A Phase 3 interventional study of Recombinant human alpha galactosidase A (agalsidase beta) and Recombinant human alpha-galactosidase A (agalsidase beta) in Fabry Disease, sponsored by Bio Sidus SA. Completed at 5 sites in Argentina. Open to participants aged 16 Years to 60 Years. Per ClinicalTrials.gov, last updated 2026-02-18.
Sponsored by Bio Sidus SA · Phase 3, Interventional, and Treatment
BIO-AGA-Fase III-001 is a Phase III, prospective, multicenter, open-label, single-group, baseline-controlled, switch over clinical trial to evaluate the efficacy and safety of AGA BETA BS in patients with FD already treated and previously stabilized with Fabrazyme®.
BIO-AGA-Fase III-001 is a Phase III, prospective, multicenter, open-label, single-group, baseline-controlled, switch over clinical trial to evaluate the efficacy and safety of AGA BETA BS in patients with FD already treated and previously stabilized with Fabrazyme®.
The study will be conducted in 2 parts: a 5-week Lead-in period (period 1) and 54 week treatment period (period 2). During period 1 all participants will receive 2 intravenous (IV) infusions of Fabrazyme®, provided by Biosidus. After that, in period 2 all participants will switch treatment to AGA BETA BS.
A total of up to 20 participants are planned for the study.
•The primary objective of the study is to evaluate the equivalence in efficacy between AGA BETA BS and Fabrazyme® after 6 months of treatment in participants with Fabry disease previously stabilized with Fabrazyme, by measuring disease biomarker (mean serum Lyso-Gb3 marker ratio after 26 weeks of treatment, defined as serum level of the marker Lyso-Gb3 after 26 weeks (6 months) divided by serum level of the marker Lyso-Gb3 at baseline).
244 studies on the registry are indexed under Fabry Disease; 55 are open to participants now.
This study's enrollment of 20 is close to the median of 22 across 108 interventional studies indexed under Fabry Disease.
Browse Fabry Disease studies →Bio Sidus SA is the lead sponsor of 6 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Sex and Age
Male or female participant with ≥16 and ≤60 years of age at the time of signing the form (ICF).
Reproduction
Confirmed previous diagnosis of FD.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
A “mini-stroke”: temporary stroke-like symptoms caused by reduced blood flow to part of the brain, spinal cord or eye, without signs of a new stroke on a scan.
Stroke symptoms need emergency care at once, even if they go away.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
TIA (transient ischaemic attack)
A “mini-stroke”: temporary stroke-like symptoms caused by reduced blood flow to part of the brain, spinal cord or eye, without signs of a new stroke on a scan.
Stroke symptoms need emergency care at once, even if they go away.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The study has a single-arm. First, all participants will receive 2 doses of Fabrazyme® with approximately 14 days between them, and afterwards all participants will switch treatment and receive AGA BETA BS for 54 weeks
Drug: Recombinant human alpha galactosidase A (agalsidase beta) · Drug: Recombinant human alpha-galactosidase A (agalsidase beta)
All participants will receive 2 doses of Fabrazyme® with approximately 14 days between them. The dose will be 1 mg/kg of body mass every 2 weeks
Also known as: Fabrazyme
All participants will receive AGA BETA BS for 54 weeks. The dose will be 1 mg/kg of body mass every 2 weeks
Also known as: AGA BETA BS
Change From Baseline in Lyso Gb3 Serum Levels
The primary objective of the study is to evaluate the equivalence in efficacy between AGA BETA BS and Fabrazyme® after 6 months of treatment in participants with Fabry disease previously stabilized with Fabrazyme, by measuring disease biomarker. Endpoint: Mean serum Lyso-Gb3 marker ratio after 26 weeks of treatment, defined as serum level of the marker Lyso-Gb3 after 26 weeks (6 months) divided by serum level of the marker Lyso-Gb3 at baseline.
Time frame: Baseline (after 5 week lead-in period on Fabrazyme®) and 26 weeks (after the switch to AGA BETA BS)
Change From Baseline in Lyso-Gb3 Serum Levels
To evaluate the difference in efficacy between AGA BETA BS and Fabrazyme® after 1 year (54 weeks) of treatment in participants with Fabry disease previously stabilized with Fabrazyme®, by measuring disease biomarker. Endpoint: Mean serum Lyso-Gb3 marker ratio after 54 weeks of treatment, defined as serum level of the marker Lyso-Gb3 after 54 weeks (12 months) divided by serum level of the marker Lyso-Gb3 at baseline.
