Clinical trials: FTD
Lysosomal Acid Lipase Deficiency, Wolman Disease
Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
This was an open-label, repeat-dose, intra-participant dose-escalation study of SBC-102 (sebelipase alfa) in children with growth failure due to lysosomal acid lipase (LAL) Deficiency.
- Trial name
- NCT01371825
- Testing
- Sebelipase alfa (SBC-102)
- Where
- 7 sites in United States, Egypt and 3 more
- Volunteers
- 9 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Primary Progressive Aphasia
Transmagnetic Stimulation Pilot in Primary Progressive Aphasia
Frontotemporal degeneration (FTD) is a non-Alzheimer's dementia that is the 2nd most common cause of dementia in the United States.
- Trial name
- TMS in PPA (NCT05860647)
- Testing
- Intermittent theta burst stimulation
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- HealthPartners Institute
Dementia, Alzheimer Disease
Efficacy Study of Kinto Care Coaching for Dementia Family Caregivers
The goal of this clinical trial is to evaluate Kinto's Care Coaching intervention for dementia caregivers. The main questions it aims to answer are: Does the intervention help caregivers to address their general caregiving goals…
- Conditions
- Dementia, Alzheimer Disease, Lewy Body Disease, Vascular Dementia
- Trial name
- NCT05916664
- Testing
- Kinto Care Coaching
- Where
- United States
- Volunteers
- 495 people, healthy volunteers welcome
- Sponsor
- Kinto
Frontotemporal Dementia, Behavioral Variant
Intranasal Insulin in Frontotemporal Dementia (FTD)
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…
- Trial name
- NCT04115384
- Testing
- Novolin-R insulin
- Where
- United States
- Volunteers
- 3 people, healthy volunteers welcome
- Sponsor
- HealthPartners Institute
Primary Progressive Aphasia
HDtDCS in Logopenic Variant PPA: Effects on Language and Neural Mechanisms
This study aims to evaluate the effectiveness of a therapy called High-Definition Transcranial Direct Current Stimulation (HD-tDCS) for the treatment of the language deficits experienced by people with a type of Primary…
- Trial name
- NCT03805659
- Testing
- HD-tDCS
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Medical College of Wisconsin
Depression, Stress, Psychological
Rural Dementia Caregiver Project
Rural caregivers face challenges of geographic isolation and lower health care access and quality. Many rural dementia caregivers experience serious health consequences due to caregiving responsibilities that can limit their…
- Conditions
- Depression, Stress, Psychological, Self Efficacy, Loneliness
- Trial name
- NCT04428112
- Testing
- Building Better Caregivers Workshop, Attention Control
- Where
- United States
- Volunteers
- 418 people, healthy volunteers welcome
- Sponsor
- University of California, San Francisco
Amyotrophic Lateral Sclerosis, ALS
Study of Safety, Tolerability, and Biological Activity of LAM-002A in C9ORF72-Associated Amyotrophic Lateral Sclerosis
This is a clinical trial to evaluate the safety, tolerability, and biological effect of LAM-002A in adults with C9ORF72-associated ALS (C9ALS).
- Trial name
- NCT05163886
- Testing
- LAM-002A
- Where
- 3 sites in United States
- Volunteers
- 15 people
- Sponsor
- IRulya Therapeutics Inc.
Frontotemporal Dementia
Flortaucipir PET Imaging in Subjects With FTD
This study is designed to assess the usefulness of flortaucipir in Positron Emission Tomography (PET) imaging for subjects diagnosed with Frontotemporal Dementia (FTD).
- Trial name
- NCT03040713
- Testing
- Flortaucipir F18, Brain PET scan
- Where
- 3 sites in United States
- Volunteers
- 16 people
- Sponsor
- Avid Radiopharmaceuticals
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia
Open-label Extension (OLE) Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)
This is an OLE study conducted to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and clinical effects of WVE-004 in adult participants with ALS, FTD, or mixed ALS/FTD phenotype with a documented…
- Trial name
- NCT05683860
- Testing
- WVE-004 10 mg Q12W
- Where
- 3 sites in Netherlands, United Kingdom
- Volunteers
- 8 people
- Sponsor
- Wave Life Sciences USA, Inc.
Frontotemporal Dementia, Right Sided Cerebral Hemisphere Cerebrovascular Accident
Efficacy of Web-Based Social-Cognitive Interventions in Right Hemisphere Stroke and Frontotemporal Dementia
This study is being done to test the feasibility and effectiveness of web-based social-cognitive training exercises in treating social-emotional deficits in patients with stroke or frontotemporal dementia (FTD).
