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Fabry Disease clinical trials

View clinical trials related to Fabry Disease.

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NCT ID: NCT05343715 Completed - Fabry Disease Clinical Trials

PK/PD Study of 2 Agalsidase Formulations in Single Dose of 1 mg/kg Administered to Healthy Volunteers as IV Infusion

Start date: October 23, 2021
Phase: Phase 1
Study type: Interventional

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

NCT ID: NCT05280548 Recruiting - Fabry Disease Clinical Trials

A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease

CARAT
Start date: May 3, 2022
Phase: Phase 3
Study type: Interventional

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) to evaluate the effect of venglustat on left ventricular mass index (LVMI) in adult participants with Fabry disease and left ventricular hypertrophy. - Study visits will take place approximately every 3 to 6 months - Participants who complete the randomized period may continue to the long-term extension (LTE) to receive venglustat for up to additional 34 months with the total study duration up to 4.4 years maximum.

NCT ID: NCT05206773 Recruiting - Fabry Disease Clinical Trials

A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease

PERIDOT
Start date: March 11, 2022
Phase: Phase 3
Study type: Interventional

This is a 12-month, parallel treatment, Phase 3, double-blind, randomized, placebo controlled study to evaluate the effect of venglustat on neuropathic and abdominal pain symptoms of Fabry disease in participants ≥16 years of age with Fabry disease who are treatment-naïve or untreated for at least 6 months. - Study visits will take place approximately every 3 months. - The double-blind period will be followed by an open-label extension (OLE) during which participants who have completed the double-blind period will be treated with venglustat for up to an additional 12 months.

NCT ID: NCT05186324 Completed - Fabry Disease Clinical Trials

Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion

PEOPLE
Start date: January 26, 2022
Phase:
Study type: Observational

Pegunigalsidase alfa (PRX-102) is a long-term enzyme replacement therapy design for the treatment of patients with Fabry disease. Although in the clinical development program patient-reported outcomes and clinician-reported outcomes have been included, this may not allow for a sufficiently accurate assessment of the quality of life in patients with Fabry Disease treated with pegunigalsidase alfa. This study will collect the patient experience on the pegunigalsidase alfa treatment administered intravenously every 4 weeks in the BRIGHT-F51 clinical study (NCT03614234).

NCT ID: NCT05106764 Active, not recruiting - Fabry Disease Clinical Trials

Early Detection of Fabry Disease

Start date: November 30, 2023
Phase:
Study type: Observational

The main aim of this study is early detection of FD using real-world data for the development of advanced natural language processing methods and to develop a predictive algorithm and to measure the performance of the algorithm in identifying participants with FD. This study is about using data from hospital Electronic Health Record database from the last 10 years to describe the ranking of participants with FD using multilevel likelihood ratios and to validate the algorithm using positive controls. No investigational medicinal product or device will be tested in this study. Hospital electronic health record data will be analyzed for a period of up to 6 months.

NCT ID: NCT05067868 Active, not recruiting - Fabry Disease Clinical Trials

A Study of Replagal in Children and Adults With Fabry Disease in India

Start date: November 1, 2022
Phase: Phase 4
Study type: Interventional

The main aim of this study is to learn more about the safety profile of Replagal. Participants will receive Replagal every 2 weeks at the clinic for about 1 year.

NCT ID: NCT05056636 Recruiting - Fabry Disease Clinical Trials

Epidemiological Study of Fabry Disease Screening in Chronic Kidney Disease Patients

Start date: June 1, 2018
Phase:
Study type: Observational

Fabry disease is a rare X-linked lysosomal storage disorder caused by deficient activity of the enzyme α-Gal A resulting from mutations affecting the GLA gene. It is characterized by severe multi-systemic involvement that leads to major organ failure and premature death in affected men and in some women. The α-Gal A deficiency results in progressive accumulation of un-degraded glycosphingolipids, predominantly globotriaosylceramide (Gb3), within cell lysosomes throughout the body. In patients at the second or third decade, progressive proteinuria, decline in glomerular filtration rate (GFR), and tubular damage occur usually, and renal failure develops in the fourth decade. Life-threatening renal, cardiac, and cerebrovascular diseases are added in later decades. In addition to that, Fabry disease patient will eventually face end-stage renal disease (ESRD) which was the most common cause of death in Fabry patients before the development of dialysis and renal transplantation. Thus it is critical to identify Fabry patient as early as possible, before reaching the stage of ESRD. Additionally, early intervention of enzyme replacement therapy for Fabry Disease patient which will help the patient to preserve a better renal function and benefit from treatment outcome. Apart from that today there is only one study published from Turkey for Fabry disease screening in CKD patient where they have screened 1453 and found that the overall prevalence of Fabry disease in CKD patient was found to be 0.2% , 3/1453 (in which 0.4% in 656 male, 0.0% in 783 female). However, there was no information available within the Asia region thereby a very low Fabry disease awareness and diagnostic awareness among nephrologist in Taiwan. Therefore in the present study the investigators are aiming to investigate the prevalence of Fabry disease in the CKD population (CKD stage 1 ~ 5) by conducting the first and largest high risk screening prevalence study among 2,000 CKD patients over 3 years in Taiwan and the investigators hope by doing such a pilot study our data would contribute to a new paradigm of Fabry disease diagnosis in the Asia region.

NCT ID: NCT05054387 Completed - Fabry Disease Clinical Trials

China Post-marketing Surveillance (PMS) Study of Fabrazyme®

Start date: October 13, 2021
Phase: Phase 4
Study type: Interventional

This is a 54-week Phase 4, open label, single arm study to evaluate the safety and the efficacy of Fabrazyme (agalsidase beta) as enzyme replacement therapy (ERT) in Chinese participants with Fabry Disease.

NCT ID: NCT05046379 Completed - Fabry Disease Clinical Trials

Studying Lipids as Potential Biomarkers in Patients With Fabry Disease

Start date: October 14, 2021
Phase:
Study type: Observational

Compare levels of lipids between well characterised enzymatically-genetically-phenotypically patients with Fabry disease and healthy controls (with no Fabry disease). Correlate levels of lipids in patients with Fabry disease to clinical outcomes/manifestations of the disease.

NCT ID: NCT05039866 Enrolling by invitation - Fabry Disease Clinical Trials

Long-Term Follow-up of Subjects Who Were Treated With ST-920

Start date: August 16, 2021
Phase:
Study type: Observational

Long-term follow-up of subjects who received ST-920 in a previous trial (ST-920-201) and completed at least 52 weeks post-infusion follow-up in their primary protocol. Enrolled subjects will be followed for a total of up to 5 years following ST-920 infusion.