Trial · NCT00084084
Replagal Enzyme Replacement Therapy for Children With Fabry Disease
NCT00084084, Phase 2, Completed, Shire; results first posted 2013-10-10
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. Replagal Enzyme Replacement Therapy for Children With Fabry Disease. Trials, record NCT00084084. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00084084
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Replagal Enzyme Replacement Therapy for Children With Fabry Disease
- NCT number
- NCT00084084
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 17 participants, actual
- Lead sponsor
- Shire
- Conditions
- Fabry Disease
- Interventions
- Agalsidase alfa
- First posted
- 2004-06-07
- Last updated
- 2021-07-30
- Results first posted
- 2013-10-10
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Patients Who Experienced At Least One Adverse Event (AE) | 362 weeks | |
| Secondary | Pharmacokinetics - Area Under the Serum Concentration-Time Curve (AUC0-∞) - Week 81 | Pre-infusion; and post-infusion at 20, 40, 50, 60, 90 minutes, and 2, 3, 4, 8 hours. | AUC0-∞ is a measure of the total exposure to a drug. |
| Secondary | Pharmacokinetics - Area Under the Serum Concentration-Time Curve (AUC0-∞) - Week 133 | Pre-infusion; and post-infusion at 20, 40, 50, 60, 90 minutes, and 2, 3, 4, 8 hours. | AUC0-∞ is a measure of the total exposure to a drug. |
| Secondary | Pharmacokinetics - Area Under the Serum Concentration-Time Curve (AUC0-∞) - Week 159 | Pre-infusion; and post-infusion at 20, 40, 50, 60, 90 minutes, and 2, 3, 4, 8 hours. | AUC0-∞ is a measure of the total exposure to a drug. |
| Secondary | Pharmacokinetics - Area Under the Serum Concentration-Time Curve (AUC0-∞) - Week 315/341 | Pre-infusion; and post-infusion at 20, 40, 50, 60, 90 minutes, and 2, 3, 4, 8 hours. | AUC0-∞ is a measure of the total exposure to a drug. |
| Secondary | Pharmacokinetics - Maximum Observed Serum Concentration (Cmax) - Week 81 | Pre-infusion; and post-infusion at 20, 40, 50, 60, 90 minutes, and 2, 3, 4, 8 hours. | Cmax is the peak plasma concentration of a drug after administration. |
| Secondary | Pharmacokinetics - Maximum Observed Serum Concentration (Cmax) - Week 133 | Pre-infusion; and post-infusion at 20, 40, 50, 60, 90 minutes, and 2, 3, 4, 8 hours. | Cmax is the peak plasma concentration of a drug after administration. |
| Secondary | Pharmacokinetics - Maximum Observed Serum Concentration (Cmax) - Week 159 | Pre-infusion; and post-infusion at 20, 40, 50, 60, 90 minutes, and 2, 3, 4, 8 hours. | Cmax is the peak plasma concentration of a drug after administration. |
| Secondary | Pharmacokinetics - Maximum Observed Serum Concentration (Cmax) - Week 315/341 | Pre-infusion; and post-infusion at 20, 40, 50, 60, 90 minutes, and 2, 3, 4, 8 hours. | Cmax is the peak plasma concentration of a drug after administration. |
| Other | Heart Rate Variability - Change From Baseline at Week 185 in SDNN | Week 185 | Heart rate variability was assessed by 2-hour Holter monitoring. Standard deviation of all filtered RR intervals over the length of the analysis (SDNN) was measured. |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Patients Who Experienced At Least One Adverse Event (AE) | 362 weeks | Safety Population: Patients in Cohort 1 who received at least one dose of Replagal RB in Phase 1. | participants | NUMBER |
| SECONDARY | Pharmacokinetics - Area Under the Serum Concentration-Time Curve (AUC0-∞) - Week 81 | Pre-infusion; and post-infusion at 20, 40, 50, 60, 90 minutes, and 2, 3, 4, 8 hours. | PK Population: All patients who received at least 1 dose of Replagal (RB or AF) and had at least 1 PK sample drawn. | min·U/mL | MEAN |
| SECONDARY | Pharmacokinetics - Area Under the Serum Concentration-Time Curve (AUC0-∞) - Week 133 | Pre-infusion; and post-infusion at 20, 40, 50, 60, 90 minutes, and 2, 3, 4, 8 hours. | PK Population: All patients who received at least 1 dose of Replagal (RB or AF) and had at least 1 PK sample drawn. | min·U/mL | MEAN |
| SECONDARY | Pharmacokinetics - Area Under the Serum Concentration-Time Curve (AUC0-∞) - Week 159 | Pre-infusion; and post-infusion at 20, 40, 50, 60, 90 minutes, and 2, 3, 4, 8 hours. | PK Population: All patients who received at least 1 dose of Replagal (RB or AF) and had at least 1 PK sample drawn. | min·U/mL | MEAN |
| SECONDARY | Pharmacokinetics - Area Under the Serum Concentration-Time Curve (AUC0-∞) - Week 315/341 | Pre-infusion; and post-infusion at 20, 40, 50, 60, 90 minutes, and 2, 3, 4, 8 hours. | PK Population: All patients who received at least 1 dose of Replagal (RB or AF) and had at least 1 PK sample drawn. | min·U/mL | MEAN |
| OTHER_PRE_SPECIFIED | Heart Rate Variability - Change From Baseline at Week 185 in SDNN | Week 185 | Number of participants present at Visit Week 185 included for analysis. | msec | MEAN |
