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Aphasia, Primary Progressive clinical trials

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NCT ID: NCT05683860 Terminated - ALS Clinical Trials

Open-label Extension (OLE) Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)

Start date: December 14, 2022
Phase: Phase 1/Phase 2
Study type: Interventional

This is an OLE study conducted to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and clinical effects of WVE-004 in adult patients with ALS, FTD, or mixed ALS/FTD phenotype with a documented mutation in the C9orf72 gene. To participate in the study, patients must have successfully completed Phase 1b/2a WVE-004-001 study.

NCT ID: NCT04931862 Terminated - ALS Clinical Trials

Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD)

FOCUS-C9
Start date: June 28, 2021
Phase: Phase 1/Phase 2
Study type: Interventional

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.

NCT ID: NCT04115384 Terminated - Clinical trials for Frontotemporal Dementia, Behavioral Variant

Intranasal Insulin in Frontotemporal Dementia (FTD)

Start date: September 9, 2019
Phase: Phase 2
Study type: Interventional

This project will study intranasal (IN) insulin in Frontotemporal dementia (FTD) in 12 patients. Study Investigators aim to evaluate the feasibility of the EXAMINER cognitive battery as a cognitive outcome measure in FTD, the ability of the HealthPartners Center for Memory and Aging's ability to sufficiently recruit subjects with FTD, and the safety of IN regular insulin administered 20 IU twice per day in two specific variants of FTD (behavioral variant frontotemporal dementia (bv-FTD), semantic dementia (SD)) over a 4 week period.

NCT ID: NCT03887533 Terminated - Clinical trials for Niemann-Pick Disease, Type C1

Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1

Start date: January 6, 2020
Phase: Phase 1/Phase 2
Study type: Interventional

Background: For people who have Niemann-Pick disease, type C1 (NPC1), cholesterol and other fats have trouble moving out of liver and other tissue cells. This makes the cells sick. Researchers want to find out if a drug called VTS-270 can help. Objective: To test if VTS-270 is safe and effective in treating chronic liver disease associated with NPC1. Eligibility: People ages 3-60 with NPC1 Design: Participants may be screened by phone or under another protocol. Participants will have visits once a month for 12 months. If they have intrathecal injections, the study may last 15 months or more. The first visit will last about 5 days. Others will last 2-3 days. Participants will get VTS-270 injected into a vein at each visit. They can also choose to have intrathecal injections. These are like spinal taps. Some visits will also include: Physical exam Urine tests Blood tests. A small tube or needle will be inserted into the participants vein to collect blood. The small tube will also be used to give the VTS-270. Hearing tests: For one test, participants will have electrodes taped to their head. These will record brain waves. Breathing tests Ultrasound of abdomen: Sounds waves will take pictures of the participant s body. Chest x-ray: This is a picture of the lungs.

NCT ID: NCT03658135 Terminated - Clinical trials for Nonfluent Aphasia, Progressive

BIIB092 in Primary Tauopathies: CBS, nfvPPA, sMAPT, and TES

TauBasket
Start date: September 12, 2018
Phase: Phase 1
Study type: Interventional

A Phase 1b, Randomized, Double-Blind, Placebo-Controlled, Parallel Cohort Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy Study of Intravenously Infused BIIB092 in Patients with Four Different Primary Tauopathy Syndromes

NCT ID: NCT03153540 Terminated - Clinical trials for Primary Progressive Nonfluent Aphasia

Transcranial Magnetic Stimulation in Nonfluent/Agrammatic Variant Primary Progressive Aphasia

Start date: September 1, 2018
Phase: N/A
Study type: Interventional

Nonfluent/agrammatic variant primary progressive aphasia (nf/avPPA) is a fatal neurodegenerative disease that begins with isolated language deficits. There is currently no cure or treatment for this disease. Repetitive Transcranial Magnetic Stimulation (rTMS), a noninvasive neuromodulatory technique, is effective in major depression, and studied in many other conditions including nf/avPPA. Here the investigators propose to study the feasibility and change in language and brain function of a newer rTMS protocol (intermittent theta-burst stimulation, iTBS) using a randomized, blinded crossover design: participants will receive active or sham iTBS for two weeks and then switch groups without them or clinicians knowing their group. The investigators hypothesize that brain function and performance with language tasks will change after active iTBS.

