Clinical trials: Storage Disease
Pediatric Emergency Medicine, Communication
PIMPmyHospital: a Mobile App to Improve Emergency Care Efficiency and Communication
This pilot study is a prospective, single-center, randomized, controlled trial in a tertiary pediatric emergency department with two parallel groups of voluntary post-graduate year 1 to 5 pediatric residents and registered…
- Trial name
- NCT05203146
- Testing
- PIMPmyHospital (mobile health tool), Conventional method
- Where
- Switzerland
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- Pediatric Clinical Research Platform
Benign Prostatic Hyperplasia
A Study to Track the Safety of Vesomni in Men in South Korea Treated in Routine Clinical Practice for the Urinary Symptoms of Benign Prostatic Hyperplasia (Enlarged Prostate)
Benign prostatic hyperplasia (BPH), also known as an enlarged prostate, happens more often in men as they age. This condition causes a sudden need to pass urine, which is hard to control.
- Trial name
- NCT07619105
- Testing
- Vesomni
- Volunteers
- 600 people
- Sponsor
- Astellas Pharma Korea, Inc.
Fabry Disease
Establishment of Biomarkers for Fabry Disease
Fabry disease, an x-linked recessive lysosomal storage disease (LSD) is commonly recognized as a cause of renal failure in involved men and more recently recognized in women too.
- Trial name
- NCT01165697
- Testing
- Fluorescein angiography
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Ohio State University
Fabry Disease
Replagal Enzyme Replacement Therapy for Adults With Fabry Disease
This study will determine the safety and effectiveness of the drug Replagal for treating people with Fabry disease, an inherited metabolic disorder.
- Trial name
- NCT00097890
- Testing
- Replagal (Agalsidase Alfa), Replagal
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Mucopolysaccharidosis I, Lysosomal Storage Diseases
Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I
The investigators are studying the use of enzyme replacement therapy into the spinal fluid for treatment of spinal cord compression in the Hurler-Scheie and Scheie forms of mucopolysaccharidosis I (MPS I).
- Conditions
- Mucopolysaccharidosis I, Lysosomal Storage Diseases, Spinal Cord Compression
- Trial name
- NCT00215527
- Testing
- laronidase
- Where
- 2 sites in United States, Finland
- Volunteers
- 4 people
- Sponsor
- Patricia I. Dickson, M.D.
Rare Disorders, Sarcoma of Bone and Connective Tissue
Characterization of the Methylation Profile of Stored Human Samples
The project, funded by the PNRR under Mission 4 "Education and Research," aims to strengthen the network of Italian biobanks by improving data management in accordance with the FAIR principles (Findable, Accessible…
- Conditions
- Rare Disorders, Sarcoma of Bone and Connective Tissue, Inflammatory Articular Diseases
- Trial name
- DNAmetCRB (NCT07573072)
- Where
- Italy
- Volunteers
- 90 people
- Sponsor
- Istituto Ortopedico Rizzoli
Pompe Disease
Frequency of Pompe Disease in Patients With Myalgia With or Without Hyper Ckemia - Data From the Reference Center (CERCA)
Pompe's disease is a lysosomal storage disease of autosomal recessive genetic transmission due to a deficiency in acid alpha glucosidase.
- Trial name
- POEM (NCT05092230)
- Where
- Martinique
- Volunteers
- 100 people
- Sponsor
- University Hospital Center of Martinique
Intentional Self-Harm by Other Specified Means
Comparison of Lethal Means Counseling and an Active Control Condition, With and Without Provision of Gun Locks
In 2013, the National Guard reported a suicide rate that was substantially higher than both the general population and the active duty component of the United States military.
- Trial name
- NCT03375099
- Testing
- Lethal Means Counseling, Health and Stress Reduction
- Where
- United States
- Volunteers
- 232 people, healthy volunteers welcome
- Sponsor
- University of Southern Mississippi
Urinary Bladder, Overactive, Pain
Myrbetriq™ (Mirabegron) to Reduce Pain and Discomfort Following Ureteral Stent Placement
The objective of this pilot study is to assess whether Myrbetriq™ will improve post-operative ureteral pain and discomfort, reduce bladder storage symptoms and increase quality of life following ureteral stenting.
