Fabry DiseasePhase 3BALANCENCT02795676
Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
A Phase 3 study testing PRX-102 (pegunigalsidase alfa) and agalsidase beta in people with Fabry Disease, run by Protalix at 29 sites in 12 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Fabry Disease
- What is being tested?
- PRX-102 (pegunigalsidase alfa) and agalsidase beta
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 78 participants
- Where
- 29 sitesin 12 countries
- Study dates
- Jun 2016to Jul 2022
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Symptomatic adult Fabry disease patients, age 18-60 years
- Males: Plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than 30% mean normal levels and one or more of the characteristic features of Fabry disease
- neuropathic pain
- cornea verticillata
- clustered angiokeratoma
- Females:
Show all 18Show fewer
- historical genetic test results consistent with Fabry pathogenic mutation and one or more of the described characteristic features of Fabry disease:
- neuropathic pain
- cornea verticillata
- clustered angiokeratoma
- or in the case of novel mutations a first degree male family member with Fabry disease with the same mutation, and one or more of the characteristic features of Fabry disease
- neuropathic pain
- cornea verticillata
- clustered angiokeratoma
- Screening eGFR by CKD-EPI equation 40 to 120 mL/min/1.73 m²
- Linear negative slope of eGFR based on at least 3 serum creatinine values over approximately 1 year (range of 9 to 18 months, including the value obtained at the screening visit) of ≥ 2 mL/min/1.73 m²/year
- Treatment with a dose of 1 mg/kg agalsidase beta per infusion every 2 weeks for at least one year and at least 80% of 13 (10.4) mg/kg total dose over the last 6 months.
- Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically accepted method of contraception, not including the rhythm method.
Who cannot join
- History of anaphylaxis or Type 1 hypersensitivity reaction to agalsidase beta
- Known non-pathogenic Fabry mutations
- History of renal dialysis or transplantation
- History of acute kidney injury in the 12 months prior to screening, including specific kidney diseases (e.g., acute interstitial nephritis, acute glomerular and vasculitic renal diseases); non-specific conditions (e.g, ischemia, toxic injury); as well as extrarenal pathology (e.g., prerenal azotemia, and acute postrenal obstructive nephropathy)
- Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated or dose changed in the 4 weeks prior to screening
- Patient with a screening eGFR value between 91-120 mL/min/1.73 m², having an historical eGFR value higher than 120 mL/min/1.73 m² (during 9 to 18 months before screening)
Show all 13Show fewer
- Urine protein to creatinine ratio (UPCR) > 0.5 g/g and not treated with an ACE inhibitor or ARB
- Cardiovascular event (myocardial infarction, unstable angina) in the 6 month period before randomization
- Congestive heart failure NYHA Class IV
- Cerebrovascular event (stroke, transient ischemic attack) in the 6 month period before randomization
- Known history of hypersensitivity to Gadolinium contrast agent that is not managed by the use of pre-medication
- Female subjects who are pregnant, planning to become pregnant during the study, or are breastfeeding
- Presence of any medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator and/or Medical Director, would interfere with the patient's compliance with the requirements of the study
Trial locations and contacts
29 sites in 12 countries.
29 sites in 12 countries
United States15 sites
Emory University School of Medicine
Atlanta, Georgia, 30322
UAB Medicine
Birmingham, Alabama, 35233
Massachusetts General Hospital
Boston, Massachusetts, 02114
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Institute of Metabolic Disease, Baylor Healthcare
Dallas, Texas, 75226
Renal Disease Research Institute, LLC - Dallas
Dallas, Texas, 75235
O+O Alpan LLC
Fairfax, Virginia, 22030
Infusion Associates
Grand Rapids, Michigan, 49525
University of Iowa Hosptials and Clinics
Iowa City, Iowa, 52242
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226-3596
University of California Irvine Center
Orange, California, 92868
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Eccles Primary Children's Outpatient Services Building
Salt Lake City, Utah, 84132
University of California San Diego
San Diego, California, 92093
United Kingdom4 sites
Addenbrooke's Hospital
Cambridge, CB2 0QQ
Institute of Metabolism and Systems Research
Edgbaston, Birmingham
The Royal Free Hospital
London, NW3 2QG
Salford Royal NHS Foundation Trust
Salford, M6 8HD
Czechia1 site
Vseobecna fakultni nemocnice v Praze
Prague, Czech Republic, 12808
Switzerland1 site
Klinik und Poliklinik für Innere Medizin UniversitätsSpital Zürich
Zurich
About this study
Official title: A Randomized, Double Blind, Active Control Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function in Patients With Fabry Disease Previously Treated With Agalsidase Beta
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. Patients who had been treated for approximately 1 year with agalsidase beta and who had been on a stable dose of that product for at least 6 months were randomized in a 2:1 ratio to either switch to PRX-102 or to continue treatment with agalsidase beta. Both treatments were delivered by intravenous infusions every two weeks, at a dosage of 1 mg/kg.
