Clinical trials: Anderson Fabry Disease
Fabry Disease
Fabry Aim Children Early (ACE) Project
The purpose of this study is to assess the frequency of Fabry disease in children with early symptoms.
- Trial name
- NCT04965467
- Testing
- Screening for Fabry disease
- Where
- 21 sites in China
- Sponsor
- Children's Hospital of Fudan University
Fabry Disease
Evaluation of Phenotypic Variability in Fabry Disease
Cerebrovascular events, such as stroke, are a devastating complication of Fabry disease that results in part from storage of complex lipids in both large and small vessels.
- Trial name
- NCT03145779
- Where
- United States
- Sponsor
- Boston Children's Hospital
Fabry Disease
Pulsatility Index, Vasomotor Reactivity and Leukoencephalopathy in Fabry Patients
We hypothesize that Fabry disease - FD is associated with elevated vascular resistance induced by cerebral small-vessel disease, indicating increased distal resistance to blood flow.
- Trial name
- NCT04577170
- Testing
- Transcranial Doppler (TCD) and Transcranial Color-Coded Duplex (TCCD) ultrasonography
- Where
- Greece
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- National and Kapodistrian University of Athens
Fabry Disease
A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics of IV migalastat HCl alone and to determine absolute bioavailability for oral migalastat HCl as compared to IV administered migalastat HCl in…
- Trial name
- NCT02082327
- Testing
- IV migalastat HCl, oral migalastat HCl
- Where
- Netherlands
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Amicus Therapeutics
Fabry Disease
Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
This was a randomized, double-blind, active control study of the enzyme replacement therapy (ERT) drug PRX-102 (pegunigalsidase alfa) in Fabry disease patients with impaired renal function.
- Trial name
- BALANCE (NCT02795676)
- Testing
- PRX-102 (pegunigalsidase alfa), agalsidase beta
- Where
- 29 sites in United States, Czechia and 10 more
- Volunteers
- 78 people
- Sponsor
- Protalix
Fabry Disease
Fabry Outcome Survey (FOS)
The purpose of this study is to collect data that will increase understanding of Fabry disease history and progression, in treated and untreated patients with Fabry disease.
- Trial name
- FOS (NCT03289065)
- Where
- United States
- Volunteers
- 4,000 people
- Sponsor
- Shire
Fabry Disease
A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease
This is an 18-month, multicenter, randomized, active-control, parallel-group Phase 3 study, in which participants will be randomized to venglustat versus standard of care therapy (agalsidase alfa, agalsidase beta, or migalastat)…
- Trial name
- CARAT (NCT05280548)
- Testing
- Venglustat (GZ402671), Agalsidase alfa, Agalsidase beta (GZ419828)
- Where
- 54 sites in United States, Austria and 17 more
- Volunteers
- 104 people
- Sponsor
- Sanofi
Fabry Disease
This Study is Designed to Evaluate PD/PK and Safety of Replagal Manufactured by Two Different Processes.
This study is designed to evaluate safety and PK/PD in Canadian Fabry patients.
- Trial name
- NCT01304277
- Testing
- agalsidase alfa
- Where
- 5 sites in Canada
- Volunteers
- 17 people
- Sponsor
- Shire
Fabry Disease, Proteinuria
Antiproteinuric Agents and Fabry Disease
Fabry disease is a rare disorder that often has kidney involvement with increased urine protein excretion. Proteinuria is recognized as an important risk factor for progression of chronic kidney disease.
- Trial name
- NCT00343577
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- University of Alabama at Birmingham
Fabry Disease
Impact of MIgalastat TheRApy on CaRdiac Function in patiEnts With Fabry's Cardiomyopathy (MIRACRE-Fabry Trial)
This is an observational study. No treatment or intervention will be assigned to the subjects. All patients will receive full standard of care concomitant medication for the treatment of their cardiac condition.
