View clinical trials related to Huntington's Disease.
Filter by:The primary purpose of this study is to evaluate the effect of SAGE-718 on cognitive performance and functioning in participants with HD.
This is the first study of AMT-130 in patients with early manifest HD and is designed to establish safety and proof-of-concept (PoC). CT-AMT-130-01 is a Phase I/II, randomized, multicenter, multiple dose, double-blind, imitation surgery, first-in-human (FIH) study. Cohort 3 participants will receive either high or low dose (1:1 randomization). Participants enrolled in Cohort 3 will also receive an immunosuppression regimen consisting of dexamethasone, sirolimus, and rituximab.
The study is designed as a multi-site, prospective, 15-month longitudinal, cohort study measuring CSF mHTT in participants with early manifest Stage I or Stage II Huntington's Disease (HD).
Huntington's disease (HD) is a rare, inherited and progressive neurodegenerative disorder for which hallmark symptoms include movement disorders, loss of cognitive faculties and psychiatric disturbances. With the progression of the disease, patients require increasing level of medical care, caregiver support, and long-term care, which lead to substantial burden of illness. Very little data are available on the direct or indirect costs for HD. The direct medical costs of HD in the US have been summarized from retrospective commercial and Medicaid claims data analysis. The indirect and out-of-pocket costs of HD in the US have not been quantified. This study will help to bridge these gaps. This study is a single-assessment, cross-sectional online survey administered to Huntington disease gene expansion carriers (HDGECs) and companions of HDGECs by HD stage to understand the indirect and out-of-pocket costs of Huntington's disease in the US.
This study will test the safety, tolerability, pharmacokinetics and pharmacodynamics of RO7234292 administered intrathecally to adult patients with Huntington's Disease.
This study will evaluate the feasibility and acceptability of a clinic-based physical activity coaching intervention in people with pre-manifest and early stage Huntington's Disease (HD). Fourteen individuals with premanifest and early stage HD will be recruited to participate in a 4 month coaching intervention. Feasibility will be assessed by recruitment and retention rates, and acceptability will be assessed by participant interviews. Participants will also be evaluate at baseline and following the coaching intervention to explore preliminary efficacy in terms of physical activity, self efficacy, disease-specific motor and cognitive function, walking endurance and strength.
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).
Cognitive impairments, especially deficits of executive function, have been well documented as a core and early feature in Huntington's disease (HD). Cognitive impairments can be considerably burdensome and devastating for people and families affected by HD. Computerised cognitive training interventions that focus on improving executive function present a potentially exciting non-pharmacological treatment option. Novel work conducted in mouse models of HD, has demonstrated that cognitive training, administered from an early stage in the disease, can improve motor performance at an older age, even in the absence of further training in the intervening time. This represents proof of principle in an animal model of HD that cognitive training can improve HD disease symptoms. Improvements associated with executive function training have also been reported in a clinical setting in a variety of neurodegenerative diseases. For example, cognitive training, can improve executive function as people age, and training specifically focused on tasks of executive function has been shown to improve both cognitive and motor outcomes in neurodegenerative diseases such as Parkinson's disease (PD) and Alzheimer's disease (AD). Therefore, this study is a feasibility study which aims to establish proof of principle for using computerised cognitive training in people with HD. The investigators propose to determine the feasibility, acceptability and gather preliminary evidence of the effectiveness of a cognitive training intervention programme, targeted for people with HD. The investigators will also aim to investigate the most appropriate outcome measures to use in this study and gather feedback on the cognitive training intervention. The investigators will also establish proof of concept via the study of brain structure and function, using MRI scanning techniques. The computerised cognitive training software and the associated outcome measures will be investigated, taking into account the views of people and families who are affected by HD. A randomised feasibility study of computerised cognitive training for people with HD will then be conducted. Participants who are randomised to the cognitive training intervention group will be asked to complete a cognitive training intervention utilising "HAPPYneuron" software. Participants in the intervention group will be asked to complete the cognitive training programme for a minimum of 30 minutes, 3 times a week for the 12 week study duration. Participants in the control group will not receive any cognitive training and will be asked to continue as normal, however they will have home visits to control for the confounding effect of social interaction. Additional monitoring and prompting for the intervention group, will be conducted via email, text or telephone reminders (as preferred by the participant) and home visits. The motor and cognitive function of participants will be assessed at the beginning and end of the study, using a range of motor and cognitive assessments. Additional cognitive measurements will be recorded as part of the HAPPYneuron programme throughout the cognitive training intervention, such as accuracy and response time measures of particular computer games. MRI scans (optional) will be conducted at the beginning and end of the study to identify any structural changes in the brain that may be associated with the cognitive training intervention. As part of the feasibility and acceptability assessment, participants, family members and carers will be invited to complete a semi-structured interview at the end of the study, if consent is obtained, focusing on using this type of software as a home based therapeutic intervention.
The aim of this study is to measure longitudinally the availability of the PDE10A enzyme in HDGECs using the radioligand [18F]MNI-659. The study will be a follow-up, examining HDGECs from the CHDIKI1201/PET-HD-PDE10A (NCT02061722) study from 18 to 28 months after the initial PET measurement.