Read the detailed description
This was a randomized, double-blind, active control study examining the safety and efficacy of pegunigalsidase alfa (PRX-102) in Fabry disease patients with impaired renal function. Participants had to have been taking the licensed ERT drug agalsidase beta (Fabrazyme®) for at least 1 year prior to study entry, and to have been on a stable dose of that product for at least the last 6 months. Since the disease expresses itself differently in males and females, gender could have an impact on the therapeutic effect; thus, there was additionally a requirement that no more than 50% of the patients could be female.
Following screening, eligible patients were randomized in a 2:1 ratio to either switch to PRX-102 or continue treatment with agalsidase beta, with randomization stratified according to whether the urine protein-to-creatinine ratio (UPCR), a measure of kidney function, was above or below a specified threshold. Both products were administered as an intravenous infusion every 2 weeks, at a dosage of 1 mg/kg, for up to 24 months. Both patients and study staff were blinded as to which treatment was being given.
Patients who completed the study were invited to continue in a long-term open-label extension study, PB-102-F60, in which all participants would receive PRX-102 1 mg/kg every 2 weeks.
- Conditions
- Fabry Disease
- Being tested
- PRX-102 (pegunigalsidase alfa), agalsidase beta
- Keywords
- Glomerular filtration rate, Proteinuria, PRX-102, pegunigalsidase alfa, Fabry Disease
- Medical subject headings
- Fabry Disease, Proteinuria
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PRX-102 (pegunigalsidase alfa)Biological
Also called pegunigalsidase alfa, Recombinant human alpha galactosidase-A
PRX-102 1 mg/kg every 2 weeks
agalsidase betaBiological
Also called Fabrazyme
agalsidase beta 1 mg/kg every 2 weeks
| Study group | Role | What they receive |
|---|---|---|
| PRX-102 (pegunigalsidase alfa) | Experimental | PRX-102 infusion every 2 weeks Receives: Biological: PRX-102 (pegunigalsidase alfa) |
| agalsidase beta | Active comparator | agalsidase beta infusion every 2 weeks Receives: Biological: agalsidase beta |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized Change (Slope) in Estimated Glomerular Filtration Rate (eGFR)
The individual annualized mean change (slope) in eGFR (mL/min/1.73 m^2/year) is an estimate of the individual patient's annualized change in eGFR, which is derived from the eGFR assessments over time, for up to 24 months.
The individual annualized mean change (slope) in eGFR is estimated for each patient with at least 4 eGFR observations. For patients with fewer than 4 eGFR observations, the slope will be missing.
Measured: 24 months
Secondary outcomes (7)
Estimated Glomerular Filtration Rate (eGFR)
eGFR was calculated based on measured serum creatinine levels according to the CKD-EPI formula. The median values obtained at baseline and at Month 24 are reported.
The change in eGFR from baseline measurement prior to first infusion to last measurement at Month 24 was summarized using descriptive statistics.
The change was calculated for each subject and the reported value is the median (full range) of these changes.
Measured: Baseline and Month 24
Plasma Lyso-Gb3
Globotriaosylsphingosine (lyso-Gb3) is a Fabry disease-specific biomarker measured in the plasma. The median concentrations obtained at baseline and at Month 24, and the change from baseline to Month 24 in median concentration, are reported.
