Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients (Bright 51)
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| ClinicalTrials.gov Identifier: NCT03614234 |
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Recruitment Status :
Enrolling by invitation
First Posted : August 3, 2018
Last Update Posted : October 1, 2019
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Sponsor:
Protalix
Information provided by (Responsible Party):
Protalix
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Brief Summary:
The objective of PB-102-F51 is to evaluate the long-term safety, tolerability, and efficacy of 2 mg/kg pegunigalsidase alfa administered intravenously every four weeks in adult Fabry patients who have successfully completed PB-102-F50.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Fabry Disease | Biological: pegunigalsidase alfa | Phase 3 |
This is an open-label study to assess the long-term safety and efficacy of pegunigalsidase alfa treatment of 2.0 mg/kg administered intravenously every 4 weeks. The duration of treatment will be up to 36 months or until pegunigalsidase alfa is available to the patient at the discretion of the Sponsor. Interim analyses may be performed for administrative purposes during the conduct of the study.
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 40 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Intervention Model Description: | Open-label extension study |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | Open Label Extension Study to Evaluate the Long-term Safety and Efficacy of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Administered by Intravenous Infusion Every 4 Weeks in Adult Patients With Fabry Disease |
| Actual Study Start Date : | November 6, 2018 |
| Estimated Primary Completion Date : | January 2023 |
| Estimated Study Completion Date : | January 2023 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Fabry disease
| Arm | Intervention/treatment |
|---|---|
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Experimental: Experimental open label
Pegunigalsidase alfa
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Biological: pegunigalsidase alfa
Recombinant human alpha galactosidase A
Other Name: PRX-102 |
Primary Outcome Measures :
- Evaluation of treatment-related adverse events [ Time Frame: Throughout the study, 156 weeks ]CTCAE v4.03
Secondary Outcome Measures :
- Kidney function [ Time Frame: At 56 and108 weeks and at the end of the study, 156 weeks ]Estimated glomerular filtration rate (eGFRCKD-EPI)
- Cardiac assessment [ Time Frame: At 56 and 108 weeks and at the end of the study, 156 weeks ]Left Ventricular Mass Index (g/m2) by echocardiogram and cardiac function stress test
- Biomarkers for Fabry disease [ Time Frame: At 56 and 108 weeks and at the end of the study, 156 weeks ]Plasma Lyso-Gb3
- Clinical assessment [ Time Frame: Every four weeks throughout the duration of the study, 156 weeks ]Record of pain medication use
- Kidney function [ Time Frame: At 56 and 108 weeks and at the end of the study, 156 weeks ]Protein/Creatinine ratio (UPCR), spot urine test
- Pain assessment [ Time Frame: At 56 and 108 weeks and at the end of the study, 156 weeks ]Short form Brief Pain Inventory (BPI)
- Symptom assessment [ Time Frame: At 56 and 108 weeks and at the end of the study, 156 weeks ]Mainz Severity Score Index (MSSI)
- Quality of life assessment [ Time Frame: At 56 and 108 weeks and at the end of the study, 156 weeks ]Quality of life (EQ-5D-5L)
Information from the National Library of Medicine
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| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Completion of study PB-102-F50.
- The patient signs informed consent.
- Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically accepted, highly effective method of contraception. These include combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable), intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomised partner, or sexual abstinence.
Exclusion Criteria:
Presence of any medical, emotional, behavioral, or psychological condition that, in the judgment of the Investigator and/or Medical Director, would interfere with patient compliance with the requirements of the study.
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03614234
Locations
| United States, Alabama | |
| UAB Medicine | |
| Birmingham, Alabama, United States, 35233 | |
| United States, Georgia | |
| Emory University School of Medicine | |
| Atlanta, Georgia, United States, 30307 | |
| United States, Iowa | |
| University of Iowa Hospitals and Clinica | |
| Iowa City, Iowa, United States, 52242 | |
| United States, Michigan | |
| Infusion Associates | |
| Grand Rapids, Michigan, United States, 49525 | |
| United States, Texas | |
| Baylor Institute of Metabolic Disease | |
| Dallas, Texas, United States, 75226 | |
| United States, Utah | |
| University of Utah Hospitals & Clinics | |
| Salt Lake City, Utah, United States, 84112 | |
| United States, Virginia | |
| O & O Alpan | |
| Fairfax, Virginia, United States, 22030 | |
| Belgium | |
| UZ Antwerpen | |
| Edegem, Belgium, 2650 | |
| Czechia | |
| Fakultní poliklinika Všeobecné fakultní nemocnice v Praze | |
| Praha, Czechia, 120 00 | |
| Denmark | |
| Rigshospitalet | |
| Copenhagen, Denmark, 2100 | |
| Italy | |
| Azienda Ospedaliera Universitaria "Federico II" | |
| Napoli, Italy | |
| Norway | |
| Helse Bergen HF Haukeland Universitetssykehus | |
| Bergen, Norway, 5021 | |
| United Kingdom | |
| Addenbrooke's Hospital | |
| Cambridge, United Kingdom, CB2 0QQ | |
| The Royal Free Hospital | |
| London, United Kingdom | |
Sponsors and Collaborators
Protalix
| Responsible Party: | Protalix |
| ClinicalTrials.gov Identifier: | NCT03614234 |
| Other Study ID Numbers: |
PB-102-F51 |
| First Posted: | August 3, 2018 Key Record Dates |
| Last Update Posted: | October 1, 2019 |
| Last Verified: | September 2019 |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Keywords provided by Protalix:
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Glomerular filtration rate Proteinuria PRX-102 pegunigalsidase alfa Fabry disease |
Additional relevant MeSH terms:
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Fabry Disease Sphingolipidoses Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Cerebral Small Vessel Diseases Cerebrovascular Disorders |
Vascular Diseases Cardiovascular Diseases Genetic Diseases, X-Linked Genetic Diseases, Inborn Metabolism, Inborn Errors Lipidoses Lipid Metabolism, Inborn Errors Lysosomal Storage Diseases Metabolic Diseases Lipid Metabolism Disorders |