Clinical trials: Fabry's 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
Fabry Disease
Study of the Spermatic Characteristics of Patients With Fabry Disease
The objective of this project is to estimate the prevalence of spermatic abnormalities in patients with Fabry disease.The main objective of this project is to estimate the prevalence of spermatic abnormalities in patients with…
- Trial name
- FERTIFABRY (NCT04073888)
- Testing
- Semen collection
- Where
- France
- Volunteers
- 22 people
- Sponsor
- University Hospital, Bordeaux
Periodontal Disease, Periodontitis
Periodontal Disease in Rare Renal Disorders (PERIO-RA-RE)
This study aims to evaluate the burden and phenotypic spectrum of periodontal disease in patients with rare kidney disorders (such as Alport syndrome, Fabry disease, and tuberous sclerosis complex) and systemic lupus…
- Conditions
- Periodontal Disease, Periodontitis, CKD, Chronic Kidney Disease
- Trial name
- PERIO-RA-RE (NCT07575347)
- Testing
- Observational assessment
- Where
- Romania
- Volunteers
- 100 people
- Sponsor
- Stefan Lujinschi
Fabry Disease
Autoimmune and Inflammatory Response Biomarkers in Fabry Disease
The goal of this observational study is to understand the immune response in Fabry disease (FD). We want to find out how the immune response is related to the severity of FD and how it affects patients' quality of life and pain.
- Trial name
- Bio-FAIR (NCT06007768)
- Where
- Spain
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Fundacion para la Investigacion Biomedica del Hospital Universitario Ramon y Cajal
Fabry Disease
Home Therapy With Replagal in Fabry Disease
The purpose of this study is to proof increasing patient satisfaction and preservation of quality of life in patients with Morbus Fabry disease receiving their Enzyme Replacement Therapy with Replagal (Agalsidase alfa) at home…
- Trial name
- NCT01355146
- Where
- 22 sites in Austria, Germany
- Volunteers
- 127 people
- Sponsor
- Shire
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
Fabry Disease
Therapeutic Implications of CMR Low Native T1 in Non-treated Non-hypertrophic Females With Fabry Disease (FD)
Females with Fabry disease are characterized by variable phenotypes regarding time of onset, severity of symptoms and progression of disease due to the random inactivation of X chromosome leading to a cellular mosaic of mutated…
- Trial name
- NATI-Fab (NCT07872852)
- Testing
- Decision tree for initiating treatment
- Where
- 12 sites in France
- Volunteers
- 60 people
- Sponsor
- University Hospital, Bordeaux
Fabry Disease
Replagal Enzyme Replacement Therapy for Children With Fabry Disease
Primary Objective(s): To assess the safety of Replagal at a dose of 0.2 mg/kg administered over 40 (+/-10) minutes in children with Fabry disease To assess the effect of Replagal on heart rate variability in patients 7 to 17…
- Trial name
- NCT00084084
- Testing
- Agalsidase alfa
- Where
- 14 sites in United States, Canada
- Volunteers
- 17 people
- Sponsor
- Shire
Fabry Disease
Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease
Primary Objective: To assess the safety, pharmacokinetics (PK), pharmacodynamics (PD), and exploratory efficacy of GZ/SAR402671 in enzyme replacement therapy treatment-naïve adult male participants diagnosed with Fabry disease.
- Trial name
- NCT02228460
- Testing
- GZ/SAR402671
- Where
- 8 sites in United States, France and 3 more
- Volunteers
- 11 people
- Sponsor
- Genzyme, a Sanofi Company
Lysosomal Storage Disorders, Gaucher Disease
Investigating Lysosomal Storage Diseases in Minority Groups
Although lysosomal storage disorders, such as Fabry disease, Gaucher disease, and Pompe disease, represent serious challenges in the healthcare system, no study has yet investigated the prevalence of these diseases in the US.
- Conditions
- Lysosomal Storage Disorders, Gaucher Disease, Fabry Disease, Pompe Disease
- Trial name
- NCT02120235
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- O & O Alpan LLC
Fabry Disease, Fabry´s Disease
The Efficacy and Safety of Switch Between Agalsidase Beta to Agalsidase Alfa for Enzyme Replacement in Patients With Anderson-Fabry Disease
The current approved treatment for Fabry disease is enzyme replacement therapy (ERT). There are actually 2 products in this therapeutic class available: Replagal® (agalsidase alfa) and Fabrazyme® (agalsidase beta).
