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Frontotemporal Dementia clinical trials at University of California Health

18 in progress, 5 open to eligible people

Showing trials for
  • First-in-Human Evaluation of an Astrocytic Glutamate Transporter (EAAT2) PET Tracer in Dementia

    open to eligible people ages 40-75

    This is a first in human study that will assess the safety and diagnostic performance of [18F]RP-115 (fluorine-18 labeled RP115), a positron emission tomography (PET) agent. This agent has the potential to identify the early changes that occur in the brains of patients with Alzheimer's disease (AD) and frontotemporal dementia (FTD).

    at UCSF

  • Veri-T: A Trial of Verdiperstat in Patients With svPPA Due to TDP-43 Pathology

    open to eligible people ages 18-85

    The purpose of the study is to test the safety and tolerability of twice daily Verdiperstat in patients with semantic variant primary progressive aphasia (svPPA) due to frontotemporal lobar degeneration with TDP-43 pathology (FTLD-TDP). Three-fourths of the participants will receive Verdiperstat and one-fourth will receive Placebo during the 24-week treatment duration.

    at UCSF

  • ARTFL LEFFTDS Longitudinal Frontotemporal Lobar Degeneration (ALLFTD)

    open to eligible people ages 18 years and up

    ARTFL LEFFTDS Longitudinal Frontotemporal Lobar Degeneration (ALLFTD) represents the formalized integration of ARTFL (U54 NS092089; funded through 2019) and LEFFTDS (U01 AG045390; funded through 2019) as a single North American research consortium to study FTLD for 2019 and beyond.

    at UCLA UCSD UCSF

  • Characterization of Inclusion Body Myopathy Associated With Paget's Disease of Bone and Frontotemporal Dementia (IBMPFD)

    open to eligible people ages 18 years and up

    The investigators are researching families with inherited inclusion body myopathy (IBM) and/or Paget disease of bone (PDB) and/or dementia (FTD) which is also called IBMPFD. IBMPFD is caused by mutations in the VCP gene. Our main goal is to understand how changes in the VCP gene cause the muscle, bone and cognitive problems associated with the disease. The investigators are collecting biological specimen such as blood and urine samples, family and medical histories, questionnaire data of patients with a personal or family history of VCP associated disease. Participants do not need to have all symptoms listed above in order to qualify. A select group of participants may be invited to travel to University of California, Irvine for a two day program of local procedures such as an MRI and bone scan. Samples are coded to maintain confidentiality. Travel is not necessary except for families invited for additional testing.

    at UC Irvine

  • Clinical Procedures to Support Research in ALS

    open to eligible people ages 18 years and up

    The purpose of the Clinical Procedures To Support Research (CAPTURE) study is to utilize information collected in the medical record to learn more about a disease called amyotrophic lateral sclerosis (ALS) and related disorders.

    at UC Irvine

  • Long-term AL001 Dosing in Frontotemporal Dementia (FTD) Patients (INFRONT-2)

    Sorry, in progress, not accepting new patients

    A Phase 2 open label study evaluating the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of AL001 in participants with a Granulin mutation or C9orf72 mutation causative of frontotemporal dementia.

    at UCSF

  • TPN-101 in Patients With C9ORF72 ALS/FTD

    Sorry, in progress, not accepting new patients

    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).

    at UC Irvine UCSD UCSF

  • AL001 in Frontotemporal Dementia (INFRONT-3)

    Sorry, in progress, not accepting new patients

    A phase 3 double blind, placebo controlled study evaluating the efficacy and safety of AL001 in participants at risk for or with frontotemporal dementia due to heterozygous mutations in the progranulin gene.

    at UCSD

  • Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C

    Sorry, in progress, not accepting new patients

    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.

    at UCSF

  • Intranasal Oxytocin for Frontotemporal Dementia

    Sorry, in progress, not accepting new patients

    The purpose of this study is to assess the safety, tolerability and effects on behaviour of Syntocinon given intranasally (by a spray into the nostrils) compared to placebo (an inactive saline substance that contains no medication) in participants with frontotemporal dementia/Pick's disease. This study will take place in approximately 15 centres across Canada and the United States. Approximately 112 patients in total will be enrolled in this study. In the first phase we will examine which of three different dosing schedules of oxytocin may be more effective. In the second phase of the study, patients entering the study will be randomized to the oxytocin dosing schedule that appeared most effective in the first phase.