- Conditions
- Urinary Bladder, Overactive, Pain, Lower Urinary Tract Symptoms
- Trial name
- NCT02462837
- Testing
- Mirabegron
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Southern Illinois University
Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II
Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III
The study quantitates behavioral challenges in mucopolysaccharidosis type I-III and parental coping strategies
- Conditions
- Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type III, Behavior Disorders
- Trial name
- NCT03161171
- Testing
- This is an observational study
- Where
- Germany
- Volunteers
- 34 people
- Sponsor
- University Hospital Heidelberg
MPS IIIA, Sanfilippo Syndrome
Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease
Open-label, clinical trial of scAAV9.U1a.hSGSH injected intravenously through a peripheral limb vein
- Conditions
- MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
- Trial name
- NCT04088734
- Testing
- ABO-102
- Where
- 3 sites in United States, Australia and 1 more
- Volunteers
- 5 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Mucopolysaccharidosis I (MPS I)
Mucopolysaccharidosis I (MPS I) Registry
The Mucopolysaccharidosis I (MPS I) Registry is an ongoing, observational database that tracks the outcomes of patients with MPS I.
- Trial name
- NCT00144794
- Where
- 141 sites in United States, Argentina and 35 more
- Volunteers
- 1,406 people
- Sponsor
- Genzyme, a Sanofi Company
Glycogen Storage Disease
Neurocognitive Function and Neuroimaging Features in Patients With Glycogen Storage Diseases
The goal of this observational study is to learn about cognitive function and brain imaging findings in people with glycogen storage disease (GSD).
- Trial name
- NEURO-GSD (NCT07908212)
- Where
- China
- Volunteers
- 318 people, healthy volunteers welcome
- Sponsor
- Peking Union Medical College Hospital
Erectile Dysfunction, Lower Urinary Tract Symptoms
Efficacy of Tadalafil (5mg) For Treatment of Early Storage Symptoms and Erectile Dysfunction After Endoscopic Enucleation of Prostate
From previous studies, the investigators found that sexual outcomes after prostate surgery may show insignificant improvement, remain unchanged or deteriorate in non-negligible number of patients especially those with high…
- Trial name
- NCT05955001
- Where
- Egypt
- Volunteers
- 110 people
- Sponsor
- Mansoura University
Diet Habit, Diet Modification
Evaluating the Relative Effectiveness of Two Front-of-pack Nutrition Labels
In efforts to promote a healthy diet, the Singapore Health Promotion Board (HPB) has attempted to use Front-of-Pack (FOP) labelling to supplement traditional nutrition labelling.
- Conditions
- Diet Habit, Diet Modification, Food Selection, Food Preferences
- Trial name
- NUSMart NM (NCT03761342)
- Testing
- Multiple Traffic Light Label, Nutri-Score Label
- Where
- Singapore
- Volunteers
- 154 people, healthy volunteers welcome
- Sponsor
- Duke-NUS Graduate Medical School
Hypochondroplasia
Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia
Phase 2, multicenter, OLE study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR (fibroblast growth factor receptor) 1-3-selective tyrosine kinase inhibitor, in participants with…
- Trial name
- NCT07393373
- Testing
- Infigratinib
- Where
- 24 sites in United States, Australia and 8 more
- Volunteers
- 135 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Asmd, Visceral Type
Magnetic Resonance Spectroscopy in Acid Sphingomyelinase Deficiency
The goal of this study is to assess the ability of MRI techniques to detect early stages of lipid accumulation in the liver of ASMD patients with the chronic visceral subtype compared to healthy subjects.
- Trial name
- MONACO (NCT05904366)
- Testing
- MR Spectroscopy (MRS), MR Elastography (MRE)
- Where
- Netherlands
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Eline C. B. Eskes
Neuroblastoma
4SCAR-T Therapy Targeting GD2, PSMA and CD276 for Treating Neuroblastoma
The purpose of this clinical trial is to assess the feasibility, safety and efficacy of multiple 4SCAR-T cell therapy which targets GD2, PSMA and CD276 surface antigens in patients with relapsed and refractory neuroblastoma (NB).
- Trial name
- NCT04637503
- Testing
- GD2, PSMA and CD276 CAR-T cells
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Sanfilippo Syndrome Type A
Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of recombinant human heparan N-sulfatase (rhHNS, GC1130A) administered via intracerebroventricular access device…
- Trial name
- NCT06567769
- Testing
- GC1130A
- Where
- 4 sites in United States, South Korea
- Volunteers
- 9 people
- Sponsor
- GC Biopharma Corp
Tay-Sachs Disease, Porphyria, Erythropoietic
Diagnostic and Screening Study of Genetic Disorders
OBJECTIVES: I. Determine the phenotypic heterogeneity of patients with genetic disorders including their clinical spectrum and natural history. II.
- Conditions
- Tay-Sachs Disease, Porphyria, Erythropoietic, Leukodystrophy, Globoid Cell, Metabolism, Inborn Errors
- Trial name
- NCT00006057
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- National Center for Research Resources (NCRR)
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