Trial · NCT01298141
A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease
NCT01298141, Phase 3, Completed, Shire; results first posted 2019-02-15
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease. Trials, record NCT01298141. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01298141
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease
- NCT number
- NCT01298141
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 171 participants, actual
- Lead sponsor
- Shire
- Conditions
- Fabry Disease
- Interventions
- agalsidase alfa
- First posted
- 2011-02-17
- Last updated
- 2021-06-08
- Results first posted
- 2019-02-15
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | From the start of study treatment up to 30 days after the last dose of study drug administration (up to 320 weeks) | An adverse event (AE) was any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in any phase of a clinical study, whether or not considered investigational product-related.Treatment-emergent adverse events (TEAEs) were defined as those events which occurred or worsened in severity after first treatment with Replagal AF until 30 days after the last dose. A serious AE (SAE) was any AE occurred at any dose that resulted in death, life-threatening, hospitalization, prolongation of existing hospitalization, persistent or significant disability or incapacity and congenital anomaly or birth defect. |
| Primary | Number of Participants With Infusion-Related Reactions (IRR) | From the start of study treatment up to 30 days after the last dose of study drug administration (up to 320 weeks) | An IRR (also referred to as infusion-related adverse event \[IRAE\]) was defined as an AE that began either during the infusion or within 12 hours after the start of the infusion and was judged as possibly or probably related to study drug. The IRRs were classified based on the severity as Mild=No limitation of usual activities, Moderate=Some limitation of usual activities, Severe=Inability to carry out usual activities and Life-threatening=Immediate risk of death. The number of participants with infusion-related reactions was reported. |
| Primary | Number of Participants Who Reported Positive to Immunoglobulin A (IgA) | Baseline (within 6 months prior to first dose) up to Week 129 | The IgA status was measured using enzyme-linked immunosorbent assay (ELISA). Number of participants who reported positive to IgA was reported. |
| Primary | Number of Participants Who Reported Positive to Immunoglobulin E (IgE) | Baseline (within 6 months prior to first dose) up to Week 129 | The IgE status was measured using ELISA. Number of participants who reported positive to IgE was reported. |
| Primary | Number of Participants Who Reported Positive to Immunoglobulin M (IgM) | Baseline (within 6 months prior to first dose) up to Week 129 | The IgM status was measured using ELISA. Number of participants who reported positive to IgM was reported. |
| Primary | Number of Participants Who Reported Positive to Anti-drug Antibody (ADA) | Baseline (within 6 months prior to first dose) up to Week 285 | The ADA status was measured using ELISA and electrochemiluminescent (ECL) immunoassay. Number of participants who reported positive to ADA was reported. |
| Primary | Number of Participants Who Reported Positive to Neutralizing Antibody (NAb) | Baseline (within 6 months prior to first dose) up to Week 285 | The NAb status was measured using enzyme activity inhibition assay. Number of participants who reported positive to NAb was reported. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | From the start of study treatment up to 30 days after the last dose of study drug administration (up to 320 weeks) | Safety Population included all participants who received at least one full or partial infusion of Replagal AF. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Infusion-Related Reactions (IRR) | From the start of study treatment up to 30 days after the last dose of study drug administration (up to 320 weeks) | Safety Population included all participants who received at least one full or partial infusion of Replagal AF. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants Who Reported Positive to Immunoglobulin A (IgA) | Baseline (within 6 months prior to first dose) up to Week 129 | Safety population included all participants who received at least one full or partial infusion of Replagal AF. