View clinical trials related to Aphasia, Primary Progressive.
Filter by:This is a phase IIa 24-week randomized, double-blind, placebo-controlled study. The study is designed to evaluate the efficacy and safety of Rotigotine (RTG) transdermal administration at the dosage of 4 mg or 6 mg per day versus Placebo (PLC) in newly diagnosed behavioural Frontotemporal Dementia (bvFTD) patients. 75 patients with a diagnosis of probable bvFTD will be randomly allocated to the 3 treatment arms (RTG 4mg/day, RTG 6mg/day or PLC), with 25 patients per group. Clinical and neurophysiological measurements and brain metabolism via FDG-PET will be collected before and after drug administration.
This is a Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, and PD of intrathecal (IT) WVE-004 in adult patients with C9orf72-associated ALS or FTD. To participate in the study, patients must have a documented mutation (GGGGCC [G4C2] repeat expansion) in the first intronic region of the C9orf72 gene and be diagnosed with ALS or FTD.
This study's goal is to use non-invasive brain stimulation (NBS) techniques to treat language impairment associated with Primary Progressive Aphasia (PPA). The purpose of this study is to combine behavioral language intervention with individualized noninvasive brain stimulation techniques, called transcranial direct current stimulation (tDCS) to help the brain reorganize around damage and improve language functions.
The purpose of this observational study is to improve understanding of the biology of why ALS, MS and FTD have different effects on different people and facilitate better measurement of the disease in future drug testing. To do this, brain and spinal cord neural network functionality will be measured over time, in addition to profiling of movement and non-movement symptoms, in large groups of patients, as well as in a population-based sample of the healthy population. Patterns of dysfunction which relate to patients' diagnosis and coinciding and future symptoms which align with categories of patients with similar prognoses will be investigated and their ability to predict incident patients' symptoms in future will be measured.
The investigators will test the feasibility of using transcranial direct current stimulation (tDCS) and speech therapy to treat participants with motor speech disorders caused by Frontotemporal Lobar Degeneration Pathology including nonfluent variant Primary Progressive Aphasia, Progressive Supranuclear Palsy, Corticobasal Syndrome, or behavioral variant Frontotemporal Dementia. The investigators will deliver transcranial direct current stimulation (tDCS) either in a clinic setting at the University of California San Francisco, or in patients' homes, via a consumer tDCS device and videoconferencing. Transcranial direct current stimulation (tDCS) is a neuromodulation technique that can enhance the benefits of speech therapy treatment. Participants will receive a dose of tDCS stimulation + speech therapy and a dose of sham tDCS + speech therapy in a randomized double blind crossover study performed either in the clinic or at home via videoconferencing. This study can be performed entirely remotely.
Primary progressive aphasia (PPA) is a progressive neurological disorder that causes a gradual decline in communication ability as a result of selective neurodegeneration of speech and language networks in the brain. PPA is a devastating condition affecting adults as young as their 40's or 50's, depriving them of the ability to communicate and function in society. There has been significant progress in discovering the neurobiological mechanisms that underlie PPA and in identifying its clinical phenotypes. With these advances, we are poised to investigate behavioral treatments that are grounded in modern cognitive and neuroanatomical concepts. Research documenting the efficacy of speech-language treatment for PPA is emerging, but limited. Systematic research is needed to establish best clinical practices in this unique patient population for whom pharmacological treatment remains elusive. The long-term objectives of this project are to provide evidence-based treatment methods addressing the speech and language deficits in PPA and to determine the neural predictors of responsiveness to intervention. The study has three main goals that build on the findings of our previous work: 1) to examine the utility of treatments designed to facilitate significant, generalized and lasting improvement of speech-language function in PPA, 2) to determine whether treatment alters the trajectory of decline in PPA by comparing performance on primary outcome measures in treated versus untreated participants after a one-year interval, and 3) to identify imaging predictors (gray matter, white matter, and functional connectivity measures) of responsiveness to behavioral intervention in individuals with PPA. In order to accomplish these aims, we will enroll 60 individuals with PPA, who will undergo a comprehensive multidisciplinary evaluation and neuroimaging. Subsequently, participants will be enrolled in treatment designed to promote lasting and generalized improvement of communicative function in core speech-language domains. Participants will be followed for up to one-year post-treatment in order to determine long-term effects of rehabilitation, and their performance will be compared with a historical cohort of untreated PPA patients. This ambitious study and the necessary recruitment will be possible due to an ongoing collaboration with the UCSF Memory and Aging Center, a leading institution in the field of PPA research. The study will broaden the evidence base supporting the efficacy of speech-language intervention in PPA and will provide novel evidence regarding neural predictors of treatment outcomes, with the potential to inform clinical decision-making and improve clinical care for individuals with this debilitating disorder.
