Trial · NCT00864851
Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease
NCT00864851, Phase 3, Completed, Shire; results first posted 2014-06-06
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease. Trials, record NCT00864851. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00864851
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease
- NCT number
- NCT00864851
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 44 participants, actual
- Lead sponsor
- Shire
- Conditions
- Fabry Disease
- Interventions
- Replagal
- First posted
- 2009-03-19
- Last updated
- 2021-06-09
- Other registries' IDs
- eudract:2007-005543-22
- Results first posted
- 2014-06-06
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline to Month 12 in Left Ventricular Mass Indexed to Height (LVMI) | Baseline, Month 12 (Week 53) | Left ventricular mass (LVM) was measured through echocardiography. |
| Secondary | Change From Baseline to Month 12 in Maximal Oxygen Consumption (VO2 Max) at Peak Exercise | Baseline, Month 12 (Week 53) | Exercise tolerance as measured by VO2 max at peak exercise using the standard exponential exercise protocol (STEEP). |
| Secondary | Change From Baseline to Month 12 in Distance Walked in 6-Minute Walk Test (6MWT) | Baseline, Month 12 (Week 53) | Exercise tolerance using the 6MWT was measured as the total distance walked in 6 minutes. |
| Secondary | Change From Baseline to Month 12 in the Minnesota Living With Heart Failure Questionnaire (MLHF-Q) Summary Score | Baseline, Month 12 (Week 53) | Quality of life (QoL) was evaluated using the MLHF-Q, version 2. The questionnaire is designed to assess the degree to which heart failure symptoms affect a patient's daily life. The summary score ranges from 0 to 105, with a score of 105 representing the highest adverse impact on a patient's QoL. |
| Secondary | Change From Baseline to Month 12 in New York Heart Association (NYHA) Functional Class | Baseline, Month 12 (Week 53) | The NYHA functional classification system relates symptoms to everyday activities and the patient's quality of life. NYHA Classification - The Stages of Heart Failure: Class I (Mild): No limitation of physical activity. Ordinary physical activity does not cause undue fatigue, palpitation, or dyspnea (shortness of breath). Class II (Mild): Slight limitation of physical activity. Comfortable at rest, but ordinary physical activity results in fatigue, palpitation, or dyspnea. Class III (Moderate): Marked limitation of physical activity. Comfortable at rest, but less than ordinary activity causes fatigue, palpitation, or dyspnea. Class IV (Severe): Unable to carry out any physical activity without discomfort. Symptoms of cardiac insufficiency at rest. If any physical activity is undertaken, discomfort is increased. |
| Secondary | Change From Baseline to Month 12 in Plasma Globotriaosylceramide (GB3) | Baseline, Month 12 (Week 53) | |
| Secondary | Change From Baseline to Month 12 in Estimated Glomerular Filtration Rate (eGFR) | Baseline, Month 12 (Week 53) | Renal function was assessed by an evaluation of change from baseline to Month 12 in eGFR as calculated using the Modification of Diet for Renal Disease (MDRD) equation. |
| Secondary | Change From Baseline to Month 12 in Urinary Albumin/Creatinine (A/Cr) Ratio | Baseline, Month 12 (Week 53) | |
