Trial · NCT05843916
Switch Over Study of Biosimilar Agalsidase Beta for Fabry Disease
NCT05843916, Phase 3, Completed, Bio Sidus SA; results first posted 2026-02-18
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. Switch Over Study of Biosimilar Agalsidase Beta for Fabry Disease. Trials, record NCT05843916. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05843916
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Switch Over Study of Biosimilar Agalsidase Beta for Fabry Disease
- NCT number
- NCT05843916
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 20 participants, actual
- Lead sponsor
- Bio Sidus SA
- Conditions
- Fabry Disease
- Interventions
- Recombinant human alpha galactosidase A (agalsidase beta), Recombinant human alpha-galactosidase A (agalsidase beta)
- First posted
- 2023-05-06
- Last updated
- 2026-02-18
- Results first posted
- 2026-02-18
- Limitations and caveats
This switch-over study was conducted in a small, heterogeneous cohort typical of rare-disease research. Limitations include the absence of a parallel control group, limited sample size, and a follow-up duration that may be insufficient for long-term assessment in a chronic progressive disease. These constraints are inherent to studies in rare diseases and were anticipated a priori.
Source ClinicalTrials.gov
Registered outcomes15
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in Lyso Gb3 Serum Levels | Baseline (after 5 week lead-in period on Fabrazyme®) and 26 weeks (after the switch to AGA BETA BS) | 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. |
| Secondary | Change From Baseline in Lyso-Gb3 Serum Levels | Baseline (after 5 week lead-in period on Fabrazyme®) and 54 weeks (after the switch to AGA BETA BS) | 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. |
| Secondary | Change From Baseline in Pain Severity as Assessed by Brief Pain Inventory-short Form Pain Severity Items Scores | Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS). | 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. |
| Secondary | Change From Baseline in Pain Interference as Assessed by BPI-short Form Pain Interference Items Scores | Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS). | 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. |
| Secondary | Change From Baseline in SF-36 Health Survey Scores | Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS). | 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. |
| Secondary | Mean Blood Urea Nitrogen (BUN) Levels at Specified Timepoints | Baseline (after 5 week lead-in period on Fabrazyme®), 14 weeks, 26 weeks and 54 weeks (after the switch to AGA BETA BS). | |
| Secondary | Mean Estimated Glomerular Filtration Rate (eGFR) at Specified Timepoints | Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS). | |
| Secondary | Mean Urine Albumin-to-Creatinine Ratio at Specified Timepoints | Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS). | |
| Secondary | Mean Electrolyte and Phosphate Levels at Specified Timepoints | Baseline (after 5 week lead-in period on Fabrazyme®), 14 weeks, 26 weeks and 54 weeks (after the switch to AGA BETA BS). | |
| Secondary | Mean Heart Rate From Electrocardiogram Assessments at Specified Timepoints | Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS). | Evaluation of Heart Rate based on the analysis of electrocardiogram exams performed at baseline, 26 weeks and 54 weeks. |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 registered outcomesOutcomes15
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in Lyso Gb3 Serum Levels | Baseline (after 5 week lead-in period on Fabrazyme®) and 26 weeks (after the switch to AGA BETA BS) | Differences are explained by the availability of samples collected within the correct protocol-defined window and the withdrawal of one subject before the start of the treatment period. | Nmol/L | MEAN |