- Trial name
- NCT04639999
- Testing
- Echocardiography
- Where
- South Korea
- Volunteers
- 20 people
- Sponsor
- Yonsei University
Fabry Disease
The Safety and Efficacy of Intravenous EXG110 in Patients With Fabry Disease
A phase 1/2, multicenter, open-label, Dose-escalation study to evaluate the safety and efficacy of intravenous EXG110 in patients with Fabry disease
- Trial name
- NCT06819514
- Testing
- EXG110 Injection
- Where
- 3 sites in China
- Volunteers
- 16 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd
Fabry Disease
Pharmacokinetics, Pharmacodynamics, and Safety of Moss-aGalactosidase A in Patients With Fabry Disease
Six patients with Fabry disease will be recruited. Patients will receive a single dose of 0.2 mg/kg recombinant human alpha-galactosidase A produced in moss (moss-aGal) as intravenous infusion.
- Trial name
- NCT02995993
- Testing
- Moss-aGal (recombinant human alpha-galactosidase A produced in moss)
- Where
- 2 sites in Germany
- Volunteers
- 6 people
- Sponsor
- Greenovation Biotech GmbH
Fabry Disease
Ophthalmic Findings During 10-year Enzyme Substitution of Danish Fabry Patients.
Fabry disease is a recessively inherited disorder due to systemic storage of abnormal metabolites (ceramide trihexocide, in particular) caused by lack of the lysosomal enzyme α-galactosidase.
- Trial name
- NCT01997489
- Testing
- Enzyme replacement
- Where
- 2 sites in Denmark
- Volunteers
- 39 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Fabry Disease
An Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal in Patients With Fabry Disease
This study will continue to evaluate the safety of using intravenous doses of Replagal for two patients with Fabry disease. Fabry disease is a genetic disorder inherited as an X-linked recessive trait.
- Trial name
- NCT00357786
- Testing
- Replagal agalsidase alfa, Replagal
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Cerebrovascular Accident
Stroke in Young Fabry Patients (sifap1): Frequency of Fabry Disease in Young Stroke Patients
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.
- Trial name
- sifap1 (NCT00414583)
- Testing
- No intervention
- Where
- 45 sites in Austria, Belgium and 12 more
- Volunteers
- 5,000 people
- Sponsor
- CENTOGENE GmbH Rostock
Fabry Disease
Safety Study of Replagal® Therapy in Children With Fabry Disease
The purpose of this study is to assess the safety of Replagal in children with Fabry disease who who have not previously been treated with enzyme replacement therapy (ERT).
- Trial name
- NCT01363492
- Testing
- Replagal (agalsidase alfa)
- Where
- 5 sites in United States
- Volunteers
- 15 people
- Sponsor
- Shire
Non-ischemic Cardiomyopathy, Dilated Cardiomyopathy (DCM)
Multimodal and Multidisciplinary Approach to Optimize Diagnostic, Prognostic, and Therapeutic Management of Patients with Non-ischemic Cardiomyopathies and Arrhythmogenic-inflammatory Phenotypes: a Multicenter, Observational, Retrospective and Prospective Registry Study.
Non-ischemic cardiomyopathies (NICM) represent a heterogeneous group of pathologies characterized by absence of obstructive disease of the epicardial coronary vessels and distinct structural and functional changes of the…
- Conditions
- Non-ischemic Cardiomyopathy, Dilated Cardiomyopathy (DCM), Hypertrophic Cardiomyopathy (HCM), Restrictive Cardiomyopathy
- Trial name
- AINICM (NCT06607471)
- Testing
- Support treatment, cardiac medical treatment, aetiology-specific treatment, device implant, arrhythmia ablation, Support treatment, cardiac medical treatment, aetiology-specific treatment
- Where
- Italy
- Volunteers
- 15,000 people
- Sponsor
- Scientific Institute San Raffaele
Fabry Disease
Cardiovasculorenal Phenotyping in Fabry Disease Through Noninvasive Testing
A longitudinal pilot study will be conducted to determine if there are additional testing modalities that are effective in broadly phenotyping subclinical dysfunction in patients with Fabry disease.
- Trial name
- VDFP (NCT05699265)
- Testing
- Measures of arterial stiffness and endothelial function, Ambulatory blood pressure monitoring, Cardiopulmonary exercise testing (CPET)
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
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
Fabry Disease
A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease
A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease. This study includes a sub-study evaluating kidney Gb3 inclusions (and other histologic lesions) in male…
- Trial name
- NCT03737214
- Testing
- Lucerastat
- Where
- 42 sites in United States, Australia and 11 more
- Volunteers
- 107 people
- Sponsor
- Idorsia Pharmaceuticals Ltd.