Active clinical trials for "Pick Disease of the Brain"

Primary progressive aphasia (PPA) is a disorder characterized by gradual decline in speech-language ability caused by underlying neurodegenerative disease. PPA is a devastating condition that can affect adults as young as their 50's, depriving them of the ability to communicate and function in society. Along with Alzheimer's Disease and other Alzheimer's Disease Related Dementias (AD/ADRD), PPA is now identified earlier and with greater precision. Increasingly, patients and families seek options for behavioral and neuromodulatory treatments to address PPA's devastating effects on communication, prolong speech-language skills, and maximize quality of life. Studies have documented the robust benefits of speech-language telerehabilitation methods for persons with PPA, with in-home treatment resulting in immediate and long-term benefits. This investigation aims to further enhance the potency of these treatment approaches by pairing them with tailored neuromodulatory intervention that targets critical brain networks supporting treatment in each clinical subtype of PPA. The study will evaluate the feasibility and preliminary benefit of home-based transcranial direct current stimulation (tDCS) combined with evidence-based speech-language telerehabilitation methods. tDCS will be delivered to patients in their own homes and site of stimulation will be tailored for each clinical subtype of PPA. This project has the potential to enhance clinical management and rehabilitation for individuals with PPA by establishing the benefit of behavioral and neuromodulatory treatment that is neurobiologically-motivated and accessible for patients and families.

A pivotal, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Niemann Pick disease type C (NPC). The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care.

A prospective, randomized, double-blind, placebo controlled therapeutic study in participants with confirmed diagnosis of Niemann-Pick disease type C (NPC). The purpose of this study is to assess the efficacy and safety of arimoclomol (compared to placebo) when it is administered as an add-on therapy to the participant's current prescribed best routine clinical care; participant's routine clinical care may, or may not, include miglustat. The CT-ORZY-NPC-002 study has been expanded to include an open label paediatric sub-study including participants aged 6 to <24 months at study enrolment.

This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Niemann-Pick type C disease (NPC). There are two phases to this study: the Parent Study, and the Extension Phase. The Parent Study evaluates the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the symptomatic treatment of NPC. The Extension Phase evaluates the long-term safety and efficacy of IB1001 for the neuroprotective, disease-modifying treatment of NPC.

Niemann-Pick disease, type C (NPC) is a lethal, autosomal recessive, lysosomal storage disorder characterized by neurodegeneration in early childhood and death in adolescence. NPC results from mutation of either the Niemann-Pick C1 disease (NPC1) (~95% of cases) or NPC2 genes. NPC is characterized by the endolysosomal storage of unesterified cholesterol and lipids in both the central nervous system and peripheral tissues such as the liver. Individuals with NPC demonstrate progressive central nervous system decline including inability to coordinate balance, gait, extremity and eye movements. Acute liver disease in the newborn/infant period is frequently observed, but subsequently resolves. However, chronic, sub-clinical liver disease persists. Intrathecal 2-Hydroxypropyl-β-Cyclodextrin (HP-β-CD, VTS-270) has proven effective in reducing the signs and prolonging life in animal models and Phase 1/2a data support efficacy in NPC1 patients. VTS-270 also has been shown to be effective in treating liver disease in the NPC1 cat. This Phase 1/2a, open-label, multiple ascending dose trial will evaluate whether VTS-270 administered intravenously is effective in treating acute liver disease in NPC1 infants.

This is a Phase 2a study to assess the the safety and tolerability of TPN-101 in patients with Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD) Associated with Hexanucleotide Repeat Expansion in the C9orf72 gene (C9ORF72 ALS/FTD).

Neuroinflammation is increasingly implicated as a potential critical pathogenic mechanism in a variety of neurologic and psychiatric disorders. This study will use hybrid PET/MRI imaging to evaluate neuroinflammation and its relationship to cerebral perfusion in frontotemporal dementia (FTD). Patients with FTD will be recruited from the Cognitive Neurology and Aging Brain clinics at Parkwood Institute and will undergo neurocognitive assessment and MRI/PET using the PET ligand FEPPA which binds to activated microglia, a marker of neuroinflammation. Correlations will be conducted to determine whether abnormal neuroinflammation is present in Frontotemporal dementia and whether differential patterns of neuroinflammation are present in different FTD clinical and molecular subtypes, and to determine the relationship between neuroinflammation, cerebral perfusion using arterial spin labeling MRI imaging techniques, and indices of brain structure including volumetric and white matter analysis.

