Clinical trials: Degenerative Brain Disorder
Wilson Disease
Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients
The main purpose of the study was to evaluate the efficacy of ALXN1840 (formerly WTX101) for 24 weeks on non-ceruloplasmin-bound copper (NCC) concentrations adjusted for molybdenum plasma concentration in participants newly…
- Trial name
- NCT02273596
- Testing
- ALXN1840
- Where
- 9 sites in United States, Austria and 3 more
- Volunteers
- 29 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Dementia-related Psychosis
Relapse Prevention Study of Pimavanserin in Dementia-related Psychosis
The purpose of this study is to evaluate the efficacy of pimavanserin compared to placebo in preventing relapse of psychotic symptoms in subjects with dementia-related psychosis who responded to 12 weeks of open label…
- Trial name
- NCT03325556
- Testing
- Pimavanserin 34 mg, Pimavanserin 20 mg
- Where
- 83 sites in United States, Bulgaria and 11 more
- Volunteers
- 392 people
- Sponsor
- ACADIA Pharmaceuticals Inc.
Friedreich Ataxia
A Clinical Study to Evaluate the Effect of MIN-102 on the Progression of Friedreich's Ataxia in Male and Female Patients
Randomized, double-blind, placebo-controlled study on the effects of MIN-102 on Biochemical, Imaging, neurophysiological, and clinical markers in patients with Friedreich's Ataxia
- Trial name
- FRAMES (NCT03917225)
- Testing
- MIN-102
- Where
- 5 sites in Belgium, France and 2 more
- Volunteers
- 39 people
- Sponsor
- Minoryx Therapeutics, S.L.
Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1
Troriluzole in Adult Participants With Spinocerebellar Ataxia
The purpose of this study is to compare the efficacy of Troriluzole (200 mg once daily) versus placebo after 48 weeks of treatment in subjects with spinocerebellar ataxia (SCA).
- Conditions
- Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3
- Trial name
- NCT03701399
- Testing
- troriluzole
- Where
- 23 sites in United States, China
- Volunteers
- 299 people
- Sponsor
- Biohaven Pharmaceuticals, Inc.
Parkinson's Disease, Primary Parkinsonism
A Trial of 18F-AV-133 Positron Emission Tomography (PET) Imaging to Differentiate Subjects With Parkinson's Disease (PD) From Other Movement Disorders
The purpose of this study is to determine whether 18F-AV-133 PET scans can be used to differentiate subjects with Parkinson's Disease from other movement disorders.
- Conditions
- Parkinson's Disease, Primary Parkinsonism, Lewy Body Parkinson's Disease
- Trial name
- NCT01550484
- Testing
- 18F-AV-133
- Where
- 15 sites in United States, Australia
- Volunteers
- 170 people
- Sponsor
- Avid Radiopharmaceuticals
NSCLC, Non Small Cell Lung Cancer, RCC, Renal Cell Cancer
A Phase 2 Study of NIR178 in Combination With PDR001 in Patients With Solid Tumors and Non-Hodgkin Lymphoma
The purpose of this phase 2 study is to evaluate the efficacy and safety of NIR178 in combination with PDR001 in multiple solid tumors and diffuse large B-cell lymphoma (DLBCL) and further explore schedule variations of NIR178 to…
- Conditions
- NSCLC, Non Small Cell Lung Cancer, RCC, Renal Cell Cancer, Pancreatic Cancer, Urothelial Cancer
- Trial name
- NCT03207867
- Testing
- NIR178, PDR001
- Where
- 21 sites in United States, Argentina and 13 more
- Volunteers
- 315 people
- Sponsor
- Novartis Pharmaceuticals
Wilson's Disease
International Wilson's Disease Patient Registry (iWilson Registry)
Longitudinal, observational, non-interventional, standard of care Registry. Data will be collected from the routinely scheduled WD clinic visits at approximately 6-12 month intervals.
- Trial name
- NCT05239858
- Where
- 16 sites in Belgium, France and 5 more
- Volunteers
- 500 people
- Sponsor
- Orphalan
Frontotemporal Dementia
A Phase 3 Study to Evaluate Efficacy and Safety of AL001 in Frontotemporal Dementia (INFRONT-3)
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.
- Trial name
- NCT04374136
- Testing
- Part 1 Blinded - AL001, Part 2 (OLE Treatment) - AL001
- Where
- 44 sites in United States, Argentina and 14 more
- Volunteers
- 119 people
- Sponsor
- Alector Inc.
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
Wilson Disease
Study of ALXN1840 on the Metabolism of a CYP2B6 Substrate in Healthy Participants
This is a Phase 1, randomized, 2-period, 2-sequence, cross-over study designed to determine the effect of ALXN1840 on the metabolism of bupropion, a sensitive cytochrome P450 2B6 (CYP2B6) substrate, in healthy male and female…
- Trial name
- NCT04526210
- Testing
- ALXN1840, Bupropion Hydrochloride
- Where
- United States
- Volunteers
- 54 people, healthy volunteers welcome
- Sponsor
- Alexion Pharmaceuticals, Inc.