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants Who Reported Positive to Immunoglobulin E (IgE) | Baseline (within 6 months prior to first dose) up to Week 129 | Safety population included all participants who received at least one full or partial infusion of Replagal AF. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants Who Reported Positive to Immunoglobulin M (IgM) | Baseline (within 6 months prior to first dose) up to Week 129 | Safety population included all participants who received at least one full or partial infusion of Replagal AF. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants Who Reported Positive to Anti-drug Antibody (ADA) | Baseline (within 6 months prior to first dose) up to Week 285 | Safety population included all participants who received at least one full or partial infusion of Replagal AF. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants Who Reported Positive to Neutralizing Antibody (NAb) | Baseline (within 6 months prior to first dose) up to Week 285 | Safety population included all participants who received at least one full or partial infusion of Replagal AF. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group12
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Replagal (Agalsidase Alfa) | Any TEAE | 163 | Participants | |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Replagal (Agalsidase Alfa) | Any Serious TEAE | 74 | Participants | |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Replagal (Agalsidase Alfa) | Any TEAE Leading to Treatment Discontinuation | 7 | Participants | |
| Number of Participants With Infusion-Related Reactions (IRR) | Replagal (Agalsidase Alfa) | Mild | 21 | Participants | |
| Number of Participants With Infusion-Related Reactions (IRR) | Replagal (Agalsidase Alfa) | Moderate | 16 | Participants | |
| Number of Participants With Infusion-Related Reactions (IRR) | Replagal (Agalsidase Alfa) | Severe | 3 | Participants | |
| Number of Participants With Infusion-Related Reactions (IRR) | Replagal (Agalsidase Alfa) | Life-threatening | 0 | Participants | |
| Number of Participants Who Reported Positive to Immunoglobulin A (IgA) | Replagal (Agalsidase Alfa) | 10 | Participants | ||
| Number of Participants Who Reported Positive to Immunoglobulin E (IgE) | Replagal (Agalsidase Alfa) | 1 | Participants | ||
| Number of Participants Who Reported Positive to Immunoglobulin M (IgM) | Replagal (Agalsidase Alfa) | 48 | Participants |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 results by groupParticipant flow4
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Replagal (Agalsidase Alfa) | 171 | |
| Overall Study | Treated | Replagal (Agalsidase Alfa) | 167 | |
| Overall Study | COMPLETED | Replagal (Agalsidase Alfa) | 140 | |
| Overall Study | NOT COMPLETED | Replagal (Agalsidase Alfa) | 31 |
Source ClinicalTrials.gov
Adverse events237
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 | Replagal (Agalsidase Alfa) | 11 | 167 | 25 |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | Replagal (Agalsidase Alfa) | 45 | 167 | 68 |
| other | Back pain | Musculoskeletal and connective tissue disorders | Replagal (Agalsidase Alfa) | 46 | 167 | 63 |
| other | Joint swelling | Musculoskeletal and connective tissue disorders | Replagal (Agalsidase Alfa) | 14 | 167 | 17 |
| other | Muscle spasms | Musculoskeletal and connective tissue disorders | Replagal (Agalsidase Alfa) | 22 | 167 | 30 |
| other | Muscular weakness | Musculoskeletal and connective tissue disorders | Replagal (Agalsidase Alfa) | 10 | 167 | 12 |
| other | Musculoskeletal pain | Musculoskeletal and connective tissue disorders | Replagal (Agalsidase Alfa) | 23 | 167 | 29 |
| other | Myalgia | Musculoskeletal and connective tissue disorders | Replagal (Agalsidase Alfa) | 21 | 167 | 22 |
| other | Neck pain | Musculoskeletal and connective tissue disorders | Replagal (Agalsidase Alfa) | 14 | 167 | 14 |
| other | Osteoarthritis | Musculoskeletal and connective tissue disorders | Replagal (Agalsidase Alfa) | 12 | 167 | 12 |
1 to 10 of 237Source ClinicalTrials.gov
See all 237 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Replagal (Agalsidase Alfa) | 6 | 167 |
| Total, other adverse events | Replagal (Agalsidase Alfa) | 160 | 167 |
| Total, serious adverse events | Replagal (Agalsidase Alfa) | 74 | 167 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 34542871 | 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 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/NCT01298141
GET https://conductscience.org/api/trials/v1/NCT01298141/events?page=2