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Dyskinesias clinical trials

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NCT ID: NCT05116813 Recruiting - Parkinson Disease Clinical Trials

Open-label Safety Study of Dipraglurant (ADX48621) in Patients With Parkinson's Disease Receiving Levodopa-based Therapy

Start date: October 25, 2021
Phase: Phase 2/Phase 3
Study type: Interventional

This open-label study is designed to assess the long-term safety and tolerability of dipraglurant in PD patients for up to 52 weeks (at doses of 150-300 mg per day) for patients that have completed an Addex sponsored double-blind clinical trial of dipraglurant.

NCT ID: NCT05103072 Recruiting - Parkinson Disease Clinical Trials

Subthalamic Nucleus Stimulation With Directional Leads on Patients With Parkinson Disease at Fluctuations and Dyskinesia Stage

NSTBOS
Start date: September 21, 2021
Phase:
Study type: Observational

Patients with subthalamic nucleus have to go through a lot of examinations and tests, before and after surgery which is difficult, sometimes painful, for the patient. The investigators used to chose the best plot of the leads for stimulation by a procedure long and exhausting for the patient and the examinator. The investigators can chose the plot using a software (Guide Xt), which can delete the exhausting test. The investigators would like to study the non inferiority of this tool to choose the best plot .

NCT ID: NCT04949308 Recruiting - Clinical trials for Primary Ciliary Dyskinesia

Nasal Nitric Oxide Across Mutations in Primary Ciliary Dyskinesia

nNO_PCD
Start date: January 1, 2021
Phase:
Study type: Observational

Primary Ciliary Dyskinesia (PCD) is a rare genetic disorder characterized by dysfunction of motile cilia associated with recurrent infections of the airways, laterality defects (Situs inversus totalis in about 50% of cases) and fertility problems. At present, mutations in > 45 genes associated with PCD and mucociliary clearance disorders have been identified, representing most likely two thirds of all human cases. The aims of this study are: 1. Correlation between nasal NO levels and distinct PCD genotypes 2. Determination of further parameters potentially associated with nasal NO levels in genotyped PCD individuals 1. course of clinical manifestations (e.g. neonatal distress, infections, bronchiectasis) 2. diagnostic results (HVMA, TEM, IF) 3. lung function outcome (FVC, FEV1)

NCT ID: NCT04929795 Recruiting - Schizophrenia Clinical Trials

Effects of Music Beat on Motor Function in Individuals at Risk for Psychotic Onset and Schizophrenia Patients

Start date: August 11, 2021
Phase: N/A
Study type: Interventional

The purpose of this project is to investigate whether a 3-week training program involving music beat (serving as a type of rhythmic auditory stimulation) reduces the severity of bradykinesia and dyskinesia in at-risk individuals and schizophrenia patients. It is hypothesized that the program is effective in reducing the severity of bradykinesia and dyskinesia in at-risk individuals and schizophrenia patients.

NCT ID: NCT04919018 Recruiting - Clinical trials for Primary Ciliary Dyskinesia

Characterizing the Upper Airway Manifestations in Primary Ciliary Dyskinesia and Primary Immunodeficiencies

Start date: June 9, 2021
Phase:
Study type: Observational

Though common, morbidities related to upper airway disease in primary ciliary dyskinesia (PCD) and primary immunodeficiencies (PID) have not been fully characterized. These conditions can be difficult to distinguish due to their overlapping phenotypes. The sinonasal and middle ear features are often identified as most problematic by patients and their families, and optimal, highly effective treatment regimens have not been established. The main objective of this project is to characterize and compare the upper airway phenotypes in individuals with confirmed diagnosis of PCD and PID, and to collect critical data to inform the design of future clinical trials of treatment of the upper airway diseases. The investigators anticipate that these investigations will discern the clinical, anatomical, and pathophysiological phenotypes of paranasal sinus disease in PCD and PID, identifying disease endpoints and biomarkers that differentiate these two overlapping disorders. Findings from these studies will also enhance our understanding of middle ear disease and associated hearing loss in a cross-sectional cohort of patients with PCD and PID. Ultimately, the long-term goal of our Consortium is to elucidate underlying phenotypes and genotypes of these diseases, potentially leading to novel therapeutics that will improve the lives of affected individuals. Given the COVID pandemic, certain procedures will have the option to be converted to telehealth visits to ensure compliance with local guidelines and participant safety.

NCT ID: NCT04915651 Recruiting - Cholecystitis Clinical Trials

Gallbladder Cryoablation in High-Risk Patients

Start date: July 1, 2021
Phase: N/A
Study type: Interventional

Benign gallbladder disease, including acute cholecystitis, chronic cholecystitis, biliary dyskinesia, and biliary colic, is very common, with over 300,000 surgical cholecystectomies performed per year in the US. Unfortunately, complication rates in elderly patients or patients with many comorbidities are high. These patients are often managed with percutaneous tube drainage of the gallbladder (percutaneous cholecystostomy). The recurrence rate of calculous cholecystitis after cholecystostomy tube removal is as high as 35% at 1 year. These patients are thus faced with permanent cholecystostomy tube drainage, high-risk surgery, or cholecystostomy tube removal and risk of repeat cholecystitis. Gallbladder cryoablation is an alternative to surgical cholecystectomy which is performed percutaneously and does not require general anesthesia. Published evidence on the outcomes of gallbladder cryoablation is however limited at this point in time. The purpose of the proposed study is to follow the outcomes of high-risk patients who undergo gallbladder cryoablation.