Time frame: Baseline (after 5 week lead-in period on Fabrazyme®) and 54 weeks (after the switch to AGA BETA BS)
Change From Baseline in Pain Severity as Assessed by Brief Pain Inventory-short Form Pain Severity Items Scores
To compare the pain severity before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the Brief Pain Inventory-short form (BPI). The Brief Pain Inventory (BPI) is a validated patient-reported outcome instrument assessing pain severity and pain-related interference with daily activities. Pain severity is assessed using four items rated on a 0-10 numeric rating scale (0 = no pain; 10 = pain as bad as imaginable), with the score calculated as the mean of items (range 0-10; higher scores indicate worse pain). Pain interference is assessed using seven items rated on a 0-10 numeric rating scale (0 = does not interfere; 10 = completely interferes), with the score calculated as the mean of items (range 0-10; higher scores indicate worse outcome). Endpoint: Change from baseline in pain severity as assessed by BPI-short form pain severity items scores after 26 and 54 weeks of treatment.
Time frame: Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS).
Change From Baseline in Pain Interference as Assessed by BPI-short Form Pain Interference Items Scores
To compare the impact of pain on daily functions before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the BPI (Brief Pain Inventory)-short form. The Brief Pain Inventory (BPI) is a validated patient-reported outcome instrument assessing pain severity and pain-related interference with daily activities. Pain severity is assessed using four items rated on a 0-10 numeric rating scale (0 = no pain; 10 = pain as bad as imaginable), with the score calculated as the mean of items (range 0-10; higher scores indicate worse pain). Pain interference is assessed using seven items rated on a 0-10 numeric rating scale (0 = does not interfere; 10 = completely interferes), with the score calculated as the mean of items (range 0-10; higher scores indicate worse outcome). Endpoint: Change from baseline in pain severity as assessed by BPI-short form pain severity items scores after 26 and 54 weeks of treatment.
Time frame: Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS).
Change From Baseline in SF-36 Health Survey Scores
To compare the participants' perception of their own health before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the Short Form-36 Health Survey (SF-36). The Short Form-36 Health Survey (SF-36) is a validated patient-reported outcome instrument consisting of 36 questions designed to assess health-related quality of life. The questionnaire generates scores across eight domains (physical functioning, role limitations due to physical health, pain, general health, energy/fatigue, social functioning, role limitations due to emotional problems, and emotional well being), each scored on a 0-100 scale. Domain scores are calculated according to standard scoring algorithms, with higher scores indicating better health status and quality of life. Endpoint: Change from baseline in SF-36 scores after 26 and 54 weeks of treatment.
Time frame: Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS).
Mean Blood Urea Nitrogen (BUN) Levels at Specified Timepoints
Time frame: Baseline (after 5 week lead-in period on Fabrazyme®), 14 weeks, 26 weeks and 54 weeks (after the switch to AGA BETA BS).
Mean Estimated Glomerular Filtration Rate (eGFR) at Specified Timepoints
Time frame: Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS).
Mean Urine Albumin-to-Creatinine Ratio at Specified Timepoints
Time frame: Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS).
Mean Electrolyte and Phosphate Levels at Specified Timepoints
Time frame: Baseline (after 5 week lead-in period on Fabrazyme®), 14 weeks, 26 weeks and 54 weeks (after the switch to AGA BETA BS).
Mean Heart Rate From Electrocardiogram Assessments at Specified Timepoints
Evaluation of Heart Rate based on the analysis of electrocardiogram exams performed at baseline, 26 weeks and 54 weeks.
Time frame: Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS).
Mean PR, QRS, QT, QTcB, and QTcF Intervals at Specified Timepoints
Evaluation of PR, QRS, QT , QTcB and QTcF intervals based on the analysis of electrocardiogram exams performed at baseline, 26 weeks and 54 weeks.
Time frame: Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS).
Mean Left Ventricular Wall Thickness at Specified Timepoints
Evaluation of cardiac function based on the analysis of bidimensional echocardiogram exams performed at baseline, 26 weeks and 54 weeks.
Time frame: Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS).
Mean Left Ventricular Mass Index at Specified Timepoints
Evaluation of cardiac function based on the analysis of bidimensional echocardiogram exams performed at baseline, 26 weeks and 54 weeks.
Time frame: Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS).
Mean Left Ventricular Ejection Fraction at Specified Timepoints
Evaluation of cardiac function based on the analysis of bidimensional echocardiogram exams performed at baseline, 26 weeks and 54 weeks.
Time frame: Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS).