| Secondary | Safety Evaluation | 56 Weeks | Adverse events were collected throughout the study, from the time of informed consent to approximately 30 days post-final infusion. |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline to Month 12 in Left Ventricular Mass Indexed to Height (LVMI) | Baseline, Month 12 (Week 53) | Intent to Treat (ITT) Population: All randomized patients who received at least 1 complete or partial dose of Replagal. | g/m^2.7 | MEAN |
| SECONDARY | Change From Baseline to Month 12 in Maximal Oxygen Consumption (VO2 Max) at Peak Exercise | Baseline, Month 12 (Week 53) | Intent to Treat (ITT) Population: All randomized patients who received at least 1 complete or partial dose of Replagal. | mL/min/kg | MEAN |
| SECONDARY | Change From Baseline to Month 12 in Distance Walked in 6-Minute Walk Test (6MWT) | Baseline, Month 12 (Week 53) | Intent to Treat (ITT) Population: All randomized patients who received at least 1 complete or partial dose of Replagal. | m | MEAN |
| SECONDARY | Change From Baseline to Month 12 in the Minnesota Living With Heart Failure Questionnaire (MLHF-Q) Summary Score | Baseline, Month 12 (Week 53) | Intent to Treat (ITT) Population: All randomized patients who received at least 1 complete or partial dose of Replagal. | scores on a scale | MEAN |
| SECONDARY | Change From Baseline to Month 12 in New York Heart Association (NYHA) Functional Class | Baseline, Month 12 (Week 53) | Intent to Treat (ITT) Population: All randomized patients who received at least 1 complete or partial dose of Replagal. | participants | NUMBER |
| SECONDARY | Change From Baseline to Month 12 in Plasma Globotriaosylceramide (GB3) | Baseline, Month 12 (Week 53) | Intent to Treat (ITT) Population: All randomized patients who received at least 1 complete or partial dose of Replagal. | nmol/ml | MEAN |
| SECONDARY | Change From Baseline to Month 12 in Estimated Glomerular Filtration Rate (eGFR) | Baseline, Month 12 (Week 53) | Intent to Treat (ITT) Population: All randomized patients who received at least 1 complete or partial dose of Replagal. | mL/min/1.73m^2 | MEAN |
| SECONDARY | Change From Baseline to Month 12 in Urinary Albumin/Creatinine (A/Cr) Ratio | Baseline, Month 12 (Week 53) | Intent to Treat (ITT) Population: All randomized patients who received at least 1 complete or partial dose of Replagal. | mg/g | MEAN |
| SECONDARY | Safety Evaluation | 56 Weeks | Intent to Treat (ITT) Population: All randomized patients who received at least 1 complete or partial dose of Replagal. Analyses were performed on the ITT population because it was identical to the safety population. | participants | NUMBER |
Source ClinicalTrials.gov
Results by group63
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline to Month 12 in Left Ventricular Mass Indexed to Height (LVMI) | Replagal 0.2 mg/kg, IV, Every Other Week | 3.2 | Standard Deviation 12.5 | g/m^2.7 | |
| Change From Baseline to Month 12 in Left Ventricular Mass Indexed to Height (LVMI) | Replagal 0.2 mg/kg, IV, Weekly | 0.5 | Standard Deviation 15.8 | g/m^2.7 | |
| Change From Baseline to Month 12 in Left Ventricular Mass Indexed to Height (LVMI) | Replagal 0.4 mg/kg, IV, Weekly | -10.3 | Standard Deviation 11.8 | g/m^2.7 | |
| Change From Baseline to Month 12 in Maximal Oxygen Consumption (VO2 Max) at Peak Exercise | Replagal 0.2 mg/kg, IV, Every Other Week | -2.0 | Standard Deviation 3.24 | mL/min/kg | |
| Change From Baseline to Month 12 in Maximal Oxygen Consumption (VO2 Max) at Peak Exercise | Replagal 0.2 mg/kg, IV, Weekly | -0.3 | Standard Deviation 4.76 | mL/min/kg | |
| Change From Baseline to Month 12 in Maximal Oxygen Consumption (VO2 Max) at Peak Exercise | Replagal 0.4 mg/kg, IV, Weekly | 2.2 | Standard Deviation 5.85 | mL/min/kg | |
