Trial · NCT00478647
Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase
NCT00478647, Phase 2, Phase 3, Completed, Shire; results first posted 2010-09-02
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase. Trials, record NCT00478647. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00478647
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase
- NCT number
- NCT00478647
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 40 participants, actual
- Lead sponsor
- Shire
- Conditions
- Gaucher Disease
- Interventions
- GA-GCB (velaglucerase alfa)
- First posted
- 2007-05-25
- Last updated
- 2021-06-10
- Other registries' IDs
- eudract:2006-006304-11
- Results first posted
- 2010-09-02
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Participants Who Experienced at Least One Adverse Event | Week 53 | Safety was assessed throughout the study by assessments including adverse events, concomitant medication use, and vital signs. Additional safety assessments, including 12-lead ECGs, physical examinations, clinical laboratory tests and determination of the presence of anti-velaglucerase alfa antibodies. Refer to Adverse event section for further details. |
| Secondary | Change From Baseline to Week 53 in Hemoglobin Concentration | Week 53 | |
| Secondary | Percent Change From Baseline to Week 53 in Platelet Count | Week 53 | |
| Secondary | Percent Change From Baseline to Week 51 in Normalized Liver Volume | Week 51 | Liver volume has been normalized for percentage (%) of body weight. Liver size relative to body weight= (Liver volume \[cc\]/Body weight \[kg\])\*100 |
| Secondary | Percent Change From Baseline to Week 51 in Normalized Spleen Volume | Week 51 | Spleen volume has been normalized for percentage (%) of body weight. Spleen size relative to body weight= (Spleen volume \[cc\]/Body weight \[kg\])\*100 |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| SECONDARY | Change From Baseline to Week 53 in Hemoglobin Concentration | Week 53 | ITT population. | g/dL | MEAN |
| SECONDARY | Percent Change From Baseline to Week 53 in Platelet Count | Week 53 | ITT population. | percent (%) change | MEAN |
| SECONDARY | Percent Change From Baseline to Week 51 in Normalized Liver Volume | Week 51 | ITT population. | Percent (%) change | MEAN |
| SECONDARY | Percent Change From Baseline to Week 51 in Normalized Spleen Volume | Week 51 | ITT population. Four splenectomized participants were excluded. | Percent (%) change | MEAN |
| PRIMARY | Participants Who Experienced at Least One Adverse Event | Week 53 | Safety population included subjects who have received at least 1 full or partial dose of study drug. | participants | NUMBER |
Source ClinicalTrials.gov
Results by group5
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 Week 53 in Hemoglobin Concentration | GA-GCB (Velaglucerase Alfa) | -0.101 | 90% Confidence Interval -0.272 to 0.070 | g/dL | |
| Percent Change From Baseline to Week 53 in Platelet Count | GA-GCB (Velaglucerase Alfa) | 7.04 | 90% Confidence Interval 0.54 to 13.53 | percent (%) change | |
| Percent Change From Baseline to Week 51 in Normalized Liver Volume | GA-GCB (Velaglucerase Alfa) | -0.03 | 90% Confidence Interval -2.62 to 2.56 | Percent (%) change | |
| Percent Change From Baseline to Week 51 in Normalized Spleen Volume | GA-GCB (Velaglucerase Alfa) | -5.56 | 90% Confidence Interval -10.77 to -0.35 | Percent (%) change | |
| Participants Who Experienced at Least One Adverse Event | GA-GCB (Velaglucerase Alfa) | 34 | participants |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | GA-GCB (Velaglucerase Alfa) | 40 | |
| Overall Study | COMPLETED | GA-GCB (Velaglucerase Alfa) | 38 | |
| Overall Study | NOT COMPLETED | GA-GCB (Velaglucerase Alfa) | 2 |
Source ClinicalTrials.gov
Adverse events46
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 | Hypertension | Vascular disorders | GA-GCB (Velaglucerase Alfa) | 3 | 40 | 15 |
| other | Fatigue | General disorders | GA-GCB (Velaglucerase Alfa) | 5 | 40 | 13 |
| other | Influenza like illness | General disorders | GA-GCB (Velaglucerase Alfa) | 4 | 40 | 5 |
| other | Malaise | General disorders | GA-GCB (Velaglucerase Alfa) | 2 | 40 | 33 |
| other | Non-Cardiac chest pain | General disorders | GA-GCB (Velaglucerase Alfa) | 2 | 40 | 3 |
| other | Pyrexia | General disorders | GA-GCB (Velaglucerase Alfa) | 5 | 40 | 7 |
| other | Anxiety | Psychiatric disorders | GA-GCB (Velaglucerase Alfa) | 2 | 40 | 2 |
| other | Fall | Injury, poisoning and procedural complications | GA-GCB (Velaglucerase Alfa) | 2 | 40 | 2 |
| other | Procedural pain | Injury, poisoning and procedural complications | GA-GCB (Velaglucerase Alfa) | 2 | 40 | 2 |
| other | Activated partial thromboplastin time prolonged | Investigations | GA-GCB (Velaglucerase Alfa) | 2 | 40 | 2 |
1 to 10 of 46Source ClinicalTrials.gov
See all 46 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | GA-GCB (Velaglucerase Alfa) | ||
| Total, other adverse events | GA-GCB (Velaglucerase Alfa) | 31 | 40 |
| Total, serious adverse events | GA-GCB (Velaglucerase Alfa) | 4 | 40 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 23339116 | 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/NCT00478647
GET https://conductscience.org/api/trials/v1/NCT00478647/events?page=2