Number of Participants With Positive Neutralizing Antibodies at Specified Timepoints
Count of participants whose serum samples showed a neutralization percentage of agalsidase beta activity greater than 50%, the predefined threshold indicating a positive result for neutralizing antibodies. Neutralizing antibody activity was evaluated using blood samples collected at Baseline, Week 14, Week 26, and Week 54. Participants with neutralization values exceeding the 50% cutoff were classified as positive for neutralizing antibodies at each timepoint.
Time frame: Baseline (after 5 week lead-in period on Fabrazyme®),14 weeks, 26 weeks and 54 weeks (after the switch to AGA BETA BS).
| Milestone | Fabrazyme Then AGA BETA BS |
|---|---|
| Started | 20 |
| Completed | 19 |
| Not completed | 1 |
| Withdrew: Withdrawal by subject | 1 |
| Milestone | Fabrazyme Then AGA BETA BS |
|---|---|
| Started | 19 |
| Completed | 18 |
| Not completed | 1 |
| Withdrew: Pregnancy | 1 |
The primary objective of the study is to evaluate the equivalence in efficacy between AGA BETA BS and Fabrazyme® after 6 months of treatment in participants with Fabry disease previously stabilized with Fabrazyme, by measuring disease biomarker. Endpoint: Mean serum Lyso-Gb3 marker ratio after 26 weeks of treatment, defined as serum level of the marker Lyso-Gb3 after 26 weeks (6 months) divided by serum level of the marker Lyso-Gb3 at baseline.
| Nmol/L | Fabrazyme Then AGA BETA BS (Intention to Treat Population) | Fabrazyme Then AGA BETA BS (Per Protocol Population) |
|---|---|---|
| Mean baseline Lyso-Gb3 | 4.99 ± 3.10 | 5.12 ± 3.15 |
| Mean 26 weeks Lyso-Gb3 | 4.95 ± 3.31 | 5.14 ± 3.45 |
To evaluate the difference in efficacy between AGA BETA BS and Fabrazyme® after 1 year (54 weeks) of treatment in participants with Fabry disease previously stabilized with Fabrazyme®, by measuring disease biomarker. Endpoint: Mean serum Lyso-Gb3 marker ratio after 54 weeks of treatment, defined as serum level of the marker Lyso-Gb3 after 54 weeks (12 months) divided by serum level of the marker Lyso-Gb3 at baseline.
| Nmol/L | Fabrazyme Then AGA BETA BS (Intention to Treat Population) | Fabrazyme Then AGA BETA BS (Per Protocol Population) |
|---|---|---|
| Mean baseline Lyso-Gb3 | 4.99 ± 3.102 | 5.12 ± 3.15 |
| Mean 54 weeks Lyso-Gb3 | 4.69 ± 3.51 | 4.71 ± 3.61 |
To compare the pain severity before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the Brief Pain Inventory-short form (BPI). The Brief Pain Inventory (BPI) is a validated patient-reported outcome instrument assessing pain severity and pain-related interference with daily activities. Pain severity is assessed using four items rated on a 0-10 numeric rating scale (0 = no pain; 10 = pain as bad as imaginable), with the score calculated as the mean of items (range 0-10; higher scores indicate worse pain). Pain interference is assessed using seven items rated on a 0-10 numeric rating scale (0 = does not interfere; 10 = completely interferes), with the score calculated as the mean of items (range 0-10; higher scores indicate worse outcome). Endpoint: Change from baseline in pain severity as assessed by BPI-short form pain severity items scores after 26 and 54 weeks of treatment.
| Scores on a scale | Fabrazyme Then AGA BETA BS |
|---|---|
| Baseline | 1.88 ± 2.02 |
| 26 weeks | 1.46 ± 2.04 |
| 54 weeks | 1.18 ± 2.24 |
To compare the impact of pain on daily functions before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the BPI (Brief Pain Inventory)-short form. The Brief Pain Inventory (BPI) is a validated patient-reported outcome instrument assessing pain severity and pain-related interference with daily activities. Pain severity is assessed using four items rated on a 0-10 numeric rating scale (0 = no pain; 10 = pain as bad as imaginable), with the score calculated as the mean of items (range 0-10; higher scores indicate worse pain). Pain interference is assessed using seven items rated on a 0-10 numeric rating scale (0 = does not interfere; 10 = completely interferes), with the score calculated as the mean of items (range 0-10; higher scores indicate worse outcome). Endpoint: Change from baseline in pain severity as assessed by BPI-short form pain severity items scores after 26 and 54 weeks of treatment.
