Clinical trials: Pick Disease of the Brain
Sphingomyelin Lipidosis
A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
The primary objective of this study was to obtain data regarding the safety of olipudase alfa in participants with acid sphingomyelinase deficiency (ASMD) who were exposed to long term treatment with olipudase alfa.
- Trial name
- NCT02004704
- Testing
- GZ402665
- Where
- 9 sites in United States, Belgium and 5 more
- Volunteers
- 25 people
- Sponsor
- Genzyme, a Sanofi Company
Dementia, Frontotemporal Dementia
Study in Asymptomatic GRN-FTD Patients to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VES001
This is an open-label, multiple dosing study in asymptomatic GRN-FTD carriers to investigate the safety, tolerability, PK, and PD of VES001.
- Conditions
- Dementia, Frontotemporal Dementia, Asymptomatic Condition
- Trial name
- SORT-IN-2 (NCT06705192)
- Testing
- VES001
- Where
- 2 sites in Netherlands, United Kingdom
- Volunteers
- 6 people
- Sponsor
- Mads Kjolby
Sphingomyelin Lipidosis
A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)
Primary Objective: To describe the clinical features and their severity at the time of diagnosis and their evolution over time in patients with confirmed chronic visceral and chronic neurovisceral forms of ASMD To describe…
- Trial name
- NCT04106544
- Testing
- Investigational Procedures
- Where
- 28 sites in United States, Argentina and 11 more
- Volunteers
- 84 people
- Sponsor
- Sanofi
Progressive Supranuclear Palsy, Corticobasal Degeneration
A Study to Evaluate the Safety, Tolerability and Pharmacodynamics of NIO752 in Primary Tauopathies
This is a phase II study intended to evaluate the safety, tolerability and pharmacodynamics of NIO752 in participants with primary tauopathies.
- Conditions
- Progressive Supranuclear Palsy, Corticobasal Degeneration, Frontotemporal Lobar Degeneration, Tauopathies
- Trial name
- NCT07868328
- Testing
- NIO752
- Volunteers
- 48 people
- Sponsor
- Novartis Pharmaceuticals
Niemann-Pick Diseases, Acid Sphingomyelinase Deficiency
A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)
US, multicenter, cohort, open label observational study with primary data collection. Ancillary protocol-specified procedures to address the study objectives (eg, assessment of ADA) may be considered outside the standard of care…
- Trial name
- NCT06192576
- Testing
- Olipudase alfa
- Where
- 6 sites in United States
- Volunteers
- 10 people
- Sponsor
- Sanofi
Glioblastoma Multiforme, Glioblastoma
Efficacy and Safety of NaviFUS System add-on Bevacizumab (BEV) in Recurrent GBM Patients
This is a prospective, single-arm, two stages, open-label, pilot study to investigate the efficacy and safety of FUS add-on bevacizumab (BEV) in rGBM patients.
- Conditions
- Glioblastoma Multiforme, Glioblastoma, Glioma, Brain Tumor
- Trial name
- NCT04446416
- Testing
- NaviFUS System, Bevacizumab
- Where
- Taiwan
- Volunteers
- 6 people
- Sponsor
- NaviFUS Corporation
Mild Cognitive Impairment, Dementia
COMPARATIVE EFFECTIVENESS OF MCI and DEMENTIA TREATMENTS IN A COMMUNITY-BASED DEMENTIA PRACTICE
This retrospective study is a more extensive, confirmatory analysis of the cognitive and functional outcomes initially seen in 2 groups of MCI/dementia patients in Springfield, MA and compares specialized dementia care and a…
- Conditions
- Mild Cognitive Impairment, Dementia, Hypoxia, Hyperhomocysteinemia
- Trial name
- NCT02860338
- Volunteers
- 900 people
- Sponsor
- Clionsky Neuro Systems Inc.
Alzheimer's Disease, Frontotemporal Dementia
Imaging Characteristics of Florbetapir 18F in Patients With Frontotemporal Dementia, Alzheimer's Disease and Normal Controls.
Study 18F-AV-45-010 is designed to evaluate the cerebral uptake of florbetapir 18F as measured by PET imaging in frontotemporal disorder (FTD) in comparison to cognitively normal volunteers and subjects with Alzheimer's disease…
- Trial name
- NCT01890343
- Testing
- florbetapir 18F, 18F-FDG
- Where
- United Kingdom
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Avid Radiopharmaceuticals
Alzheimer's Disease
24 Months Safety and Efficacy Study of AADvac1 in Patients With Mild Alzheimer's Disease
This study evaluates the safety and efficacy of AADvac1 in the treatment of patients with mild Alzheimer's disease. 60% of participants will receive AADvac1 and 40% of participants will receive placebo.
