Trial · NCT02795676
Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
NCT02795676, Phase 3, Completed, Protalix; results first posted 2023-08-02
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function. Trials, record NCT02795676. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02795676
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
- NCT number
- NCT02795676
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 78 participants, actual
- Lead sponsor
- Protalix
- Conditions
- Fabry Disease
- Interventions
- PRX-102 (pegunigalsidase alfa), agalsidase beta
- First posted
- 2016-06-10
- Last updated
- 2023-09-13
- Results first posted
- 2023-08-02
- Limitations and caveats
- * Small sample size~* Randomization of 2:1
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Annualized Change (Slope) in Estimated Glomerular Filtration Rate (eGFR) | 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. |
| Secondary | Estimated Glomerular Filtration Rate (eGFR) | Baseline and Month 24 | 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. |
| Secondary | Plasma Lyso-Gb3 | Baseline and Month 24 | 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. |
| Secondary | Short Form Brief Pain Inventory (BPI) | Baseline and Month 24 | 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. |
| Secondary | Mainz Severity Score Index (MSSI) | Baseline and Month 24 | 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. |
| Secondary | Urine Protein/Creatinine Ratio (UPCR) | Baseline and Month 24 | 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. |
| Secondary | Left Ventricular Mass Index With Hypertrophy at Baseline | Baseline and Month 24 | 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. |
| Secondary | Left Ventricular Mass Index Without Hypertrophy at Baseline | Baseline and Month 24 | 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. |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Annualized Change (Slope) in Estimated Glomerular Filtration Rate (eGFR) | 24 months | The Intent to Treat (ITT) population includes all randomized patients who received at least one dose of the assigned study medication with at least 4 eGFR observations.~For 1 patient with fewer than 4 eGFR observations, the slope was missing. | mL/min/1.73 m^2/year | MEDIAN |
| SECONDARY | Estimated Glomerular Filtration Rate (eGFR) | Baseline and Month 24 | The Intent to Treat (ITT) population includes all randomized patients who received at least one dose of the assigned study medication. | mL/min/1.73 m^2 | MEDIAN |
| SECONDARY | Plasma Lyso-Gb3 | Baseline and Month 24 | The Intent to Treat (ITT) population includes all randomized patients who received at least one dose of the assigned study medication. | nM | MEDIAN |
| SECONDARY | Short Form Brief Pain Inventory (BPI) | Baseline and Month 24 | The Intent to Treat (ITT) population includes all randomized patients who received at least one dose of the assigned study medication. | score on a scale | MEAN |
| SECONDARY | Mainz Severity Score Index (MSSI) | Baseline and Month 24 | The Intent to Treat (ITT) population includes all randomized patients who received at least one dose of the assigned study medication. | score on a scale | MEAN |
| SECONDARY | Urine Protein/Creatinine Ratio (UPCR) | Baseline and Month 24 | The Intent to Treat (ITT) population includes all randomized patients who received at least one dose of the assigned study medication. | percentage of participants | NUMBER |
| SECONDARY | Left Ventricular Mass Index With Hypertrophy at Baseline | Baseline and Month 24 | The Intent to Treat (ITT) population includes all randomized patients who received at least one dose of the assigned study medication.~This analysis included the subset of patients in each treatment arm who had hypertrophy at baseline. | g/m^2 | MEDIAN |
