Trial · NCT00635427
An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease
NCT00635427, Phase 3, Completed, Shire; results first posted 2014-01-28
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease. Trials, record NCT00635427. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00635427
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease
- NCT number
- NCT00635427
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 95 participants, actual
- Lead sponsor
- Shire
- Conditions
- Gaucher Disease, Type 1
- Interventions
- VPRIV®
- First posted
- 2008-03-13
- Last updated
- 2021-06-10
- Other registries' IDs
- eudract:2008-001965-27
- Results first posted
- 2014-01-28
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Overall Summary of Treatment Emergent Adverse Events | Baseline to termination of study | Safety was evaluated by an analysis of adverse events (AEs), concomitant medication use, clinical laboratory tests, vital signs during the infusion of study drug, physical examination, and the development of anti-velaglucerase alfa. No formal comparisons or statistical tests were applied for the safety analyses, including for differences between the groups. |
| Secondary | Change From Baseline to 24 Months in Hemoglobin Concentration for Each Treatment Group | Baseline to 24 months | |
| Secondary | Change From Baseline to 24 Months in Platelet Counts for Each Treatment Group | Baseline to 24 months | |
| Secondary | Change From Baseline to 24 Months in Normalized Liver Volume for Each Treatment Group | Baseline to 24 months | |
| Secondary | Percentage Change From Baseline to 24 Months in Normalized Spleen Volume for Each Treatment Group | Baseline to 24 months |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Overall Summary of Treatment Emergent Adverse Events | Baseline to termination of study | All participants who received at least 1 infusion (full or partial) of study drug were evaluated for safety (ie, were included in the safety population). There were 95 participants in the safety population. | Participants | NUMBER |
| SECONDARY | Change From Baseline to 24 Months in Hemoglobin Concentration for Each Treatment Group | Baseline to 24 months | (g/dL) | MEAN | |
| SECONDARY | Change From Baseline to 24 Months in Platelet Counts for Each Treatment Group | Baseline to 24 months | (10^9/L) | MEAN | |
| SECONDARY | Change From Baseline to 24 Months in Normalized Liver Volume for Each Treatment Group | Baseline to 24 months | % Body weight | MEAN | |
| SECONDARY | Percentage Change From Baseline to 24 Months in Normalized Spleen Volume for Each Treatment Group | Baseline to 24 months | Precent (%) change | MEAN |
Source ClinicalTrials.gov
Results by group48
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Overall Summary of Treatment Emergent Adverse Events | VPRIV 60 U/kg(Parent Study VPRIV(45 or 60 U/kg) TKT032,GCB039) | Experienced no AEs | 3 | Participants | |
| Overall Summary of Treatment Emergent Adverse Events | VPRIV 60 U/kg (Parent Study-imiglucerase (60 U/kg)HGT-GCB-039) | Experienced no AEs | 1 | Participants | |
| Overall Summary of Treatment Emergent Adverse Events | VPRIV 15-60 U/kg (Parent Study VPRIV (15-60 U/kg) TKT034) | Experienced no AEs | 3 | Participants | |
| Overall Summary of Treatment Emergent Adverse Events | VPRIV 60 U/kg(Parent Study VPRIV(45 or 60 U/kg) TKT032,GCB039) | Experienced at least 1 AE | 38 | Participants | |
| Overall Summary of Treatment Emergent Adverse Events | VPRIV 60 U/kg (Parent Study-imiglucerase (60 U/kg)HGT-GCB-039) | Experienced at least 1 AE | 15 | Participants | |
| Overall Summary of Treatment Emergent Adverse Events | VPRIV 15-60 U/kg (Parent Study VPRIV (15-60 U/kg) TKT034) | Experienced at least 1 AE | 35 | Participants | |
| Overall Summary of Treatment Emergent Adverse Events | VPRIV 60 U/kg(Parent Study VPRIV(45 or 60 U/kg) TKT032,GCB039) | Experienced at least 1 drug-related AE | 9 | Participants | |
| Overall Summary of Treatment Emergent Adverse Events | VPRIV 60 U/kg (Parent Study-imiglucerase (60 U/kg)HGT-GCB-039) | Experienced at least 1 drug-related AE | 7 | Participants | |