NCT ID: NCT04901715 Recruiting - Clinical trials for Primary Ciliary Dyskinesia

Functional Studies of Novel Genes Mutated in Primary Ciliary Dyskinesia II: Genotype to Phenotype

Start date: June 10, 2021
Phase: Early Phase 1
Study type: Interventional

The purpose of this study is to measure mucociliary clearance (MCC) in groups of subjects with the disease Primary Ciliary Dyskinesia (PCD) caused by mutations in different genes, and compare to healthy subjects. Some of these genes are associated with a milder clinical phenotype. This study seeks to determine if the milder phenotype is a result of mutations in a set of specific genes. The hypothesis is that subjects with PCD caused by mutations in the milder group will maintain a low, but significant rate of mucociliary clearance, while patients with mutations in genes in the more severe group will have a complete absence of mucociliary clearance. These studies will help inform future treatment strategies.

NCT ID: NCT04857359 Recruiting - Parkinson Disease Clinical Trials

Dipraglurant (ADX48621) for the Treatment of Patients With Parkinson's Disease Receiving Levodopa-based Therapy

Start date: August 6, 2021
Phase: Phase 2/Phase 3
Study type: Interventional

This study is designed to evaluate the safety and efficacy of dipraglurant in PD patients with dyskinesia (randomized 1:1 to receive active or placebo) for 12 weeks (1 week at 150 mg per day and 11 weeks at 300 mg per day). The primary efficacy assessment will be based on the Unified Dyskinesia Rating Scale (UDysRS). Patients who complete the 12-week blinded treatment period may have the option to roll into an open-label safety extension study for an additional 12-month treatment period.

NCT ID: NCT04825119 Recruiting - Tremor Clinical Trials

Hyperkinetic Movements in Patients With Disease of Motor Neurons and Their Response to Treatment With Nusinersen

Start date: October 1, 2017
Phase:
Study type: Observational

Hyperkinetic movement disorders in patients with diseases of motor neurons will be studied. Patients with spinal muscular atrophy (SMA) and motor neuron disease patients will be studied. Involuntary movements will be video recorded and accelerometry with electromyography (EMG) will be recorded in a subset of patients. Hyperkinetic involuntary movements studied will be tremor and minipolymyoclonus. Tremor is defined as involuntary, rhythmic, oscillatory movements of a body part, and minipolymyoclonus are intermittent and irregular movements, with amplitudes sufficient to produce visible movements of the joints. Hyperkinetic movement disorders may be of central or peripheral origin and using accelerometry with EMG may help distinguish the two mechanisms. In patients with SMA the investigators will explore the effect of Nusinersen treatment on phenomenology and amplitude of tremor and minipolymyoclonus. Aims: To explore the prevalence and phenomenology of hyperkinetic movement disorders in patients with MND and SMA and to study the underlying pathological mechanisms with the use of accelerometry and EMG. To explore the effect of Nusinersen treatment on phenomenology and amplitude of involuntary movements. Hypotheses: Based on clinical observations the investigators believe it will proven that hyperkinetic movement disorders are common in patients with disease of motor neurons. The investigators hypothesize that hyperkinetic movement disorders in MND and SMA patients are of peripheral origin, being caused by uneven graduation of contraction in the wasted muscles with large motor units being active with no sufficient previous recruitment of small units to smooth contraction of large motor units. If tremor and minipolymyoclonus in SMA are due to the activation of enlarged motor units which are caused by reinnervation of muscle fibers, the treatment with Nusinersen will increase the amplitude of tremor and minipolymyoclonus. Methods: Presence, quality, and regularity of hyperkinetic movement disorders will be defined using clinical examination, accelerometry and EMG. Hyperkinetic movements will be classified as minipolymyoclonus or tremor. In patients with SMA, the measurements will be repeated 6-12 months after initiation of treatment with Nusinersen.

NCT ID: NCT04717115 Recruiting - Clinical trials for Primary Ciliary Dyskinesia

Genotype/Phenotype Correlation With Focus on Lung Function in Primary Ciliary Dyskinesia (PCD)

LuFu_PCD
Start date: November 1, 2019
Phase:
Study type: Observational

Primary Ciliary Dyskinesia (PCD) is a rare genetic disorder characterized by dysfunction of motile cilia associated with recurrent infections of the airways, laterality defects (Situs inversus totalis in about 50% of cases) and fertility problems. At present, mutations in > 45 genes associated with PCD and mucociliary clearance disorders have been identified, representing most likely two thirds of all human cases. Aim of this study are: - Correlation between genotype and lung function of patients with genetically confirmed PCD in an international cohort on a longitudinal basis - Determination of further parameters, such as body mass index (BMI), possibly associated with lung function in genetically confirmed PCD patients