Trial · NCT00081497
A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
NCT00081497, Phase 4, Completed, Genzyme, a Sanofi Company; results first posted 2010-08-18
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease. Trials, record NCT00081497. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00081497
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
- NCT number
- NCT00081497
- Status
- Completed
- Phases
- Phase 4
- Enrollment
- 67 participants, actual
- Lead sponsor
- Genzyme, a Sanofi Company
- Conditions
- Fabry Disease
- Interventions
- agalsidase beta
- First posted
- 2004-04-16
- Last updated
- 2015-04-02
- Results first posted
- 2010-08-18
- Limitations and caveats
No valid conclusions can be made from the predefined primary efficacy analysis. The sponsor believes that it is more statistically appropriate to compare placebo patients with Fabrazyme patients as they were randomized in the original Phase 4 trial.
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Difference in Inverse Serum Creatinine Within Patients' Slopes Between the Placebo AGAL-008-00 (NCT00074984) and Fabrazyme AGAL02503 (NCT00081497) Periods | Placebo period AGAL-008-00 (up to 35 months) through Fabrazyme period AGAL02503 (18 months) | The primary efficacy analysis was the summary of change in slope of inverse serum creatinine for Placebo/Fabrazyme patients in the Intent to Treat (ITT) Population. It compared the placebo period slope with the Fabrazyme period slope. |
| Secondary | Serum Creatinine at Pre-Fabrazyme and 6, 12, and 18 Months | Pre-Fabrazyme, 6, 12, and 18 months | Pre-Fabrazyme=baseline visit of AGAL-008-00 (NCT00074984) for Fabrazyme patients; assessment prior to open-label for placebo patients who transitioned to Fabrazyme in AGAL-008-00 (NCT00074984); assessment prior to first Fabrazyme infusion in AGAL02503 (NCT00081497) for placebo patients who did not transition to Fabrazyme in AGAL-008-00 (NCT00074984). |
| Secondary | Estimated Glomerular Filtration Rate (eGFR) at Pre-Fabrazyme and 6, 12, and 18 Months | Pre-Fabrazyme, 6, 12, and 18 months | Pre-Fabrazyme=baseline visit of AGAL-00-800 (NCT00074984) for Fabrazyme patients; assessment prior to open-label for placebo patients who transitioned to Fabrazyme in AGAL-008-00 (NCT00074984); assessment prior to first Fabrazyme infusion in AGAL02503 (NCT00081497) for placebo patients who did not transition to Fabrazyme in AGAL-008-00 (NCT00074984). |
| Secondary | Plasma Globotriaosylceramide (GL-3) (Normal Plasma GL-3 Level is ≤ 7.03 µg/mL) at Pre-Fabrazyme and 6, 12, and 18 Months | Pre-Fabrazyme and 6, 12, and 18 months | Pre-Fabrazyme=baseline visit of AGAL00800 for Fabrazyme patients; assessment prior to open-label for placebo patients who transitioned to Fabrazyme in AGAL00800; assessment prior to first Fabrazyme infusion in AGAL02503 for placebo patients who did not transition to Fabrazyme in AGAL00800. |
| Secondary | Proteinuria at Pre-Fabrazyme and 6, 12, and 18 Months | Pre-Fabrazyme and 6, 12, and 18 months | Pre-Fabrazyme=baseline visit of AGAL-008-00 (NCT00074984) for Fabrazyme patients; assessment prior to open-label for placebo patients who transitioned to Fabrazyme in AGAL-008-00 (NCT00074984); assessment prior to first Fabrazyme infusion in AGAL02503 (NCT00081497) for placebo patients who did not transition to Fabrazyme in AGAL-008-00 (NCT00074984). |
Source ClinicalTrials.gov
Outcomes6