The purpose of this study is to learn more about amyotrophic lateral sclerosis (ALS) and other related neurodegenerative diseases, including frontotemporal dementia (FTD), primary lateral sclerosis (PLS), hereditary spastic paraplegia (HSP), progressive muscular atrophy (PMA) and multisystem proteinopathy (MSP). More precisely, the investigator wants to identify the links that exist between the disease phenotype (phenotype refers to observable signs and symptoms) and the disease genotype (genotype refers to your genetic information). The investigator also wants to identify biomarkers of ALS and related diseases.
ECOCAPTURE@HOME is a study which is currently being developed with the objective to capture the behavioral signature of apathy in everyday life context through remote monitoring of participants' behavior for about one month. Participants will not only be patients with apathy but also their spouse caregiver. Behavioral markers of apathy will be extracted from a combination of: 1/ objective physiological data from sensors on a bracelet worn by participants; 2/ subjective data filled by the caregiver through an application. Thus investigators will collect a pool of metrics and show they can measure three assumed behavioral markers of apathy (daytime activity, quality of sleep and emotional arousal), which in turn allow to predict caregiver's perception of the dyad's psychological state. The final goal is to lay the foundations for the development of a clinical tool for the remote follow-up of patient-caregiver couples.
A prospective, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 3 and older with confirmed diagnosis of Niemann Pick disease type C1 (NPC1). The objective of this study is to evaluate the safety, tolerability and efficacy of 2000 mg/kg dose of Trappsol Cyclo (hydroxypropyl betacyclodextrin) administered intravenously compared to standard of care. An open-label sub-study in countries following European Medicines Agency (EMA) guidance will enroll asymptomatic or symptomatic patients from infancy up to age 3 to evaluate safety in that population.
Cognitive neurodegenerative diseases are a major public health issue. At present, the diagnosis of certainty is still based on anatomopathological analyses. Even if the diagnostic tools available to clinicians have made it possible to improve probabilistic diagnosis during the patient's lifetime, there are still too many diagnostic errors and sub-diagnostic in this field. The arrival of biomarkers has made it possible to reduce these diagnostic errors, which were of the order of 25 to 30%. This high error rate is due to different parameters. These diseases are numerous and often present common symptoms due to the fact that common brain structures are affected. These diseases evolve progressively over several years and their early diagnosis, when the symptoms are discrete, makes them even more difficult to diagnose at this stage. In addition, co-morbidities are common in the elderly, further complicating the diagnosis of these diseases. At present, the only cerebrospinal fluid (CSF) biomarkers that are routinely used for the biological diagnosis of neurodegenerative cognitive pathologies are those specific to Alzheimer's disease: Aβ42, Aβ40, Tau-total and Phospho-Tau. These biomarkers represent an almost indispensable tool in the diagnosis of dementia. It is therefore important to determine whether Alzheimer's biomarkers can be disrupted in other neurodegenerative cognitive pathologies, but also to find biomarkers specific to these different pathologies by facilitating the implementation of clinical studies which will thus make it possible to improve their diagnosis.