Clinical trials: Brain Disorder, Metabolic
Pompe Disease
First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
This is an international, multi-center, open-label study designed to evaluate if the co-administration of investigational new drugs ATB200 and AT2221 is safe in adults with Pompe disease.
- Trial name
- NCT02675465
- Testing
- ATB200, AT2221
- Where
- 19 sites in United States, Australia and 4 more
- Volunteers
- 29 people
- Sponsor
- Amicus Therapeutics
Frontotemporal Dementia, Right Sided Cerebral Hemisphere Cerebrovascular Accident
Efficacy of Web-Based Social-Cognitive Interventions in Right Hemisphere Stroke and Frontotemporal Dementia
This study is being done to test the feasibility and effectiveness of web-based social-cognitive training exercises in treating social-emotional deficits in patients with stroke or frontotemporal dementia (FTD).
- Trial name
- NCT02506036
- Testing
- Brain HQ
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Johns Hopkins University
Type 1 Diabetes, Hypoglycemia Unawareness
Naltrexone and Hypoglycemia in Type 1 Diabetes
Low blood sugar is also called hypoglycemia. Usually, it is mild and can be treated quickly and easily by eating or drinking a small amount of a sugar-rich food.
- Trial name
- Naltrexone (NCT01053078)
- Testing
- Naltrexone
- Where
- United States
- Volunteers
- 29 people, healthy volunteers welcome
- Sponsor
- University of Minnesota
Stroke, Myocardial Infarction
Insulin Resistance Intervention After Stroke Trial
The purpose of this study is to determine if pioglitazone is effective in preventing future strokes or heart attacks among non-diabetic persons who have had a recent ischemic stroke.
- Conditions
- Stroke, Myocardial Infarction, Diabetes
- Trial name
- IRIS (NCT00091949)
- Testing
- pioglitazone
- Where
- 183 sites in United States, Australia and 6 more
- Volunteers
- 3,876 people
- Sponsor
- Yale University
Fabry Disease
Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks
This open-label switchover study will assess the safety, efficacy, and pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg administered every 4 weeks for 52 weeks in Fabry patients previously treated with ERT: agalsidase…
- Trial name
- BRIGHT (NCT03180840)
- Testing
- Pegunigalsidase alfa
- Where
- 14 sites in United States, Belgium and 5 more
- Volunteers
- 30 people
- Sponsor
- Protalix
Ornithine Transcarbamylase (OTC) Deficiency
Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency
This is a Phase 1/2, open-label, single arm, multicenter, safety and dose finding study of DTX301 in adults with late-onset OTC deficiency.
- Trial name
- CAPtivate (NCT02991144)
- Testing
- scAAV8OTC, Reactive Corticosteroid Taper Regimen, Prophylactic Corticosteroid Taper Regimen
- Where
- 9 sites in United States, Canada and 2 more
- Volunteers
- 16 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Pompe Disease
A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease
The objective of this study was to evaluate the efficacy and safety of treatment with 4000 litre (L) alglucosidase alfa (Lumizyme®) in Pompe participants.
- Trial name
- NCT01526785
- Testing
- Alglucosidase alfa
- Where
- 51 sites in United States
- Volunteers
- 113 people
- Sponsor
- Genzyme, a Sanofi Company
Pompe Disease, Glycogen Storage Disease Type II (GSD-II)
Growth and Development Study of Alglucosidase Alfa
Pompe disease (also known as glycogen storage disease Type II) is a rare autosomal recessive metabolic muscle disease caused by the deficiency of acid α glucosidase (GAA), an enzyme that degrades lysosomal glycogen.
- Conditions
- Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease
- Trial name
- NCT00486889
- Testing
- alglucosidase alfa
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- Genzyme, a Sanofi Company
Frontotemporal Lobar Degeneration
Effects of Tolcapone on Frontotemporal Dementia
This study will test the effects of a medication called tolcapone on cognitive, behavioral, and language problems seen in patients with frontotemporal dementia (FTD).
- Trial name
- NCT00604591
- Testing
- Tolcapone
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Columbia University
Cardiovascular Disease
The Evaluation of Bococizumab (PF-04950615;RN316) in Reducing the Occurrence of Major Cardiovascular Events in High Risk Subjects
This study evaluates the PCSK9 inhibitor, Bococizumab (PF-04950615;RN316), compared to placebo, in reducing the occurrence of major cardiovascular events, including cardiovascular death, myocardial infarction, stroke, and…
- Trial name
- SPIRE-1 (NCT01975376)
- Testing
- bococizumab (PF-04950615)
- Where
- 1,745 sites in United States, Argentina and 34 more
- Volunteers
- 16,784 people
- Sponsor
- Pfizer
Frontotemporal Dementia (FTD)
Low-Dose Lithium for the Treatment of Behavioral Symptoms in Frontotemporal Dementia
Frontotemporal dementia (FTD) is a progressive neurodegenerative illness that affects the frontal and anterior temporal lobes of the brain.