| SECONDARY | Pharmacokinetics - Maximum Observed Serum Concentration (Cmax) - Week 81 | Pre-infusion; and post-infusion at 20, 40, 50, 60, 90 minutes, and 2, 3, 4, 8 hours. | U/mL | MEAN | |
| SECONDARY | Pharmacokinetics - Maximum Observed Serum Concentration (Cmax) - Week 133 | Pre-infusion; and post-infusion at 20, 40, 50, 60, 90 minutes, and 2, 3, 4, 8 hours. | U/mL | MEAN | |
| SECONDARY | Pharmacokinetics - Maximum Observed Serum Concentration (Cmax) - Week 159 | Pre-infusion; and post-infusion at 20, 40, 50, 60, 90 minutes, and 2, 3, 4, 8 hours. | U/mL | MEAN | |
| SECONDARY | Pharmacokinetics - Maximum Observed Serum Concentration (Cmax) - Week 315/341 | Pre-infusion; and post-infusion at 20, 40, 50, 60, 90 minutes, and 2, 3, 4, 8 hours. | U/mL | MEAN |
Source ClinicalTrials.gov
Results by group10
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Patients Who Experienced At Least One Adverse Event (AE) | Agalsidase Alfa (Cohort 1) | 17 | participants | ||
| Pharmacokinetics - Area Under the Serum Concentration-Time Curve (AUC0-∞) - Week 81 | Agalsidase Alfa (Cohort 1) | 245282 | Standard Deviation 159071 | min·U/mL | |
| Pharmacokinetics - Area Under the Serum Concentration-Time Curve (AUC0-∞) - Week 133 | Agalsidase Alfa (Cohort 1) | 295779 | Standard Deviation 161058 | min·U/mL | |
| Pharmacokinetics - Area Under the Serum Concentration-Time Curve (AUC0-∞) - Week 159 | Agalsidase Alfa (Cohort 1) | 218078 | Standard Deviation 73560 | min·U/mL | |
| Pharmacokinetics - Area Under the Serum Concentration-Time Curve (AUC0-∞) - Week 315/341 | Agalsidase Alfa (Cohort 1) | 213193 | Standard Deviation 119581 | min·U/mL | |
| Heart Rate Variability - Change From Baseline at Week 185 in SDNN | Agalsidase Alfa (Cohort 1) | 20.256 | Standard Deviation 29.060 | msec | |
| Pharmacokinetics - Maximum Observed Serum Concentration (Cmax) - Week 81 | Agalsidase Alfa (Cohort 1) | 3173 | Standard Deviation 969 | U/mL | |
| Pharmacokinetics - Maximum Observed Serum Concentration (Cmax) - Week 133 | Agalsidase Alfa (Cohort 1) | 3842 | Standard Deviation 1235 | U/mL | |
| Pharmacokinetics - Maximum Observed Serum Concentration (Cmax) - Week 159 | Agalsidase Alfa (Cohort 1) | 3842 | Standard Deviation 1235 | U/mL | |
| Pharmacokinetics - Maximum Observed Serum Concentration (Cmax) - Week 315/341 | Agalsidase Alfa (Cohort 1) | 3568 | Standard Deviation 1492 | U/mL |
Source ClinicalTrials.gov
Participant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Phase 1 (Treatment With Replagal RB) | STARTED | Agalsidase Alfa (Cohort 1) | 17 | |
| Phase 1 (Treatment With Replagal RB) | COMPLETED | Agalsidase Alfa (Cohort 1) | 16 | |
| Phase 1 (Treatment With Replagal RB) | NOT COMPLETED | Agalsidase Alfa (Cohort 1) | 1 | |
| Phase 2 (Transition to Replagal AF) | STARTED | Agalsidase Alfa (Cohort 1) | 11 | |
| Phase 2 (Transition to Replagal AF) | COMPLETED | Agalsidase Alfa (Cohort 1) | 10 | |
| Phase 2 (Transition to Replagal AF) | NOT COMPLETED | Agalsidase Alfa (Cohort 1) | 1 |
Source ClinicalTrials.gov
Adverse events436
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 | Gastroenteritis Viral | Infections and infestations | Safety Population RB | 8 | 17 | 12 |
| other | Gastroenteritis Viral | Infections and infestations | Transition Safety Population | 5 | 11 | 6 |
| other | Nasopharyngitis | Infections and infestations | Safety Population RB | 7 | 17 | 12 |
| other | Nasopharyngitis | Infections and infestations | Transition Safety Population | 6 | 11 | 12 |
| other | Upper Respiratory Tract Infection | Infections and infestations | Safety Population RB | 4 | 17 | 11 |
| other | Upper Respiratory Tract Infection | Infections and infestations | Transition Safety Population | 5 | 11 | 16 |
| other | Influenza | Infections and infestations | Safety Population RB | 3 | 17 | 3 |
| other | Influenza | Infections and infestations | Transition Safety Population | 3 | 11 | 3 |
| other | Bronchitis | Infections and infestations | Safety Population RB | 2 | 17 | 2 |
| other | Bronchitis | Infections and infestations | Transition Safety Population | 0 | 11 | 0 |
1 to 10 of 436Source ClinicalTrials.gov
See all 436 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Safety Population RB | ||
| Total, all-cause mortality | Transition Safety Population | ||
| Total, other adverse events | Safety Population RB | 17 | 17 |
| Total, other adverse events | Transition Safety Population | 11 | 11 |
| Total, serious adverse events | Safety Population RB | 2 | 17 |
| Total, serious adverse events | Transition Safety Population | 2 | 11 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 25425121 | DERIVED |
Source PubMed
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 comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Safety Population RB vs Transition Safety Population: serious adverse events |
Source 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/NCT00084084
GET https://conductscience.org/api/trials/v1/NCT00084084/events?page=2