Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients

Status Completed

Clinical Trial Summary

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). The safety, tolerability, and exploratory efficacy will be evaluated in this study of increasing doses. Patients will be treated with infusions every two weeks for 12 months.

Clinical Trial Description

Under the PB-102-F01 study protocol, patients will be enrolled into one of three PRX-102 dosing groups (0.2 mg/kg, 1.0 mg/kg, 2.0 mg/kg) and receive PRX-102 as an intravenous infusion every 2 weeks for 12 weeks (3 months). Patients who finish the PB-102-F01 study will be enrolled in the PB-102-F02 extension study and receive the same dose they had received in the PB-102-F01 study for an additional 38 weeks (9 months). ;

Study Design

Related Conditions & MeSH terms

Fabry Disease

Clinical Trial Details

NCT number	NCT01678898
Study type	Interventional
Source	Protalix
Contact
Status	Completed
Phase	Phase 1/Phase 2
Start date	October 2012
Completion date	March 6, 2016

View Details

See also
Status	Clinical Trial	Phase
Recruiting	`NCT04893889` - Substudy (NCT04456582): Noninvasive Assessment of Myocardial Stiffness by 2D-SWE Ultrasound Technique (Two-dimensional Shear Wave Elastography) in Patients With Amyloidosis and Fabry Disease.	N/A
Completed	`NCT04455230` - A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190	Phase 1/Phase 2
Completed	`NCT01218659` - Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease	Phase 3
Completed	`NCT00304512` - A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease	Phase 2
Withdrawn	`NCT04189601` - Complement Activation in the Lysosomal Storage Disorders
Completed	`NCT03500094` - Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)	Phase 3
Withdrawn	`NCT04143958` - To Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Versus Continuing on Agalsidase Alfa (Replagal) in Male Patients With Classic Fabry Disease	Phase 4
Recruiting	`NCT02994303` - Podocyturia - Predictor of Renal Dysfunction in Fabry Nephropathy	N/A
Completed	`NCT01947634` - Sleepiness and Sleep-disordered Breathing in Fabry Disease. A Prospective Cohort Study.	N/A
Recruiting	`NCT01695161` - Non-invasive Assessment of Intraocular Pressure in MPS by Use of the Ocular Response Analyzer.	N/A
Completed	`NCT01853852` - A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Single Oral Doses of GR181413A/AT1001 in Healthy Japanese Subjects	Phase 1
Completed	`NCT00701415` - A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms	Phase 3
Completed	`NCT00068107` - Dosing Study of Replagal in Patients With Fabry Disease	Phase 2
Completed	`NCT01997489` - Ophthalmic Findings During 10-year Enzyme Substitution of Danish Fabry Patients.	Phase 4
Recruiting	`NCT06007768` - Autoimmune and Inflammatory Response Biomarkers in Fabry Disease
Recruiting	`NCT05698901` - Biomarkers and Cardiac Imaging Diagnostic Assay for Monitoring Patients With Fabry Disease
Active, not recruiting	`NCT03305250` - Arrhythmia Burden, Risk of Sudden Cardiac Death and Stroke in Patients With Fabry Disease	N/A
Terminated	`NCT00526071` - Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study	Phase 2
Active, not recruiting	`NCT03566017` - Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease	Phase 3
Recruiting	`NCT06065605` - Assess Urine Biomarkers to Predict Nephropathy in Fabry Disease