| Scores on a scale | Fabrazyme Then AGA BETA BS |
|---|---|
| Baseline | 2.56 ± 3.03 |
| 26 weeks | 1.64 ± 2.36 |
| 54 weeks | 2.25 ± 2.8 |
To compare the participants' perception of their own health before and after the treatment with AGA BETA BS in participants with Fabry disease previously stabilized with Fabrazyme®, as measured by the Short Form-36 Health Survey (SF-36). The Short Form-36 Health Survey (SF-36) is a validated patient-reported outcome instrument consisting of 36 questions designed to assess health-related quality of life. The questionnaire generates scores across eight domains (physical functioning, role limitations due to physical health, pain, general health, energy/fatigue, social functioning, role limitations due to emotional problems, and emotional well being), each scored on a 0-100 scale. Domain scores are calculated according to standard scoring algorithms, with higher scores indicating better health status and quality of life. Endpoint: Change from baseline in SF-36 scores after 26 and 54 weeks of treatment.
| Scores on a scale | Fabrazyme Then AGA BETA BS |
|---|---|
| Physical Functioning Score - Mean Baseline | 79.5 ± 21.01 |
| Physical Functioning Score - Mean 26 weeks | 78.7 ± 24.99 |
| Physical Functioning Score - Mean 54 weeks | 76.7 ± 24.43 |
| Role limitations due to physical health- Mean Baseline | 71.1 ± 41.05 |
| Role limitations due to physical health-Mean 26 weeks | 73.7 ± 38.62 |
| Role limitations due to physical health- Mean 54 weeks | 76.4 ± 42.42 |
| Role limitations due to emotional problems-Mean Baseline | 50.88 ± 44.95 |
| Role limitations due to emotional problems- Mean 26 weeks | 70.18 ± 45.67 |
| Role limitations due to emotional problems- Mean 54 weeks | 62.96 ± 46.99 |
| Energy/fatigue- Mean Baseline | 51.10 ± 21.38 |
| Energy/fatigue- Mean 26 weeks | 60.30 ± 22.27 |
| Energy/fatigue - Mean 54 weeks | 55 ± 16.36 |
| Emotional well-being - Mean Baseline | 68.6 ± 20.54 |
| Emotional well-being- Mean 26 weeks | 71.2 ± 17.21 |
| Emotional well-being- Mean 54 weeks | 64.7 ± 15.84 |
| Social functioning- Mean Baseline | 79.61 ± 19.92 |
| Social functioning- Mean 26 weeks | 77.24 ± 27.22 |
| Social functioning- Mean 54 weeks | 79.17 ± 20.29 |
| Pain- Mean Baseline | 69.61 ± 27.03 |
| Pain- Mean 26 weeks | 74.21 ± 25.90 |
| Pain- Mean 54 weeks | 84.17 ± 23.87 |
| General Health-Mean Baseline | 60.30 ± 22.28 |
| General Health-Mean 26 weeks | 62.40 ± 20.77 |
| General Health- Mean 54 weeks | 57.80 ± 24.27 |
| mmol/L | Fabrazyme Then AGA BETA BS |
|---|---|
| Mean Baseline Blood Urea Nitrogen | 5.03 ± 2.19 |
| Mean 14 weeks Blood Urea Nitrogen | 4.70 ± 1.76 |
| Mean 26 weeks Blood Urea Nitrogen | 4.34 ± 1.40 |
| Mean 54 weeks Blood Urea Nitrogen | 4.78 ± 1.82 |
| ml/min/1.73m2 | Fabrazyme Then AGA BETA BS |
|---|---|
| Mean Baseline eGFR | 106.05 ± 25.32 |
| Mean 26 weeks eGFR | 107.99 ± 27.02 |
| Mean 54 weeks eGFR | 100.47 ± 26.54 |
| g/mol | Fabrazyme Then AGA BETA BS |
|---|---|
| Mean Baseline Urine Albumin/Creatinine ratio | 9.26 ± 13.95 |
| Mean 26 weeks Urine Albumin/Creatinine ratio | 9.14 ± 12.16 |
