Fabry Disease
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2024-10-01Clinical Trial Update: Studying Lipids as Potential Biomarkers in Patients With Fabry Disease
A completed clinical trial update studying lipids as potential biomarkers in patients with Fabry disease.
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CLINICAL_TRIALS trial_result · 2024-09-19Clinical Trial Update: Clinical Investigation on the Blood Oxygenation at the Optic Nerve Head in Fabry Patients
This entry reports the completion and posting of results from a clinical investigation examining blood oxygenation at the optic nerve head i…
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CLINICAL_TRIALS trial_result · 2024-08-20Clinical Trial Update: Screening of Fabry Disease in Patients With Left Ventricular Hypertrophy Detected in Echocardiography
The study completed screening of patients with left ventricular hypertrophy detected by echocardiography for Fabry disease. It assessed the …
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CLINICAL_TRIALS trial_result · 2024-08-09Clinical Trial Update: Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease [Phase 3]
Phase 3 trial evaluating oral lucerastat as monotherapy in adults with Fabry disease; results assessing efficacy and safety have been posted…
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CLINICAL_TRIALS trial_result · 2024-07-31Clinical Trial Update: Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients
Open-label expanded access treatment with pegunigalsidase alfa for Fabry disease is described, providing therapy to patients outside of a tr…
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CLINICAL_TRIALS trial_result · 2024-06-17Clinical Trial Update: A Study of Replagal in Treatment-naïve Adults With Fabry Disease [Phase 3]
This clinical trial update reports that the Phase 3 study of Replagal (agalsidase alfa) in treatment-naïve adults with Fabry disease was ter…
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CLINICAL_TRIALS trial_result · 2024-05-23Clinical Trial Update: French Prospective, Observational Cohort Study of Patients With Fabry Disease Treated With Migalastat
A completed French prospective observational cohort study evaluating outcomes in Fabry disease patients treated with migalastat. Sponsor: Am…
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CLINICAL_TRIALS trial_result · 2024-04-10Clinical Trial Update: Study of the Effects of Fabrazyme Treatment on Lactation and Infants [Phase 4]
Phase 4 trial update on Fabrazyme (agalsidase beta) evaluating effects on lactation and infants in Fabry disease, with terminated status and…
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CLINICAL_TRIALS trial_result · 2024-04-08Clinical Trial Update: An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease [Phase 1, Phase 2]
Open-label Phase 1/2 trial exploring gene therapy 4D-310 in adults with Fabry disease.
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CLINICAL_TRIALS trial_result · 2024-03-15Clinical Trial Update: PK/PD Study of 2 Agalsidase Formulations in Single Dose of 1 mg/kg Administered to Healthy Volunteers as IV Infusion [Phase 1]
Phase 1 pharmacokinetic/pharmacodynamic study comparing two agalsidase formulations given as a single 1 mg/kg intravenous dose in healthy vo…
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CLINICAL_TRIALS trial_result · 2024-01-05Clinical Trial Update: Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease [Phase 1, Phase 2]
Open-label study of AVR-RD-01 for treatment-naive Fabry disease subjects in Phase 1/2 was terminated with results posted.
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CLINICAL_TRIALS trial_result · 2023-12-07Clinical Trial Update: A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190 [Phase 1, Phase 2]
A long-term follow-up study of FLT190 in Fabry disease subjects across Phase 1/2 has been completed.
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CLINICAL_TRIALS trial_result · 2023-08-02Clinical Trial Update: Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function [Phase 3]
Phase 3 study comparing PRX-102 to Agalsidase Beta in Fabry disease completed with results posted.
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CLINICAL_TRIALS trial_result · 2023-03-27Clinical Trial Update: Evaluation of the Gastrointestinal Manifestation of Fabry's Disease [NA]
Clinical trial update reporting completed status and posted results for evaluation of gastrointestinal manifestations in Fabry's disease.
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CLINICAL_TRIALS trial_result · 2023-02-08Clinical Trial Update: Extension Study of PRX-102 for up to 60 Months [Phase 1, Phase 2]
Extension study update for PRX-102 in Fabry disease following Phase 1/2, with results posted after completion.
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CLINICAL_TRIALS trial_result · 2023-02-02Clinical Trial Update: Effects Of Sodium Glucose Cotranspoter 2 Inhibitors On Heart And Kidneys In Fabry Disease Patients [Phase 2]
Phase 2 trial update investigating SGLT2 inhibitors for effects on heart and kidneys in Fabry disease patients.
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CLINICAL_TRIALS trial_result · 2023-01-05Clinical Trial Update: Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks [Phase 3]
Phase 3 trial assessing safety, efficacy, and pharmacokinetics of PRX-102 given IV every 4 weeks in Fabry disease completed with posted resu…
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CLINICAL_TRIALS trial_result · 2022-06-29Clinical Trial Update: Safety and Efficacy of PRX-102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa) [Phase 3]
Phase 3 trial updating safety and efficacy of PRX-102 in Fabry disease patients already treated with agalsidase alfa (REPLAGAL).
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CLINICAL_TRIALS trial_result · 2021-11-30Clinical Trial Update: Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years) [Phase 3]
Phase 3 pediatric study assessing migalastat safety, pharmacokinetics/pharmacodynamics, and efficacy in adolescents with Fabry disease has c…
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CLINICAL_TRIALS trial_result · 2021-10-07Clinical Trial Update: Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan [NA]
A completed clinical trial assessing small fiber neuropathy in Fabry disease and related conditions using Sudoscan; results posted in 2021.
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CLINICAL_TRIALS trial_result · 2020-12-21Clinical Trial Update: Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease [Phase 3]
Phase 3 open-label extension study reporting long-term effects of migalastat HCL in Fabry disease has completed with results posted.
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CLINICAL_TRIALS trial_result · 2020-01-27Clinical Trial Update: Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients [Phase 1, Phase 2]
Dose-ranging study of PRX-102 in adult Fabry disease patients completed; results posted in 2020.
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CLINICAL_TRIALS trial_result · 2019-12-17Clinical Trial Update: Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease [Phase 2]
Phase 2 clinical trial evaluating safety, pharmacodynamics, pharmacokinetics, and exploratory efficacy of GZ/SAR402671 in treatment-naïve ad…
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CLINICAL_TRIALS trial_result · 2019-12-09Clinical Trial Update: Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease [Phase 2]
Phase 2 trial evaluating long-term safety, pharmacodynamics, and exploratory efficacy of GZ/SAR402671 in treatment-naïve adult male Fabry di…
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CLINICAL_TRIALS trial_result · 2019-02-15Clinical Trial Update: A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease [Phase 3]
A multicenter open-label trial assessing the safety of agalsidase alfa enzyme replacement therapy in Canadian Fabry disease patients; study …