Clinical trials: Storage Disease
Fabry Disease
Efficacy and Safety of Enzyme Replacement Therapy in Patients With Fabry Disease
The goal of this observational study is to learn how enzyme replacement therapy works in patients with Fabry disease. The main question it aims to answer is: What medical problems do participants have? Participants already on…
- Trial name
- FAVORIT (NCT06880250)
- Where
- 23 sites in Russia
- Volunteers
- 100 people
- Sponsor
- NPO Petrovax
Stroke, Multiple Sclerosis
Development and Application of Tele-Neuro Rehabilitation System
Disability due to neurological diseases is increasing in our country and world. With regular physiotherapy and rehabilitation, as well as patients' symptoms are brought under control, their affected functions in daily life are…
- Conditions
- Stroke, Multiple Sclerosis, Parkinson Disease
- Trial name
- NCT05031403
- Testing
- Telerehabilitaton, Home Exercise
- Where
- Turkey (Türkiye)
- Volunteers
- 114 people
- Sponsor
- Istanbul University - Cerrahpasa
Sickle Cell Trait
Mobilization and Handling of Stem Cells for Transplant From Healthy Volunteers With Sickle Cell Trait
This study will examine the effects of granulocyte colony-stimulating factor (G-CSF) on bone marrow stem cells in healthy volunteers with sickle cell trait and determine if cells collected for transplantation from donors with…
- Trial name
- NCT00005782
- Testing
- Granulocyte colony-stimulating factor, Leukapheresis
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Fabry Disease
Study of the Relation Between Lipid Myocardial Overload Evaluated by Cardiac Magnetic Resonance Imaging (MRI), Alteration of Longitudinal Myocardial Deformations by Echocardiography, and Clinical Achievements (Functional, Biological and Electrical) in Fabry Disease, and Its Outcomes.
Anderson-Fabry disease is a genetic lysosomal storage disease, linked to chromosome X (gene GLA), responsible of enzyme synthesis deficit in α-galactosidase A with intracellular sphingolipids accumulation and multiorganic…
- Trial name
- FABRY-Image (NCT03123523)
- Testing
- Echocardiography at T0, Exercise test, Biological assays
- Where
- France
- Volunteers
- 55 people, healthy volunteers welcome
- Sponsor
- University Hospital, Bordeaux
Hypertension, Pulmonary, Hemolysis
Use of Inhaled Nitric Oxide to Prevent Pulmonary Hypertension Associated to Stored Blood Transfusion
The objective of this study is to assess if prolonged storage time of a packed red blood cell unit may cause pulmonary vasoconstriction after transfusion, in a susceptible population such as cardiac surgery patients.
- Trial name
- NCT02217683
- Testing
- Nitric Oxide
- Where
- United States
- Sponsor
- Massachusetts General Hospital
Cerebrovascular Accident, Stroke, Acute
Fabry and Stroke Epidemiological Protocol (FASEP): Risk Factors In Ischemic Stroke Patients With Fabry Disease
More than one million people in Europe suffer from a stroke every day. Normally older people have a stroke, but also a significant number of younger people between 18 and 55 years.
- Conditions
- Cerebrovascular Accident, Stroke, Acute, Cerebral Stroke
- Trial name
- FASEP (NCT01182818)
- Where
- 14 sites in Germany, Poland
- Volunteers
- 1,000 people
- Sponsor
- University of Rostock
Pompe Disease
Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies
Hypothesis: the effectiveness of treatment of Pompe Disease with rhGAA enzyme replacement therapy (ERT) is limited at least in part because patients develop antibodies against the provided rhGAA enzyme.
- Trial name
- NCT01451879
- Testing
- Rituximab, Miglustat
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- University of Florida
Inhalation; Gas
Sevoflurane Wash in Method Using Conventional Flows Versus Low Flows of 0.5L
The goal of this observational study is to to evaluate a technique of initiating Sevoflurane low-flow anesthesia which is simple, has a quick wash-in and minimises gas consumption without compromising the safety when compared to…
- Trial name
- NCT06209112
- Testing
- Sevoflurane
- Where
- Oman
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Royal Hospital, Oman
Fabry Disease, Cerebrovascular Accident
Stroke in Young Fabry Patients (sifap2): Characterization of the Stroke Rehabilitation
New studies indicate that in about 1 - 2 percent of the younger stroke patients the cause could have been an undiagnosed genetic disease, the so called Fabry disease.