| Overall Summary of Treatment Emergent Adverse Events | VPRIV 15-60 U/kg (Parent Study VPRIV (15-60 U/kg) TKT034) | Experienced at least 1 drug-related AE | 8 | Participants | |
| Overall Summary of Treatment Emergent Adverse Events | VPRIV 60 U/kg(Parent Study VPRIV(45 or 60 U/kg) TKT032,GCB039) | Experienced at least 1 infusion-related AE | 5 | Participants |
1 to 10 of 48Source ClinicalTrials.gov
See all 48 results by groupParticipant flow15
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | VPRIV 60 U/kg (Parent Study VPRIV (45 U/kg) -TKT032) | 13 | |
| Overall Study | STARTED | VPRIV 60 U/kg (Parent Study- VPRIV (60 U/kg)-TKT032) | 12 | |
| Overall Study | STARTED | VPRIV 60 U/kg (Parent Study- VPRIV (60U/kg) HGT-GCB-039) | 16 | |
| Overall Study | STARTED | VPRIV 60 U/kg (Parent Study-imiglucerase(60 U/kg)HGT-GCB-039) | 16 | |
| Overall Study | STARTED | VPRIV 15-60 U/kg (Parent Study VPRIV(15-60 U/kg)TKT034) | 38 | |
| Overall Study | COMPLETED | VPRIV 60 U/kg (Parent Study VPRIV (45 U/kg) -TKT032) | 1 | |
| Overall Study | COMPLETED | VPRIV 60 U/kg (Parent Study- VPRIV (60 U/kg)-TKT032) | 6 | |
| Overall Study | COMPLETED | VPRIV 60 U/kg (Parent Study- VPRIV (60U/kg) HGT-GCB-039) | 5 | |
| Overall Study | COMPLETED | VPRIV 60 U/kg (Parent Study-imiglucerase(60 U/kg)HGT-GCB-039) | 7 | |
| Overall Study | COMPLETED | VPRIV 15-60 U/kg (Parent Study VPRIV(15-60 U/kg)TKT034) | 30 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 participant flowAdverse events375
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 | Abdominal pain | Gastrointestinal disorders | VPRIV 15-60 U/kg (Parent Study VPRIV (15-60 U/kg) TKT034) | 2 | 38 | 2 |
| other | Abdominal pain | Gastrointestinal disorders | VPRIV 60 U/kg(VPRIV Parent Study 45 or 60 U/kg- TKT032,GCB039) | 3 | 41 | 3 |
| other | Abdominal pain | Gastrointestinal disorders | VPRIV 60 U/kg (Parent Study-imiglucerase(60 U/kg) HGT-GCB-039) | 1 | 16 | 1 |
| other | Abdominal pain upper | Gastrointestinal disorders | VPRIV 15-60 U/kg (Parent Study VPRIV (15-60 U/kg) TKT034) | 3 | 38 | 4 |
| other | Abdominal pain upper | Gastrointestinal disorders | VPRIV 60 U/kg(VPRIV Parent Study 45 or 60 U/kg- TKT032,GCB039) | 4 | 41 | 6 |
| other | Abdominal pain upper | Gastrointestinal disorders | VPRIV 60 U/kg (Parent Study-imiglucerase(60 U/kg) HGT-GCB-039) | 2 | 16 | 2 |
| other | Diarrhoea | Gastrointestinal disorders | VPRIV 15-60 U/kg (Parent Study VPRIV (15-60 U/kg) TKT034) | 2 | 38 | 2 |
| other | Diarrhoea | Gastrointestinal disorders | VPRIV 60 U/kg(VPRIV Parent Study 45 or 60 U/kg- TKT032,GCB039) | 2 | 41 | 3 |
| other | Diarrhoea | Gastrointestinal disorders | VPRIV 60 U/kg (Parent Study-imiglucerase(60 U/kg) HGT-GCB-039) | 1 | 16 | 1 |
| other | Dyspepsia | Gastrointestinal disorders | VPRIV 15-60 U/kg (Parent Study VPRIV (15-60 U/kg) TKT034) | 2 | 38 | 4 |
1 to 10 of 375Source ClinicalTrials.gov
See all 375 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | VPRIV 15-60 U/kg (Parent Study VPRIV (15-60 U/kg) TKT034) | ||
| Total, all-cause mortality | VPRIV 60 U/kg(VPRIV Parent Study 45 or 60 U/kg- TKT032,GCB039) | ||
| Total, all-cause mortality | VPRIV 60 U/kg (Parent Study-imiglucerase(60 U/kg) HGT-GCB-039) | ||
| Total, other adverse events | VPRIV 15-60 U/kg (Parent Study VPRIV (15-60 U/kg) TKT034) | 35 | 38 |
| Total, other adverse events | VPRIV 60 U/kg(VPRIV Parent Study 45 or 60 U/kg- TKT032,GCB039) | 35 | 41 |
| Total, other adverse events | VPRIV 60 U/kg (Parent Study-imiglucerase(60 U/kg) HGT-GCB-039) | 15 | 16 |
| Total, serious adverse events | VPRIV 15-60 U/kg (Parent Study VPRIV (15-60 U/kg) TKT034) | 6 | 38 |
| Total, serious adverse events | VPRIV 60 U/kg(VPRIV Parent Study 45 or 60 U/kg- TKT032,GCB039) | 6 | 41 |
| Total, serious adverse events | VPRIV 60 U/kg (Parent Study-imiglucerase(60 U/kg) HGT-GCB-039) | 4 | 16 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
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/NCT00635427
GET https://conductscience.org/api/trials/v1/NCT00635427/results?page=2