Behavioural Problems in Huntington Disease : Analysis and Enhancement of Caregiver and Patient Experience...
Huntington DiseaseThe study hypothesis is that caregivers experience allows them to develop a lot of knowledge and know-how about behavioural problems in Huntington Disease and its consequences. The purpose of this study is to collect, describe and analyse caregivers experience in order to find and enhance their knowledge related to the adaptation to behavioural problems of Huntington Disease patients.
Multicentric Trial of the Treatment of Huntington's Disease by Cysteamine (RP103)
Huntington's DiseaseThe purpose of this study is to evaluate the effect of cysteamine in patients with symptomatic Huntington's disease by comparing two groups of patients (cysteamine vs placebo) on the results of the Unified Huntington's Disease Rating Scale (UHDRS, Huntington study group 1996).
Ursodiol in Huntington's Disease
Huntington DiseaseThe purpose of this study is to evaluate the safety of the drug ursodiol (ursodeoxycholic acid, UDCA) in people with Huntington's disease (HD) and to explore how the compound is processed by the body.
Efficacy of Deutetrabenazine to Control Symptoms of Dysphagia Associated With HD
Huntington DiseaseTo determine the efficacy of deutetrabenazine to control symptoms of dysphagia associated with HD.
Pharmacokinetic (PK) and Pharmacodynamics (PD) Study of Ilera Specific Products
ALSAutism Spectrum Disorder17 moreThis 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.
Assessing the Symptomatic Benefit of Acoustic Slow Wave Enhancement in Parkinson Disease
Parkinson DiseaseMild Cognitive Impairment1 moreThe study is a randomized, double-blind, sham-controlled cross-over trial to assess the efficacy as well as safety and tolerability of auditory SWS enhancement on measured outcomes in Parkinson disease (PD) patients with disturbed nighttime sleep. Additionally, the investigators will assess the feasibility and efficacy of auditory slow-wave sleep (SWS) enhancement in Mild Cognitive Impairment (MCI) and Huntington Disease (HD) patients in a pilot study.
CKD-504 in SAD and MAD in Healthy Korean and Caucasian Adult Male and Female Subjects
Huntington DiseaseTo assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single and multiple ascending oral doses of CKD-504 compared to placebo in healthy Korean and Caucasian adult subjects
Safety and Efficacy of Bone Marrow Derived MNCs for Treatment of Cells for the Treatment of Hunting...
Huntington DiseaseThis study is Single arm, Single Centre trial to check the Safety and Efficacy of Bone Marrow Derived Autologous mononuclear cell {MNC} (100 Million per dose).trial to be conducted for 36 months in patients with diabetes Mellitus in India,Primary outcome measure are Improvement in cognitive and Psychiatric Symptoms and Improvement in Jerky,random, and Uncontrollable Movements called Chorea.
Nilotinib in Huntington's Disease
Huntington DiseaseBased on strong pre-clinical evidence of the effects of Nilotinib on neurodegenerative pathologies, including autophagic clearance of neurotoxic proteins, neurotransmitters (dopamine and glutamate), immunity and behavior, the investigators conducted an open label pilot clinical trial in mid-to-advanced PD with dementia (PDD) and Dementia with Lewy Bodies (DLB) (stage 3-4) patients. Participants (N=12) were randomized 1:1 to once daily oral dose of 150mg and 300mg Nilotinib for 6 months. The investigators data suggests that Nilotinib penetrates the brain and inhibits CSF Abelson (Abl) activity via reduction of phosphorylated Abl in agreement with pre-clinical data. Several studies suggest that CSF alpha-Synuclein and Abeta42 are decreased and CSF total Tau and p-Tau are increased in PD and DLB. The investigators data shows attenuation of loss of CSF alpha-Synuclein and Abeta40/42 with 300mg (50% of the CML dose) compared to 150mg Nilotinib after 6 months treatment. CSF homovanillic acid (HVA), which is a by-product of dopamine metabolism, is significantly increased; and CSF total Tau and p-Tau are significantly reduced (N=5, P<0.05) with 300mg Nilotinib between baseline and 6 months treatment. Despite the reduction of L-Dopa replacement therapies in our study, the Unified Parkinson's Disease Rating Scale (UDPRS) I-IV scores improved with 150mg (3.5 points) and 300mg (11 points) from baseline to 6 months and worsened (13.7 points and 11.4 points) after 3 months withdrawal of 150mg and 300mg, respectively. Other non-motor functions e.g. constipation was resolved in all patients and cognition was also improved (3.5 points) using both the Mini-Mental Status Exam (MMSE) or the Scales for Outcomes in Parkinson's Disease-Cognition (SCOPA-Cog) between baseline and 6 months. MMSE scores returned to baseline after 3 months of Nilotinib withdrawal. These data are very compelling to evaluate the effects of Nilotinib in an open label proof-of-concept study in patients with HD.
Non-invasive Brain Stimulation in Huntington's Disease
Huntington DiseaseHuntington's disease is a disabling neurodegenerative disease. Patients present with involuntary movements of the body and cognitive symptoms like behavioral problem, difficulty in planning tasks, etc. No effective treatment is currently available for them apart from symptomatic therapy. Currently, studies are going on the effectiveness of noninvasive brain stimulation (NIBS) in neurodegenerative diseases. It is a mode of brain stimulation technique where current is delivered into the brain by placing electrodes into their scalp. Transcranial pulsed current stimulation (tPCS) is a new modality of noninvasive brain stimulation. It is a device that generates electrical impulses. The current generated from this device can stimulate underlying brain areas. The investigators have planned to study the feasibility of non-invasive brain stimulation via tPCS in these patients of Huntington's disease. At first, the investigators will rate their involuntary body movement by a standard scale, assess arm movements by a robotic device, assess speech with standard protocol, assess brain electrophysiology by electroencephalography (EEG) and will also do gait (walking) analysis. Next, the investigators will give them 2 week (20 min/day) stimulation via real tPCS (active stimulation) or placebo (sham stimulation). After 2 weeks of stimulation, the investigators will re-assess the patient using the same tools mentioned pre-stimulation. Each patient will receive real and placebo stimulation with a 1-month gap in between. They will not know whether they are receiving real stimulation or placebo. Assessment will be done before stimulation, after 2 week real stimulation and after 2 week sham stimulation.