The change in Lyso-Gb3 from baseline measurement prior to first infusion to last measurement at Month 24 was summarized using descriptive statistics.
The change was calculated for each subject and the reported value is the median (full range) of these changes.
Measured: Baseline and Month 24
Short Form Brief Pain Inventory (BPI)
The Short Form Brief Pain Inventory ( BPI) questioner is self-completed by patients regarding pain severity and interference.
Descriptive statistics summarize the findings for the change from baseline at Week 104 for "Pain at Its Worst in Last 24 Hours".
The severity of various aspects of pain scored on a scale of 0 to 10 ( no pain / pain as bad as you can imagine).
The change in BPI from baseline measurement prior to first infusion to the last measurement at Month 24 was summarized using descriptive statistics.
The change was calculated for each subject and the reported value is the mean (Standard Error) of these changes.
Measured: Baseline and Month 24
Mainz Severity Score Index (MSSI)
The Mainz Severity Score Index (MSSI) is an instrument that is specifically designed to measure the severity of Fabry disease signs/symptoms and to monitor the clinical course of the disease. The MSSI is administered by the investigator, and yields scores for general, neurological, cardiovascular, renal, and overall assessments. The overall score range from 0 to 76. An overall score of less than 20 points is considered mild signs and symptoms of Fabry disease, 20 to 40 is considered moderate, and greater than 40 is considered severe.
The change in overall MSSI score from baseline measurement prior to first infusion to the last measurement at Month 24 was summarized using descriptive statistics.
The change was calculated for each subject and the reported value is the mean (Standard Error) of these changes.
Measured: Baseline and Month 24
Urine Protein/Creatinine Ratio (UPCR)
The UPCR provides an estimate of protein excretion in urine, and is used as an indicator of the extent of chronic kidney disease. It was classified into three categories: 1) UPCR ≤ 0.5 gr/gr, 2) 0.5 gr/gr < UPCR < 1 gr/gr, 3) 1 gr/gr ≤ UPCR. The results are presented as the percentage of patients (%) in each category at baseline and Month 24.
There are no statistical analyses for this endpoint.
Measured: Baseline and Month 24
Left Ventricular Mass Index With Hypertrophy at Baseline
Left Ventricular Mass Index (LVMI) based on cardiac MRI for patients with hypertrophy at baseline (for males, hypertrophy is above 91 g/m^2 and for females, above 77 g/m^2).
The change in LVMI from baseline measurement prior to first infusion to the last measurement at Month 24 was summarized using descriptive statistics.
The change was calculated for each subject and the reported value is the median (full range) of these changes.
Measured: Baseline and Month 24
Left Ventricular Mass Index Without Hypertrophy at Baseline
Left Ventricular Mass Index (LVMI) based on cardiac MRI for patients without hypertrophy at baseline (for males, hypertrophy is above 91 g/m^2 and for females, above 77 g/m^2).
The change in LVMI from baseline measurement prior to first infusion to the last measurement at Month 24 was summarized using descriptive statistics.
The change was calculated for each subject and the reported value is the median (full range) of these changes.
Measured: Baseline and Month 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jun 2016 (estimated)
- Study startJun 2016
- Primary completionOct 2021
- Study completionJul 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 78 participants (actual)
Sponsor and investigators
- Lead sponsor
- Protalix
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Chiesi Farmaceutici S.p.A.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02795676
- Sponsor's protocol ID
- PB-102-F20
- Acronym
- BALANCE
- Status last verified
- Sep 2023
- First submitted
- 2 Jun 2016
- Last updated
- 13 Sep 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
Azimpour K, Dorling P, Koulinska I, Kunduri S, Lan Z, Poritz J, Tremblay G, Raad-Faherty A. Health State Utility Values in Fabry Disease: Insights from the Pegunigalsidase Alfa Clinical Trials. Adv Ther. 2025 Mar;42(3):1421-1434. doi: 10.1007/s12325-024-03095-2. Epub 2025 Jan 23.
