Clinical trials: Aphasia Primary Progressive
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
Frontotemporal Dementia, FTD
A Study of PBFT02 in Participants With FTD and Mutations in the Granulin Precursor (GRN) or C9ORF72 Genes
PBFT02 is a gene therapy for frontotemporal dementia intended to deliver a functional copy of the GRN gene to the brain. This study will assess the safety, tolerability and efficacy of this treatment in patients with…
- Conditions
- Frontotemporal Dementia, FTD, FTD-GRN, Dementia Frontotemporal
- Trial name
- upliFT-D (NCT04747431)
- Testing
- PBFT02
- Where
- 10 sites in United States, Australia and 3 more
- Volunteers
- 30 people
- Sponsor
- Passage Bio, Inc.
Niemann-Pick Disease, Niemann-Pick Disease, Type C
Biomarker for Niemann Pick Type C Disease (BioNPC)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Niemann Pick Type C disease from Blood (plasma)
- Trial name
- BioNPC (NCT01306604)
- Where
- 4 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
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
Frontotemporal Dementia, Pick Complex
A Pilot Study to Explore the Safety and Tolerability of Galantamine HBr in the Treatment of Pick Complex/Frontotemporal Dementia
The purpose of this study is to explore the safety and tolerability and the efficacy of galantamine treatment in subjects with Pick Complex/ Frontotemporal Dementia (PC/FTD).
- Trial name
- NCT00416169
- Testing
- galantamine hydrobromide
- Volunteers
- 41 people
- Sponsor
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Hurler Syndrome (MPS I), Hurler-Scheie Syndrome
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
The goal of this research study is to establish chimerism and avoid graft-versus-host-disease (GVHD) in patients with inherited metabolic disorders.
- Conditions
- Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III)
- Trial name
- NCT01372228
- Testing
- hematopoietic stem cell infusion
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Talaris Therapeutics Inc.
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.
Alzheimer Disease
A First-In-Human Study of LY3954068 in Participants With Early Symptomatic Alzheimer's Disease
The main purpose of this study is to evaluate the safety of LY3954068 in participants with early symptomatic Alzheimer's Disease (AD).
- Trial name
- NCT06297590
- Testing
- LY3954068, Flortaucipir F18
- Where
- 10 sites in United States, Japan and 1 more
- Volunteers
- 48 people
- Sponsor
- Eli Lilly and Company
Frontotemporal Dementia (FTD), Amyotrophic Lateral Sclerosis (ALS)
[18F]ACI-19626 PET in TDP-43 Proteinopathies
The goal of this clinical trial is to test whether we can reliably and safely measure the accumulation of pathological protein TDP-43 [involved in rare forms of dementia such as frontotemporal dementia (FTD) and in amyotrophic…
- Conditions
- Frontotemporal Dementia (FTD), Amyotrophic Lateral Sclerosis (ALS), TDP-43 Proteinopathies, Suspected Limbic Predominant Age-related TDP-43 Encephalopathy (LATE)
- Trial name
- NCT06891716
- Testing
- [18F]ACI-19626
- Where
- Netherlands
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- AC Immune SA