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Difference in Inverse Serum Creatinine Within Patients' Slopes Between the Placebo AGAL-008-00 (NCT00074984) and Fabrazyme AGAL02503 (NCT00081497) Periods | Placebo period AGAL-008-00 (up to 35 months) through Fabrazyme period AGAL02503 (18 months) | ITT Population - Analysis compares results during the placebo period with those during the Fabrazyme period and includes only the 28 patients who were randomized to placebo in the AGAL-008-00 (NCT00074984) study; as such no formal sample size calculations were performed. | dL/mg/year | LEAST_SQUARES_MEAN |
| POST_HOC | Differences in Slopes of Estimated Glomerular Filtration Rate (eGFR) Comparing Randomized Placebo vs Fabrazyme Patients (Based on the Original Randomization in AGAL-008-00 (NCT00074984)) by Baseline eGFR Subgroups of >60 and ≤60 mL/Min/1.73 m^2. | Throughout study; 18 months | ITT population. For subgroup Estimated Glomerular Filtration Rate (eGFR) \>60, there were 9 placebo patients and 15 Fabrazyme patients. For subgroup eGFR ≤60, there were 19 placebo patients and 24 Fabrazyme patients. For randomized Fabrazyme patients, both the double-blind and open-label data was used. | mL/min/1.73m^2/year | LEAST_SQUARES_MEAN |
| SECONDARY | Serum Creatinine at Pre-Fabrazyme and 6, 12, and 18 Months | Pre-Fabrazyme, 6, 12, and 18 months | ITT population-67 patients had assessments at 6 and 12 months while 65 patients had assessments at 18 months in the open-label extension study. | mg/dL | MEAN |
| SECONDARY | Estimated Glomerular Filtration Rate (eGFR) at Pre-Fabrazyme and 6, 12, and 18 Months | Pre-Fabrazyme, 6, 12, and 18 months | ITT population-67 patients had assessments at 6 and 12 months while 65 patients had assessments at 18 months in the open-label extension study. | ml/min/1.73m^2 | MEAN |
| SECONDARY | Plasma Globotriaosylceramide (GL-3) (Normal Plasma GL-3 Level is ≤ 7.03 µg/mL) at Pre-Fabrazyme and 6, 12, and 18 Months | Pre-Fabrazyme and 6, 12, and 18 months | ITT population-64 patients had assessments at 6 and 18 months while 65 patients had assessments at 12 months in the open-label extension study. | µg/mL | MEAN |
| SECONDARY | Proteinuria at Pre-Fabrazyme and 6, 12, and 18 Months | Pre-Fabrazyme and 6, 12, and 18 months | ITT population-62 patients had assessments at 6 months, 61 patients had assessments at 12 months, and 54 patients had assessments at 18 months in the open-label extension study. | urine protein(mg/dL) / creatinine(mg/dL) | MEAN |
Source ClinicalTrials.gov
Results by group22
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Difference in Inverse Serum Creatinine Within Patients' Slopes Between the Placebo AGAL-008-00 (NCT00074984) and Fabrazyme AGAL02503 (NCT00081497) Periods | Placebo Period - AGAL-008-00 (NCT00074984) | -0.044 | Standard Error 0.012 | dL/mg/year | |
| Difference in Inverse Serum Creatinine Within Patients' Slopes Between the Placebo AGAL-008-00 (NCT00074984) and Fabrazyme AGAL02503 (NCT00081497) Periods | Fabrazyme Period - AGAL02503 (NCT00081497) | -0.073 | Standard Error 0.011 | dL/mg/year | |
Differences in Slopes of Estimated Glomerular Filtration Rate (eGFR) Comparing Randomized Placebo vs Fabrazyme Patients (Based on the Original Randomization in AGAL-008-00 (NCT00074984)) by Baseline eGFR Subgroups of >60 and ≤60 mL/Min/1.73 m^2. | Placebo - AGAL-008-00 (NCT00074984) eGFR >60 | -7.685 | Standard Error 1.836 | mL/min/1.73m^2/year | |
Differences in Slopes of Estimated Glomerular Filtration Rate (eGFR) Comparing Randomized Placebo vs Fabrazyme Patients (Based on the Original Randomization in AGAL-008-00 (NCT00074984)) by Baseline eGFR Subgroups of >60 and ≤60 mL/Min/1.73 m^2. | Fabrazyme - AGAL02503 (NCT00081497) eGFR >60 | -0.898 | Standard Error 1.129 | mL/min/1.73m^2/year | |