    at UCLA UCSF

  • Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1

    Sorry, in progress, not accepting new patients

    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.

    at UCSF

  • Speech-Language Treatment With Remotely Supervised Transcranial Direct Current Stimulation in Primary Progressive Aphasia

    Sorry, in progress, not accepting new patients

    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.

    at UCSF

  • Care Ecosystem Consortium Effectiveness Study

    Sorry, accepting new patients by invitation only

    The Care Ecosystem is an accessible, remotely delivered team-based dementia care model, designed to add value for patients, providers and payers in complex organizational and reimbursement structures. Care is delivered via the phone and web by unlicensed Care Team Navigators, who are trained and supervised by a team of dementia specialists with nursing, social work, and pharmacy expertise. The evidence base to date suggests that the Care Ecosystem improves outcomes important to people with dementia, caregivers, and payers when delivered in a controlled research environment, including reduced emergency department visits, higher quality of life for patients, lower caregiver depression, and reduced potentially inappropriate medication use (Possin et al., 2019; Liu et al., 2022). The investigators propose a rapid pragmatic trial in 6 health systems currently offering the Care Ecosystem program in geographically and culturally diverse populations. The investigators will leverage technology, delivering care via the phone and web and using electronic health records to monitor quality improvements and evaluate outcomes while maximizing external validity. The investigators will evaluate the effectiveness of the Care Ecosystem on outcomes important to patients, caregivers, healthcare providers, and health systems during the pandemic. By evaluating the real-world effectiveness in diverse health systems that are already providing this model of care, this project will bridge the science-practice gap in dementia care during an unprecedented time of heightened strain on family caregivers, healthcare providers and health systems.

    at UCLA UCSF

  • Treatment for Speech and Language in Primary Progressive Aphasia

    Sorry, in progress, not accepting new patients

    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.

    at UCSF

  • 4-Repeat Tauopathy Neuroimaging Initiative - Cycle 2

    Sorry, in progress, not accepting new patients

    The goal of this study is to identify the most reliable methods of analysis for tracking CBD, PSP, and o/vPSP over time. The results from this study may be used in the future to calculate statistical power for clinical drug trials. The study will also provide information about the relative value of novel imaging techniques for diagnosis, as well as the value of imaging techniques versus testing of blood, urine, and cerebrospinal fluid (CSF) 'biomarkers'.

    at UCSD UCSF

  • Early Access Program With Arimoclomol in US Patients With NPC

    Sorry, not accepting new patients

    NPC is a rare, relentlessly progressive, neurological disease and associated with serious morbidity and shortened life expectancy. The purpose of this Expanded Access Program is to provide early access to arimoclomol for patients with Niemann-Pick Type C disease who, in the opinion and the clinical judgement of the treating physician, may benefit from treatment with arimoclomol. Participants will receive treatment with arimoclomol until their doctor finds it does not help them anymore, they withdraw, or the study is stopped for any reason.

    at UCSF

  • Neurofilament Surveillance Project (NSP)

    Sorry, in progress, not accepting new patients

    This is a biomarker study designed to collect and analyze blood specimens from individuals carrying known familial frontotemporal lobar degeneration (f-FTLD) mutations compared to a control group of individuals without known f-FTLD mutations. The NSP is an ancillary study to the ARTFL LEFFTDS Longitudinal Frontotemporal Lobar Degeneration" (ALLFTD) study, NCT04363684. More information can be found at https://www.allftd.org/.

    at UCSF

  • Phenotype, Genotype & Biomarkers in ALS and Related Disorders

    Sorry, accepting new patients by invitation only

    The goals of this study are: (1) to better understand the relationship between the phenotype and genotype of amyotrophic lateral sclerosis (ALS) and related diseases, including primary lateral sclerosis (PLS), hereditary spastic paraplegia (HSP), progressive muscular atrophy (PMA), and frontotemporal dementia (FTD); and (2) to develop biomarkers that might be useful in aiding therapy development for this group of disorders.

    at UCSD

Our lead scientists for Frontotemporal Dementia research studies include .

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