- Trial name
- NCT02506036
- Testing
- Brain HQ
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Johns Hopkins University
Neimann-Pick Disease
Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1
Niemann-Pick disease type C (NPC) is a lethal, autosomal recessive, lysosomal storage disorder characterized by neurodegeneration in early childhood and death in adolescence.
- Trial name
- NCT02124083
- Testing
- Vorinostat
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Alzheimer's Disease
A Study to Evaluate the Efficacy and Safety of ABBV-8E12 in Participants With Early Alzheimer's Disease
This study seeks to evaluate the efficacy and safety of ABBV-8E12 in participants with early Alzheimer's disease (AD).
- Trial name
- NCT02880956
- Testing
- ABBV-8E12
- Where
- 68 sites in United States, Australia and 9 more
- Volunteers
- 453 people
- Sponsor
- AbbVie
Niemann-Pick Disease, Type C
Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease
Due to different study designs, the sponsor separated Part C into this separate registration (NCT04958642), leaving Parts A/B in NCT02534844.
- Trial name
- NCT04958642
- Testing
- Adrabetadex
- Where
- 30 sites in United States, Australia and 7 more
- Volunteers
- 66 people
- Sponsor
- Mandos LLC
Hurler's Syndrome, Maroteaux-Lamy Syndrome
Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
The primary objective of this clinical trial is to evaluate the ability to achieve and sustain donor engraftment in patients with lysosomal and peroxisomal inborn errors of metabolism undergoing hematopoietic stem cell…
- Conditions
- Hurler's Syndrome, Maroteaux-Lamy Syndrome, Sly Syndrome, Alpha Mannosidosis
- Trial name
- NCT00668564
- Testing
- Stem Cell Transplantation, Cyclophosphamide, Campath-1H
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Alzheimer Disease
Alzheimer's PET Imaging in Racially/Ethnically Diverse Adults
The study employs tau positron emission tomography (PET) imaging in a well-characterized multi-racial/ethnic cohort to examine the extent to which tau pathology is associated with cognition, differences in tau pathology across…
- Trial name
- NCT03706261
- Testing
- 18F-MK-6240, 18F-Florbetaben
- Where
- United States
- Volunteers
- 145 people, healthy volunteers welcome
- Sponsor
- Adam Brickman
Aphasia, Primary Progressive Aphasia
CB3 Pilot (Communication Bridge: A Person-centered Internet-based Intervention for Individuals With Primary Progressive Aphasia)
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…
- Conditions
- Aphasia, Primary Progressive Aphasia, Dementia, Communication
- Trial name
- CB3 Pilot (NCT05697380)
- Testing
- Communication Bridge™
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- University of Chicago
Primary Progressive Aphasia
Remotely Supervised Transcranial Direct Current Stimulation (tDCS) for Primary Progressive Aphasia (PPA)
The purpose of this study is to establish the feasibility of a program of remotely supervised transcranial direct current stimulation (RS-tDCS) paired with language skills practice for people living with the semantic or logopenic…
- Trial name
- NCT05615922
- Testing
- Transcranial Direct Current Stimulation (tDCS), Word-Naming Activity
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- NYU Langone Health
Alzheimer's Disease, Frontotemporal Dementia, Behavioral Variant
Non-invasive Stimulation of Brain Networks and Cognition in Alzheimer's Disease and Frontotemporal Dementia
This pilot study aims to test clinical and connectivity changes following non-invasive stimulation of disease-specific networks in Alzheimer's disease (AD) and behavioral variant frontotemporal dementia (bvFTD).
- Trial name
- NetCogBs (NCT03422250)
- Testing
- transcranial direct current stimulation (tDCS)
- Where
- Italy
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- IRCCS Centro San Giovanni di Dio Fatebenefratelli
Sphingomyelin Lipidosis
Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency
Primary Objective: To evaluate the safety and tolerability of olipudase alfa administered intravenously in pediatric participants every 2 weeks for 64 weeks.
- Trial name
- ASCEND-Peds (NCT02292654)
- Testing
- Olipudase alfa
- Where
- 6 sites in United States, Brazil and 4 more
- Volunteers
- 20 people
- Sponsor
- Genzyme, a Sanofi Company
Frontotemporal Dementia
A Phase 2 Study to Evaluate Safety of Long-term AL001 Dosing in Frontotemporal Dementia (FTD) Patients (INFRONT-2)
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.
- Trial name
- NCT03987295
- Testing
- AL001
- Where
- 12 sites in United States, Canada and 4 more
- Volunteers
- 33 people
- Sponsor
- Alector Inc.