| SECONDARY | Change From Baseline in Lyso-Gb3 Serum Levels | Baseline (after 5 week lead-in period on Fabrazyme®) and 54 weeks (after the switch to AGA BETA BS) | Differences are explained by the availability of samples collected within the correct visit window, the withdrawal of one subject before the start of the treatment period, and the discontinuation of one subject due to pregnancy before the Week 54 study visit. | Nmol/L | MEAN |
| SECONDARY | Change From Baseline in Pain Severity as Assessed by Brief Pain Inventory-short Form Pain Severity Items Scores | Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS). | The difference in N at Week 54 is explained by the discontinuation of one subject due to pregnancy. | Scores on a scale | MEAN |
| SECONDARY | Change From Baseline in Pain Interference as Assessed by BPI-short Form Pain Interference Items Scores | Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS). | The difference in N reflects the discontinuation of one subject due to pregnancy before week 54 visit and the availability of determinations that were either missing at specific visits or collected outside the correct protocol-defined window. | Scores on a scale | MEAN |
| SECONDARY | Change From Baseline in SF-36 Health Survey Scores | Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS). | The difference in N at Week 54 is explained by the discontinuation of one subject due to pregnancy. | Scores on a scale | MEAN |
| SECONDARY | Mean Blood Urea Nitrogen (BUN) Levels at Specified Timepoints | Baseline (after 5 week lead-in period on Fabrazyme®), 14 weeks, 26 weeks and 54 weeks (after the switch to AGA BETA BS). | The differences in N across visits reflect the discontinuation of one subject due to pregnancy before week 54 visit and the availability of laboratory determinations at each scheduled visit. | mmol/L | MEAN |
| SECONDARY | Mean Estimated Glomerular Filtration Rate (eGFR) at Specified Timepoints | Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS). | The difference in N at Week 54 is explained by the discontinuation of one subject due to pregnancy. | ml/min/1.73m2 | MEAN |
| SECONDARY | Mean Urine Albumin-to-Creatinine Ratio at Specified Timepoints | Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS). | The differences in N across visits reflect the discontinuation of one subject due to pregnancy and the availability of laboratory determinations at each scheduled visit. | g/mol | MEAN |
| SECONDARY | Mean Electrolyte and Phosphate Levels at Specified Timepoints | Baseline (after 5 week lead-in period on Fabrazyme®), 14 weeks, 26 weeks and 54 weeks (after the switch to AGA BETA BS). | The differences in N across visits reflect the discontinuation of one subject due to pregnancy and the availability of laboratory determinations at each scheduled visit. | mmol/L | MEAN |
| SECONDARY | Mean Heart Rate From Electrocardiogram Assessments at Specified Timepoints | Baseline (after 5 week lead-in period on Fabrazyme®), 26 weeks and 54 weeks (after the switch to AGA BETA BS). | The difference in N at Week 54 is explained by the discontinuation of one subject due to pregnancy. | Beats/min | MEAN |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 outcomesResults by group107
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in Lyso Gb3 Serum Levels | Fabrazyme Then AGA BETA BS (Intention to Treat Population) | Mean baseline Lyso-Gb3 | 4.99 | Standard Deviation 3.10 | Nmol/L |
| Change From Baseline in Lyso Gb3 Serum Levels | Fabrazyme Then AGA BETA BS (Per Protocol Population) | Mean baseline Lyso-Gb3 | 5.12 | Standard Deviation 3.15 | Nmol/L |
| Change From Baseline in Lyso Gb3 Serum Levels | Fabrazyme Then AGA BETA BS (Intention to Treat Population) | Mean 26 weeks Lyso-Gb3 | 4.95 | Standard Deviation 3.31 | Nmol/L |
