Clinical trials: Brain Disorder, Metabolic
Phenylketonuria (PKU)
Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
This is a Phase 3 open-label randomized controlled study enrolling approximately 54 adolescents with PKU. The study is designed to assess the safety and efficacy of pegvaliase injections.
- Trial name
- PEGASUS (NCT05270837)
- Testing
- Pegvaliase, Diet Only
- Where
- 16 sites in United States, Germany
- Volunteers
- 55 people
- Sponsor
- BioMarin Pharmaceutical
Phenylketonurias
Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet
The primary objective of this trial is to evaluate the ability of Phenoptin to increase phenylalanine (phe) tolerance in children with phenylketonuria who are following a phe-restricted diet.
- Trial name
- NCT00272792
- Testing
- Sapropterin Dihydrochloride
- Where
- 9 sites in United States
- Volunteers
- 45 people
- Sponsor
- BioMarin Pharmaceutical
Neurodegenerative Disease, Weight Loss
Electronic-health Application To Measure Outcomes REmotely Clinical Trial
This is a phase II feasibility, safety, tolerability and preliminary efficacy study of an e-Health application versus in-person nutritional counseling to maintain or increase weight in patients with neurodegenerative diseases…
- Conditions
- Neurodegenerative Disease, Weight Loss, Amyotrophic Lateral Sclerosis, Parkinson's Disease
- Trial name
- EAT MORE (NCT02418546)
- Testing
- In-Person Nutritional Counseling by a Registered Dietitian, Nutritional counseling using an e-Health Application
- Where
- United States
- Volunteers
- 78 people
- Sponsor
- Massachusetts General Hospital
Fabry Disease
Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)
This is the first in human treatment with ST-920, an adeno-associated virus (AAV2/6) vector encoding the complementary deoxyribonucleic acid (cDNA) for human a-Gal A.
- Trial name
- NCT04046224
- Testing
- ST-920
- Where
- 18 sites in United States, Australia and 5 more
- Volunteers
- 36 people
- Sponsor
- Sangamo Therapeutics
Sialorrhea
Efficacy and Safety Study of MYOBLOC® in the Treatment of Sialorrhea in Adult Subjects
This study will evaluate the efficacy and safety of MYOBLOC in the treatment of Sialorrhea (drooling), which can be a symptom of many disease conditions. MYOBLOC will be injected directly into the salivary glands.
- Trial name
- MYSTICOL (NCT01994109)
- Testing
- MYOBLOC
- Where
- 34 sites in United States, Russia and 1 more
- Volunteers
- 187 people
- Sponsor
- Solstice Neurosciences, LLC, a subsidiary of US WorldMeds, LLC
Late Infantile Metachromatic Leukodystrophy
Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD
This is a multi-center, open-label, extension study of patients with late infantile MLD who have previously completed clinical study HGT-MLD-048 (NCT00633139), defined as the completion of all Week 52 procedures.
- Trial name
- NCT00681811
- Testing
- HGT-1111
- Where
- Denmark
- Volunteers
- 11 people
- Sponsor
- Shire
Alzheimer Disease
Evaluating Simvastatin's Potential Role in Therapy
The purpose of this study is to see how simvastatin, a cholesterol-lowering drug, affects processes related to the development of Alzheimer's disease, including: 1) levels of a substance called beta-amyloid-42 found in the spinal…
- Trial name
- ESPRIT (NCT00486044)
- Testing
- Simvastatin
- Where
- United States
- Volunteers
- 103 people, healthy volunteers welcome
- Sponsor
- University of Wisconsin, Madison
Cerebral Amyloid Angiopathy
Study Evaluating the Safety,Tolerability and Efficacy of PF-04360365 in Adults With Probable Cerebral Amyloid Angiopathy
Cerebral Amyloid Angiopathy (CAA) is a condition caused by the build-up of a protein called amyloid, predominantly Aβ40, within the walls of brain blood vessels, especially those blood vessels in the occipital lobe of the brain.
- Trial name
- NCT01821118
- Testing
- Ponezumab
- Where
- 13 sites in United States, Canada and 3 more
- Volunteers
- 36 people
- Sponsor
- Pfizer
Diabetes Type 2, Dementia
The Interaction Between Diabetes and Estradiol on Human Brain Metabolism in Postmenopausal Women
The primary aim of this study is to test whether type 2 diabetes interacts with estradiol on brain metabolism in vivo in humans.
- Trial name
- E2T2D (NCT03681691)
- Testing
- Estradiol patch
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Wake Forest University Health Sciences
Small Fiber Neuropathy, Fabry Disease
Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan
Sudoscan™ (Impeto Medical, Paris France) uses electrochemical skin conductance as a novel noninvasive method to detect sudomotor dysfunction.
- Conditions
- Small Fiber Neuropathy, Fabry Disease, Ehlers Danlos Syndrome, Mitochondrial Disease
- Trial name
- NCT02985710
- Testing
- Sudoscan, Skin biopsy, QSART
- Where
- United States
- Volunteers
- 102 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Parkinson Disease
Absorption, Distribution, Metabolism and Excretion of [14C]-Labeled BIA 9-1067 and Metabolites
To determine the absorption, metabolism and excretion of BIA 9-1067.