| SECONDARY | Left Ventricular Mass Index Without Hypertrophy at Baseline | Baseline and Month 24 | The Intent to Treat (ITT) population includes all randomized patients who received at least one dose of the assigned study medication.~This analysis included the subset of patients in each treatment arm who did not have hypertrophy at baseline. | g/m^2 | MEDIAN |
Source ClinicalTrials.gov
Results by group50
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Annualized Change (Slope) in Estimated Glomerular Filtration Rate (eGFR) | PRX-102 (Pegunigalsidase Alfa) | -2.514 | 95% Confidence Interval -3.788 to -1.240 | mL/min/1.73 m^2/year | |
| Annualized Change (Slope) in Estimated Glomerular Filtration Rate (eGFR) | Agalsidase Beta | -2.155 | 95% Confidence Interval -3.805 to -0.505 | mL/min/1.73 m^2/year | |
| Estimated Glomerular Filtration Rate (eGFR) | PRX-102 (Pegunigalsidase Alfa) | Baseline | 73.45 | Full Range 30.2 to 125.9 | mL/min/1.73 m^2 |
| Estimated Glomerular Filtration Rate (eGFR) | Agalsidase Beta | Baseline | 74.85 | Full Range 34.1 to 107.6 | mL/min/1.73 m^2 |
| Estimated Glomerular Filtration Rate (eGFR) | PRX-102 (Pegunigalsidase Alfa) | Month 24 | 69.35 | Full Range 27.6 to 113.7 | mL/min/1.73 m^2 |
| Estimated Glomerular Filtration Rate (eGFR) | Agalsidase Beta | Month 24 | 74.48 | Full Range 24.4 to 114.8 | mL/min/1.73 m^2 |
| Estimated Glomerular Filtration Rate (eGFR) | PRX-102 (Pegunigalsidase Alfa) | Change from Baseline to Month 24 | -2.39 | Full Range -36.9 to 21.8 | mL/min/1.73 m^2 |
| Estimated Glomerular Filtration Rate (eGFR) | Agalsidase Beta | Change from Baseline to Month 24 | -3.20 | Full Range -18.0 to 16.8 | mL/min/1.73 m^2 |
| Plasma Lyso-Gb3 | PRX-102 (Pegunigalsidase Alfa) | Baseline | 15.20 | Full Range 0.8 to 143.9 | nM |
| Plasma Lyso-Gb3 | Agalsidase Beta | Baseline | 17.60 | Full Range 2.1 to 142.0 | nM |
1 to 10 of 50Source ClinicalTrials.gov
See all 50 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | PRX-102 (Pegunigalsidase Alfa) | 53 | |
| Overall Study | STARTED | Agalsidase Beta | 25 | |
| Overall Study | Treated | PRX-102 (Pegunigalsidase Alfa) | 52 | |
| Overall Study | Treated | Agalsidase Beta | 25 | |
| Overall Study | COMPLETED | PRX-102 (Pegunigalsidase Alfa) | 48 | |
| Overall Study | COMPLETED | Agalsidase Beta | 24 | |
| Overall Study | NOT COMPLETED | PRX-102 (Pegunigalsidase Alfa) | 5 | |
| Overall Study | NOT COMPLETED | Agalsidase Beta | 1 |
Source ClinicalTrials.gov
Adverse events190
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Hypertension | Vascular disorders | PRX-102 (Pegunigalsidase Alfa) | 3 | 52 | 4 |
| other | Hypertension | Vascular disorders | Agalsidase Beta | 1 | 25 | 1 |
| other | Hypotension | Vascular disorders | PRX-102 (Pegunigalsidase Alfa) | 0 | 52 | 0 |
| other | Hypotension | Vascular disorders | Agalsidase Beta | 2 | 25 | 2 |
| other | Fatigue | General disorders | PRX-102 (Pegunigalsidase Alfa) | 9 | 52 | 10 |
| other | Fatigue | General disorders | Agalsidase Beta | 4 | 25 | 6 |
| other | Pyrexia | General disorders | PRX-102 (Pegunigalsidase Alfa) | 5 | 52 | 5 |
| other | Pyrexia | General disorders | Agalsidase Beta | 3 | 25 | 4 |
| other | Oedema peripheral | General disorders | PRX-102 (Pegunigalsidase Alfa) | 4 | 52 | 9 |
| other | Oedema peripheral | General disorders | Agalsidase Beta | 3 | 25 | 3 |
1 to 10 of 190Source ClinicalTrials.gov
See all 190 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | PRX-102 (Pegunigalsidase Alfa) | 0 | 52 |
| Total, all-cause mortality | Agalsidase Beta | 0 | 25 |
| Total, other adverse events | PRX-102 (Pegunigalsidase Alfa) | 47 | 52 |
| Total, other adverse events | Agalsidase Beta | 24 | 25 |
| Total, serious adverse events | PRX-102 (Pegunigalsidase Alfa) | 8 | 52 |
| Total, serious adverse events | Agalsidase Beta | 6 | 25 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| PRX-102 (Pegunigalsidase Alfa) vs Agalsidase Beta: deaths from any cause | Trial comparisons |
| PRX-102 (Pegunigalsidase Alfa) vs Agalsidase Beta: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02795676
GET https://conductscience.org/api/trials/v1/NCT02795676/results?page=2