- Trial name
- Lithium (NCT02862210)
- Testing
- Lithium Carbonate
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Columbia University
Cardiovascular Disease
The Evaluation of Bococizumab (PF-04950615; RN316) in Reducing the Occurrence of Major Cardiovascular Events in High Risk Subjects
This study evaluates the PCSK9 inhibitor, Bococizumab (PF-04950615;RN316), compared to placebo, in reducing the occurrrence of major cardiovascular events, including cardiovascular death, myocardial infarction, stroke, and…
- Trial name
- SPIRE-2 (NCT01975389)
- Testing
- bococizumab (PF-04950615)
- Where
- 1,595 sites in United States, Argentina and 33 more
- Volunteers
- 10,564 people
- Sponsor
- Pfizer
Chronic Cerebral Ischemia
Safety and Efficacy of Sequential Therapy With Mexidol® in Patients With Chronic Cerebral Ischemia
The purpose of this study is to evaluate safety and efficacy of sequential treatment with Mexidol® in patients with chronic cerebral ischemia (CCI).
- Trial name
- MEMO (NCT06834490)
- Testing
- Mexidol
- Where
- 14 sites in Russia, Uzbekistan
- Volunteers
- 318 people
- Sponsor
- Pharmasoft
Friedreich's Ataxia
(+) Epicatechin to Treat Friedreich's Ataxia
This 24-week study will test the safety and effectiveness of synthetically produced (+) Epicatechin in treating patients who have Friedreich's Ataxia, a neurological disorder.
- Trial name
- NCT02660112
- Testing
- (+)-Epicatechin
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Ralitza Gavrilova
Hemochromatosis, Cerebrovascular Accident
Stroke With Transfusions Changing to Hydroxyurea
The purpose of this study is to compare standard therapy (transfusions and chelation) with alternative therapy (hydroxyurea and phlebotomy) for the prevention of secondary stroke and management of iron overload in children with…
- Conditions
- Hemochromatosis, Cerebrovascular Accident, Anemia, Sickle Cell, Hematologic Diseases
- Trial name
- NCT00122980
- Testing
- Red Cell Transfusions, Iron Chelation, Hydroxyurea
- Where
- 28 sites in United States
- Volunteers
- 134 people
- Sponsor
- St. Jude Children's Research Hospital
Parkinson's Disease (PD)
An Open-label Study in Healthy Male Subjects to Assess the Absorption, Distribution, Metabolism and Excretion of [14C]-Labelled BIA 9-1067 and Metabolites
The purpose of this study is to determine the rate and routes of excretion of OPC and the mass balance in urine, faeces and expired air.
- Trial name
- NCT02169427
- Testing
- OPC
- Where
- Netherlands
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Bial - Portela C S.A.
Ataxia Telangiectasia
Evaluate the Neurological Effects of EryDex on Subjects With A-T
This is an international, multi-center, randomized, prospective, double-blind, placebo-controlled, Phase 3 study, designed to assess the effect of EryDex (dexamethasone sodium phosphate [DSP] in autologous erythrocytes)…
- Trial name
- NEAT (NCT06193200)
- Testing
- Dexamethasone sodium phosphate
- Where
- 19 sites in United States, Denmark and 7 more
- Volunteers
- 105 people
- Sponsor
- Quince Therapeutics S.p.A.
Cardiovascular Disease, Peripheral Arterial Disease
Treatment of HDL to Reduce the Incidence of Vascular Events HPS2-THRIVE
The primary aim is to assess the effects of raising HDL cholesterol (the good type) with extended release niacin/laropiprant 2g (previously known as MK-0524A) versus matching placebo on the risk of heart attack or coronary death…
- Conditions
- Cardiovascular Disease, Peripheral Arterial Disease, Diabetes Mellitus, Coronary Heart Disease
- Trial name
- HPS2-THRIVE (NCT00461630)
- Testing
- ER niacin/laropiprant, simvastatin, ezetimibe/simvastatin
- Where
- United Kingdom
- Volunteers
- 25,673 people
- Sponsor
- University of Oxford
Gaucher Disease
A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
A protocol to extend the assessment of the safety and efficacy of taliglucerase alfa in pediatric subjects (2 to <18 years old) with symptoms and clinical manifestations of Gaucher disease who completed treatment in Protocols…
- Trial name
- NCT01411228
- Testing
- Taliglucerase alfa
- Where
- 3 sites in Israel, Paraguay and 1 more
- Volunteers
- 15 people
- Sponsor
- Pfizer
Wilson Disease
Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840
The main objective of the study is to evaluate the change in liver copper (Cu) concentration following 48 weeks of treatment with ALXN1840 in adult participants with Wilson Disease (WD) who have been previously treated for at…
- Trial name
- NCT04422431
- Testing
- Bis-Choline Tetrathiomolybdate
- Where
- 12 sites in United States, Canada and 6 more
- Volunteers
- 31 people
- Sponsor
- Alexion Pharmaceuticals, Inc.