- Conditions
- Fabry Disease, Fabry´s Disease, Anderson-Fabry Disease
- Trial name
- SWITCH (NCT01268241)
- Where
- 9 sites in Argentina, Belgium and 6 more
- Volunteers
- 200 people
- Sponsor
- CENTOGENE GmbH Rostock
Fabry Disease
A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease
The purpose of this study is to observe the safety of agalsidase alfa in Canadian patients with Fabry disease.
- Trial name
- NCT01298141
- Testing
- agalsidase alfa
- Where
- 12 sites in Canada
- Volunteers
- 171 people
- Sponsor
- Shire
Fabry Disease
Pre-Clinical White Matter Changes and Associated Connectivity Effects in Fabry Disease
The purpose of this research project is: to use an advanced quantitative MRI technique (FBFI) to detect and quantify brain lesion in patients with FD to use fMRI to identify altered brain function to use FBFI and fMRI together to…
- Trial name
- NCT03678324
- Testing
- Functional MRI and fast bound-pool fraction imaging
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- University of Utah
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)
Fabry Disease
Detection of Fabry Disease in Chronic Renal Failure Patients in Area Provence - Alpes - Côte d'Azur
Fabry disease is a rare genetic disease characterized by an enzyme deficiency, called alpha-galactosidase A, which normally breaks down a lipid, is missing or does not function properly.
- Trial name
- NCT01374997
- Testing
- micromethod from samples taken from blood spots on filter paper
- Where
- France
- Volunteers
- 6 people
- Sponsor
- Centre Hospitalier Universitaire de Nice
Fabry Disease, Pregnancy
Maternal and Postnatal Outcomes Study (MOS): A Global Observational Registry Assessing the Safety of Elfabrio® in Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
The goal of this observational registry is to evaluate the safety and outcomes of pregnancy and lactation in women with Fabry disease who are exposed to pegunigalsidase alfa within 30 days prior to conception and/or during…
- Conditions
- Fabry Disease, Pregnancy, Pregnancy Complications
- Trial name
- MOS (NCT06941025)
- Testing
- Not applicable- observational study
- Where
- 5 sites in United States, Germany and 3 more
- Volunteers
- 10 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Fabry Disease
Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients
The objective of this treatment protocol is to provide guidance to Treating Physicians who seek access to pegunigalsidase alfa for Fabry patients whose clinical condition, in the opinion of the Treating Physician, requires…
- Trial name
- NCT04552691
- Testing
- Pegunigalsidase Alfa
- Where
- 11 sites in United States
- Sponsor
- Chiesi Farmaceutici S.p.A.
Fabry Disease
PK/PD Study of 2 Agalsidase Formulations in Single Dose of 1 mg/kg Administered to Healthy Volunteers as IV Infusion
Pharmacokinetic/Pharmacodynamic Study of 2 agalsidase beta Formulations in Single Dose Administered to Healthy Volunteers as intravenous infusion, at a concentration of 1 mg/kg
- Trial name
- NCT05343715
- Testing
- Agalsidase beta from Biosidus 1 mg/kg, Fabrazyme (agalsidase beta) 1 mg/kg
- Where
- Argentina
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Bio Sidus SA
Fabry Disease
Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients
This is the first human treatment with PRX-102, an enzyme being developed as a long-term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry disease (alpha galactosidase deficiency).
- Trial name
- NCT01678898
- Testing
- PRX-102
- Where
- 14 sites in United States, Australia and 4 more
- Volunteers
- 18 people
- Sponsor
- Protalix
Fabry Disease
Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease
This was a multinational, open-label study to assess the efficacy and safety of AVR-RD-01 in approximately 15 male subjects, who were 16 years of age or older and postpubertal with a confirmed diagnosis of classic Fabry disease…
- Trial name
- NCT03454893
- Testing
- AVR-RD-01
- Where
- 5 sites in United States, Australia and 1 more
- Volunteers
- 15 people
- Sponsor
- AVROBIO