Clinical trials: Primary Progressive Aphasia (PPA)
Niemann-Pick Disease, Type C1
Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1
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).
- Trial name
- TransportNPC (NCT04860960)
- Testing
- Hydroxypropyl-beta-cyclodextrin
- Where
- 35 sites in United States, Argentina and 11 more
- Volunteers
- 94 people
- Sponsor
- Cyclo Therapeutics, Inc.
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.
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
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.
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
Niemann-Pick Disease, Type C
Early Access Program With Arimoclomol in US Patients With NPC
NPC is a rare, relentlessly progressive, neurological disease and associated with serious morbidity and shortened life expectancy.
- Trial name
- NCT04316637
- Testing
- Arimoclomol
- Where
- 13 sites in United States
- Sponsor
- ZevraDenmark
Niemann-Pick Type C Disease, GM1 Gangliosidosis
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease and in…
- Conditions
- Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
- Trial name
- NCT07054515
- Testing
- AZ-3102
- Where
- 39 sites in United States, Argentina and 15 more
- Volunteers
- 147 people
- Sponsor
- Azafaros B.V.
Alzheimer's Disease
Transcranial Electromagnetic Treatment (TEMT) Against Alzheimer's Disease
This is a second extension of EM 1000-1 wherein mild/moderate AD subjects who participated in the original study have completed participation in a first extension of 4-months.
- Trial name
- NCT04271163
- Testing
- MemorEM
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- NeuroEM Therapeutics, Inc.
Frontotemporal Dementia
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL593 in Healthy Participants and Participants With Frontotemporal Dementia (FTD-GRN)
This is a Phase 1/2, multicenter, randomized, placebo-controlled, double-blind study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple doses of DNL593 in two parts…
- Trial name
- NCT05262023
- Testing
- DNL593
- Where
- 26 sites in United States, Belgium and 10 more
- Volunteers
- 85 people, healthy volunteers welcome
- Sponsor
- Denali Therapeutics Inc.
Granulin Mutation
Study to Assess the Safety, Tolerability, and Pharmacodynamic (PD) Effects of FRM-0334 in Subjects With Prodromal to Moderate Frontotemporal Dementia With Granulin Mutation
The purposes of this study are to investigate the safety, tolerability, and pharmacodynamics of FRM-0334 in subjects with prodromal to moderate frontotemporal dementia with granulin mutation.
- Trial name
- NCT02149160
- Testing
- FRM-0334
- Where
- 13 sites in United States, Belgium and 4 more
- Volunteers
- 30 people
- Sponsor
- FORUM Pharmaceuticals Inc
Alzheimer Disease, Parkinson Disease
Research Study for Patients With Neurological Diseases Which Evaluates the Patient Experience of the MemorEM Device
Primary Objective: The primary objective of this pilot study is to gain voluntary feedback from patients with neurological diseases and their caregivers regarding their experiences using the MemorEM head device.
- Conditions
- Alzheimer Disease, Parkinson Disease, Frontotemporal Dementia, Cortico Basal Degeneration
- Trial name
- MemorEM (NCT07222605)
- Testing
- MemorEM
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- eQ8Health Corporation d/b/a CareONE Concierge
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
Acid Sphingomyelinase Deficiency, Niemann-Pick Disease
Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)
The purpose of this study is to determine the safe range of single doses of rhASM administered to adults with ASM deficiency.
- Trial name
- NCT00410566
- Testing
- rhASM
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Genzyme, a Sanofi Company
Niemann-Pick Diseases
Induced Pluripotent Stem Cells for Niemann Pick Disease
Establishment of individualized human cellular disease models based on induced pluripotent stem cells that reflect the broad heterogeneous phenotypic spectrum of Niemann Pick disease
- Trial name
- IPSNPABC (NCT03883750)
- Where
- Pakistan
- Volunteers
- 40 people
- Sponsor
- CENTOGENE GmbH Rostock
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.
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
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
Epidemiological Study in FRONtoTemporal Dementia
An international, multicenter, epidemiological observational study aims to investigate the prevalence of genetic etiologies in patients diagnosed with FTD or clinically suspected for FTD.
- Trial name
- EFRONT (NCT05075187)
- Testing
- Genetic Screening
- Where
- 37 sites in Belgium, Germany and 5 more
- Volunteers
- 2,287 people, healthy volunteers welcome
- Sponsor
- CENTOGENE GmbH Rostock
ALS, FTD
Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD)
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.
- Trial name
- FOCUS-C9 (NCT04931862)
- Testing
- WVE-004
- Where
- 17 sites in Australia, Belgium and 6 more
- Volunteers
- 35 people
- Sponsor
- Wave Life Sciences USA, Inc.