Clinical trials: Storage Disease
Gaucher Disease, Patient Reported Outcome
Digital Health Platform Customized for Patients With Gaucher Disease
The entry of digitization into the world in recent years is helping the health care system to operate more efficiently than in the past and has increased the participation of patients and their families in managing their health…
- Trial name
- NCT04997772
- Testing
- Digital Gaucher Platform
- Where
- Israel
- Volunteers
- 80 people
- Sponsor
- Shoshana Vilk
Gaucher Disease, Type 1, Gaucher Disease, Type 3
Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3
Multicenter, doubleblinded, randomized placebo-controlled study of arimoclomol in patients with Gaucher Disease Type 1 or 3
- Trial name
- NCT03746587
- Testing
- Arimoclomol
- Where
- 8 sites in India
- Volunteers
- 39 people
- Sponsor
- ZevraDenmark
Diarrhea, Waterborne Disease
Trial of Safe Water Storage Among People Living With HIV
Environmental health-related pathogens include faecal-oral, diarrhoeagenic microbes that may be transmitted via drinking water and are related to sanitation and hygiene.
- Conditions
- Diarrhea, Waterborne Disease, HIV
- Trial name
- NCT01376336
- Testing
- Safe storage device
- Where
- Zambia
- Volunteers
- 1,600 people, healthy volunteers welcome
- Sponsor
- London School of Hygiene and Tropical Medicine
Benign Prostatic Hyperplasia, Lower Urinary Tract Symptoms
Study of Solifenacin Succinate and Tamsulosin Hydrochloride OCAS in Males With Lower Urinary Tract Symptoms
Clinical study to examine the efficacy, safety and tolerability of combination therapy of tamsulosin hydrochloride and solifenacin succinate compared to monotherapy of tamsulosin hydrochloride in the treatment of males with LUTS…
- Trial name
- Neptune (NCT01018511)
- Testing
- tamsulosin hydrochloride OCAS 0.4 mg, tamsulosin hydrochloride/solifenacin succinate fixed dose combination (0.4 mg/6 mg)
- Where
- 118 sites in Austria, Belarus and 11 more
- Volunteers
- 1,334 people
- Sponsor
- Astellas Pharma Europe B.V.
Developmental Delay, Skeletal Abnormalities
Biomarker for Sly Disease (MPS VII) (BioSly)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Sly disease from blood (plasma)
- Conditions
- Developmental Delay, Skeletal Abnormalities, Hepatomegaly, Splenomegaly
- Trial name
- BioSly (NCT02298699)
- Where
- 5 sites in Egypt, Germany and 2 more
- Sponsor
- CENTOGENE GmbH Rostock
Multiple Sclerosis
Prevalence of Fabry Disease in a Defined Population at Risk - Patients Formerly Diagnosed With Multiple Sclerosis
The association of Multiple Sclerosis (MS) and Fabry disease is known from own clinical experiences as well as from case reports in the literature, where symptoms and suspicious results in the brain MRI led to the misdiagnosis of…
- Trial name
- NCT01271699
- Volunteers
- 250 people
- Sponsor
- CENTOGENE GmbH Rostock
Pompe Disease, Glycogen Storage Disease Type II
An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease
The purpose of this study was to evaluate the efficacy, clinical benefits and safety of a prophylactic immunomodulatory regimen given prior to first treatment with alglucosidase alfa (Myozyme®) in patients with infantile-onset…
- Trial name
- NCT00701129
- Testing
- Alglucosidase Alfa, Methotrexate, Rituximab
- Where
- 2 sites in United States
- Volunteers
- 4 people
- Sponsor
- Genzyme, a Sanofi Company
Sanfilippo Syndrome Type A (MPS IIIA)
A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients
MPS IIIA, also known as Sanfilippo A, is an inherited lysosomal storage disease (LSD). MPS IIIA is caused by a deficiency in sulfamidase, one of the enzymes involved in the lysosomal degradation of the glycosaminoglycan (GAG)…
- Trial name
- NCT03423186
- Testing
- SOBI003
- Where
- 4 sites in United States, Germany and 1 more
- Volunteers
- 6 people
- Sponsor
- Swedish Orphan Biovitrum
Gaucher Disease, Type 1
Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs
The objective of this study is to assess Elelyso treatment on bone disease in Gaucher patients currently treated with other enzyme replacement therapy.