| Change From Baseline in Lyso Gb3 Serum Levels | Fabrazyme Then AGA BETA BS (Per Protocol Population) | Mean 26 weeks Lyso-Gb3 | 5.14 | Standard Deviation 3.45 | Nmol/L |
| Change From Baseline in Lyso-Gb3 Serum Levels | Fabrazyme Then AGA BETA BS (Intention to Treat Population) | Mean baseline Lyso-Gb3 | 4.99 | Standard Deviation 3.102 | Nmol/L |
| Change From Baseline in Lyso-Gb3 Serum Levels | Fabrazyme Then AGA BETA BS (Per Protocol Population) | Mean baseline Lyso-Gb3 | 5.12 | Standard Deviation 3.15 | Nmol/L |
| Change From Baseline in Lyso-Gb3 Serum Levels | Fabrazyme Then AGA BETA BS (Intention to Treat Population) | Mean 54 weeks Lyso-Gb3 | 4.69 | Standard Deviation 3.51 | Nmol/L |
| Change From Baseline in Lyso-Gb3 Serum Levels | Fabrazyme Then AGA BETA BS (Per Protocol Population) | Mean 54 weeks Lyso-Gb3 | 4.71 | Standard Deviation 3.61 | Nmol/L |
| Change From Baseline in Pain Severity as Assessed by Brief Pain Inventory-short Form Pain Severity Items Scores | Fabrazyme Then AGA BETA BS | Baseline | 1.88 | Standard Deviation 2.02 | Scores on a scale |
| Change From Baseline in Pain Severity as Assessed by Brief Pain Inventory-short Form Pain Severity Items Scores | Fabrazyme Then AGA BETA BS | 26 weeks | 1.46 | Standard Deviation 2.04 | Scores on a scale |
1 to 10 of 107Source ClinicalTrials.gov
See all 107 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Lead in (Fabrazyme) | STARTED | Fabrazyme Then AGA BETA BS | 20 | |
| Lead in (Fabrazyme) | COMPLETED | Fabrazyme Then AGA BETA BS | 19 | |
| Lead in (Fabrazyme) | NOT COMPLETED | Fabrazyme Then AGA BETA BS | 1 | |
| Treatment Period (AGA BETA BS) | STARTED | Fabrazyme Then AGA BETA BS | 19 | |
| Treatment Period (AGA BETA BS) | COMPLETED | Fabrazyme Then AGA BETA BS | 18 | |
| Treatment Period (AGA BETA BS) | NOT COMPLETED | Fabrazyme Then AGA BETA BS | 1 |
Source ClinicalTrials.gov
Adverse events102
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 | Influenza | Infections and infestations | Lead-in Period (Fabrazyme) | 1 | 20 | 1 |
| other | Influenza | Infections and infestations | Treatment Period (AGA BETA BS) | 4 | 19 | 5 |
| other | Pharyngotonsillitis | Infections and infestations | Lead-in Period (Fabrazyme) | 0 | 20 | 0 |
| other | Pharyngotonsillitis | Infections and infestations | Treatment Period (AGA BETA BS) | 3 | 19 | 4 |
| other | Urinary tract infection | Infections and infestations | Lead-in Period (Fabrazyme) | 0 | 20 | 0 |
| other | Urinary tract infection | Infections and infestations | Treatment Period (AGA BETA BS) | 3 | 19 | 4 |
| other | Bronchitis | Infections and infestations | Lead-in Period (Fabrazyme) | 0 | 20 | 0 |
| other | Bronchitis | Infections and infestations | Treatment Period (AGA BETA BS) | 2 | 19 | 2 |
| other | Gastroenteritis | Infections and infestations | Lead-in Period (Fabrazyme) | 0 | 20 | 0 |
| other | Gastroenteritis | Infections and infestations | Treatment Period (AGA BETA BS) | 1 | 19 | 1 |
1 to 10 of 102Source ClinicalTrials.gov
See all 102 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Lead-in Period (Fabrazyme) | 0 | 20 |
| Total, all-cause mortality | Treatment Period (AGA BETA BS) | 0 | 19 |
| Total, other adverse events | Lead-in Period (Fabrazyme) | 4 | 20 |
| Total, other adverse events | Treatment Period (AGA BETA BS) | 17 | 19 |
| Total, serious adverse events | Lead-in Period (Fabrazyme) | 0 | 20 |
| Total, serious adverse events | Treatment Period (AGA BETA BS) | 5 | 19 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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 |
|---|---|
| Lead-in Period (Fabrazyme) vs Treatment Period (AGA BETA BS): deaths from any cause | Trial comparisons |
| Lead-in Period (Fabrazyme) vs Treatment Period (AGA BETA BS): 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/NCT05843916
GET https://conductscience.org/api/trials/v1/NCT05843916/results?page=2