PubMed 39847314Wallace EL, Goker-Alpan O, Wilcox WR, Holida M, Bernat J, Longo N, Linhart A, Hughes DA, Hopkin RJ, Tondel C, Langeveld M, Giraldo P, Pisani A, Germain DP, Mehta A, Deegan PB, Molnar MJ, Ortiz D, Jovanovic A, Muriello M, Barshop BA, Kimonis V, Vujkovac B, Nowak A, Geberhiwot T, Kantola I, Knoll J, Waldek S, Nedd K, Karaa A, Brill-Almon E, Alon S, Chertkoff R, Rocco R, Sakov A, Warnock DG. Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study. J Med Genet. 2024 May 21;61(6):520-530. doi: 10.1136/jmg-2023-109445.
PubMed 37940383
Study at a glance
From the registry record, last updated by the sponsor on 13 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double Blind, Active Control Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function in Patients With Fabry Disease Previously Treated With Agalsidase Beta
- ClinicalTrials.gov ID
- NCT02795676
- Acronym / sponsor ID
- BALANCE / PB-102-F20
- Conditions
- Fabry Disease
- Keywords
- Glomerular filtration rate, Proteinuria, PRX-102, pegunigalsidase alfa, Fabry Disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 78 participants (actual)
- Lead sponsor
- ProtalixIndustry (pharma, biotech, devices)
- Collaborators
- Chiesi Farmaceutici S.p.A.
- Study start
- Jun 2016 (actual)
- Primary completion
- Oct 2021 (actual)
- Study completion
- Jul 2022 (actual)
- First posted
- 10 Jun 2016 (estimated)
- Results
- Posted 2 Aug 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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. Patients who had been treated for approximately 1 year with agalsidase beta and who had been on a stable dose of that product for at least 6 months were randomized in a 2:1 ratio to either switch to PRX-102 or to continue treatment with agalsidase beta. Both treatments were delivered by intravenous infusions every two weeks, at a dosage of 1 mg/kg.
Detailed description
This was a randomized, double-blind, active control study examining the safety and efficacy of pegunigalsidase alfa (PRX-102) in Fabry disease patients with impaired renal function. Participants had to have been taking the licensed ERT drug agalsidase beta (Fabrazyme®) for at least 1 year prior to study entry, and to have been on a stable dose of that product for at least the last 6 months. Since the disease expresses itself differently in males and females, gender could have an impact on the therapeutic effect; thus, there was additionally a requirement that no more than 50% of the patients could be female.
Following screening, eligible patients were randomized in a 2:1 ratio to either switch to PRX-102 or continue treatment with agalsidase beta, with randomization stratified according to whether the urine protein-to-creatinine ratio (UPCR), a measure of kidney function, was above or below a specified threshold. Both products were administered as an intravenous infusion every 2 weeks, at a dosage of 1 mg/kg, for up to 24 months. Both patients and study staff were blinded as to which treatment was being given.
Patients who completed the study were invited to continue in a long-term open-label extension study, PB-102-F60, in which all participants would receive PRX-102 1 mg/kg every 2 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| PRX-102 (pegunigalsidase alfa) | Experimental | Biological: PRX-102 (pegunigalsidase alfa)PRX-102 infusion every 2 weeks |
| agalsidase beta | Active comparator | Biological: agalsidase betaagalsidase beta infusion every 2 weeks |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (18 listed)
- Symptomatic adult Fabry disease patients, age 18-60 years
- Screening eGFR by CKD-EPI equation 40 to 120 mL/min/1.73 m²
- Linear negative slope of eGFR based on at least 3 serum creatinine values over approximately 1 year (range of 9 to 18 months, including the value obtained at the screening visit) of ≥ 2 mL/min/1.73 m²/year
- Treatment with a dose of 1 mg/kg agalsidase beta per infusion every 2 weeks for at least one year and at least 80% of 13 (10.4) mg/kg total dose over the last 6 months.
- Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically accepted method of contraception, not including the rhythm method.
