Clinical trials: Degenerative Brain Disorder
Friedreich Ataxia
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
The primary objective of the study is to evaluate the efficacy (using the modified Friedreich Ataxia Rating Scale [mFARS]) and safety of vatiquinone in participants with Friedreich ataxia (FA).
- Trial name
- MOVE-FA (NCT04577352)
- Testing
- Vatiquinone
- Where
- 14 sites in United States, Australia and 7 more
- Volunteers
- 146 people
- Sponsor
- PTC Therapeutics
Age-Related Macular Degeneration, Macular Degeneration
Repeated Eye Injections of Aflibercept for Treatment of Wet Age Related Macular Degeneration
To evaluate the visual outcome and number of injections required during an optical coherence tomography (OCT)-guided treat and extend regimen with intravitreal aflibercept for treatment of subfoveal neovascular age-related…
- Trial name
- ATLAS (NCT01773954)
- Testing
- Intravitreal Aflibercept Injection
- Where
- 3 sites in United States
- Volunteers
- 40 people
- Sponsor
- MidAtlantic Retina
Friedreich's Ataxia
A First in Human Study of RT001 in Patients With Friedreich's Ataxia
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of RT001 in patients with Friedreich's ataxia.
- Trial name
- NCT02445794
- Testing
- Low dose cohort, High dose cohort
- Where
- 2 sites in United States
- Volunteers
- 19 people
- Sponsor
- Biojiva LLC
Wilson's Disease
Study Comparing Once Daily Dose of 900mg of TETA 4HCL Against Cuprior® (450mg Trientine Base, Twice Daily).
A randomised, open-label study evaluating the pharmacokinetics, safety, and tolerability of a new once daily dose of 900mg of TETA 4HCL by comparing it against the current marketed Cuprior® formulation (450mg trientine base…
- Trial name
- NCT06128954
- Testing
- 900mg TETA 4HCl Once Daily Formulation, 900mg TETA 4HCl Cuprior®
- Where
- United Kingdom
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Orphalan
Primary Progressive Aphasia, MCI
tDCS Intervention in Primary Progressive Aphasia
Primary progressive aphasia (PPA) is a neurodegenerative disease that affects first and foremost language abilities. Mild cognitive impairment (MCI) is slowly progressive decline in a single domain of cognition (e.g.
- Conditions
- Primary Progressive Aphasia, MCI, FTD
- Trial name
- NCT02606422
- Testing
- Active HD-tDCS plus Speech-Language Therapy
- Where
- United States
- Volunteers
- 61 people, healthy volunteers welcome
- Sponsor
- Johns Hopkins University
High Tumor Mutation Burden, High TMB (Tumor Mutation Burden)
Study of MK-3475 (Pembrolizumab) in Patients With Microsatellite Unstable (MSI) Tumors (Cohort D)
This study will be looking at whether MK-3475 (pembrolizumab) is effective (anti-tumor activity) and safe in patients with MSI (Microsatellite Unstable) negative cancer with a mutator phenotype.
- Conditions
- High Tumor Mutation Burden, High TMB (Tumor Mutation Burden), MSS (Microsatellite Stable)
- Trial name
- NCT04098068
- Testing
- MK-3475
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Metastatic Colorectal Cancer
Safety and Efficacy of Pembrolizumab (MK-3475) Plus Binimetinib Alone or Pembrolizumab Plus Chemotherapy With or Without Binimetinib in Metastatic Colorectal Cancer (mCRC) Participants (MK-3475-651/KEYNOTE-651)
The purpose of this study is to determine safety and tolerability and to establish a preliminary recommended Phase 2 dose (RP2D) for the following combinations: pembrolizumab plus binimetinib (Cohort A), pembrolizumab plus…
- Trial name
- NCT03374254
- Testing
- Pembrolizumab, Binimetinib, Oxaliplatin
- Where
- 14 sites in United States, Canada
- Volunteers
- 116 people
- Sponsor
- Merck Sharp & Dohme LLC
Primary Progressive Aphasia, Behavioral Variant of Frontotemporal Dementia
Assessment of Hyperphosphorylated Tau PET Binding in Primary Progressive Aphasia and Frontotemporal Dementia
This study is designed to learn more about overall tau burden in the brain of patients with Primary Progressive Aphasia (PPA) and Frontotemporal Dementia.
- Conditions
- Primary Progressive Aphasia, Behavioral Variant of Frontotemporal Dementia, Frontotemporal Dementia, Behavioral Variant
- Trial name
- SLD3 (NCT02736695)
- Testing
- F-18 AV 1451
- Where
- United States
- Volunteers
- 81 people
- Sponsor
- Mayo Clinic
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.
Cerebellar Ataxia
Training in Ataxia - Individuals With Degenerative Cerebellar Diseases
Balance and aerobic training show promise as treatments for degenerative cerebellar diseases, but the neural effects of both training methods are unknown.
- Trial name
- NCT05002218
- Testing
- Aerobic Training, Balance Training
- Where
- United States
- Volunteers
- 64 people
- Sponsor
- Columbia University
Friedreich Ataxia
Neurology Measures in FA Children
The purpose of this study is to identify ways to follow progression of Friedreich's Ataxia (FA) and be able to measure changes over time in children with FA.
- Trial name
- NCT03418740
- Where
- 3 sites in United States
- Volunteers
- 108 people
- Sponsor
- Children's Hospital of Philadelphia
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.
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.
Multiple Sclerosis
Augmenting Cerebral Blood Flow to Treat Established Multiple Sclerosis
This study will evaluate how improved cerebral blood flow affects the way in which newly formed MS lesions evolve and whether tissue repair is improved.
- Trial name
- perfuseMS (NCT02466074)
- Testing
- Acetazolamide
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- The University of Texas Health Science Center, Houston
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
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.
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
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
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