| Change From Baseline to Month 12 in Distance Walked in 6-Minute Walk Test (6MWT) | Replagal 0.2 mg/kg, IV, Every Other Week | -10.4 | Standard Deviation 87.7 | m | |
| Change From Baseline to Month 12 in Distance Walked in 6-Minute Walk Test (6MWT) | Replagal 0.2 mg/kg, IV, Weekly | 37.9 | Standard Deviation 70.5 | m | |
| Change From Baseline to Month 12 in Distance Walked in 6-Minute Walk Test (6MWT) | Replagal 0.4 mg/kg, IV, Weekly | 24.7 | Standard Deviation 45.7 | m | |
| Change From Baseline to Month 12 in the Minnesota Living With Heart Failure Questionnaire (MLHF-Q) Summary Score | Replagal 0.2 mg/kg, IV, Every Other Week | -3.1 | Standard Deviation 16.7 | scores on a scale |
1 to 10 of 63Source ClinicalTrials.gov
See all 63 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Replagal 0.2 mg/kg, IV, Every Other Week | 20 | |
| Overall Study | STARTED | Replagal 0.2 mg/kg, IV, Weekly | 19 | |
| Overall Study | STARTED | Replagal 0.4 mg/kg, IV, Weekly | 5 | |
| Overall Study | COMPLETED | Replagal 0.2 mg/kg, IV, Every Other Week | 17 | |
| Overall Study | COMPLETED | Replagal 0.2 mg/kg, IV, Weekly | 18 | |
| Overall Study | COMPLETED | Replagal 0.4 mg/kg, IV, Weekly | 5 | |
| Overall Study | NOT COMPLETED | Replagal 0.2 mg/kg, IV, Every Other Week | 3 | |
| Overall Study | NOT COMPLETED | Replagal 0.2 mg/kg, IV, Weekly | 1 | |
| Overall Study | NOT COMPLETED | Replagal 0.4 mg/kg, IV, Weekly | 0 |
Source ClinicalTrials.gov
Adverse events924
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 | Nasopharyngitis | Infections and infestations | Replagal 0.2 mg/kg, IV, Every Other Week | 2 | 20 | 2 |
| other | Nasopharyngitis | Infections and infestations | Replagal 0.2 mg/kg, IV, Weekly | 6 | 19 | 11 |
| other | Nasopharyngitis | Infections and infestations | Replagal 0.4 mg/kg, IV, Weekly | 0 | 5 | 0 |
| other | Nasopharyngitis | Infections and infestations | Overall | 8 | 44 | 13 |
| other | Bronchitis | Infections and infestations | Replagal 0.2 mg/kg, IV, Every Other Week | 2 | 20 | 2 |
| other | Bronchitis | Infections and infestations | Replagal 0.2 mg/kg, IV, Weekly | 3 | 19 | 3 |
| other | Bronchitis | Infections and infestations | Replagal 0.4 mg/kg, IV, Weekly | 1 | 5 | 1 |
| other | Bronchitis | Infections and infestations | Overall | 6 | 44 | 6 |
| other | Respiratory tract infection | Infections and infestations | Replagal 0.2 mg/kg, IV, Every Other Week | 0 | 20 | 0 |
| other | Respiratory tract infection | Infections and infestations | Replagal 0.2 mg/kg, IV, Weekly | 4 | 19 | 4 |
1 to 10 of 924Source ClinicalTrials.gov
See all 924 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Replagal 0.2 mg/kg, IV, Every Other Week | ||
| Total, all-cause mortality | Replagal 0.2 mg/kg, IV, Weekly | ||
| Total, all-cause mortality | Replagal 0.4 mg/kg, IV, Weekly | ||
| Total, all-cause mortality | Overall | ||
| Total, other adverse events | Replagal 0.2 mg/kg, IV, Every Other Week | 19 | 20 |
| Total, other adverse events | Replagal 0.2 mg/kg, IV, Weekly | 17 | 19 |
| Total, other adverse events | Replagal 0.4 mg/kg, IV, Weekly | 5 | 5 |
| Total, other adverse events | Overall | 41 | 44 |
| Total, serious adverse events | Replagal 0.2 mg/kg, IV, Every Other Week | 8 | 20 |
| Total, serious adverse events | Replagal 0.2 mg/kg, IV, Weekly | 5 | 19 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 20730727 | RESULT |
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 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/NCT00864851
GET https://conductscience.org/api/trials/v1/NCT00864851/results?page=2