Background and objects Amyloid plaques and tau protein are the landmarks of neurodegeneration in Alzheimer's disease (AD). On the other hand, it is reported that cerebral ischemia may induce amyloid plaques and tau protein accumulation. However, it was difficult to in vivo disentangle the complex and dynamic interactions between AD pathophysiology and cerebral vascular injury during the post-stroke cognitive impairment development in the past. With the advent of novel radiotracers specific to cerebral amyloid plaques and tau protein, we aim to conduct a prospective multimodal neuroimaging cohort study to investigate the contribution of vascular injury, amyloid plaque and tau protein to cognitive impairment. Subjects and methods The prospective project plans to recruit patients with vascular cognitive impairment (VCI) (Group A, n=80), Alzheimer's disease/mild cognitive impairment (MCI) (Group B, n = 120), fronto-temporal dementia (FTD) (Group C, n =30), and progressive supranuclear palsy (PSP) (Group E, n = 80). In addition, another 30 healthy people will be recruited as the control group (Group D, n=30). [18F]AV45 and [18F]MNI-958(PMPBB3) PET will be done for imaging cerebral amyloid plaque and tau protein distribution, brain MRI for obtaining structural and functional information, and neuropsychological tests for cognitive performance. Cognitive evaluation will be repeated 18 months after recruitment. In addition, APOE genotyping will be performed as well. By obtaining the neuroimaging information, such as severity of white matter change and infarction, cortical and hippocampal atrophy, and SUVRs of [18F]AV-45 and [18F]MNI-958(PMPBB3) PET, the study will be able to investigate the composite influence of cerebrovascular disease and neurodegenerative pathology on the trajectory of cognitive impairment. Group comparisons will be performed using the Chi-square test, independent t test, Mann-Whitney U test, ANOVA test, and multiple linear regression, where appropriate. Anticipation In this project, we will be able to explore the distribution patterns of amyloid plaque and tau protein among dementia patients with different etiologies, and also evaluate their influence on cognition

The goal of this study is to remediate word-finding problems in patients who have Primary Progressive Aphasia (PPA) or Alzheimer's Disease and to delay the further progression of word-finding impairment. The current approach is novel in that it contains a prophylaxis component in which the investigators attempt to strengthen neural connections that remain functional, making them more resistant to degradation as the disease progresses. While the study is specific in its targeting of word-finding problems, a successful outcome would bode well for other studies aimed at prevention or reversal of declining cognitive functions in dementia. One set of participants with PPA will receive practice with picture naming in two conditions: viewing the picture and repeating the name; and viewing the picture with its written name, plus reading and writing the name. Another set of participants with PPA or Alzheimer's Disease will be trained in two different conditions: learning about the word's semantic features (meaning); and learning about the word's lexical features (letters and sounds). Naming of pictures trained in each of these conditions will be compared, at three time intervals post-training, with naming of pictures tested before the study but never trained. It is predicted that the pairing of the picture with its written name, combined with the motor task of writing the name, will result in a greater ability to name the picture at a later date than simple practice viewing the picture and repeating the name. Furthermore, it is predicted that participants who have difficulty understanding concepts will be more likely to respond to semantic treatment, while participants who have difficulty connecting words with concepts will be more likely to respond to lexical treatment.

This study will evaluate evidence-based treatments for adults with mild Primary Progressive Aphasia (PPA). The aim of the study is to help identify efficacious communication and quality of life interventions for those with PPA and their care-partners. Participants with a diagnosis of PPA and their actively-engaged care partners will be involved in the study for 12 months. Each participant will receive a iPad equipped with the necessary applications and features for the study. Participants will complete evaluations, speech therapy sessions with a speech and language therapist, and sessions with a licensed social worker or related clinician. They will have access to Communication Bridge, a personalized web application to practice home exercises that reinforce treatment strategies. There are no costs to participate in this study.