Differences in Slopes of Estimated Glomerular Filtration Rate (eGFR) Comparing Randomized Placebo vs Fabrazyme Patients (Based on the Original Randomization in AGAL-008-00 (NCT00074984)) by Baseline eGFR Subgroups of >60 and ≤60 mL/Min/1.73 m^2. | Placebo - AGAL-008-00 (NCT00074984) eGFR ≤60 | -2.575 | Standard Error 1.188 | mL/min/1.73m^2/year | |
Differences in Slopes of Estimated Glomerular Filtration Rate (eGFR) Comparing Randomized Placebo vs Fabrazyme Patients (Based on the Original Randomization in AGAL-008-00 (NCT00074984)) by Baseline eGFR Subgroups of >60 and ≤60 mL/Min/1.73 m^2. | Fabrazyme - AGAL02503 (NCT00081497) eGFR ≤60 | -4.906 | Standard Error 0.900 | mL/min/1.73m^2/year | |
| Serum Creatinine at Pre-Fabrazyme and 6, 12, and 18 Months | Fabrazyme 1.0 mg/kg Every 2 Weeks | Serum Creatinine at Pre-Fabrazyme | 1.7 | Standard Deviation 0.60 | mg/dL |
| Serum Creatinine at Pre-Fabrazyme and 6, 12, and 18 Months | Fabrazyme 1.0 mg/kg Every 2 Weeks | Serum Creatinine at 6 months post-treatment | 1.8 | Standard Deviation 0.77 | mg/dL |
| Serum Creatinine at Pre-Fabrazyme and 6, 12, and 18 Months | Fabrazyme 1.0 mg/kg Every 2 Weeks | Serum Creatinine at 12 months post-treatment | 1.9 | Standard Deviation 0.98 | mg/dL |
| Serum Creatinine at Pre-Fabrazyme and 6, 12, and 18 Months | Fabrazyme 1.0 mg/kg Every 2 Weeks | Serum Creatinine at 18 months post-treatment | 2.1 | Standard Deviation 1.21 | mg/dL |
1 to 10 of 22Source ClinicalTrials.gov
See all 22 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Placebo/Fabrazyme | 28 | |
| Overall Study | STARTED | Fabrazyme/Fabrazyme | 39 | |
| Overall Study | COMPLETED | Placebo/Fabrazyme | 25 | |
| Overall Study | COMPLETED | Fabrazyme/Fabrazyme | 37 | |
| Overall Study | NOT COMPLETED | Placebo/Fabrazyme | 3 | |
| Overall Study | NOT COMPLETED | Fabrazyme/Fabrazyme | 2 |
Source ClinicalTrials.gov
Adverse events1,545
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 | Anaemia | Blood and lymphatic system disorders | Fabrazyme/Fabrazyme | 5 | 39 | |
| other | Anaemia | Blood and lymphatic system disorders | Placebo/Fabrazyme | 5 | 28 | |
| other | Anaemia | Blood and lymphatic system disorders | Total | 10 | 67 | |
| other | Leukopenia | Blood and lymphatic system disorders | Fabrazyme/Fabrazyme | 0 | 39 | |
| other | Leukopenia | Blood and lymphatic system disorders | Placebo/Fabrazyme | 1 | 28 | |
| other | Leukopenia | Blood and lymphatic system disorders | Total | 1 | 67 | |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Fabrazyme/Fabrazyme | 2 | 39 | |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Placebo/Fabrazyme | 0 | 28 | |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Total | 2 | 67 | |
| other | Nephrogenic anaemia | Blood and lymphatic system disorders | Fabrazyme/Fabrazyme | 1 | 39 |
1 to 10 of 1,545Source ClinicalTrials.gov
See all 1,545 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Fabrazyme/Fabrazyme | ||
| Total, all-cause mortality | Placebo/Fabrazyme | ||
| Total, all-cause mortality | Total | ||
| Total, other adverse events | Fabrazyme/Fabrazyme | 39 | 39 |
| Total, other adverse events | Placebo/Fabrazyme | 27 | 28 |
| Total, other adverse events | Total | 66 | 67 |
| Total, serious adverse events | Fabrazyme/Fabrazyme | 19 | 39 |
| Total, serious adverse events | Placebo/Fabrazyme | 12 | 28 |
| Total, serious adverse events | Total | 31 | 67 |
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT00081497
GET https://conductscience.org/api/trials/v1/NCT00081497/results?page=2