- Trial name
- NCT01515891
- Testing
- BIA 9-1067
- Where
- Switzerland
- Volunteers
- 4 people, healthy volunteers welcome
- Sponsor
- Bial - Portela C S.A.
Breast Cancer, Coronary Artery Disease
Reducing Metabolic Dysregulation in Obese Latina Breast Cancer Survivors Using Physical Activity
This study is about testing whether exercise will improve fitness and lessen risk factors related to heart disease, diabetes, and obesity in Latina breast cancer survivors.
- Conditions
- Breast Cancer, Coronary Artery Disease, Stroke, Type2 Diabetes
- Trial name
- NCT04717050
- Testing
- Progressive combine training (PCT), Attention Control (AC)
- Where
- United States
- Volunteers
- 64 people
- Sponsor
- Dana-Farber Cancer Institute
Gaucher Disease
A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the…
- Trial name
- NCT00376168
- Testing
- Plant cell expressed recombinant glucocerebrosidase (prGCD)
- Where
- 11 sites in United States, Canada and 6 more
- Volunteers
- 32 people
- Sponsor
- Pfizer
Frontal Lobe Dementia, Frontotemporal Lobe Dementia
Memantine (10mg BID) for the Frontal and Temporal Subtypes of Frontotemporal Dementia
The primary objective of the study is to determine whether memantine is effective in slowing the rate of behavioral decline in frontotemporal dementia.
- Conditions
- Frontal Lobe Dementia, Frontotemporal Lobe Dementia, Semantic Dementia
- Trial name
- NCT00545974
- Testing
- memantine
- Where
- 9 sites in United States
- Volunteers
- 81 people
- Sponsor
- University of California, San Francisco
Syndrome of Inappropriate Antidiuretic Hormone Secretion (SIADH)
A PK Study of 3 Dosages of Tolvaptan in Patients With Syndrome of Inappropriate Antidiuretic Hormone Secretion (SIADH)
This is a study to evaluate how the body handles and metabolizes (PK) the various doses of the drug Tolvaptan, and what the effect (PD) of the various doses of Tolvaptan are on the content of "salt" in blood and urine
- Trial name
- NCT02009878
- Testing
- tolvaptan
- Where
- 14 sites in Czechia, Denmark and 5 more
- Volunteers
- 30 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Fabry Disease
Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease
This is an open-label extension study intended to provide continued treatment with migalastat hydrochloride (HCl) for participants with Fabry disease who completed treatment of a previous migalastat HCl study.
- Trial name
- NCT02194985
- Testing
- migalastat HCl 150 mg
- Where
- 28 sites in United States, Argentina and 13 more
- Volunteers
- 84 people
- Sponsor
- Amicus Therapeutics
Hyponatremia, Dilutional Hyponatremia
Effects of Tolvaptan vs Fluid Restriction in Hospitalized Subjects With Dilutional Hyponatremia
The purpose of this study is to determine if hospitalized patients with symptomatic hyponatremia treated with tolvaptan are in the hospital for less time than patients treated with fluid restriction.
- Conditions
- Hyponatremia, Dilutional Hyponatremia, Inappropriate ADH Syndrome
- Trial name
- SALACIA (NCT01227512)
- Testing
- tolvaptan, Fluid Restriction
- Where
- 56 sites in United States
- Volunteers
- 124 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Healthy, Amyotrophic Lateral Sclerosis
P300 Brain Computer Interface Keyboard to Operate Assistive Technology
The purpose of this research is to develop tools enable people who are paralyzed to operate technology and access computers. These tools are called brain computer interfaces (BCIs).
- Conditions
- Healthy, Amyotrophic Lateral Sclerosis, Neuromuscular Disease, Spinal Cord Injury
- Trial name
- NCT00860951
- Testing
- Brain Computer Interface Keyboard
- Where
- 2 sites in United States
- Volunteers
- 29 people, healthy volunteers welcome
- Sponsor
- University of Michigan
Fabry Disease
A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease
People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme.
- Trial name
- NCT00074984
- Testing
- Fabrazyme (agalsidase beta)
- Where
- 26 sites in United States, Canada and 4 more
- Volunteers
- 82 people
- Sponsor
- Genzyme, a Sanofi Company
Alzheimer's Disease
Rasagiline Rescue in Alzheimer's Disease Clinical Trial
This is a Phase II, randomized, double blind, placebo controlled, parallel group, proof of concept three-site study, to evaluate the effect of Rasagiline in the regional brain metabolism on 18F-2-fluoro-2-deoxy-D-glucose…
- Trial name
- R2 (NCT02359552)
- Testing
- Rasagiline
- Where
- 3 sites in United States
- Volunteers
- 50 people
- Sponsor
- The Cleveland Clinic