| Mean 54 weeks Urine Albumin/Creatinine ratio | 9.8 ± 20.52 |
| mmol/L | Fabrazyme Then AGA BETA BS |
|---|---|
| Mean Baseline Sodium | 141.10 ± 3.16 |
| Mean 14 weeks Sodium | 140.30 ± 2.91 |
| Mean 26 weeks Sodium | 140.40 ± 2.89 |
| Mean 54 weeks Sodium | 140.80 ± 4.71 |
| Mean Baseline Potassium | 4.22 ± 0.41 |
| Mean 14 weeks Potassium | 4.36 ± 0.45 |
| Mean 26 weeks Potassium | 4.31 ± 0.37 |
| Mean 54 weeks Potassium | 4.25 ± 0.58 |
| Mean Baseline Magnesium | 0.84 ± 0.13 |
| Mean 14 weeks Magnesium | 0.83 ± 0.11 |
| Mean 26 weeks Magnesium | 0.83 ± 0.09 |
| Mean 54 weeks Magnesium | 0.85 ± 0.12 |
| Mean Baseline Calcium | 2.43 ± 0.14 |
| Mean 14 weeks Calcium | 2.37 ± 0.12 |
| Mean 26 weeks Calcium | 2.36 ± 0.14 |
| Mean 54 weeks Calcium | 2.40 ± 0.13 |
| Mean Baseline Chloride | 104.28 ± 4.26 |
| Mean 14 weeks Chloride | 103.71 ± 3.51 |
| Mean 26 weeks Chloride | 104.49 ± 4.82 |
| Mean 54 weeks Chloride | 104.72 ± 5.24 |
| Mean Baseline Bicarbonate | 24.18 ± 2.84 |
| Mean 14 weeks Bicarbonate | 24.47 ± 2.33 |
| Mean 26 weeks Bicarbonate | 24.25 ± 2.44 |
| Mean 54 weeks Bicarbonate | 23.78 ± 2.50 |
| Mean Baseline Phosphate | 1.12 ± 0.40 |
| Mean 14 weeks Phosphate | 1.11 ± 0.28 |
| Mean 26 weeks Phosphate | 1.21 ± 0.25 |
| Mean 54 weeks Phosphate | 1.28 ± 0.22 |
Evaluation of Heart Rate based on the analysis of electrocardiogram exams performed at baseline, 26 weeks and 54 weeks.
| Beats/min | Fabrazyme Then AGA BETA BS |
|---|---|
| Baseline Mean ECG-assesed Heart Rate | 61.80 ± 10.06 |
| 26 weeks Mean ECG-assesed Heart Rate | 64.50 ± 10.93 |
| 54 weeks Mean ECG-assesed Heart Rate | 63.10 ± 12.43 |
Evaluation of PR, QRS, QT , QTcB and QTcF intervals based on the analysis of electrocardiogram exams performed at baseline, 26 weeks and 54 weeks.
| msec | Fabrazyme Then AGA BETA BS |
|---|---|
| Mean Baseline PR interval | 130.30 ± 45.71 |
| Mean 26 weeks PR interval | 119.2 ± 36.72 |
| Mean 54 weeks PR interval | 127 ± 37.98 |
| Mean Baseline QRS interval | 79.5 ± 10.05 |
| Mean 26 weeks QRS interval | 80.50 ± 8.77 |
| Mean 54 weeks QRS interval | 85.50 ± 16.40 |
| Mean Baseline QT interval | 416.60 ± 36.95 |
| Mean 26 weeks QT interval | 398.90 ± 46.90 |
| Mean 54 weeks QT interval | 411.10 ± 37.79 |
| Mean Baseline QTcB interval | 417.20 ± 22.05 |
| Mean 26 weeks QTcB interval | 407.30 ± 34.80 |
| Mean 54 weeks QTcB interval | 420.20 ± 41.39 |
| Mean Baseline QTcF interval | 421.50 ± 28.04 |
| Mean 26 weeks QTcF interval | 404.30 ± 42.06 |
| Mean 54 weeks QTcF interval | 415.60 ± 35.21 |
Evaluation of cardiac function based on the analysis of bidimensional echocardiogram exams performed at baseline, 26 weeks and 54 weeks.
| mm | Fabrazyme Then AGA BETA BS |
|---|---|
| Mean Baseline Left Ventricle wall thickness | 9.04 ± 2.27 |
| Mean 26 weeks Left Ventricle wall thickness | 8.38 ± 2.47 |
| Mean 54 weeks Left Ventricle wall thickness | 9.65 ± 2.29 |
Evaluation of cardiac function based on the analysis of bidimensional echocardiogram exams performed at baseline, 26 weeks and 54 weeks.
