Trial · NCT00430625
A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease
NCT00430625, Phase 3, Completed, Shire; results first posted 2010-09-10
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease. Trials, record NCT00430625. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00430625
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease
- NCT number
- NCT00430625
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 25 participants, actual
- Lead sponsor
- Shire
- Conditions
- Gaucher Disease, Type 1
- Interventions
- VPRIV ®,
- First posted
- 2007-02-02
- Last updated
- 2021-06-29
- Results first posted
- 2010-09-10
- Limitations and caveats
- Due to small sample sizes no conclusions could be drawn on Quality of life (QOL).
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline to 12 Months in Hemoglobin Concentration for the 60 U/kg Treatment Group. | Week 53 | Efficacy endpoint |
| Secondary | Change From Baseline to 12 Months in Hemoglobin Concentration in 45 U/kg Treatment Group | Week 53 | |
| Secondary | Change From Baseline to 12 Months in Platelet Counts for Each Treatment Group. | Week 53 | intent to treat (ITT) Population |
| Secondary | Change From Baseline to 12 Months in Normalized Liver Volume (Percent Body Weight) for Each Treatment Group (Measured by Magnetic Resonance Imaging (MRI) | Week 51 | Liver Volume has been normalized for percentage of body weight for each treatment arm. Liver size relative to body weight = (Liver volume \[cc\]/Body weight \[kg\])\*100 |
| Secondary | Change From Baseline to 12 Months in Normalized Spleen Volume (Percent Body Weight) for Each Treatment Group (Measured by Magnetic Resonance Imaging (MRI)) | Week 51 | 12 patients in the 60 U/kg group and 13 patients in the 45 U/kg group were analyzed for efficacy in the intent to treat (ITT) population. Spleen Volume has been normalized for percent of body weight for each treatment arm. Spleen size relative to body weight = (Spleen volume \[cc\]/Body weight \[kg\])\*100 |
| Secondary | Percent Change From Baseline to 12 Months in Plasma Chitotriosidase for Each Treatment Group | Week 53 | Percent Change from Baseline to Weeks 53 by Randomized velaglucerase alfa Treatment Group - Subset of intent to treat (ITT) Population who were wild type homozygous for chitotriosidase. |
| Secondary | Percent Change From Baseline to 12 Months in Chemokine (C-C Motif) Ligand 18 (CCL18) | Week 53 |
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 | Change From Baseline to 12 Months in Hemoglobin Concentration for the 60 U/kg Treatment Group. | Week 53 | 12 patients in the 60 U/kg group were analyzed for efficacy in the intent to treat (ITT) population. | g/dL | MEAN |
| SECONDARY | Change From Baseline to 12 Months in Hemoglobin Concentration in 45 U/kg Treatment Group | Week 53 | 13 patients in the 45 U/kg group were analyzed for efficacy in the intent to treat (ITT) population. | (g/dL) | MEAN |
| SECONDARY | Change From Baseline to 12 Months in Platelet Counts for Each Treatment Group. | Week 53 | 12 patients in the 60 U/kg group and 13 patients in the 45 U/kg group were analyzed for efficacy in the intent to treat (ITT) population. | x10^9/L | MEAN |
| SECONDARY | Change From Baseline to 12 Months in Normalized Liver Volume (Percent Body Weight) for Each Treatment Group (Measured by Magnetic Resonance Imaging (MRI) | Week 51 | 12 patients in the 60 U/kg group and 13 patients in the 45 U/kg group were analyzed for efficacy in the intent to treat (ITT) population. | Percent body weight | MEAN |
| SECONDARY | Change From Baseline to 12 Months in Normalized Spleen Volume (Percent Body Weight) for Each Treatment Group (Measured by Magnetic Resonance Imaging (MRI)) | Week 51 | 12 patients in the 60 U/kg group and 13 patients in the 45 U/kg group were analyzed for efficacy in the intent to treat (ITT) population. | Percent body weight | MEAN |
| SECONDARY | Percent Change From Baseline to 12 Months in Plasma Chitotriosidase for Each Treatment Group | Week 53 | 2 patients in the 60 U/kg group and 7 patients in the 45 U/kg group who were wild type for the chitotriosidase mutation were analyzed; remaining patients were deficient in chitotriosidase activity. | Percent | NUMBER |
| SECONDARY | Percent Change From Baseline to 12 Months in Chemokine (C-C Motif) Ligand 18 (CCL18) | Week 53 | 12 patients in the 60 U/kg group and 13 patients in the 45 U/kg group were analyzed. | Percent | NUMBER |
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 |
|---|---|---|---|---|---|
| Change From Baseline to 12 Months in Hemoglobin Concentration for the 60 U/kg Treatment Group. | VPRIV® (60 U/kg, IV, Every Other Week) | 2.429 | 95% Confidence Interval 1.717 to 3.141 | g/dL | |