- Trial name
- sifap2 (NCT00413595)
- Testing
- No intervention
- Where
- 20 sites in Austria, Croatia and 5 more
- Volunteers
- 100 people
- Sponsor
- CENTOGENE GmbH Rostock
Mucopolysaccharidosis I, Mucopolysaccharidosis II
Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI
Randomized, double-blind, placebo-controlled, parallel-group, single-center study followed by open-label phase, to evaluate the effects of adalimumab compared to placebo on the change from baseline in joint and skeletal disease…
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VI
- Trial name
- NCT03153319
- Testing
- Adalimumab Injection [Humira], Saline Solution for Injection
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Gaucher Disease
Miglustat on Gaucher Disease Type IIIB
evaluate the combination therapy with Miglustat and enzyme replacement therapy (ERT) on Gaucher disease
- Trial name
- NCT02520934
- Testing
- Miglustat, ERT
- Where
- Taiwan
- Volunteers
- 19 people, healthy volunteers welcome
- Sponsor
- National Taiwan University Hospital
Mucopolysaccharidosis VI
Study of rhASB in Patients With Mucopolysaccharidosis VI
The purpose of this study is to evaluate the long-term efficacy and safety of rhASB treatment in patients with Mucopolysaccharidosis VI.
- Trial name
- NCT00104234
- Testing
- N-acetylgalactosamine 4-sulfatase
- Where
- United States
- Volunteers
- 39 people
- Sponsor
- BioMarin Pharmaceutical
Mucopolysaccharidoses
Validation and Reliability of Iris Cameras in Mucopolysaccharidoses
To assess the reliablity and validity of 2 new iris cameras in the assement of corneal opacification in mucopolysaccharidoses
- Trial name
- NCT05354219
- Testing
- Imaging
- Where
- United Kingdom
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Manchester University NHS Foundation Trust
Glycogen Storage Disease Type III
Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With Glycogen Storage Disease Type III (GSD III)
The primary objective of this study is to evaluate potential biomarkers of GSD III.
- Trial name
- NCT04574830
- Testing
- No Intervention
- Where
- 4 sites in United States
- Volunteers
- 18 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Hypovitaminosis D
Vitamin D and Adipose Tissue: a Complex Relationship
The goal of this observational study is to verify the role of adipose tissue in determining the vitamin D serum level after monthly oral administration in subjects with vitamin D deficiency.
- Trial name
- ADIPOVITAD (NCT05957692)
- Where
- Italy
- Volunteers
- 58 people
- Sponsor
- Azienda Ospedaliera Universitaria Integrata Verona
Lysosomal Storage Disease
The Classification and Cause of Leukodystrophies of Unknown Cause
Leukodystrophy is a disease of the white matter of the brain. White matter is the portion of the brain responsible for conducting electrical impulses from one area of the brain to the other.
- Trial name
- NCT00001671
- Where
- 5 sites in United States, France and 2 more
- Volunteers
- 400 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
End Stage Renal Disease on Dialysis, Hemodialysis
Interdialytic Peritoneal UltraFiltration in HemoDialysis Patients
Sodium accumulates in the tissues of patients with kidney disease and impairs survival. Three-times weekly, standard hemodialysis alone cannot remove excess sodium efficiently.
- Conditions
- End Stage Renal Disease on Dialysis, Hemodialysis, Peritoneal Dialysis, Nonosmotic Sodium Storage
- Trial name
- IPUF-HD (NCT04603014)
- Testing
- Interdialytic peritoneal ultrafiltration with 10% dextrose solution
- Where
- Canada
- Volunteers
- 10 people
- Sponsor
- London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's
Opioid Misuse
Text Intervention to Facilitate Secure Storage and Disposal of Prescription Opioids
The proposed study aims to develop and implement a text message intervention that will instruct and motivate individuals to securely store opioid medications during treatment and subsequently dispose of unused medications…
- Trial name
- NCT05503186
- Testing
- Text Message, Standard of Care
- Where
- United States
- Volunteers
- 479 people, healthy volunteers welcome
- Sponsor
- Wake Forest University Health Sciences
Overactive Bladder Syndrome
Impact of TTNS on Bladder Symptoms Among People With MS, A RCT
Background: Neurogenic lower urinary tract dysfunction is common among people with multiple sclerosis (MS). Recent studies showed that bladder storage symptoms are predominant among MS with a pooled prevalence of frequency at…
- Trial name
- NCT06341673
- Testing
- Active Transcutaneous Electrical Nerve Stimulation (TENS)
- Where
- Saudi Arabia
- Volunteers
- 72 people
- Sponsor
- Hawra Al-Dandan
Pompe Disease (Late-onset)
A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)
This is a multicenter, international open-label extension study of ATB200/AT2221 in adult subjects with late-onset Pompe disease (LOPD) who completed Study ATB200-03.
- Trial name
- NCT04138277
- Testing
- AT2221, ATB200
- Where
- 60 sites in United States, Argentina and 21 more
- Volunteers
- 119 people
- Sponsor
- Amicus Therapeutics
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