- Trial name
- ADAMANT (NCT02579252)
- Testing
- AADvac1
- Where
- 42 sites in Austria, Czechia and 6 more
- Volunteers
- 208 people
- Sponsor
- Axon Neuroscience SE
Neurodegenerative Diseases, Osteoarthritis
Safety and Clinical Outcomes Study: SVF Deployment for Orthopedic, Neurologic, Urologic, and Cardio-pulmonary Conditions
To evaluate for any adverse effects that may be related to the administration and reception of autologous adipose derived stromal vascular fraction (SVF).
- Conditions
- Neurodegenerative Diseases, Osteoarthritis, Erectile Dysfunction, Autoimmune Diseases
- Trial name
- NCT01953523
- Testing
- Administration of autologous adipose derived SVF
- Where
- United States
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Elliot Lander
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
Primary Progressive Nonfluent Aphasia
A 24-month Phase 1 Pilot Study of AADvac1 in Patients With Non Fluent Primary Progressive Aphasia
This study is a pilot trial evaluating the safety and immunogenicity of AADvac1 in patients with the non-fluent variant of Primary Progressive Aphasia.
- Trial name
- AIDA (NCT03174886)
- Testing
- AADvac1 40 µg, AADvac1 160 µg
- Where
- 3 sites in Germany
- Volunteers
- 33 people
- Sponsor
- Axon Neuroscience SE
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia
A Phase 2a Study of TPN-101 in Patients With C9ORF72 ALS/FTD
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…
- Trial name
- NCT04993755
- Testing
- TPN-101, 400 mg/day
- Where
- 19 sites in United States, Belgium and 3 more
- Volunteers
- 42 people
- Sponsor
- Transposon Therapeutics, Inc.
Glioblastoma Multiforme, Brain Neoplasms
Study of [68Ga]-FF58 in Patients With Selected Solid Tumors Expected to Overexpress αvβ3 and αvβ5 Integrins.
This is a First-In-Human (FIH) study of [68Ga]-FF58 to characterize the imaging properties, safety, biodistribution and dosimetry properties of [68Ga]-FF58 in adults with relapsed or refractory (r/r) glioblastoma multiforme…
- Conditions
- Glioblastoma Multiforme, Brain Neoplasms, Gastroesophageal Adenocarcinoma, Pancreatic Ductal Adenocarcinoma
- Trial name
- NCT04712721
- Testing
- 68Ga-FF58
- Where
- 2 sites in France, Germany
- Volunteers
- 14 people
- Sponsor
- Novartis Pharmaceuticals
Niemann-Pick Disease, Type C
Safety and Efficacy of Miglustat in Chinese NPC Patients
This is a prospective, multi-center, open-label, non-randomized, single-arm Phase IV confirmatory study. Approximately 19 subjects with Niemann Pick Type C disease (NPC) will be enrolled in this study.
- Trial name
- NCT03910621
- Testing
- Miglustat
- Where
- 2 sites in China
- Volunteers
- 17 people
- Sponsor
- Actelion
Alzheimer's Disease
18-months Safety Follow-up Study of AADvac1, an Active Tau Vaccine for Alzheimer's Disease
This follow-up study continues to observe patients who have completed the phase 1 trial of AADvac1, for another 18 months. Long-term safety and behavior of the immune response to AADvac1 over time are the main points of interest.
- Trial name
- FUNDAMANT (NCT02031198)
- Testing
- AADvac1
- Where
- 4 sites in Austria
- Volunteers
- 25 people
- Sponsor
- Axon Neuroscience SE
Niemann-Pick Disease Type C, GM2 Gangliosidosis
Long-Term Safety and Tolerability of Nizubaglustat in Participants With Rare Lysosomal Disorders
This is an open-label extension trial to evaluate the long-term safety and tolerability of nizubaglustat (AZ-3102) in participants with rare lysosomal disorders who have participated in a previous nizubaglustat trial and wish to…
- Conditions
- Niemann-Pick Disease Type C, GM2 Gangliosidosis, GM1 Gangliosidosis, Gaucher Disease Type 3
- Trial name
- HARBOR (NCT07885917)
- Testing
- AZ-3102
- Volunteers
- 218 people
- Sponsor
- Azafaros B.V.
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.
Inherited Metabolic Diseases, Lysosomal Storage Disorders
ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases
Eligible research subjects will receive an unrelated umbilical cord blood transfusion as a possible cure for their inherited metabolic disease.
- Conditions
- Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism
- Trial name
- UCBT-002 (NCT00654433)
- Testing
- ALD-101
- Where
- 3 sites in United States
- Volunteers
- 40 people
- Sponsor
- Aldagen
GM2 Gangliosidosis, Niemann-Pick Type C Disease
A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
This open-label study aims to gather long-term safety, tolerability, PK, biomarker, and clinical efficacy data relating to daily administration of Nizubaglustat in participants previously enrolled in the Phase 2 RAINBOW study…
- Trial name
- PRISMA (NCT07399704)
- Testing
- AZ-3102
- Where
- 3 sites in Brazil
- Volunteers
- 21 people
- Sponsor
- Azafaros B.V.