| Change From Baseline to 12 Months in Hemoglobin Concentration in 45 U/kg Treatment Group | VPRIV® (45 U/kg, IV, Every Other Week) | 2.438 | 95% Confidence Interval 1.488 to 3.389 | (g/dL) | |
| Change From Baseline to 12 Months in Platelet Counts for Each Treatment Group. | VPRIV® (45 U/kg, IV, Every Other Week) | 40.92 | 95% Confidence Interval 11.20 to 70.64 | x10^9/L | |
| Change From Baseline to 12 Months in Platelet Counts for Each Treatment Group. | VPRIV® (60 U/kg, IV, Every Other Week) | 50.88 | 95% Confidence Interval 23.97 to 77.78 | x10^9/L | |
| Change From Baseline to 12 Months in Normalized Liver Volume (Percent Body Weight) for Each Treatment Group (Measured by Magnetic Resonance Imaging (MRI) | VPRIV® (45 U/kg, IV, Every Other Week) | -0.30 | 95% Confidence Interval -0.92 to 0.32 | Percent body weight | |
| Change From Baseline to 12 Months in Normalized Liver Volume (Percent Body Weight) for Each Treatment Group (Measured by Magnetic Resonance Imaging (MRI) | VPRIV® (60 U/kg, IV, Every Other Week) | -0.84 | 95% Confidence Interval -1.58 to -0.11 | Percent body weight | |
| Change From Baseline to 12 Months in Normalized Spleen Volume (Percent Body Weight) for Each Treatment Group (Measured by Magnetic Resonance Imaging (MRI)) | VPRIV® (45 U/kg, IV, Every Other Week) | -1.87 | 95% Confidence Interval -3.17 to -0.57 | Percent body weight | |
| Change From Baseline to 12 Months in Normalized Spleen Volume (Percent Body Weight) for Each Treatment Group (Measured by Magnetic Resonance Imaging (MRI)) | VPRIV® (60 U/kg, IV, Every Other Week) | -1.92 | 95% Confidence Interval -3.04 to -0.79 | Percent body weight | |
| Percent Change From Baseline to 12 Months in Plasma Chitotriosidase for Each Treatment Group | VPRIV® (45 U/kg, IV, Every Other Week) | -61.16 | 7.768 | Percent | |
| Percent Change From Baseline to 12 Months in Plasma Chitotriosidase for Each Treatment Group | VPRIV® (60 U/kg, IV, Every Other Week) | -69.65 | 17.65 | Percent |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | VPRIV® (45 U/kg, IV, Every Other Week) | 13 | |
| Overall Study | STARTED | VPRIV® (60 U/kg, IV, Every Other Week) | 12 | |
| Overall Study | COMPLETED | VPRIV® (45 U/kg, IV, Every Other Week) | 13 | |
| Overall Study | COMPLETED | VPRIV® (60 U/kg, IV, Every Other Week) | 12 | |
| Overall Study | NOT COMPLETED | VPRIV® (45 U/kg, IV, Every Other Week) | 0 | |
| Overall Study | NOT COMPLETED | VPRIV® (60 U/kg, IV, Every Other Week) | 0 |
Source ClinicalTrials.gov
Adverse events50
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 | Tinea cruris | Infections and infestations | VPRIV® (45 U/kg, IV, Every Other Week) | 0 | 13 | 0 |
| other | Tinea cruris | Infections and infestations | VPRIV® (60 U/kg, IV, Every Other Week) | 1 | 12 | 1 |
| other | Thrombocytopenia | Blood and lymphatic system disorders | VPRIV® (45 U/kg, IV, Every Other Week) | 1 | 13 | 1 |
| other | Thrombocytopenia | Blood and lymphatic system disorders | VPRIV® (60 U/kg, IV, Every Other Week) | 1 | 12 | 1 |
| other | Hyperproteinaemia | Metabolism and nutrition disorders | VPRIV® (45 U/kg, IV, Every Other Week) | 0 | 13 | 0 |
| other | Hyperproteinaemia | Metabolism and nutrition disorders | VPRIV® (60 U/kg, IV, Every Other Week) | 1 | 12 | 1 |
| other | Headache | Nervous system disorders | VPRIV® (45 U/kg, IV, Every Other Week) | 2 | 13 | 2 |
| other | Headache | Nervous system disorders | VPRIV® (60 U/kg, IV, Every Other Week) | 2 | 12 | 2 |
| other | Dizziness | Nervous system disorders | VPRIV® (45 U/kg, IV, Every Other Week) | 1 | 13 | 1 |
| other | Dizziness | Nervous system disorders | VPRIV® (60 U/kg, IV, Every Other Week) | 1 | 12 | 1 |
1 to 10 of 50Source ClinicalTrials.gov
See all 50 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | VPRIV® (45 U/kg, IV, Every Other Week) | ||
| Total, all-cause mortality | VPRIV® (60 U/kg, IV, Every Other Week) | ||
| Total, other adverse events | VPRIV® (45 U/kg, IV, Every Other Week) | 9 | 13 |
| Total, other adverse events | VPRIV® (60 U/kg, IV, Every Other Week) | 6 | 12 |
| Total, serious adverse events | VPRIV® (45 U/kg, IV, Every Other Week) | 0 | 13 |
| Total, serious adverse events | VPRIV® (60 U/kg, IV, Every Other Week) | 1 | 12 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 27839979 | 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 |
|---|
| VPRIV® (45 U/kg, IV, Every Other Week) vs VPRIV® (60 U/kg, IV, Every Other Week): 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/NCT00430625
GET https://conductscience.org/api/trials/v1/NCT00430625/events?page=2