK, PD, and clinical effects of WVE-120102 in adult patients with early manifest HD who carry a targeted single nucleotide polymorphism, rs362331 (SNP2). To participate in the study, patients must have completed the Phase 1b/2a clinical study WVE-HDSNP2-001.
WVE-HDSNP1-002 is an open-label extension (OLE) study to evaluate the safety, tolerability, PK, PD, and clinical effects of WVE-120101 in adult patients with early manifest HD who carry a targeted single nucleotide polymorphism, rs362307 (SNP1). To participate in the study, patients must have completed the Phase 1b/2a clinical study WVE-HDSNP1-001.
This is a double-blind, placebo-controlled 2-period 10-week treatment within-subject crossover study of neflamapimod in early-stage Huntington disease (HD). The primary objective is to determine whether neflamapimod can reverse hippocampal dysfunction in patients with early-stage HD, as assessed by the virtual water-maze-test for evaluating spatial learning and selected tests on the Cambridge Neuropsychological Test Automated Battery (CANTAB).
This is an observational study of medical marijuana manufactured and dispensed by Ilera and given as standard treatment for a variety of approved serious medical conditions as defined by individual state law. All patients who are receiving one of the four formulations (Dream, Soothe, Shine and Ease) of medical marijuana will be provided a study flyer and asked to contact the study team via phone or email. Once the study team confirms eligibility, the study team will meet the subject face-to-face most likely at their dispensary (or other mutually agreeable location) and obtain informed consent, and assent when appropriate. Initial baseline demographic information, medical history and medication inventory will be completed. Also, since it is possible that the Investigators will enroll subjects across the region, Investigators anticipate the need to seek consent over the phone for many patients. This will be done via Skype, Go to Meeting, Facetime or similar platforms so that the Investigators can have a face to face interaction with the potential subjects. Regardless of where this discussion takes place (i.e., in person or via the web), all reasonable safeguards to ensure patient privacy will be taken. Patients or their legally authorized representative (LAR) will be given sufficient (i.e., up to several hours/days) to make a decision to participate in this study. Study staff will fax or email the consent form for their signature and no study procedures will begin until the signed consent form is received by the study team. The subjects or their LARs will be instructed on obtaining the blood samples. Blood draws will be completed in the subjects' home after one of their standard doses is taken.
This is a FIH (first-in-human) adaptive PET (Positron Emission Tomography) imaging study to explore the binding and kinetic properties of two potential mutant huntingtin (mHTT) radioligands; [¹¹C]CHDI-00485180-R and [¹¹C]CHDI-00485626. The binding characteristics of these radioligands will be evaluated first in young (< 35 years of age) healthy control (HC) participants (Phase 1a) and subsequently in young HCs and Huntington's disease gene-expansion carriers (HDGECs) with Stage II HD (Phase 1b). Subsequent phases will continue to explore the radioligands' binding characteristics in HDGECs with Stage II HD (Phase 2a), Stage I HD (Phase 2b) and pre-manifest HDGECs (Phase 2c) and their age matched healthy controls. All phases are cross-sectional and will include comparisons between HCs and HDGECs. Test-retest (TRT) evaluations will be done during Phase 2a, with the option of including further test-retests in Phases 2b, and 2c after review of data by the iMagemHTT Executive Committee. TRT is only applicable to HDGEC participants. There are four planned interim analyses at which either radioligand may be dropped if its characteristics are shown to be suboptimal. If successful, the study will establish [¹¹C]CHDI-00485180-R and/or [¹¹C]CHDI-00485626 as fit for further development as drug development tools to measure mHTT levels in HDGECs. This development is intended to demonstrate the value of PET imaging with these radioligands as a disease progression biomarker, predictive biomarker, pharmacodynamic biomarker, and eventually as an efficacy biomarker. All HDGEC participants will be invited to provide an optional cerebrospinal fluid (CSF) sample that will be collected after the imaging visits are complete. These samples will be processed to evaluate CSF mHTT levels and other potential biomarkers and to explore potential relationships between soluble CSF mHTT levels and mHTT binding identified by [¹¹C]CHDI-00485180-R and/or [¹¹C]CHDI-00485626 PET imaging. Potential CSF biomarkers that might be co-expressed or accumulated in HD may also be examined.
PRECISION-HD2 is a Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple doses of WVE-120102 in adult patients with early manifest Huntington's disease (HD) who carry a targeted single nucleotide polymorphism (SNP) rs362331 (SNP2).
PRECISION-HD1 is a Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple doses of WVE-120101 in adult patients with early manifest Huntington's disease (HD) who carry a targeted single nucleotide polymorphism (SNP) rs362307 (SNP1).
The role of disorders of socio-emotional processes in cerebral diseases such as Alzheimer's disease, frontal temporal dementia, Parkinson's disease, Huntington's disease, traumatic brain injury, stroke, focal lesions, has been recognized recently. Social cognition refers to a large group of emotional and cognitive abilities regulating inter-individuals relationships and it includes mainly theory of mind, emotional information processing and empathy. However, assessment of socio-emotional processes is still largely based on experimental tests that are not validated for clinical purpose. In addition their long duration of administration is not adapted to clinical examination. Finally these tests have not been standardized and normalized in French-speaking population.
To assess the safety and tolerability of five doses of PBF-999 (160 mg and 320mg) after repeated (8 days) single daily oral dose administration in young male and female healthy subjects.
The purpose of the study is to collect and assess long term data on the safety, tolerability, and efficacy of pridopidine in patients with Huntington's disease (HD).