Key exclusion criteria (13 listed)
- History of anaphylaxis or Type 1 hypersensitivity reaction to agalsidase beta
- Known non-pathogenic Fabry mutations
- History of renal dialysis or transplantation
- History of acute kidney injury in the 12 months prior to screening, including specific kidney diseases (e.g., acute interstitial nephritis, acute glomerular and vasculitic renal diseases); non-specific conditions (e.g, ischemia, toxic injury); as well as extrarenal pathology (e.g., prerenal azotemia, and acute postrenal obstructive nephropathy)
- Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated or dose changed in the 4 weeks prior to screening
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PRX-102 (pegunigalsidase alfa)Also: pegunigalsidase alfa, Recombinant human alpha galactosidase-APRX-102 1 mg/kg every 2 weeks | Biological | PRX-102 (pegunigalsidase alfa) |
| agalsidase betaAlso: Fabrazymeagalsidase beta 1 mg/kg every 2 weeks | Biological | agalsidase beta |
Primary outcome
Annualized Change (Slope) in Estimated Glomerular Filtration Rate (eGFR)
Time frame: 24 months
The individual annualized mean change (slope) in eGFR (mL/min/1.73 m^2/year) is an estimate of the individual patient's annualized change in eGFR, which is derived from the eGFR assessments over time, for up to 24 months. The individual annualized mean change (slope) in eGFR is estimated for each patient with at least 4 eGFR observations. For patients with fewer than 4 eGFR observations, the slope will be missing.
7 secondary outcomes
Estimated Glomerular Filtration Rate (eGFR)
Time frame: Baseline and Month 24
Plasma Lyso-Gb3
Time frame: Baseline and Month 24
Short Form Brief Pain Inventory (BPI)
Time frame: Baseline and Month 24
Mainz Severity Score Index (MSSI)
Time frame: Baseline and Month 24
Urine Protein/Creatinine Ratio (UPCR)
Time frame: Baseline and Month 24
Left Ventricular Mass Index With Hypertrophy at Baseline
Time frame: Baseline and Month 24
Left Ventricular Mass Index Without Hypertrophy at Baseline
Time frame: Baseline and Month 24
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Sep 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 39847314Azimpour K, Dorling P, Koulinska I, Kunduri S, Lan Z, Poritz J, Tremblay G, Raad-Faherty A. Health State Utility Values in Fabry Disease: Insights from the Pegunigalsidase Alfa Clinical Trials. Adv Ther. 2025 Mar;42(3):1421-1434. doi: 10.1007/s12325-024-03095-2. Epub 2025 Jan 23.
- Derived from this study: PubMed 37940383Wallace EL, Goker-Alpan O, Wilcox WR, Holida M, Bernat J, Longo N, Linhart A, Hughes DA, Hopkin RJ, Tondel C, Langeveld M, Giraldo P, Pisani A, Germain DP, Mehta A, Deegan PB, Molnar MJ, Ortiz D, Jovanovic A, Muriello M, Barshop BA, Kimonis V, Vujkovac B, Nowak A, Geberhiwot T, Kantola I, Knoll J, Waldek S, Nedd K, Karaa A, Brill-Almon E, Alon S, Chertkoff R, Rocco R, Sakov A, Warnock DG. Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study. J Med Genet. 2024 May 21;61(6):520-530. doi: 10.1136/jmg-2023-109445.
- Individual participant dataSharing planned: No
Related trials for Fabry Disease
RecruitingPhase 3
A Study to Learn How Well Lucerastat Works and How Safe it is in Untreated Adult Male Participants With Fabry Disease
Fabry Disease
RecruitingObservational study
A Study of Patients With Fabry Disease (US Specific)
Fabry Disease
RecruitingObservational study
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
Fabry Disease
Active, not recruiting
Evaluation of the Effect of a Low-FODMAP Diet on Gastrointestinal Symptoms and Inflammatory Markers in Fabry Patients
Fabry Disease
RecruitingPhase 3
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Fabry Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
29 sites in 12 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing PRX-102 (pegunigalsidase alfa) and agalsidase beta in people with Fabry Disease, run by Protalix at 29 sites in 12 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 18 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 29 sites in United States, United Kingdom, Czechia, Finland, France and 7 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 2 Aug 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annualized Change (Slope) in Estimated Glomerular Filtration Rate (eGFR) (time frame: 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02795676, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Sep 2023.