NCT ID: NCT03028324 Terminated - Apraxia Speech Clinical Trials

Transcranial Magnetic Stimulation (TMS) for Primary Progressive Apraxia of Speech (PPAOS)

Start date: August 9, 2017
Phase: N/A
Study type: Interventional

The purpose of this study is to assess the influence of transcranial magnetic stimulation (TMS) on speech performance in individuals with primary progressive apraxia of speech.

NCT ID: NCT02841358 Terminated - Clinical trials for Psychiatric Adults Patients

Screening of Niemann-Pick Disease, Type C in a Psychiatric Population

NPCPsy
Start date: December 2013
Phase: N/A
Study type: Interventional

Niemann-Pick disease, Type C is a rare genetic disorder characterized by a failing in intracellular cholesterol transport, inducing an accumulation of sphingolipids in the brain. Neurological signs are at the forefront of the disease. There are also psychiatric signs of psychotic kind among 28 to 45 % of patients according to studies, and a thirty cases were published. These signs can be concomitant with neurological signs or precede them. Is is likely that psychotic disorders are the first signs of a Niemann-Pick disease not yet non encore diagnosed for some patients. Yet, no prevalence study for this disease in a psychiatric population of patients currently exists. In response to this problem this study proposes to search patients whose disease could be of organic origin or patients whose disease is suspected, based on clinical data. The diagnosis will be confirmed certified with a genetic and/or biochemical test.

NCT ID: NCT02245568 Terminated - Alzheimer's Disease Clinical Trials

Open-Label Study of Leuco-methylthioninium Bis(Hydromethanesulfonate) (LMTM) in Subjects With Alzheimer's Disease or Behavioral Variant Frontotemporal Dementia (bvFTD)

Start date: August 2014
Phase: Phase 3
Study type: Interventional

The purpose of this study is to provide subjects who have completed participation in a Phase 2 or Phase 3 trial of LMTM continued access to therapy and to evaluate the long-term safety of LMTM.

NCT ID: NCT01738386 Terminated - Adaptation Clinical Trials

Living With Frontotemporal Dementia

Start date: June 13, 2012
Phase:
Study type: Observational

Background: - Frontotemporal dementia (FTD) is the second most common cause of early-onset dementia. Alzheimer s disease is the most common. Alzheimer s disease happens most often in the elderly, but FTD typically appears between 40 and 60 years of age. It also has a strong genetic component: Up to 40% of FTD cases are linked to positive family histories. Earlier diagnoses and genetic tests mean that people with FTD will spend more years in earlier stages of disease, aware that they have it. However, few studies have looked at the personal experiences or coping styles of people with FTD. Researchers want to interview people with FTD and their caregivers to understand their experiences with the disease. This information will help create better treatments and therapies for those affected by FTD. Objectives: - To study the experiences of persons with FTD and their primary caregivers. Eligibility: - Individuals at least 18 years of age who have been diagnosed with FTD. - Primary caregivers (spouse or partner at least 18 years of age) of individuals who have been diagnosed with FTD. Design: - Before FTD participants are recruited, a pilot study will test the interview questions. This pilot study will be given to people with Alzheimer s disease and their caregivers. It will study how well people with dementia understand the interview questions. - FTD study participants will be recruited through dementia care centers. - All participants will have in-person interviews. These interviews will take up to 1 hour. - Participants with FTD will answer questions about their experience with the disease. They will talk about their mental abilities, challenges, and coping strategies. - Caregivers will answer questions about their experience in caring for someone with FTD. They will talk about their challenges and coping strategies. They will also talk about the person with FTD, and how aware they believe that the person is of the dementia symptoms. - All participants will receive a small gift card as compensation for their time. - No treatment will be provided as part of this study.