Mitochondrial Diseases, Drug Resistant Epilepsy
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
This is a parallel-arm, double-blind, placebo-controlled study with a screening phase that includes a 28-day run-in phase to establish baseline seizure frequency, followed by a 24-week, randomized, placebo-controlled phase.
- Conditions
- Mitochondrial Diseases, Drug Resistant Epilepsy, Leigh Disease, Leigh Syndrome
- Trial name
- MIT-E (NCT04378075)
- Testing
- Vatiquinone
- Where
- 27 sites in United States, Canada and 7 more
- Volunteers
- 68 people
- Sponsor
- PTC Therapeutics
Spinocerebellar Degeneration
Phase II Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD)
To evaluate the efficacy, safety and pharmacokinetics profile of KPS-0373 in patients with SCD
- Trial name
- NCT00863538
- Testing
- KPS-0373
- Where
- 3 sites in Japan
- Volunteers
- 40 people
- Sponsor
- Kissei Pharmaceutical Co., Ltd.
SCA, Spinocerebellar Ataxias
Expanded Access Protocol of Troriluzole in Patients With Spinocerebellar Ataxia (SCA)
The purpose of this expanded access protocol is to provide access to the investigational drug troriluzole in patients with spinocerebellar ataxia (SCA).
- Trial name
- NCT06034886
- Testing
- Troriluzole
- Sponsor
- Biohaven Pharmaceuticals, Inc.
Spinocerebellar Ataxia
Effects of Oral Administration of Antrodia Cinnamomea Products for Clinical Symptoms in Spinocerebellar Ataxia Patients
This study aims to assess the effect of Antrodia cinnamomea on clinical symptoms in spinocerebellar ataxia patients. To investigate the advancements in neurodegenerative diseases.
- Trial name
- NCT06633003
- Testing
- Antrodia cinnamomea Products
- Where
- Taiwan
- Volunteers
- 60 people
- Sponsor
- ALPS Biotech CO. LTD
Memory Deficit Aquired Due to the Disease, e.g. Stroke, Neurodegeneration, Inflammatory Disease of CNS, Traumatic Brain Injury
Vestibular Stimulation in Sleep for Neurorehabilitation Patients
1. Why are we conducting this study? You are a patient of the neurorehabilitation clinic with one of the therapy focuses on memory and learning. Neuropsychological training is a standard therapeutic approach in this case.
- Trial name
- NCT07658027
- Testing
- Somnomat - rocking bed providing vestibular stimulation during sleep
- Where
- Switzerland
- Volunteers
- 20 people
- Sponsor
- Cereneo AG
Friedreich Ataxia
NAD+ Precursor Supplementation in Friedreich's Ataxia
The primary objective is to test the safety and tolerability of short-term therapy with a nicotinamide adenine dinucleotide (NAD+) precursor (MIB-626) in adults with Friedreich's Ataxia (FA) without overt heart failure and with a…
- Trial name
- NCT04817111
- Testing
- MIB-626
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Metro International Biotech, LLC
Friedreich Ataxia
A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's Ataxia
To evaluate the safety and tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of subcutaneous (SC) administration of CTI-1601 over 28 days in subjects with Friedreich's ataxia (FRDA).
- Trial name
- NCT05579691
- Testing
- CTI-1601
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Larimar Therapeutics, Inc.
SCD
A Phase II Double Blind Comparative Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD)
The purpose of this study is to evaluate the efficacy and safety of KPS-0373 compared to placebo in patients with Spinocerebellar Degeneration (SCD).
- Trial name
- NCT01384435
- Testing
- KPS-0373
- Where
- Japan
- Volunteers
- 200 people
- Sponsor
- Kissei Pharmaceutical Co., Ltd.
Alzheimer's Disease, Dementia
Blood Gene Expression Signature in Patients Diagnosed With Probable Alzheimer's Disease Compared to Patients Suffering From Other Types of Dementia
The objective of the study is to define the performance of blood-based signatures for Alzheimer's Disease (AD) in different patients populations including AD, non-AD dementia, and non-demented controls.
- Trial name
- NCT00880347
- Testing
- Blood sampling
- Where
- 21 sites in France
- Volunteers
- 550 people, healthy volunteers welcome
- Sponsor
- Exonhit
Machado-Joseph Disease / Spinocerebellar Ataxia 3
Study To Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta in Patients With Machado-Joseph Disease
This is an exploratory, randomized, parallel-group, dose escalation and dose-controlled study without a placebo arm. Eligible patients will be randomized in a 1:1 ratio (double-blind) to receive Cabaletta in 2 doses, once weekly…
- Trial name
- NCT02147886
- Testing
- Cabaletta for IV infusion once weekly during 24 weeks
- Where
- Israel
- Volunteers
- 15 people
- Sponsor
- Bioblast Pharma Ltd.