- Trial name
- NCT04353466
- Testing
- quantitative chemical shift imaging (QCSI), Elelyso
- Volunteers
- 30 people
- Sponsor
- Shaare Zedek Medical Center
Diarrhoeal Disease
Health Impact of Treating and Safely Storing Shallow Tubewell Drinking Water
When shallow tubewells replaced highly contaminated surface water as the primary source of drinking water in Bangladesh in the late 20th century, contemporary studies demonstrated no reduction in diarrheal disease with this…
- Trial name
- NCT01350063
- Testing
- Sodium dichloroisocyanurate (NaDCC) tablet (Aquatab), Water storage container (Jerry can), No intervention
- Where
- Bangladesh
- Volunteers
- 1,800 people, healthy volunteers welcome
- Sponsor
- International Centre for Diarrhoeal Disease Research, Bangladesh
Glycogen Storage Disease Type III
French Observatory for Patients with Type 3 Glycogenosis
Glycogen storage disease type III (GSD-III) or Cori/Forbes disease, is caused by autosomal recessive mutations in the AGL gene, which codes for the glycogen debranching enzyme (GDE) involved in the release of glucose-1P from…
- Trial name
- NCT06616545
- Where
- 3 sites in France
- Volunteers
- 150 people
- Sponsor
- Institut de Myologie, France
Fabry Disease
A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.
This is an international, non-interventional research study of adult patients with Fabry Disease and their caregivers. The study comprised a prospective time and motion evaluation and a cross-sectional evaluation of patient and…
- Trial name
- NCT04281537
- Testing
- Enzyme Replacement Therapy (ERT)
- Where
- 9 sites in United States, Brazil and 3 more
- Volunteers
- 82 people
- Sponsor
- Amicus Therapeutics
Gut Health
A Study to Evaluate the Effects of a Butyrate-Polyphenol Formulation on Gut Health and Associated Symptoms
The purpose of the study is to evaluate the efficacy of a butyrate/polyphenol formulation on modulation of the gut microbiome and gastrointestinal symptoms using questionnaires in individuals with self-reported gastrointestinal…
- Trial name
- NCT07371975
- Testing
- Butyrate + Polyphenol Formulation
- Where
- United States
- Volunteers
- 124 people, healthy volunteers welcome
- Sponsor
- Supplement Formulators, Inc.
Fabry Disease
Evaluation of Efficacy and Safety of Agalsidase Beta in Heterozygous Females for Fabry Disease
Fabry disease (OMIM 301500) is an X-linked inborn error of sphingolipid metabolism resulting from the deficiency of the lysosomal enzyme alpha-galactosidase A.
- Trial name
- HEART (NCT00487630)
- Testing
- recombinant alpha-galactosidase A
- Where
- France
- Volunteers
- 34 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Metachromatic Leukodystrophy
The Natural History of Metachromatic Leukodystrophy Study (HOME Study)
The primary aims of the HOME Study are to: Design and implement a natural history study for metachromatic leukodystrophy to serve as a source of external control data, to augment or replace concurrent controls in clinical trials…
- Trial name
- NCT04628364
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- National Organization for Rare Disorders
Parkinson Disease, GBA Gene Mutation
The GBA Multimodal Study in Parkinson's Disease
This study plans to analyze the molecular and clinical mechanisms of the relationship between the GBA mutations and Parkinson's disease.
- Conditions
- Parkinson Disease, GBA Gene Mutation, Gaucher Disease
- Trial name
- NCT04101968
- Testing
- PET scan, neuroQWERTY
- Where
- 3 sites in United States, Canada
- Volunteers
- 30 people
- Sponsor
- Pacific Parkinson's Research Centre
Acute Kidney Injury, Anemia
Urinary NGAL Acute Kidney Injury After Stored Red Blood Cells Transfusion in Critically Ill Patients.
The study aims to explore the association of the novel urinary bio marker NGAL with the age of PRBCs(packed red blood cells) transfusion in critically ill patients.
- Trial name
- NCT03560921
- Testing
- urinary NGAL
- Where
- Egypt
- Volunteers
- 147 people
- Sponsor
- Kasr El Aini Hospital
MPS 7, Sly Syndrome
A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
The Phase 3 study will use a novel randomized, intra-subject placebo-controlled, single crossover design, referred to as Blind Start, to evaluate the safety and efficacy of UX003.
- Conditions
- MPS 7, Sly Syndrome, Mucopolysaccharidosis, MPS VII
- Trial name
- NCT02230566
- Testing
- UX003
- Where
- 4 sites in United States
- Volunteers
- 12 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Pompe Disease, Hypersensitivity Reaction
A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction
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
- NCT02185651
- Testing
- Zavesca® Prescription
- Volunteers
- 2 people
- Sponsor
- University of Florida
Prostate Cancer
Studying Stored Samples From Patients With Localized Prostate Cancer Treated on Clinical Trial NCI-00-C-0154
RATIONALE: Studying samples of blood in the laboratory from patients before and after treatment may help doctors learn more about the effects of the treatment on cells.
- Trial name
- NCT00535834
- Testing
- diagnostic procedure, molecular diagnostic method, protein expression analysis
- Volunteers
- 48 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
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