| g/m² | Fabrazyme Then AGA BETA BS |
|---|---|
| Mean Baseline Left Ventricular Mass Index | 85.50 ± 35.33 |
| Mean 26 weeks Left Ventricular Mass Index | 95.96 ± 37.31 |
| Mean 54 weeks Left Ventricular Mass Index | 110.13 ± 43.37 |
Evaluation of cardiac function based on the analysis of bidimensional echocardiogram exams performed at baseline, 26 weeks and 54 weeks.
| percentage of ejection fraction | Fabrazyme Then AGA BETA BS |
|---|---|
| Mean Baseline Left Ventricle Ejection Fraction | 65.16 ± 6.21 |
| Mean 26 weeks Left Ventricle Ejection Fraction | 64.42 ± 7.27 |
| Mean 54 weeks Left Ventricle Ejection Fraction | 63.28 ± 11.22 |
Count of participants whose serum samples showed a neutralization percentage of agalsidase beta activity greater than 50%, the predefined threshold indicating a positive result for neutralizing antibodies. Neutralizing antibody activity was evaluated using blood samples collected at Baseline, Week 14, Week 26, and Week 54. Participants with neutralization values exceeding the 50% cutoff were classified as positive for neutralizing antibodies at each timepoint.
| Participants | Fabrazyme Then AGA BETA BS |
|---|---|
| Baseline | 1 |
| 14 weeks | 1 |
| 26 weeks | 1 |
| 54 weeks | 1 |
Collected over 13 months. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Lead-in Period (Fabrazyme) | 0/20 (0%) | 0/20 (0%) | 4/20 (20%) |
| Treatment Period (AGA BETA BS) | 0/19 (0%) | 5/19 (26.3%) | 17/19 (89.5%) |
| Event | Lead-in Period (Fabrazyme) | Treatment Period (AGA BETA BS) |
|---|---|---|
| Renal AtrophyRenal and urinary disorders | 0/20 | 1/19 |
| CholelithiasisHepatobiliary disorders | 0/20 | 1/19 |
| Ischaemic StrokeNervous system disorders | 0/20 | 1/19 |
| Atrial fibrillationCardiac disorders | 0/20 | 1/19 |
| Chest painGeneral disorders | 0/20 | 1/19 |
| Urinary tract infectionInfections and infestations | 0/20 | 1/19 |
| Event | Lead-in Period (Fabrazyme) | Treatment Period (AGA BETA BS) |
|---|---|---|
| InfluenzaInfections and infestations | 1/20 | 4/19 |
| HeadacheNervous system disorders | 0/20 | 4/19 |
| PharyngotonsillitisInfections and infestations | 0/20 | 3/19 |
| Urinary tract infectionInfections and infestations | 0/20 | 3/19 |
| Pyrexia (fever)General disorders | 1/20 | 3/19 |
| BronchitisInfections and infestations | 0/20 | 2/19 |
| Adverse drug reactionGeneral disorders | 0/20 | 2/19 |
| ChillsGeneral disorders | 0/20 | 2/19 |
| MyalgiaMusculoskeletal and connective tissue disorders | 0/20 | 2/19 |
| Drug HypersensitivityImmune system disorders | 0/20 | 2/19 |
| Age, Continuous(years) | Fabrazyme Then AGA BETA BS |
|---|---|
| Mean | 35.6 ± 12.6 |
| Sex: Female, Male(Participants) | Fabrazyme Then AGA BETA BS |
|---|---|
| Female | 15 |
| Male | 5 |
| Ethnicity (NIH/OMB)(Participants) | Fabrazyme Then AGA BETA BS |
|---|---|
| Hispanic or Latino | 20 |
| Not Hispanic or Latino | 0 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | Fabrazyme Then AGA BETA BS |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 20 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(participants) | Fabrazyme Then AGA BETA BS |
|---|---|
| Argentina | 20 |
| Height(Centimeters) | Fabrazyme Then AGA BETA BS |
|---|---|
| Mean | 162.2 ± 7.7 |
| Weight(Kilograms) | Fabrazyme Then AGA BETA BS |
|---|---|
| Mean | 69.9 ± 15.3 |
| BMI(Kg/m2) | Fabrazyme Then AGA BETA BS |
|---|---|
| Mean | 26.4 ± 6.2 |
1 further baseline measures are reported on the registry.
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Dec 2022 to primary completion in Sep 2024, 21 months.
Results were first posted in Feb 2026, 1 year 4 months after primary completion.
Of 22 phase 3 interventional studies under Fabry Disease, half reached primary completion within 41 months of starting (the median), counting those still running; a quarter within 31 and three quarters within 64. This study's 21 months is below that.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Jan 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Agalsidase beta
Bio Sidus SA